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Adam Feuerstein

Columnist Adam Feuerstein covers biotech stocks-in plain English. Also included is his popular weekend biotech-stocks mailbag.

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Immunomedics Kicked Out of Prestigious ASCO Cancer Conference

Fri, 03 Jun 2016 15:42 GMT

Updates article from late Thursday, June 2, with stock price, company statement and analysis.  Immunomedics was caught trying to sneak old, previously presented clinical data on its triple-negative breast cancer drug IMMU-132 into the American Society of Clinical Oncology (ASCO) annual meeting, which starts Friday. That's a violation of ASCO's rules. As a result, ASCO informed Immunomedics Thursday that the IMMU-132 breast cancer abstract was removed from the meeting. "Since the confidentiality of this abstract was violated, the abstract and presentation have been removed from the meeting and will no longer be featured in our press program," Alise Fisher, program coordinator in ASCO's science communications department, said in an email Thursday night. The Immunomedics screw-up was entirely self-inflicted. ASCO accepted the company's late-breaking abstract for IMMU-132 into the annual meeting on the premise that it contained updated and previously undisclosed results from a mid-stage study in triple-negative breast cancer. But Immunomedics failed to tell ASCO that the IMMU-132 data were not updated or new at all. Immunomedics Founder and Chief Scientific Officer David Goldenberg had already presented the same IMMU-132 trial results at an industry networking meeting in April, ASCO learned. The company issued a press release when these IMMU-132 data were presented in April, posted the presentation slides on its web site and discussed the data with investors on a conference call.  Immunomedics also never told Dr. Aditya Bardia of Harvard Medical School, the principal investigator of the IMMU-132 breast cancer study and the scheduled ASCO presenter, about the April data reveal, Bardia told me in an interview on Wednesday. Investors look to the ASCO meeting, held in June each year, for the latest developments and newest clinical data on experimental cancer drugs. On a recent conference call with investors, Goldenberg and his wife, Immunomedics CEO Cynthia Sullivan, played up the importance of the IMMU-132 ASCO presentation. Coupled with the Breakthrough Therapy Designation granted to the drug by FDA in February, the company hopes to win approval of its first drug after 34 years of research efforts. [Immunomedics was founded in 1982.] Immunomedics stock price has risen by 70% since April 20, when ASCO announced the inclusion of the IMMU-132 breast cancer data as part of its media briefing program for the annual meeting. But unbeknownst to ASCO and investors, the updated IMMU-132 data hyped by Immunomedics executives turned out to be old and previously seen. For that transgression, Immunomedics was kicked out of the most prestigious and closely watched cancer conference of the year. Immunomedics shares recently fell 18% to $4.35 Friday.  The company issued a statement confirming ASCO's decision to remove the IMMU-132 presentation from the annual meeting. CEO Sullivan added: The presenter, Dr. Bardia, and I are attempting to reverse this with ASCO, because we believe the patient population and results reported in April were different from those in the ASCO abstract submitted last February. Both we and our many investigators involved are disappointed that our excellent therapeutic results, achieving an interim median survival of about 14 months, may not be presented at this meeting. These results are very encouraging for TNBC patients with metastatic disease who failed multiple prior therapies. IMMU-132, also known as sacitizumab govitecan, consists of a monoclonal antibody targeting Trop-1, a protein found on cancer cells, linked to a metabolite of the common chemotherapy irinotecan. When injected into patients, the antibody portion of IMMU-132 is supposed to find and attach to Trop-1 receptors on the outside of cancer. The chemotherapy payload is then delivered inside the cancer cell, killing it. Immunomedics enrolled 80 women with metastatic, triple-negative breast cancer as part of a larger phase I/II clinical trial of additional c[...]

Ziopharm Subpar Cancer Therapies Can't Support Lofty Market Valuation

Fri, 20 May 2016 11:00 GMT

Ziopharm Oncology and Oncosec Medical  are developing similar but competing gene therapies to deliver interleukin-12, a protein which stimulates the immune system to kill cancer cells, directly into tumors. Ziopharm's current enterprise value is almost $800 million, while Oncosec has a negative enterprise value of $2 million. But guess which company has better clinical data, to date? Oncosec. Ziopharm is also developing other anti-cancer therapies using engineered T cells, commonly referred to as CAR-T. Does this research justify Ziopharm's valuation? No, because Ziopharm's CAR-T pipeline is inferior and straggling behind competitors like Novartis , Kite Pharma and Juno Therapeutics . All three companies are expected to secure regulatory approvals and reach the commercial market with their CAR-T products years ahead of Ziopharm. Ziopharm is its own mini biotech bubble, held aloft unjustifiably because of its interlocking relationship with Intrexon , the synthetic biology company majority owned by R.J. Kirk, the deal-making health care billionaire. If tracking stocks were still in vogue like they were in the late 1990s, Ziopharm would be the biotech tracking stock of Intrexon. Ziopharm is dependent on Intrexon scientifically and financially. Ziopharm's interleukin-12 gene therapy technology was licensed from Intrexon. Kirk is a major investor in Ziopharm and helps the company raise additional money. Ziopharm acquired its CAR-T platform in partnership with Intrexon. The Intrexon relationship has been beneficial to Ziopharm so far because Kirk spins a good story about using synthetic biology to invent new drugs to fight cancer. Ziopharm owes a lot of its $800 million enterprise value to Kirk's ability to woo investors. But the Kirk-Intrexon cancer-fighting story doesn't hold up to scrutiny when you examine Ziopharm's clinical data compared to its competitors like the much smaller Oncosec. Ziopharm's lead anti-cancer gene therapy Ad-RTS-hIL-12 uses a virus encoded with DNA to express interleukin-12, or IL-12, a powerful protein known to stimulate T cells in the immune system to kill cancer cells. This virus is injected directly into a patient's tumor. Once there, the gene and the production of IL-12 are controlled using an oral drug, veledimex. By raising or lowering the veledimex dose swallowed by patients, Ziopharm says it can control the production of IL-12 to optimize the killing of cancer cells in both injected and non-injected tumors, while also limiting the dangerous side effects (sometimes fatal) caused when IL-12 is administered systemically. Melanoma is known to be a cancer most susceptible to immune-stimulating drugs, so this is where Intrexon first focused the development of Ad-RTS-hIL-12 + veledimex. Ziopharm took over that work when it licensed the gene therapy from Intrexon in January 2011. Last September, Ziopharm and Intrexon presented results from a phase II study of Ad-RTS-hIL-12 + veledimex in melanoma patients at a cancer immunotherapy conference in New York City. In a press release, Ziopharm described the results this way: Ad-RTS-hIL-12 and veledimex in patients with melanoma was found to increase in the immune cytokine IL-12 and downstream cytokines, IFNg, IP-10 and IL-10, resulting in a significant increase in tumor infiltrating lymphocytes both locally, in injected lesions, and systemically, in non-injected lesions. The actual poster presented at the meeting tells a much different, more negative story: Ad-RTS-hIL-12 and veledimex was almost completely ineffective at shrinking melanoma tumors in patients enrolled in the study. Ziopharm and Intrexon enrolled 26 melanoma patients into the study. All were treated with Ad-RTS-hIL-12 and veledimex. Inexplicably, seven patients were omitted from the efficacy analysis. Of the remaining 19 patients, only two patients achieved a partial response following treatment with Ad-RTS-hIL-12 and veledimex. Two melanoma patients with objective tumor shrinkage out of 19 is a 10% partial respon[...]

NewLink Pancreatic Cancer Vaccine Fails, May Even Harm Patients

Mon, 09 May 2016 20:53 GMT

An experimental cancer vaccine developed by NewLink Genetics failed to prolong survival in patients with pancreatic cancer compared to a standard therapy, according to results from a phase III study disclosed Monday. NewLink shares were halted at $16.50 ahead of the trial results being announced. When trading re-opened, NewLink shares fell 36% to $10.60. The NewLink vaccine, known as algenpantucel-L, was designed to stimulate a patient's immune system to recognize and kill cancer cells. It did no such thing. Pancreatic cancer patients treated with algenpantucel-L lived for a median of 27.3 months in NewLink's phase III trial compared to median survival of 30.4 months for patients treated with standard therapy. The three-month median survival difference went in the wrong direction -- suggesting patients might have been harmed by treatment with algenpantucel-L. NewLink's statistical plan prohibited independent data monitors from performing an interim futility analysis on algenpantucel-L. Had one been conducted last May during a pre-scheduled safety check, the study might have been terminated early. Newlink has never explained why it didn't allow independent data monitors to conduct a futility analysis of the algenpantucel-L phase III study, but the absence of one has been profitable for NewLink CEO Charles Link Jr. He sold approximately $9.5 million in company stock since last May, according to SEC filings. Now that NewLink's therapeutic cancer vaccine platform looks to be a zero, the company's dwindling valuation relies even more heavily on its so-called IDO inhibitors -- another form of cancer immunotherapy -- in early stages of clinical development. The field of companies developing cancer immunotherapies is competitive and crowded. NewLink shares have lost 70% of their value since hitting an all-time high of $57 last April.

