Um... Wasn't an early eteplirsen filing supposed to be good news? I've often said stocks prices can lie, especially over the short term. To say this about the current Sarepta situation would be facile. Even bulls (hand raised) need to acknowledge the significant risks to the eteplirsen early approval thesis. Feel free to label me delusional, but I was pleased with Sarepta's July 24 announcement. It was everything I expected, including the cautious language describing the FDA's apparent uncertainty with dystrophin as an approvable endpoint. The only disappointment, minor, is the eteplirsen filing potentially not being completed until the second quarter 2014. I still expect FDA to approve eteplirsen on the current phase II data, most likely the six-minute walk benefit rather than purely on dystrophin production. (Although dystrophin data will support the approval.) On the company's earnings call Thursday, Sarepta CEO Chris Garabedian did a nice job explaining the routes FDA can use to review and approve eteplirsen, including granting full approval. If you've been following the Sarepta story closely, you knew this already. Hopefully, though, Garabedian's explanation resolved the confusion which exists. Before the eteplirsen filing and FDA review happens, GlaxoSmithKline ( GSK) and Prosensa ( RNA) will be announcing results from a phase III study of drisapersen during the fourth quarter. The companies may also release dystrophin production data from a previous drisapersen phase II study. I can conjure multiple scenarios for how Sarepta's stock price reacts to these data. Best case for Sarepta: Drisapersen data that are "meh" -- meaning efficacy and toxicity in line with the current view of the drug: Drisapersen improves walking ability but at the cost of serious side effects, including kidney toxicity and thrombocytopenia, with extended use. Worst case for Sarepta: The drisapersen data are super strong, meaning better-than-expected walking improvements with few reported side effects. If this scenario plays out, expect the Sarepta short story to switch to something like this: "With drisapersen data so strong, FDA is no longer under any pressure to approve eteplirsen early. DMD patients will be happy if just drisapersen is approved."
@adamfeuerstein Adam, what are your observations concerning $GALE?— Terrence A. Valko (@ValkoUSA) August 7, 2013We're into the segment of the current biotech bubble where old and failed stock-promotion stories get recycled. Galena Biopharma ( GALE) is one of these tired stories getting new life. In the past week, three Seeking Alpha contributors have published cringe-worthy ecomiums to Galena and its experimental breast cancer vaccine. The timing is not coincidental. With $27 million in cash and securities on hand at the end of the first quarter, Galena needs more money. The company last sold stock in December 2012, but needs to beef up the bank account to continue funding the NeuVax phase III study and hire a sales team to sell Abstral, its recently acquired cancer pain narcotic. Abstral will be a commercial flop, but Galena will waste a ton of money on it, nonetheless, because the company has nothing else to do. I've spent quite a bit of time in the past explaining why NeuVax will be a zero, too. Read the interview with Galena CEO Mark Ahn published by "Regarded Solutions" to get a taste of company-sponsored stock promotion in action. Excellent work, Claire Sojda! This chart says a lot: Galena totally left out of this summer's big move in the biotech sector: GALE data by YCharts
Speaking of stock promotion, Ken Luskin of Intrinsic Value Asset Management has been on a mission to convince every retail investor in the land to buy shares of Vical ahead of the announcement of results from a phase III study of allovectin-7, the company's skin cancer immunotherapy.
@adamfeuerstein Would love to have you do $INO article. While agree, co has been touting for a while, things have changed in last 3 yrs, no?— rockingrollingfun (@rocknrollingfun) August 6, 2013As far as I can tell, nothing has changed at Inovio Pharmaceuticals ( INO). The company is still flogging the synthetic vaccine and electroporation vaccine delivery technologies of 30 years ago. The other constant is Inovio's relentless self promotion. Two big changes over this time period: 1) Inovio's pipeline products fail or disappear, and 2) one group of disgruntled and money-losing retail investors is replaced by a new crowd of doe-eyed investors believing Inovio has turned the corner. Of course, this is never true. Rinse. Repeat.
Inovio's institutional ownership is 8 percent. Retail investors own 80% of the stock, according to CapitalIQ. And when Seeking Alpha prints irresponsible, nonsense articles like Inovio May Prove To Be Merck's Solution, speculators and momentum traders jump on board. Incredibly, Inovio fans the flames by tweeting a link to the article from its corporate Twitter account: @InovioPharma A looming partnership with Merck provides Inovio shareholders with a worthwhile catalyst. Looming Merck partnership?!?! Except there isn't one, which probably freaked out the lawyers charged with keeping Inovio out of trouble. The offending tweet was quickly taken down, replaced with this:
Inovio has deleted a previous post linking to a SeekingAlpha article. Inovio does not endorse or refute third party opinions or articles.— Inovio (@InovioPharma) July 31, 2013Perhaps some of these shenanigans could be forgiven if Inovio possessed a pipeline of therapeutic and prophylactic vaccines worth investing in. It doesn't. The company's pipeline is a mix of preclinical and early-stage vaccines lacking meaningful clinical data. Inovio's therapeutic vaccine to treat cervical dysplasia in women is the furthest along, but a phase II study has been trying to enroll 148 patients for almost two and half years. Inovio has promised data early next year, but the study isn't designed to provide any definitive answers about the vaccine's efficacy. A study of Inovio's leukemia vaccine comes back "status unknown" on ClinicalTrials.gov. Inovio's hepatitis C vaccine failed a proof-of-concept study last spring. No matter because a Hep C vaccine is obsolete already given the looming market entry of remarkably effective pills which will cure the disease in 8-12 weeks. An HIV vaccine? Seriously? A flu vaccine? Sure, maybe, but Inovio has no partner. Don't believe in the program unless and until a larger company with credibility takes interest. None have. Paul L. asks: Adam, I appreciate the heads up you provide when it comes to FDA approval decisions. It seems like we're not seeing as many of these decisions lately. Do you have an update for your calendar? I agree, the FDA drug approval cupboard has been a bit bare this summer. Not much action. Here's an updated list of significant FDA drug approval decisions through the end of the year: GlaxoSmithKline ( GSK)
Drug/indication: Dolutegravir, HIV
Approval decision date: Aug. 17
Drug/indication: Xiaflex, Peyronie's Disease
Approval decision date: Sept. 6 Delcath Systems ( DCTH)
Drug/indication: Meblez, liver tumors due to ocular melanoma
Approval decision date: Sept. 13 Antares Pharma ( ATRS)
Drug/indication: Otrexup, rheumatoid arthritis
Approval decision date: Oct. 14 Alimera Sciences ( ALIM), Psivida ( PSDV)
Drug/indication: Iluvien, diabetic macular edema
Approval decision date: Oct. 17 AMAG Pharmaceuticals ( AMAG)
Drug/indication: Feraheme, iron deficient anemia
Approval decision date: Oct. 21 Cubist Pharmaceuticals ( CBST)
Drug/indication: Entereg, post-operative constipation
Approval decision date: Oct. 21 Gilead Sciences ( GILD)
Drug/indication: Sofosbuvir, hepatitis C
Approval decision date: Dec. 6 Amarin ( AMRN)
Drug/indication: Vascepa, mixed dyslipidemia
Approval decision date: Dec. 20 Also remember a few very important and stock-moving FDA advisory panels scheduled for the fall: Amarin's Vascepa on Oct. 16, Gilead's sofosbuvir on Oct. 25 and Vanda Pharmaceuticals' ( VNDA) tasimelteon on Nov. 14. -- Reported by Adam Feuerstein in Boston. Follow @AdamFeuerstein