This Week in Biotech: Biogen Idec Gets a Key Approval While Insmed Confuses Investors

An FDA approval, a terrible review from regulators on a breakthrough therapy designated drug, and a trio of mid-stage clinical data are this week's top biotech stories.

Mar 29, 2014 at 3:15PM

With the SPDR S&P Biotech Index up 38% over the trailing-12-month period, it's evident that investment dollars are willingly flowing into the biotech sector. Keeping that in mind, let's have a look at some of the rulings, studies, and companies that made waves in the sector last week.

A two-sided coin
In terms of regulatory actions it was a mixed week with one company receiving a long-awaited approval from the Food and Drug Administration while another got buried under a unanimous vote against recommending approval by the FDA's panel.

The lucky victor this week is Biogen Idec (NASDAQ:BIIB) which, on Friday, announced that the FDA had approved its long-acting hemophilia B injectable drug Alprolix. Hemophilia B is a rare inherited disorder affecting roughly 4,000 people in the U.S. that doesn't allow their blood to clot properly and can lead to excessive bleeding and bruising. Aprolix works as a prophylactic infusion given once weekly, or every 10-14 days, to stay ahead of possible bleed complications. In its B-LONG study, for instance, the overall median annualized bleeding rates was 2.95 for the weekly prophylaxis arm compared to 17.69 for the on-demand treatment group. Alprolix was also approved in Canada last week. With peak sales potential of around $400 million in the U.S. -- assuming a six-digit annual therapy price tag -- Biogen continues to impress with its pipeline innovation.

The other side of the coin featured Novartis (NYSE:NVS) having its breakthrough therapy designated drug serelaxin absolutely buried by the FDA's advisory panel. According to a Tuesday release by the FDA's Cardiovascular and Renal Drugs Advisory Committee, it voted 11-0 against recommending Novartis' acute heart failure therapy serelaxin for approval. While finding no fault with its safety or tolerability, the panel did find holes in its RELAX-AHF trial which led to it stating that "there is insufficient evidence to support the proposed indication." This was a study with only a single independent trial where two are usually needed to get a drug approved. Furthermore, per Forbes, an FDA reviewer questioned the claim that serelaxin reduced mortality rate at day 180 and suggested a follow-up study be conducted. All told, with the EU sending Novartis away empty-handed earlier this year with regard to serelaxin, it now looks as if its ongoing lengthier trial testing serelaxin's safety and efficacy that's scheduled to be completed in 2016 will be necessary if it's to ever be approved.

Mid-stage madness
It was also a busy week for phase 2 pipeline updates with two positive and one mixed clinical study release.

Late in the week Idera Pharmaceuticals (NASDAQ:IDRA) reported positive top-line data from its phase 2 study involving IMO-8400 as a treatment for moderate-to-severe plaque psoriasis. In its 32-patient study Idera noted that IMO-8400 met its primary endpoint of safety and tolerability, and its secondary endpoint of efficacy by delivering a lesion improvement of 50% or greater as measured by the Psoarisis Area and Severity Index in nine of 20 patients compared to just one in seven for the control arm. What's more intriguing is that Idera is a clinical-stage DNA and RNA drug developer, so this success could help validate the effectiveness of RNA-based therapeutics. It's probably best to wait until we have the full data set before getting too excited, but this was a good way for shareholders to end the week.

Pfizer (NYSE:PFE) also got in on the action this week by reporting positive results in its phase 2b study involving its PCSK9 inhibitor bococizumab (formerly RN316) for the treatment of hyperlipidemia. According to its Thursday press release, bococizumab injected once or twice monthly led to a placebo-adjusted mean change in baseline in LDL-cholesterol reduction at week 12 of minus 53.4 mg/dL for the 150 mg twice monthly dose and minus 44.9 mg/dL for the once monthly 300 mg dose. PCSK9 inhibitors represent a truly unique pathway to treating high cholesterol, and Pfizer's bococizumab has certainly impressed in trials thus far. If I were you, I would strongly suggest keeping a close eye on all PCSK9 inhibitors as they could represent the future of cardiovascular disease prevention when statins either aren't enough, when they cause serious adverse side effects, or in patients who are genetically predisposed to extremely high-cholesterol levels.

Finally, clinical-stage biopharmaceutical company Insmed (NASDAQ:INSM) fell 7% on the week after reporting mixed phase 2 data on Wednesday for Arikayce, its inhaled therapy for treatment-resistant nontuberculous mycobacterial lung infections. Insmed's press release notes that Arikayce failed to meet its primary endpoint of a change in mycobacterial density on a seven-point scale from baseline to the end of the randomized portion of the trial (which was week 12). It did, however, meet a key secondary endpoint of culture conversion with 11 of 44 patients demonstrating negative cultures after 12 weeks compared to just three of 45 patients in the control arm. There were also more adverse events noted in the Arikayce intent-to-treat group than the control arm. Ultimately, Arikayce's important secondary endpoint data should allow it to continue onto phase 3 studies, but it's not a slam dunk by any means. Although the serious adverse events profile was similar to the control arm, Arikayce is really going to have to demonstrate a statistical benefit to outweigh any efficacy and safety concerns the FDA may have if it ever hopes to be approved.

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Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.

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