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Vertex Forecasts Billion-Dollar Sales for Cystic Fibrosis Drug in '16

Wed, 27 Apr 2016 21:03 GMT

Vertex Pharmaceuticals on Wednesday tried to soothe investor fears about the commercial ramp of its cystic fibrosis drug franchise by providing Orkambi sales guidance for the first time since the drug launched last summer. The company also raised slightly the sales guidance for its other cystic fibrosis drug Kalydeco. Vertex forecast 2016 Orkambi sales in the range of $1 billion to $1.1 billion, which is on the lower end of investor expectations. The company said approximately 15% of cystic fibrosis patients who start on Orkambi discontinue after three months. Over time, Vertex believes the Orkambi patient persistence rate will stabilize at 70-80%. The drug's compliance rate, meaning the number of prescribed pills actually taken by patients each month, will also stabilize at 70-80%, Vertex said. The clinical trials upon which Orkambi was approved demonstrated a modest improvement in lung function, but the drug also causes chest tightness, which can lead some patients, particularly those with poor lung function, to discontinue treatment. For similar reasons, cystic fibrosis patients prescribed Orkambi may not take all their doses. Orkambi is a particularly important product for Vertex because revenue is expected to help deliver sustainable profits after years of losses. But the drug's commercial launch has been slower than investors had expected, leading to multiple downward revisions in sales estimates. Uncertainty over how fast Orkambi sales could grow has hurt Vertex's stock price, which is down 33% this year. In the first quarter, Vertex reported Kalydeco sales of $171 million and Orkambi sales of $223 million, the latter weaker than the Wall Street consensus. On an adjusted basis, Vertex reported net income of $22.4 million, or 9 cents per share in the first quarter, compared to a net loss of $148.4 million, or 62 cents per share, one year ago. Vertex shares closed Wednesday at $84.50 and were down 1% to $83.40 in the after-hours trading session.

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Sarepta Bolstered by Doctor Letter Urging FDA Approval of DMD Drug

Mon, 21 Mar 2016 17:07 GMT

Thirty-six doctors, all experts in Duchenne muscular dystrophy, or DMD, have written a letter to the U.S. Food and Drug Administration urging the agency to approve Sarepta Therapeutics' experimental therapy eteplirsen. The lobbying effort seeking to bolster support for eteplirsen and Sarepta -- and counter previously released FDA criticism of the drug -- is ramping up as an advisory committee meeting on April 25 nears. Last month, 109 members of Congress sent their own letter to the FDA urging the agency to accelerate the approval of a DMD drug, without mentioning eteplirsen by name. Sarepta shares rose 16% to $20.92 Monday after the scientific letter was released publicly. The letter was dated Feb. 24 and was addressed to Billy Dunn, the director of the FDA's neurology division in charge of the eteplirsen clinical review. "We suggest that the most scientifically robust way forward and the most ethical choice for the Duchenne community is in the context of an accelerated approval followed by a confirmatory trial," wrote Dr. M. Carrie Miceli and Dr. Stanley Nelson, co-director of the Center for Muscular Dystrophy at UCLA, referring to eteplirsen. The full letter can be downloaded and read here. In the letter, Miceli and Nelson rebut some of the criticism leveled against the eteplirsen clinical data by FDA staff when the medical review of the drug was made public in January. The letter also expresses support for updated eteplirsen data submitted by Sarepta to the FDA. The FDA accepted Sarepta's supplemental eteplirsen data and extended the drug's review period by three months from February to May. As previously reported, the FDA's decision to extend the eteplirsen review period could be a signal that the new data satisfy the agency's initial concerns.

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Lilly Alzheimer's Study Change Piles Risk on Risk; Jim Cramer Comments

Wed, 16 Mar 2016 15:16 GMT

Updates from Tuesday at 3:30 p.m. ET to include comments from Jim Cramer and Jack Mohr of Actions Alert PLUS. Clinical trials of Alzheimer's drugs are always risky. Alzheimer's drug clinical trials altered midstream without the consent of regulators are, therefore, super risky. The super risky situation Eli Lilly  is where finds itself in Tuesday after disclosing a change to the primary endpoint of its most important Alzheimer's disease clinical trial involving the beta-amyloid antibody solanezumab. Lilly consulted with the U.S. Food and Drug Administration and its counterpart in Europe about the change but acknowledges regulators were not necessarily in agreement with the the company's plan. The disappointing result prompted TheStreet's Jim Cramer, manager of the Action Alerts PLUS charitable portfolio, to drop Lilly. "We believe LLY's disclosure is significant and reduces the already slim possibility of achieving regulatory approval for mild Alzheimer's," said Cramer and Research Director Jack Mohr on Wednesday. "In doing so, we believe the stock's risk/reward has been negatively skewed, with higher risk containing the upside and steepening the downside. The investment has become an increasingly speculative one, and we want to avoid what we view as an increasingly binary outcome amid diminishing clarity." Exclusive Look Inside: You see Jim Cramer on TV. Now, see where he invests his money and learn why LLY is no longer a core holding of his multi-million dollar portfolio. Learn more now. Solanezumab ("sola" for short) has already failed to demonstrate a convincing benefit for mild to moderate Alzheimer's patients in previous, large clinical trials, so investor confidence is already shaky ahead of results from the EXPEDITION3 study in mild Alzheimer's patients expected in the fourth quarter. Add the uncertainty surrounding a change to the primary endpoint of EXPEDITION3 and the 4% drop in Lilly's stock price Tuesday is understandable. As announced, the new primary endpoint of the EXPEDITION3 study will measure only the change in cognition over time between sola and placebo. The study's origin design called for co-primary endpoints measuring change to cognition and function. Under the amended trial design, function is now a secondary endpoint, Lilly said. Lilly says the change was made because of "emerging scientific evidence" which suggests cognitive decline precedes and predicts functional decline in mild Alzheimer's patients. The decision to amend the primary endpoint was not based on an early look at data from the ongoing study, the company says. But Lilly's decision to change the sola study, particularly so close to top-line results, does suggests that internally, the company lacks the confidence in the drug's ability to achieve a co-primary endpoint of cognition and function. By demoting function to a secondary endpoint, Lilly can claim victory in EXPEDITION3 if sola improves cognition only. By the way, Biogen , another Action Alerts PLUS holding, is conducting a large, phase III study of its Alzheimer's drug aducanumab, which has a primary endpoint which combines measures of cognition and function. The most important paragraph in Lilly's announcement Tuesday was this one focused on how regulators view the threshold for approving a disease-modifying Alzheimer's drug: Lilly understands that regulators globally will continue to view both cognitive and functional endpoints as necessary for clinical trials in people with mild Alzheimer's dementia, and regulatory guidance has been to include these as co-primary endpoints. Lilly is submitting the EXPEDITION3 amendment to all appropriate regulatory authorities. In other words, Lilly might declare the sola study a win on cognition alone, but regulators still view improvement in function as a requirement for approval. Lilly believes cognition is the bes[...]

Aerie Pharma Falls on Glaucoma Eye Drop Safety Concerns

Thu, 03 Mar 2016 17:37 GMT

Aerie Pharmaceuticals disclosed new details Thursday about the safety and tolerability of its experimental glaucoma eye drop Rhopressa. The data spooked investors, sending Aerie shares down 15%. Rhopressa is Aerie's lead product and will be submitted for U.S. approval in the third quarter. But the drug's concerning safety profile, including elevated rates of eye redness, corneal deposits and blurry vision, could turn off doctors and glaucoma patients who already have access to cheaper and better-tolerated eye drops. Aerie has previously announced results from two phase III studies comparing once-daily Rhopressa to twice-daily timolol, a generic glaucoma medicine. The studies demonstrated Rhopressa lowered intra-ocular eye pressure equally as well as timolol, with a "positive safety profile," according to Aerie. New data disclosed for the first time Thursday show more glaucoma patients treated with Rhopressa experienced significantly higher eye-related side effects compared to timolol-treated patients in the two phase III studies. When Rhopressa was given as a single dose, more than half of patients experienced conjunctival hyperemia, or eye redness, compared to 8% to 10% of timolol patients. More complaints of eye redness with Rhopressa occurred even though the drug is used before bedtime to minimize the impact of the side effect. In addition, 9% and 5% of Rhopressa once-daily patients reported corneal deposits across the two phase III studies compared to 0.4% and 0% of the timolol patients. Blurry vision was reported by 7% and 5% of Rhopressa patients compared to 3% and 0.5% of timolol patients in the studies. The new Rhopress data are contained in a scientific poster being presented at the American Glaucoma Society annual meeting.  Arie shares are down $2.57 to $15.06 in Thursday trading. The stock has lost 38% of its value year to date. 

