Last week, major events in the biotech sector included an acquisition announcement, a positive FDA panel review for an insomnia candidate and a delay in the filing of a new drug application (:NDA) for a Duchenne muscular dystrophy (DMD) candidate.

Vanda the Clear Winner: Vanda (VNDA) was the clear winner last week with shares shooting up 115% on positive feedback from the FDA regarding insomnia candidate, tasimelteon (proposed trade name: Hetlioz). The FDA’s advisory panel voted resoundingly in favor of approving the candidate for the treatment of Non-24-Hour Disorder in the totally blind.

While the FDA is not required to follow the advice of its advisory panel, it usually does so. So no surprises if tasimelteon is approved by Jan 31, 2014 (FDA action date).

Sarepta Plunges on Eteplirsen Update: Sarepta’s (SRPT) hopes of filing an NDA for its DMD candidate, eteplirsen, based on current data were quashed with the FDA saying that the NDA filing would be considered premature. The company lost about 64% of its market value. Sarepta has a long way to go – first up, the company will be meeting with the FDA later this month to discuss the confirmatory study design.

With the FDA asking for a placebo-controlled study, patient enrolment will be a challenge. As things stand now, Sarepta is looking at a delay of at least two years in the potential approval of eteplirsen.

Shire Zeroes In on ViroPharma: Rumors regarding ViroPharma (VPHM) being an acquisition target surfaced earlier this year in June. At that time, the shares were trading in the $27 - $28 range. But with acquisition rumors gaining traction, shares shot up to the late $30s range with a deal finally being struck with Shire at $50 per share (representing a premium of 27%). Apart from Shire, companies like Sanofi were also rumored to be interested in ViroPharma which is well known for its focus on rare diseases.

Impact of New ACC/AHA Guidelines: The new guidelines issued by The American College of Cardiology (ACC) and the American Heart Association (:AHA) released last week created a lot of buzz with the main discussion being the impact of the guidelines on PSCK9 inhibitors. The guidelines, which will benefit statins and lead to an increase in their use, have left a question mark on the regulatory process for PSCK9 inhibitors, a new class of cholesterol management drugs currently in development.

The main concern is that the FDA will require cardiovascular outcome trials to be conducted before granting approval to PSCK9 inhibitors – this would delay the approval process significantly.

However, according to a Bloomberg article, U.S. regulators will not require companies developing PSCK9 inhibitors to conduct cardiovascular outcome trials to gain approval. The drugs need to meet the FDA’s current standards for approval which include cholesterol/blood pressure reduction etc. But, results from a study (IMPROVE-IT) being conducted by Merck could very well change this equation. Some biotech companies with a stake in PSCK9 inhibitors are Amgen, Regeneron and Alnylam (ALNY).

A Look at the Major Players

The table below captures the price movement of the major biotech players last week and during the past 6 months. There wasn’t much of an update from these companies last week. The only news of significance was an update on Biogen’s (BIIB) hemophilia A candidate, Eloctate. Eloctate could be facing a bit of a delay as the FDA has asked the company for additional information on certain steps in the validation process in the manufacturing plan. So, Eloctate will now most likely be launched in mid-2014 instead of sometime in the second quarter of 2014 - not much of a delay to lose sleep over.

Other Developments:

CADX Shoots Up on Favorable Ruling: Cadence (CADX) investors heaved a sigh of relief with the company receiving a favorable ruling in its patent infringement lawsuit against Exela Pharma Sciences for Ofirmev. Shares shot up 34%. The favorable ruling removes a significant overhang and Cadence should rest assured that generic competition will not be entering the market in the near future. The company can now focus on driving Ofirmev revenues.

ImmunoGen Gains on ADC Deal: ImmunoGen continues to sign deals with major pharma companies for its antibody-drug conjugate (ADC) technology. The latest deal is with Novartis – ImmunoGen’s third with this company - for the exclusive right to use the ADC technology to develop anticancer therapeutics to an undisclosed target. Shares rallied 10.3%. This agreement marks ImmunoGen’s fifth this year with a major healthcare company.

FDA Approves Rare Blood Cancer Drug: Last week, Pharmacyclics’ (PCYC) much-awaited blood cancer drug, Imbruvica gained FDA approval. Imbruvica, developed in collaboration with Johnson and Johnson, is approved for use as a single agent in patients with mantle cell lymphoma (:MCL) who have received at least one prior therapy. The drug works by blocking signals that make the cancer grow and spread. Shares were up 3.5% on the news. Imbruvica is also under review for chronic lymphocytic leukemia (:CLL) with a response due by Feb 28, 2014.

The Week So Far:

Amgen presented new data on its cholesterol management candidate, evolocumab (AMG 145) at the American Heart Association (:AHA) Scientific Sessions 2013. Evolocumab works by inhibiting PCSK9, a protein that reduces the liver's ability to remove "bad" cholesterol from the blood. The company also presented encouraging interim overall survival data on its phase III cancer candidate - talimogene laherparepvec.

Gilead gained EU approval for its HIV drug Vitekta - the FDA had refused to approve it in April this year.

Regeneron presented data from several studies on Eylea at the annual meeting of the American Academy of Ophthalmology (:AAO).

BioMarin’s Vimzim was backed by the FDA’s advisory panel. Vimzim is under regulatory review for the treatment of patients with the lysosomal storage disorder Morquio A syndrome.

What Next?

With quite a few companies attending scientific conferences like the AHA Scientific Sessions, more data should be out through the remainder of the week.

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