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Germany Slaps PTC Therapeutics With Big Drug Price Cut

Fri, 26 Feb 2016 21:17 GMT

The reimbursed price of PTC Therapeutics' rare disease drug Translarna was reduced by 65% in a ruling on Thursday by a German insurance arbitration board. If PTC Therapeutics is unable to reverse the German pricing decision, the company's ability to generate revenue in Europe, the only geography where the drug is approved, will be significantly diminished. The company's efforts to get Translarna approved in the U.S. look dim after the U.S. Food and Drug Administration issued a "refuse to file" letter on Feb. 23, essentially rejecting the drug without a full clinical review. European regulators granted conditional approval to Translarna to treat a form of Duchenne muscular dystrophy. The Translarna price-cut decision was handed down Feb. 25 in an arbitration proceeding between PTC Therapeutics and Germany's Central Federal Association of Health Insurance. A copy of the decision, filed in a German court, was obtained by TheStreet. The German arbitration panel decided that the new, lowered price for a 30-pill bottle of Translarna (125 mg dose) would be 1,312 euros, which represents a 65% reduction from the previous price of 3,822 euros. The new pricing decision is final and retroactive to December 2015, although PTC Therapeutics can try to sue the German government to overturn the ruling. PTC Therapeutics issued a statement on the adverse Translarna pricing decision, stating, "As a result, PTC is considering delisting Translarna from the German pharmacy ordering system. Under these circumstances, patients and healthcare professionals may be able to access Translarna through a reimbursed importation pathway possible under German law, thus minimizing any access issues for German patients while maintaining a sustainable price." PTC Therapeutics launched Translarna in Germany in December 2014. The drug is also sold in several other European countries but a majority of the company's sales come from Germany. In the first nine months of 2015, Translarna sales totaled $21 million. PTC Therapeutics reports fourth quarter and 2015 earnings on Monday. RBC Capital analyst Simos Simeonidis is forecasting fourth quarter and 2015 Translarna sales of $12.6 million and $33.6 million, respectively. Simeonidis expects 2016 Translarna sales to reach $90 million. If the German price cut sticks, the Street's Translarna sales numbers going forward are in for a significant downward revision. PTC Therapeutics closed Friday down 29% to $7.44.

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A Lack of News and Buzz at 'JPM16' Sinks Biotech Stocks

Mon, 11 Jan 2016 21:25 GMT

Investors navigated cramped hotel hallways and hustled between company presentations on the opening day of the J.P. Morgan Healthcare Conference on Monday while staring incredulously at their smartphones. Biotech stocks large and small were plummeting, and the painful start to 2016 was turning torturous. The largest and most important healthcare conference held in San Francisco at this time each year is supposed to generate news and excitement to get investors in the mood to buy biotech stocks. But this year, there were very few market-moving announcements.a Shire finally clinched its $32 billion acquisition of Baxalta , but the deal had been discussed for months and was therefore widely expected by investors. No other significant biotech M&A deals were announced Monday. Celgene offered 2016 revenue and earnings guidance at the conference, but it was largely in line with current consensus expectations, so investors shrugged. None of the other large-cap biotech companies provided guidance Monday.a Vertex Pharmaceuticals offered a limited and conservative sales outlook for one of its cystic fibrosis drugs, Kalydeco, while holding off on providing guidance on a second, more important drug, Orkambi.a Nearly every other company deciding to disclose 2016 sales guidance Monday told investors what they already knew. Almost across the board, company guidance was "in line" with consensus expectations.a A dull start to the J.P Morgan Healthcare conference contributed to a bad day for biotech stocks. The SPDR S&P Biotech ETF fell 6% Monday to 57.08, a level it had not visited since November 2014. In the early afternoon Monday, the XBI was down more than 8%.a The XBI, the ETF with the broadest portfolio of biotech stocks, has now lost more than 18% to start 2016.a Among large cap biotech stocks, Celgene closed down 5%. Vertex lost 6% and Alexion Pharmaceuticals fell 3%. Gilead Sciencesa managed to close flat, which should be considered a major victory.a Bad news announced Monday was punished severely. Genvec fell 61% because enrollment for clinical trial of a gene therapy for hearing loss was stopped for a patient safety check.a Bluebird Bio dropped 18% after the company said it would wait until next December to present updated results on its gene therapy clinical trials in beta-thalassemia and sickle cell disease. "Biotech stocks were on sale today," a healthcare investor remarked to me while waiting for a company presentation to begin. "The scarier thing is that prices could be marked down even lower in the coming days."

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Editas' IPO Challenge: Edit Investor Expectations as Well as Diseased Genes

Tue, 05 Jan 2016 13:27 GMT

Editas Medicine is most definitely the hottest biotech initial public offering of a very young 2016. But the challenge for the gene-editing startup will be to find public investors willing to wait the two years estimated before its therapies are ready to start human clinical trials. The Cambridge, Mass.-based company filed papers Monday night for an IPO of $100 million, although the amount to be raised will most certainly change. Editas' proposed ticker symbol is EDIT -- a near-perfect encapsulation of the company's underlying technology, which aims to treat genetic diseases with a type of molecular scissors capable of cutting out defective, disease-causing genes in patients. This gene-editing technology, known as Crispr, has shown enormous potential in the laboratory and in limited studies in mice, but has not yet been tested in humans. Editas' most advanced product is a gene-editing therapy to treat leber congenital amaurosis, a genetic disease which causes progressive blindness. But Editas is still conducting pre-clinical work and doesn't expect to begin human studies until 2017. The scientific buzz around the Crispr gene-editing technology will almost certainly generate a tremendous amount of interest from institutional investors for the Editas IPO. The challenge for the company will be to find investors with the patience and risk tolerance necessary to wait out the long clinical development timelines. Editas "was incredibly selective in terms of their current investors and I think that will continue to be the case with the IPO," a health care investment fund portfolio manager (and early Editas investor) told me via email Monday night. "I have no idea what percent of the deal will be oversubscribed, but I hope the company doesn't make the same mistake that others have made and allocate broadly to a bunch of flippers, but rather focus on tightly allocating to long-term oriented folks," he added. The big but botched Axovant Sciences' IPO is what Editas needs to avoid. Axovant leveraged a speculative frenzy for Alzheimer's drugs last June when it priced its initial offering at $15 per share. The stock doubled in value on the first day of trading but the excitement faded fast. By the end of summer, Axovant was trading at $11. Today, the stock is barely above the original IPO offering price. Editas should benefit from the star power and long-term outlook of some of its early backers. Last year, the company raised $163 million in a preferred stock offering bought by Google Ventures, Bill Gates, Fidelity Investments and venture capital firms Flagship Ventures and Polaris Partners. Editas also has an ongoing cancer therapy partnership with Juno Therapeutics . "The existing syndicate can buy all of the [Editas] IPO and then some, so they will set the price," a venture capitalist not involved with Editas told me. "If there's lots of demand above that, great. If not, the deal will still price well." But Editas is also coming public at a time when biotech investing has lost some of its luster. The NYSE ARCA Biotechnology Index (BTK) closed 2015 up 10% but down 15% from the July high. A net outflow of investment dollars leaving biotech stocks contributed to the sector's weakness in the second half of last year, as did concerns about valuations and drug pricing. "It's not clear to me that Editas will see the same demand as IPOs like Juno Therapeutics or Kite Pharma . I would be surprised," said a health care fund manager to me, referring to two of the hottest biotech IPOs of 2015. "I have seen some pretty dramatic changes occurring in the crossover market which could be seen as a leading indicator. Valuations and demand have come in a lot. Fidelity and one or two others are still doing a lot but must have scaled back." If turning novel, groundbreaking science like Cri[...]

MannKind Elder Abuse Is Replacing Inept CEO With Founder

Fri, 20 Nov 2015 15:36 GMT

Finally, there's been a development in the MannKind saga which bulls and bears agree is for the best: Chief Executive Officer Hakan Edstrom resigned. Edstrom was a terribly ineffective CEO. Even handcuffed with the deeply flawed Afrezza inhaled insulin device, Edstrom still came across as a guy with no imagination. Never once did he articulate a viable strategy for lifting MannKind out of its doldrums. And so, Edstrom is gone. His replacement: Al Mann, the company's founder. Mann is 90. Granted, Mann is only taking the CEO job on an interim basis, but that doesn't diminish how desperate this executive transition makes MannKind look. It's almost cruel to see Mann being forced back into the CEO role at MannKind, but the company's near-total collapse demands it. The Afrezza commercial launch has been terrible, and it's not getting any better. MannKind's marketing partner, Sanofi , is signaling to everyone that it has run out of patience and is nearing a decision to exit. MannKind's balance sheet remains in poor shape even with the recent, small infusion of new money. The window of opportunity to develop the Technosphere technology underlying Afrezza into new products has closed. Mann will be more entertaining than Edstrom on investor conference calls, but he won't be any more effective. MannKind, like its founder, is in its twilight.

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BioMarin Duchenne Drug Receives Harsh Review From FDA

Fri, 20 Nov 2015 13:33 GMT

The U.S. Food and Drug Administration is highly critical of BioMarin Pharmaceuticals'  drisapersen, concluding that efficacy data from three clinical trials "does not reach the level of substantial evidence" necessary for approval. The FDA's review of drisapersen was posted Friday morning to the agency's Web site ahead of Tuesday's advisory committee meeting. BioMarin is seeking FDA approval for drisapersen to treat Duchenne muscular dystrophy, a rare, genetic muscle-wasting disease. BioMarin shares are down 7% to $95.70 in premarket Friday trading. Here's how the FDA review team summed up the efficacy data from three clinical trials submitted by BioMarin to support drisapersen's approval: "This review concludes that, while there may be some evidence suggestive of efficacy of drisapersen, the evidence is inconsistent and in some cases contradictory, and does not reach the level of substantial evidence." In addition to raising serious questions about drisapersen's efficacy, the FDA reviewers also have concerns about the drug's safety "Even in the context of an invariably disabling and fatal disease such as DMD, the safety profile of drisapersen is concerning ... Severe toxicity across many organ systems was encountered in the nonclinical studies, and appeared to predict a number of the adverse events that were subsequently observed in the clinical studies." FDA drug reviews are known to be harsh, so the concerns raised about drisapersen do not necessarily spell doom for BioMarin at Tuesday's advisory panel meeting. The experts on the panel will be weighing drisapersen's efficacy and safety data in the context of Duchenne being a fatal disease with no currently approved treatments. The panel might also be swayed by two hours of testimony from DMD patients, their families and advocates -- most, if not all, will be urging the FDA to approve drisapersen. Interestingly, the voting questions posed to the panel Tuesday by the FDA do not specifically ask for a yes or no vote on a recommendation for approval. The FDA wants the panel to address each of the three clinical trials individually and assess if they add, substract or have no impact on the overall strength of evidence necessary for approval. Sarepta Therapeutics is developing a competing Duchenne drug, eteplirsen, which will undergo its own FDA advisory panel review in January. Shares of Sarepta are up 28% to $33.43 in reaction to the negative FDA review of drisapersen.

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Juno, Kite, Novartis Yelp 'Oh Merde!' at Cellectis Off-the-Shelf CAR-T Data

Thu, 05 Nov 2015 16:21 GMT

The buzzworthy but still experimental blood-cancer therapies known as CAR-T being developed by Juno Therapeutics , Kite Pharma and Novartis are all personalized for individual patients. They're complicated, time consuming and expensive to manufacture. And now, possibly, obsolete. On Thursday, investors got the first peek at patient data from an off-the-shelf CAR-T from French drug company Cellectis . The results are impressive but super preliminary. An 11-month-old girl with a form of treatment-resistant leukemia was put into remission by doctors using Cellectis' "universal" CAR-T, known as UCART19, under an emergency, compassionate-use protocol. These are data from a single patient, so it's not much more than an anecdote. The follow-up is also so short that it's impossible to say if the remission induced by Cellectis' off-the-shelf CAR-T will be durable. Still, the new data, anecdote or whatever you want to call it, is intriguing because it provides a possible answer to one of the major concerns with cellular cancer therapies: How do you turn transformative science into a viable business? Next year, Novartis, Juno and Kite Pharma are all expected to seek regulatory approvals for their first CAR-Ts in relatively rare forms of blood cancer. If approved, they will face the challenge of producing these individualized therapies for each patient who needs them. The price of the CAR-T therapies and their manufacturing costs will be closely scrutinized to see if the businesses can scale and be profitable. Dendreon tried something similar with a personalized prostate cancer vaccine but ended in bankruptcy. Valeant Pharmaceuticals a (Yes, that Valeant!) later bought Dendreon's assets in a fire sale. Some of the executives responsible for the Dendreon failure now run Juno. It's way too early to declare Cellectis' technology the thing that kills Juno, Kite and Novartis' therapies before they even get off the ground. But you can bet that all three companies are probably looking over their shoulder just a little bit. The Cellectis UCART19 data can be found in this research abstract released today for the American Society of Hematology annual meeting.

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Zafgen Discloses Patient Death, Cause Unknown, in Obesity Drug Study.

Wed, 14 Oct 2015 16:07 GMT

Update from 7:09 am EST: Zafgen finally disclosed substantive information. Here is the company's statement: "Zafgen recently learned of a patient death which occurred in the Company's ongoing double-blind, randomized, placebo-controlled Phase 3 bestPWS study of beloranib in Prader-Willi Syndrome, a rare genetic disorder with a high rate of mortality linked to obesity and its co-morbidities.a The cause of death remains unknown at this time. aAccording to normal practice, the event was reported to the U.S. Food and Drug Administration, at which point the Agency initiated a discussion with the Company.a The Company is working with the Agency to expedite a review and understanding of this event, and to determine implications of the event on the conduct of the trial, and anticipates providing an update as its discussions with the Agency progress.a The thoughts of the Company are with the family of the patient at this time.a Zafgen remains committed to ensuring the safety of all patients enrolled in its studies." BOSTON (TheStreet) -- Zafgen afinally reacted to the two-day, 54% plunge in the company's stock price. On Tuesday night, Zafgen CFO Patty Allen sent the following email to analysts, some shareholders and reporters: We have received a number of inquiries from the investment community. As you are aware, we do not comment on share price movement or market speculation. We appreciate the concerns of our stakeholders. Zafgen remains committed to developing novel therapies to improve the health and well-being of patients affected by obesity and complex metabolic disorders. Wow. Not helpful at all. There is something very wrong at Zafgen. No rational company allows its shareholders to suffer extreme losses because of a "no comment" policy on market speculation. If the reasons for Zafgen cancelling two investor meetings this week were benign, the company would have said so quickly and this trainwreck would have been avoided. Some have speculated Zafgen's undisclosed "news" might be positive, but again, if that were so, the company would have stepped in to stop the extreme losses in its stock price. Zafgen shares closed Tuesday at $15.75, a loss of 29% on top of the 35% slide Monday. More than $500 million in shareholder value has been wiped out. Zafgen might be unable to tell investors what's happening inside the company because it hasn't collected all the information it needs. But even if this is true, allowing the stock to trade in the panic-stricken interregnum is insane. Zafgen should halt trading in its stock until it can communicate with shareholders. I've been writing about biotech stocks for almost 15 years and I can't remember a situation like Zafgen's. In the absence of information, investors will speculate on what's going on inside the company. Investors are assuming the meetings were called off because something has possibly gone wrong with Zafgen's lead drug beloranib. A phase III study of beloranib is underway with initial results expected in the first quarter of next year. Beloranib is an obesity drug which works by altering the way the body metabolizes fat. The initial phase III study is targeted at patients with Prader-Willi Syndrome, a rare disorder which causes life-threatening overeating.

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BioMarin Secures FDA Panel Date for Duchenne Drug but Where is Sarepta? (Update)

Wed, 14 Oct 2015 15:47 GMT

Update from 10 am EST: The FDA has listed Jan. 22, 2016 as a tentative date for a Sarepta FDA advisory panel, according to theFDA web site. BOSTON (TheStreet) -- There is yet another twist in the crazy Duchenne muscular disease drug development story. On Wednesday morning, the U.S. Food and Drug Administration confirmed Nov. 24 as the date for an advisory committee meeting to review BioMarin Pharmaceuticals' Duchenne drug drisapersen. Notice of the meeting was posted in the Federal Register. Where is the concurrent FDA advisory panel for Sarepta Therapeutics and its Duchenne drug eteplirsen? Everyone expected FDA to schedule back-to-back panels on Nov. 23 and 24, but there is no discussion of a Sarepta meeting in Wednesday's Federal Register. Strange. Sarepta shares wereadown 8% to $30.42 Wednesday on the omission. Before investors panic, remember, FDA already instructed Sarepta to prepare for an advisory panel to discuss eteplirsen. It's entirely possible Thursday's Federal Register will communicate details of the Nov. 23 FDA panel date for Sarepta. There is precedent. In 2012, FDA held back-to-back advisory panels to review competing cholesterol-lowering drugs from Aegerion Pharmaceuticals and Genzyme. Advance details about the two panels were posted in Federal Registers on consecutive days. One knock on this theory: In the case of Aegerion and Genzyme, the separate Federal Register postings mirrored the order of the FDA advisory panels. That would not true in for Biomarin and Sarepta, since Biomarin's panel is Nov. 24, presumably the second of the two-day meeting.a It's also entirely possible the FDA schedules an eteplirsen review at a later date. Sarepta filed its new drug application for eteplirsen two months after BioMarin, so perhaps FDA didn't have time to prepare for a back-to-back advisory panels. That's just speculation, of course. I reached out to Sarepta for comment but have not heard back yet. The FDA is holding the BioMarin drisapersen panel on its D.C.-area campus, so presumably, the meeting will be webcast for free. The FDA has also allotted two hours for public comments during the meeting, twice as much time as usually given. BioMarin shares were up 4% to $102.98. The Duchenne drug development story is never boring.

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Advaxis Cancer Immunotherapy on Hold Due to Patient Death

Tue, 06 Oct 2015 21:07 GMT

PRINCETON, N.J. ( TheStreet) -- The U.S. Food and Drug Administration placed a halt on clinical trials involving a cancer immunotherapy from Advaxis because of concerns a bacteria used to deliver the drug may have contributed to a patient death. Advaxis believes the patient died due to the progression of cervical cancer, but FDA is asking for additional information to confirm the company's experimental therapy, axalimogene filolisbac, played no role. Until the FDA's concerns are resolved, four clinical trials involving Advaxis' axalimogene filolisbac are on clinical hold. Shares of Advaxis fell 25% to $7.63 in after-hours trading on news of the clinical trials halt. The bad news couldn't come at a worse time as investors are already in a sour mood over a prolonged and painful retrenchment in biotech stocks. Advaxis' cancer immunotherapy technology uses an engineered form of listeria, a common bacteria, to trick the immune system into viewing tumors as bacterial infections requiring elimination. Advaxis is developing three immunotherapy products which pair listeria with different cancer targets -- human papillomavirus (HPV), prostate-specific antigen (PSA) and HER-2. Tuesday's safety issue cropped up with the HPV-targeted therapy. The cervical cancer patient was last treated with axalimogene filolisbac in early 2013. In July 2015, the patient was hospitalized and tests discovered the presence of the Advaxis-engineered listeria bacteria in her bloodstream. The patient died in August. The listeria bacteria used to make axalimogene filolisbac is modified to prevent harmful infection. But the presence of the Advaxis listeria in the patient's bloodstream two years after treatment with axalimogene filolisbac caused enough alarm bells at the FDA to warrant a closer look. Advaxis was informed by FDA about the clinical hold last Thursday but didn't disclose the news to investors until Tuesday night. On a conference call Tuesday night, Advaxis called the case an isolated incident. An investigation into the patient's death has not found any evidence that the listeria played a harmful role. The company has already provided FDA with the information requested. Advaxis has ongoing cancer immunotherapy collaborations with the Medimmune arm of AstraZeneca and Merck .

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Drug Pricing Fears Erase Biotech Stock Gains for the Year

Mon, 28 Sep 2015 17:33 GMT

Updated with new information at 1:33 pm EDT. NEW YORK (TheStreet) -- The bloodletting in biotech stocks continued Monday with aggressive selling erasing the year's gains in a key sector exchange-traded fund. Another closely-followed ETF is barely hanging on to its outperformance. Fears that the next president or Congress might push through legislation to rein in ever-escalating drug prices is the immediate trigger for the biotech sell off. But the sector has been on shaky ground since this summer because of lofty (some say over-inflated) stock valuations and troubles in the global markets. The latest confidence shaker came mid-day Monday when Democrats in Congress sent a letter to Valeant Pharmaceuticals requesting CEO Michael Pearson's appearance at a hearing investigating drug price increases. Valeant shares have lost 12% to $175.46. At midday, the iShares Nasdaq Biotechnology ETF was losing 7%. The fund is now in the red for 2015 and is down 27% from its July peak. Likewise, the SPDR S&P Biotech ETF  is off 8% Monday and is just barely hanging on to a tiny gain for the year. From its July peak, the XBI is down 31%. Biotech analysts tried to calm investors' jangled nerves Monday morning with reams of research notes and conference calls arguing the fundamental strengths of the biotech sector remain intact and the odds of politicians doing anything to regulate drug prices remained low. Generalist investors piling into biotech stocks seeking growth and outsized returns have kept the biotech bull market run alive for several years. But non-stop headlines and political posturing about drug-price controls raises the risk of a generalist flight from the biotech sector, says J.P. Morgan biotech analyst Cory Kasimov. "Generalists are more concerned that these headlines will keep a lid on broader sector performance," Kasimov wrote in a Monday research note. "As a result, we also hear from some specialists concerned not about pricing, but rather that generalists will look elsewhere to invest for the balance of 2015 and into 2016." So far, bio-pharma executives and their representative lobbying groups in Washington, D.C. have done little to staunch fears about the implementation of drug-price controls, other than to slam Turing Pharmaceuticals CEO Martin Shkreli for raising the price of an obscure infectious disease drug by 5,000%. On Friday, Biogen CEO George Scangos said Turing's business strategy was a "perversion of the system" and criticized Shkreli's actions as "arrogant" and "naive" in an interview with Bloomberg. But Scangos' own company raises the price of its decades-old multiple sclerosis drug Avonex every year, sometimes twice a year. Avonex now costs more than $60,000 per year, up from less than $10,000 per year when it was first approved in 1996. Newer, more effective MS drugs have reached the market recently, but Biogen and its competitors continue to raise the price of their older MS drugs in lockstep with each other. In addition to running Biogen, Scangos is about to take over the chairmanship of the pharma industry's main lobbying group, the Pharmaceutical Research and Manufacturers of America. In recent years, PhRMA and the biotech industry group BIO have worked to prevent Medicare from being granted the power to negotiate for drug-price discounts. The lobbying groups have also sought to slow down the development of generic versions of biologic drugs. Click to view a price quote on VRX. Click to research the Drugs industry.[...]

Clinton Drug Plan Cuts Market Exclusivity, Sets Mandatory R&D Spending

Tue, 22 Sep 2015 11:31 GMT

NEW YORK (TheStreet) -- For biotech companies and their investors, the scariest part of Hillary Clinton's multi-part plan to rein in the escalating cost of prescription drugs is a proposal to reduce the period of sales exclusivity for biotechnology drugs to seven years from 12. If Clinton's proposal, announced Tuesday, is implemented,abiotech companies would have less time to generate big profits from their expensive biologic and specialty drugs before other companies could enter the market with cheaper copies, known as biosimilars. Clinton, seeking the Democratic nomination for president, also wants to force drugmakers to spend a set portion of their revenue on research and development, or risk losing federal grants or tax credits. Biotech companies already spend a generous portion of their revenue on R&D, so this proposal isn't likely to impact the sector all that much. The R&D requirement could have a larger, negative effect onaspecialty pharma companies like Valeant Pharmaceuticals , which eschew new drug development but grow instead through acquisitions and raising the prices of older drugs. A smaller but similar company, Turing Pharma, run by a former hedge fund manager Martin Shkreli, was theatarget of intense criticism Monday because of a decision to buy an older infectious disease drug and raise its price from $13.50 a pill to $750 a pill. A tweet from Clinton on Monday calling Turing's plan "outrageous" and promising to rein in high drug prices sent biotech and drug stocks into freefall. Both the iShares Nasdaq Biotechnology ETF and the SPDR S&P Biotech ETF closed Monday down 5%. Now that the details of Clinton's drug-pricing plan are public, investors mightaview Monday's biotech stockafreefall as an overreaction. There's little in Clinton's proposals that hasn't been floated before by her election-year rival Bernie Sanders or in previous attempts to legislate some control over prescription drug prices. Clinton wants to grant Medicare the power to negotiate drug discounts just like private insurance companies can do already. But even this proposal has been pushed by Democrats for years but never implemented due to Republican opposition. Circling back to Monday's brouhaha over Turing Pharma, its CEO aShkreli and the 5,000% price hike for the infectious disease drug Daraprim.aA biotech CEO (he asked I not share his name) had this to say about Shkreli: Please make the point that Turing is NOT representative of our industry. A CEO that maligns other CEOs, journalists and anyone who opposes him in our industry is a disappointment. What he is doing does not even come close to what our industry is about. He is not a genius, a leader or a visionary. It's disgusting to see what he is doing. There's a desire to differentiate Shkreli and his business strategy at Turing from the biotech industry as a whole. The CEO quoted above believes the biotech industry works hard to develop drugs to improve the lives of patients (and at prices that can be justified). He doesn't want to be tarred with the same profiteering brush used to vilify Shkreli. I understand his desire to remain anonymous, but his argument would be stronger if he identified himself. I reached out to a few other biotech companies and CEOs for comment Monday. No one wanted to talk about Shkreli, even though they were as frustrated as the CEO above. I asked BIO, the biotech industry trade group in Washington, D.C., if it agreed with Shkreli's justification for increasing the price of Daraprim by 5,000%. I also wanted to know if Turing's decision would help or hurt the biotec[...]

Biotech Stock Mailbag: Bristol, Exelixis, Lion Bio, GW Pharma

Fri, 18 Sep 2015 10:11 GMT

BOSTON (TheStreet) -- Thank you in advance for reading the latest Biotech Stock Mailbag. This week, I tackle questions about cancer drugs from Bristol-Myers Squibb , Exelixis and Lion Biotechnologies . Also covered: GW Pharma , Rockwell Medical  and Genfit .  @megtirrell @adamfeuerstein Today's Opdivo news makes $BMY that more valuable. Big potential here. — Jared (@JDR1024) September 16, 2015 Earlier this week, Bristol-Myers Squibb announced the U.S. Food and Drug Administration granted Breakthrough Therapy Designation to Opdivo for the treatment of advanced renal cell (kidney) cancer. This is certainly good news for Bristol, but not much a surprise given that a phase III study in previously treated kidney cancer was stopped early in July following an interim analysis demonstrating a survival benefit for Opdivo over Afinitor (everolimus), the current standard of care for second-line kidney cancer patients. Must Read: The Fed Will Raise Interest Rates -- Are You Ready? Smart Moves to Make Now Full results from this Bristol study, known as Checkmate-025, are being presented on Sept. 26 at the 2015 European Cancer Congress. Bristol has already announced plans to submit Opdivo for approval in second-line kidney cancer this year. If approved, Opdivo will be the first checkpoint inhibitor therapy for kidney cancer patients. Opdivo is already approved for melanoma and non-small cell lung cancer. Novartis secured U.S. approval for Afinitor in second-line kidney cancer based on a study demonstrating a 67% reduction in the risk of disease progression or death compared to placebo. At the median, Afinitor progression-free survival (PFS) was 4.9 months compared to 1.9 months for placebo. Keep these data in mind for the Opdivo Checkmate-025 data presentation next week. At next week's European Cancer Congress, investors will also compare the Opdivo kidney cancer data against a presentation of results from Exelixis' phase III "METEOR" study of Cometriq. In July, Exelixis announced that Cometriq reduced the risk of disease progression or death by 42% compared to Afinitor in second-line kidney cancer patients. Cometriq also demonstrated a non-significant 33% reduction in the risk of death compared to Afinitor (with survival data still maturing and subject to change.) Full details from the METEOR study are being presented on Sept. 26. Exelixis plans to seek approval for Cometriq in second-line kidney cancer early next year, setting up a potential for doctors to have two new treatment options. Investors don't often get the opportunity to compare two large studies of different cancer drugs in the same patient population, especially when the the same drug is used as the comparator in both studies. The Opdivo and Cometriq presentations next week should generate a good deal of attention. @adamfeuerstein maybe i missed it but surprised I didn't see you laugh at some almost statistically significant results out of $GWPH — Walt Hudson (@WaltHudson) September 16, 2015   My general view of "proof of concept" clinical trials is that the promoted results (usually via an optimistically penned press release) are like the people at Lake Wobegon -- strong, good looking and above average. Drug companies design proof-of-concept studies to find something positive about whatever drug is being investigated. This is not to say all proof-of-concept studies are B.S. You just shouldn't get too excited about them. Unsurprisingly, GW Pharma said this week that cannabidiol (CBD) demonstrated su[...]

Intra-Cellular Surges on Optimistic Outlook for Schizophrenia Drug

Wed, 16 Sep 2015 15:10 GMT

NEW YORK (TheStreet) -- Schizophrenia patients treated with an experimental drug from Intra-Cellular Therapies reported fewer antipsychotic symptoms compared to a placebo in a large, phase III study, the company said Wednesday. Intra-Cellular shares climbed 73% to $45.36, an all-time high, on the positive results from the study and the belief that the drug, ITI-007, could generate billions of dollars in schizophrenia sales if eventually approved. Before that happens, ITI-007 must also score a victory in a second, more challenging phase III study comparing the drug against a currently approved antipsychotic. Results from the next ITI-007 study are expected next year. According to Wednesday's study results, four weeks of treatment with a 60 mg dose of ITI-007 demonstrated a statistically significant improvement in antipsychotic symptoms compared to placebo, scored using the Positive and Negative Syndrome Scale, or PANSS. The effect size of ITI-007 over placebo was smaller in the phase III study than what was seen in a previous phase II study. A 40 mg dose of ITI-007 was also tested but did not beat placebo. The 60 mg dose of ITI-007 also achieved key secondary measures of efficacy against placebo in the phase III study. Must Read: How to Invest in Dividend Stocks The most common side effects attributable to ITI-007 were drowsiness, fatigue and mild sedation. Some currently approved antipsychotics used to treat schizophrenia can cause weight gain or high cholesterol. Intra-Cellular said there was no difference in weight gain or metabolic measures between ITI-007 and placebo observed in the study. "Today's data does not suggest ITI-007 will be a strong drug. Nevertheless, the p value suggests this effect is consistent and real, as do the secondary efficacy endpoints," Cowen analyst Ritu Baral wrote in a research note Wednesday. She continued, "Overall, today's data set supports the promising profile our key opinion leader consultants suspected this drug would have.... ITI-007 might be a less strong anti-psychotic that could be best for use in disease control maintenance, especially because of its weight/metabolic neutrality." Intra-Cellular is running a second phase III study in schizophrenia comparing ITI-007 to placebo and risperidone, a currently approved antipsychotic. The primary efficacy endpoint of the ongoing study, like the first, compares ITI-007 against placebo. From a commercial perspective, seeing how the efficacy and safety of ITI-007 compares against risperidone will be equally important. Schizophrenia patients tend to cycle through multiple drugs, so even inferior products, like Pfizer's Geodon, can generate substantial sales. However, Vanda Pharmaceuticals' schizophrenia drug Fanapt is not seen as different enough from cheaper alternatives and has struggled commercially.

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Tetraphase Executive Profits Hours Before Stock Plummets

Mon, 14 Sep 2015 10:27 GMT

WATERTOWN, Mass. (TheStreet) -- Craig Thompson's stock sale was very well timed. Miraculous might be the best way to describe it. On Sept. 8, Thompson, chief operating officer at the biotech firm Tetraphase Pharmaceuticals , exercised 10,000 company stock options priced at $14.45. He immediately sold the Tetraphase shares at an average price of $44.08, according to an SEC filing. The insider sale netted Thompson just under $300,000. On the same day, just after the market closed and hours after Thompson's stock sale, Tetraphase announced negative results from a pivotal study of its experimental antibiotic eravacycline. Within minutes, the bad news sent Tetraphase's stock price plummeting 70% from its $44.78 close. The next day, Sept. 9, Tetraphase opened for trading at $9.49 per share, erasing $1.3 billion in market value. Tetraphase shareholders lost a lot of money. Thompson, an executive at the company since February 2014, profited. Must Read: What Happens When the Fed Hikes Interest Rates? Thompson's insider sale was fortuitous but legal because it was conducted under a 10b5-1 trading plan. 10b5-1 plans allow corporate executives and other insiders to schedule sales of company stock in advance, thereby insulating themselves from the risk of insider trading. 10b5-1 plans can be set up to trigger stock sales on a specific pre-specified date or when a stock hits a certain price. The 10b5-1 plan covering Thompson's Tetraphase stock holdings was established on March 13, 2015, according to an SEC filing. The use of 10b5-1 trading plans is a common Wall Street practice, but the timing of Thompson's insider sale of Tetraphase stock -- just hours before the company's market value crashed -- still raises questions about ethics, fairness and whether these plans are truly independent. Tetraphase was in possession of the eravacycline phase III study results "at the end of last week," meaning Sept. 3 or 4, according to CEO Guy McDonald, speaking on a conference call held after the news of the failed study was disseminated on the night of Sept. 8. The eravacycline study results were material, stock-moving information known to executives inside the company days before the information was released to the public. Yet Tetraphase still maintained an open trading window for its executives, through which Thompson exercised options and sold stock. "As stated in the Form 4, Mr. Thompson sold shares under a 10b5-1 Plan which he put in place in March 2015, prior to IGNITE2 completing patient enrollment in May 2015," said Tetraphase spokesman Mike Beyer in response to a question about the timing and circumstances of Thompson's stock sale. IGNITE2 was the name given to the eravacycline phase III study. But ordinary investors also holding shares of Tetraphase but not privy to the details of the eravacycline study results before Sept. 8 were not as lucky as Thompson. 10b5-1 plans are supposed to inure executive against allegations they use inside information to enrich themselves at the expense of ordinary shareholders. Too often, however, these plans seem to do the opposite. Earlier this month, Ariad Pharmaceuticals stock price soared on market reports about a possible takeover by pharma company Baxalta. Ariad shares fell almost as fast when the takeover chatter was debunked, but not before Ariad Pharma Chief Financial Officer Ed Fitzgerald was able to sell company stock through a 10b5-1 plan, according to an SEC filing.. The timing of Fitzgerald's stock sale -- at the a[...]

Santhera Drug Wins European Approval for Rare Blindness Disease

Wed, 09 Sep 2015 05:01 GMT

LIESTAL, Switzerland (TheStreet) -- European regulators on Wednesday approved a new drug fromaSanthera Pharmaceuticals (SANN.SW) to treat a rare, inherited disease which causes progressive vision loss and blindness. The drug, Raxone, is the first approved treatment for Leber hereditary optic neuropathy (LHON), which affects approximately 10,000 patients (mostly males) in Europe. The Raxone commercial launch will commence in the coming weeks, said Santhera CEO Thomas Meier. He declined to discuss how much the companyexpects to charge for Raxone, except to say that it will be "priced as a typical orphan disease drug." Raxone is a pill designed to stimulate mitochondria, the energy-producing organelles found inside cells. In LHON, a defect in the mitochondrial DNA causes retinal ganglion cells -- a type of nerve cells connecting the eye to the brain -- to lose energy and stop functioning. When retinal ganglion cells stop working, patients experience progressive vision loss and eventual blindness. Raxone doesn't correct the defective mitochondrial DNA underlying LHON. Instead, the drug circumvents the genetic defect and restores energy levels to the retinal ganglion cells. European regulators approved Raxone based on data from a clinical trial and an expanded access program showing the drug mitigates and reverses vision loss in LHON patients. RBC Capital analyst Simos Simeonidis forecasts $29 million in peak Raxone LHON sales based on annual pricing of $43,000. The LHON indication makes up just a fraction of RBC's Santhera valuation because the company is also developing Raxone to improve the lung function of Duchenne muscular dystrophy (DMD) patients. The progressive weakening of muscles in the chest of DMD patients leads to respiratory disease and breathing problems. Santhera hopes to submit Raxone approval filings for DMD in the U.S. and Europe by the end of the year, said Meier. Must Read:aWill This $5 Biotech Stock Go To $22 Or $1.50?

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Santhera Drug Win European Approval for Rare Blindness Disease

Tue, 08 Sep 2015 19:22 GMT

Liestal, Switzerland (TheStreet) -- European regulators approved Wednesday a new drug from Santhera Pharmaceuticals (SANN.SW) to treat a rare, inherited disease which causes progressive vision loss and blindness. The Santhera drug, Raxone, is the first approved treatment for Leber hereditary optic neuropathy (LHON), which affects approximately 10,000 patients (mostly males) in Europe. The Raxone commercial launch will commence in the coming weeks, said Santhera CEO Thomas Meier. He declined to discuss how much the company expects to charge for Raxone except to say that it will be "priced as a typical orphan disease drug." Raxone is a pill designed to stimulate mitochondria, the energy-producing organelle found inside cells. In LHON, a defect in the mitochondrial DNA causes retinal ganglion cells -- a type of nerve cell connecting the eye to the brain -- to lose energy and stop functioning. When retinal ganglion cells stop working, patients experience progressive vision loss and eventual blindness. Raxone doesn't correct the defective mitochondrial DNA underlying LHON. Instead, the drug circumvents the genetic defect and restores energy levels to the retinal ganglion cells. European regulators approved Raxone based on data from a clinical trial and an expanded access program showing the drug mitigates and reverses vision loss in LHON patients. RBC Capital analyst Simos Simeonidis forecasts $29 million in peak Raxone LHON sales based on annual pricing of $43,000. The LHON indication makes up just a fraction of RBC's Santhera valuation because the company is also developing Raxone to improve the lung function of Duchenne muscular dystrophy (DMD) patients. The progressive weakening of muscles in the chest of DMD patients leads to respiratory disease and breathing problems. Santhera hopes to submit Raxone approval filings for DMD in the U.S. and Europe by the end of the year, said Meier.

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Global Blood Valuation Gives Goldman Analyst Pause

Tue, 08 Sep 2015 16:12 GMT

SOUTH SAN FRANCISCO, Calif. (TheStreet) -- One of these sell-side analyst initiation reports issued Tuesday on Global Blood Therapeutics is not like the others. Morgan Stanley: Overweight, $67 price target.Cowen: Outperform, $80 target.Wedbush Securities: Outperform, $94 price target.Goldman Sachs: Neutral, $44 target. Global Blood shares are down 3% to $51.20 in Tuesday trading. One lukewarm Goldman analyst appears to carry more weight than three bulls from Morgan Stanley, Cowen and Wedbush. All four investment banks handled Global Blood's successful IPO in August, which makes Goldman's neutral initiation stand out. Sell-side cheerleading on behalf of their employer's banking clients has become such a standard practice that anything remotely bearish is news. Goldman isn't negative on the fundamentals supporting Global Therapeutics, which is in the early stages of developing a new drug for sickle cell disease. The bank's neutral rating and $44 price target -- 15% lower than the stock's current trading price -- is a valuation call. "We see valuation as fair given existing data and stage of development," writes Goldman analyst Terrence Flynn. Global Blood's lead drug, GBT440, has the potential to deliver $2.4 billion in peak sales, says Flynn, but the tiny amount of data from a single phase I/II study on hand only justifies a 40% probability of success at this point. New, updated results from the GBT440 study are expected in December at the American Society of Hematology (ASH) annual meeting. By comparison, Cowen's $80 price target is derived, in part, by assuming a 60% probability of success for GBT440, with the similar $2.5 billion peak sales. Must Read: Why You Shouldn't Panic About Fed Rate Hikes

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Biotech Stock Mailbag: Sarepta, Biomarin, Spark, Raptor

Thu, 03 Sep 2015 10:23 GMT

BOSTON (TheStreet) -- Welcome back to the Biotech Stock Mailbag. Tony B. writes, "I appreciate your prediction of the approval for the DMD drugs but I think you're too optimistic about Sarepta Therapeutics . I believe FDA approves Biomarin but tells Sarepta that another trial must be run. In your article, you should have provided more explanation for the risk facing Sarepta and the possibility its drug is rejected." Before I wrote my last column predicting the approvals of Duchenne muscular dystrophy drugs drisapersen and eteplirsen, I spoke to a health care investor I've known and respected for a long time. This investor agrees with Tony B. He says the FDA is more likely to approve Biomarin's drisapersen than Sarepta's eteplirsen, although at this point, he lacks the confidence to invest in either stock. So, why Biomarin over Sarepta? Because what Biomarin is seeking to do -- make a convincing argument for drisapersen's approval based on messy clinical data including a failed phase III study -- doesn't set a regulatory precedent. It's something that the FDA has done before with drugs for rare diseases and would probably be comfortable doing again. Examples include Vanda Pharma's Hetlioz and Chelsea Therapeutics' Northera. (Chelsea was later acquired by Lundbeck .) Biomarin even has prior experience with its orphan-disease drug Aldurazyme, which passed FDA muster despite study results which failed to hit statistical significance. To approve Sarepta's eteplirsen, by contrast, requires the FDA to set a regulatory precedent which the agency might regret later. The data from Sarepta's 10-patient eteplirsen study are compelling, but is that enough to justify an approval? If the FDA says yes to Sarepta, must it also say yes to any future company which seeks approval on tiny uncontrolled studies? Does giving Sarepta a pass lower regulatory standards too much? Sarepta bears argue that the FDA won't approve eteplirsen this time around for these reasons. It's easier for the FDA to approve Biomarin's drisapersen on its own. DMD patients and their families finally get a drug for which they've long lobbied, making the FDA look good. And the door is left open for Sarepta's eteplirsen to eventually reach the market if the ongoing phase III study is positive. @adamfeuerstein what explains the relative strength of $QURE compared to $ONCE? Qure + 1.2% in August, ONCE -27%! — Superwoester (@sparklybluequre) September 1, 2015     The simplistic answer is investors are nervous about the soon-to-be released outcome of Spark Therapeutics' phase III study. Recent actions taken by Spark management have exacerbated investor concerns. Uniqure will also be announcing some study results of its own this fall, but the data are early and preliminary, so not as pivotal to the future of the company. Both Spark and Uniqure are developing gene therapies for serious diseases. The recent clinical setbacks at Avalanche Biotechnologies and Celadon -- also gene therapy stocks -- put a chill in the market's love-a-thon with the emerging technology. It's a mistake to lump all gene therapy stocks together, but it would also be a confidence boost for the market to see positive results from a pivotal gene therapy study. Spark is next in line. Must Read: 13 Stocks Set for Huge Revenue Growth Even When Interest Rates Rise Spark's lead gene therapy[...]

Sage Psychiatric Drug Shows Activity Hint in Small Muscle Tremor Study

Thu, 03 Sep 2015 10:01 GMT

CAMBRIDGE, Mass. ( TheStreet) -- Sage Therapeutics announced Thursday that an injection of the experimental psychiatric drug SAGE-547 was more effective than a placebo in treating patients with essential tremors, a neurological condition which causes involuntary, rhythmic muscle shaking. Tempered optimism is probably the best way to interpret the data from the small (only 25 patients were enrolled) exploratory study. On one measure of muscle tremor reduction assessed at 12 hours, twice the number of patients treated with SAGE-547 responded compared to placebo. The difference between SAGE-547 and placebo, however, was not statistically significant. On other measures of efficacy, SAGE-547 demonstrated a benefit over placebo that was barely statistically significant. In an interview Wednesday afternoon, Sage CEO Jeff Jonas said the intention of the study from the beginning was to evaluate the feasibility of treating essential tremor patients with a drug like SAGE-547 that acts on the GABA neurotransmitter system in the brain, hoping to see clinical evidence of positive drug activity. "The data that we're reporting is as clear a signal as one can get to move a drug forward. We're excited by it," said Jonas. The signal of activity Jonas believes Sage sees with SAGE-547 in essential tremor may turn out to be real or a mirage. There's nothing definitive enough about Thursday's study results to know the true answer. Sage doesn't intend to develop SAGE-547 any further in essential tremor. Instead, the company will likely turn to a similar but oral compound, SAGE-217, which will be ready for human, healthy volunteer safety studies later this year. Sage is developing SAGE-547 as a treatment for a severe form of epilepsy known as refractory status epilepticus. A phase III study is underway. Earlier this year, Sage also reported on a small, proof-of-concept study in which an injection of SAGE-547 eliminated severe postpartum depression in four women. In the study reported Thursday, 25 patients with moderate-to-severe essential tremor were randomized to treatment with an injection of SAGE-547 or a placebo injection. Doctors and patients were blinded to the treatments. The amplitude and frequency of tremor motion was measured using an accelerometer device attached to the patients' arms. In a test of upper limb kinetic tremor (patients were asked to touch their finger to their nose), the SAGE-547 injection resulted in a statistically significant reduction in tremors compared to placebo after 12 hours. A composite assessment of various arm movements, also measured with an accelerometer, showed after 12 hours a 33% response rate for patients treated with SAGE-547 compared to 16% for placebo. The difference, however, was not statistically significant. Must Read: Bank of America's 10 Best Stock Ideas for the Third Quarter Likewise, a subjective clinician rating scale of tremor reduction trended in favor of SAGE-547 over placebo but was also not statistically significant. The highest blood levels of SAGE-547 matched the drug's peak therapeutic activity over the course of the short study. In a second stage of the study, 17 of the 25 essential tremor patients were treated with a dose of SAGE-547 twice as high as what was used in stage one. The response rate to SAGE-547 was higher although there was no placebo arm for a comparison. One [...]

The Numbers Behind Biotech's Horrible, No Good, Awful August

Tue, 01 Sep 2015 11:27 GMT

NEW YORK (TheStreet) -- August was a horrible month for biotech stocks. Excluding penny stocks, no biotech company fared worse than NantKwest , the cancer immunotherapy vanity project fronted by health care billionaire Patrick Soon-Shiong. The market's been a nasty place for Nantworks since the record-setting IPO at $25 per share in late July. The stock closed Monday at $17.12 -- a 44% loss for the month of August, according to Sorento Therapeutics , another biotech stock in which Soon-Shiong owns a large stake, fell 39% during August. Biotech's other billionaires didn't fare much better during the August doldrums, notes health care investor and biotech-Twitter commentator Brad Loncar. R.J. Kirk's Intrexon and Ziopharm fell 32% and 35% during the month, respectively. Phil Frost's Opko Health shares dropped 42%. MannKind , the eponymous biotech of Al Mann, escaped August rather easily, losing just 12%. All these stocks underperformed the benchmark iShares Nasdaq Biotechnology ETF  , which lost 11% during August. The breadth of the selling during the month was impressive. Among all U.S.-listed biotech stocks regardless of market cap, only 68 companies managed to end August with higher stock prices, while 267 biotech stocks lost ground, according to Other notable losing stocks during August: Celldex Therapeutics down 35%, OvaSciences   down 29%, Intercept Pharmaceuticals down 28% and Spark Therapeutics   down 27%. The once-hot CAR-T cancer stocks Kite Pharma and Juno Therapeutics lost 27% and 26%, respectively, in August. Gene therapy darling Bluebird Bio fell 20%.  Of course, none of this matters today, Sept. 1, the unofficial start of the rest of the year. It's good to note the dreadful sell-off in biotech stocks for the historical record, but it doesn't help investors pick winning stocks (or avoid further losing stocks) for the remainder of 2015 and beyond. If you want to be an optimist, note the IBB ETF is still outperforming the broader market for the year. Happy biotech investing! Must Read: The Biotech Bubble Debate: Solid Science vs. Sky-High Valuations

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Amicus Acquires Scioderm, Adds Drug for Rare Skin Disorder to Pipeline

Mon, 31 Aug 2015 10:01 GMT

CRANBURY, N.J. (TheStreet) -- Amicus Therapeutics is expanding its pipeline of rare-disease drugs by acquiring privately held Scioderm, the developer of a topical cream to treat patients with a genetic disorder that causes extremely fragile skin susceptible to blisters and tears from even the lightest touch. Scioderm is being purchased for $229 million in cash and Amicus stock, according to deal terms announced by the companies Monday. Amicus will pay up to $618 million in additional cash and stock to Scioderm investors if future clinical, regulatory and sales milestones are met. Acquiring Scioderm gives Amicus another late-stage orphan disease drug expected to have pivotal trial data in the first half of next year. Amicus' current lead product Galafold is under review in Europe for the treatment of Fabry disease and could be approved and launched commercially in 2016. The company plans to submit the same drug for U.S. approval before the end of the year, which also sets up the possibility of a 2016 approval and launch. For much of the past year, Amicus has been looking for first-in-class or best-in-class rare-disease drugs to bring into the company, but "there are not a lot of opportunities out there," said John Crowley, Amicus' chairman and CEO. Eventually, Crowley found the desired acquisition target close to home. He's been a director on Scioderm's board for the past two years and got to know the company and the desperately ill patients its drug is trying to help quite well. "We proposed this deal to [Scioderm] a few months ago. It was not a competitive process. We entered into an exclusive negotiating period and convinced them that we were best suited to drive them forward," said Crowley. Scioderm's lead product Zorblisa is a novel, topical cream for the treatment of epidermolysis bullosa, a rare connective tissue disorder with no approved treatment options. EB can stem from different genetic mutations and vary in severity, but all EB patients have fragile skin that blisters and tears from minor friction or trauma. Patients with severe EB suffer from blisters and lesions covering a large portion of their bodies that can lead to infection, scarring and death. Children with the disease often die as young adults. EB patients are treated today with bandaging and bathing meant to keep torn skin and wounds free from infections. Zorblisa works by inhibiting inflammatory response and promoting the growth of epithelial cells and tissues to promote wound healing, says Crowley. An estimated 30,000 to 40,000 patients are currently diagnosed with EB in major markets, Amicus said. The company believes Zorblisa, if approved, could generate sales of $1 billion or more. The current palliative treatments of bandaging, wound cleaning and pain management for EB patients cost between $10,000 and $15,000 per month. In 2012, Scioderm conducted a small study in which eight children with EB had Zorblisa cream applied to their entire bodies once per day. After three months, the treatment resulted in the closure of 85% of targeted chronic skin lesions and a 57% reduction in the body surface area of skin lesions and erosions. Based on these data, the U.S. Food and Drug Administration in April 2013 awarded Zorblisa a Breakthrough Therapy Designation for the treatment of EB. [...]

Predicting the Outcome of the BioMarin and Sarepta FDA Drug Reviews

Thu, 27 Aug 2015 16:45 GMT

Updated with more information throughout. BOSTON (TheStreet) -- The two leading drugs to treat Duchenne muscular dystrophy, drisapersen and eteplirsen, are accepted officially for priority review by the U.S. Food and Drug Administration. With the intrigue about regulatory filings out of the way, investors can now focus solely on what really matters -- the outcome of the FDA reviews and the impact on the drugs' respective sponsors BioMarin Pharmaceuticals  and Sarepta Therapeutics . I won't bury the lede any further. I believe FDA will approve drisapersen and eteplirsen. BioMarin and Sarepta both win. This doesn't mean I believe the drugs are equivalent. I'm still in the "eteplirsen is better than drisapersen" camp, although I acknowledge my Sarepta favoritism comes with a generous helping of blind faith in the relatively scant eteplirsen clinical data. Must Read: 7 Stocks Warren Buffett Is Buying in 2015 Approving both drugs is the most politically expedient decision FDA can make. The drisapersen and eteplirsen reviews are still nominally about the science, but in this case, equally about making good on promises (explicit and implied) to the families of kids with Duchenne muscular dystrophy (DMD) to approve first-of-its-kind treatments as soon as possible. The FDA is essentially approving every drug that comes before it. There are multiple reasons for why the agency has adopted a warm-hug policy on new drug reviews, but the desire for good press and bending to public pressure are certainly on the list. Put it another way, FDA just approved the female libido drug Addyi based largely on an astroturf campaign of women's sexual fairness orchestrated and paid for by the drug's sponsor. Never mind Addyi barely works and is quite possibly risky and dangerous for the women who might take it. With this disturbing regulatory precedent fresh in everyone's mind, how can FDA say no to two drugs, drisapersen and eteplirsen, aiming to improve the lives of kids with a devastating, fatal disease? The FDA can't and won't. This is not to say affirmative decisions by the FDA are a slam dunk. The respective clinical datasets for drisapersen and eteplirsen are flawed. BioMarin comes to the FDA with a large, phase III study of drisapersen which failed to demonstrate a walking benefit over placebo. The company's pitch for approval requires throwing out data on patients where drisapersen didn't work, believing it works for other patients, not worrying too much about side effects and totally overlooking the absence of mechanistic data on dystrophin production. [The absence of dystrophin, a protein necessary for muscle function, is what causes DMD.] Must Read: Stocks Survive Bungee Jump but Technicals Continue to Deteriorate Sarepta's eteplirsen data look better and durable but there's not much of it. Can FDA really trust the significant improvement in walking ability seen with 10 DMD patients in a phase II study are real? Eteplirsen's has proven to be remarkably safe to date, but again, side effects often only show up when a drug is studied in a large number of patients, which Sarepta has not yet done. [A larger phase III study is underway.] Sarepta has gathered additional data showing eteplirsen helps produce a functional form of dystrophin in treated p[...]

An Inside Look at Biotech Investor Pros Dealing With Market Meltdown

Tue, 25 Aug 2015 11:38 GMT

BOSTON (TheStreet) -- Last night, I exchanged emails with a number of investors who run dedicated health care funds. I wanted to know how they navigated Monday's market selloff. Were they selling? Buying? Did the extreme volatility in stock prices Monday force them to do anything different? Almost to a man (I didn't speak to any women, unfortunately), these professional health care investors told me they spent most of Monday pretty much sitting on their hands. Must Read: Warren Buffett's Top 10 Dividend Stocks "When it's this crazy, we actually tend to do not much," one biotech hedge fund manager told me. His fund did a little bit of buying and covered a few shorts, but otherwise, it was a slow day for the portfolio. "Can't really do anything on days like [Monday]," a portfolio manager at a large mutual fund told me. "What can you really do? Nibble at your best longs. Consider covering your most beaten down shorts or press those with a catalyst. But, also, do actual work and try to set up for the fall season," said another health care hedge-fund manager. I wish I had something more exciting to report, but I didn't hear anything substantially different from the other investors I emailed with last night. Collectively, they manage billions of dollars in investment capital, but everyone basically remained calm and ignored the volatility. Nobody panicked during the Monday morning flash crash when the iShares Nasdaq Biotechnology Index  gave up all its gains for the year and sentinel stocks like Celgene  and Gilead Sciences plunged. Granted, all the investors I contacted take a fundamental approach to investing in biotech stocks, and they tend to manage their health care books for the long term. I didn't speak with any traders or investors who have a much shorter investment horizon. If I had, the story might be a little different. To augment my relatively small sample size of investors, I also reached out to a few institutional brokers and sell-side analysts, asking them to rate the sentiment of the buyside investors they spoke with Monday. On a scale of 1 (snooze) to 10 (OMFG, We're DOOMED!), how were their investor clients feeling Monday? "Probably a 3 or 4," one sell-side analyst told me. "Certainly no panicking in biotech. People get that this sell off is all macro and has little to do with our industry. Yes, people are feeling the pain, but still like the group's fundamentals. Generalists are no different and tell us they have no where else to invest for growth." "I think the vast majority of folks saw this as a macro issue, not biotech specific, so I'd put it at a 3 to 4," said another biotech analyst. "Even hedgies I talked to were going to 'sit it out,' maybe taking down some risk but by no means bailing on the whole group. Generalists took down a lot of biotech risk after Q2 when Biogen puked it." On a day when markets worldwide were in turmoil and a lot of biotech stocks were down 5% to 6%, these investors did nothing. Boring doesn't make for a good story but it might be the wisest health care investment lesson. Must Read: 3 Charts That Will Tell You When It's Safe to Buy Stocks Again Click to view a price quote on[...]