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Late last week, companies developing PCSK9 inhibitors saw a fall in their share price with the FDA raising concerns about this class of drugs. PCSK9 inhibitors work by inhibiting PCSK9, a protein that reduces the liver's ability to remove "bad" cholesterol from the blood.

Although statins (the current standard of treatment) are effective, many patients have problems achieving their cholesterol goals. Moreover, several patients are unable to tolerate statin therapy. In such a scenario, the successful development of PCSK9 inhibitors would be a major breakthrough in the area of cholesterol management. While results on PCSK9 inhibitors in development have been encouraging so far, this is the first time that serious safety concerns have been raised.

The FDA has raised concerns about neurocognitive adverse events in the PCSK9 inhibitor class. These are events that are also associated with the use of statins. So will PCSK9 inhibitors eventually prove to be better than statins as was assumed before?

Sanofi and Regeneron (REGN - Analyst Report), who are developing PCSK9 inhibitor alirocumab, disclosed that they have been advised by the FDA regarding the issues though neither company is aware of the circumstances under which the FDA became aware of these adverse events.  So questions like did the FDA notice these events when the PCSK9 inhibitor was being tested as a monotherapy or when it was being tested in combination with a statin or another cholesterol treatment remain.

These companies have been told to evaluate potential neurocognitive adverse events across the global development program for the experimental drug, especially in long-term studies. The FDA has also raised the possibility of incorporating neurocognitive testing in at least a subset of patients in late-stage studies.

With the news about the FDA’s concerns spreading like wildfire, Regeneron saw its shares sliding 3.1%. Amgen’s (AMGN - Analyst Report) shares were down 1.6%. Amgen’s PCSK9 inhibitor, evolocumab, is currently in late-stage development with regulatory filing expected this year itself. The FDA will most likely ask all these companies to evaluate neurocognitive adverse events in long-term studies.

InterMune Soars on Takeover Rumors: InterMune shares were up 10.8% on rumors that it is an acquisition target. The acquisition rumors do not come as a surprise; just a few days back, InterMune scored a huge win in a late-stage study conducted on its lung disease drug, Esbriet. The results are compelling enough for the company to succeed in gaining FDA approval for the product which is already approved in the EU.

Approval in the U.S. would boost sales of the drug significantly. Esbriet sales were $70.3 million in 2013. Given Esbriet’s improving prospects, quite a few pharma companies would be interested in acquiring the company.

Pipeline Setback Overshadows (XOMA - Snapshot Report)’s Fourth Quarter Results: XOMA’s fourth quarter results were overshadowed by the company’s announcement that it will not be moving gevokizumab into phase III studies for erosive osteoarthritis of the hand. Shares were down 27.8%.

Company Last Week Last 6 Months
AMGN -11.31% 12.99%
BIIB -3.50% 45.99%
GILD -3.88% 30.20%
CELG -2.61% 6.53%
REGN -1.32% 22.63%
ALXN -4.95% 50.64%
^BTK 0.20% 30.60%


Other Developments:

Investors Not Impressed with Interim Analysis of NewLink (NLNK - Snapshot Report) IMPRESS Study: Based on the recommendation an independent data safety monitoring committee, NewLink said that it will continue with a phase III study (IMPRESS) on cancer therapy, algenpantucel-L. Although there is nothing negative in the company’s statement, usually when an interim analysis is conducted expectations are high that the study will be halted prematurely on positive signals. Investors seemed to have been expecting such a positive outcome and expressed disappointment at the news sending the shares down 16.2%.

Biogen (BIIB - Analyst Report) – Eisai Tie Up: Biogen is expanding its efforts to develop a treatment for Alzheimer’s disease. The company, which already has a couple of Alzheimer’s disease candidates in its pipeline, is tying up with Eisai for the development and commercialization of two of Eisai’s candidates for Alzheimer’s disease. The companies will pool in their resources and expertise for developing these candidates, which are BACE inhibitors.

The Alzheimer’s disease market represents huge commercial potential and a successfully developed product could generate billions of dollars in sales once launched. However, the successful development of therapies for the treatment of Alzheimer’s disease is challenging and several companies like Eli Lilly, Pfizer and Johnson & Johnson have had pipeline setbacks in this area.

The Week So Far:

Alexion (ALXN - Analyst Report) Ups Guidance: Alexion boosted its 2014 earnings and revenue guidance based on a reimbursement deal in France for Soliris. Shares were up 7.1%.

Regado Gains on Fast Track Status: Regado’s shares popped 38.1% on news that the FDA has given fast track status to the company’s anticoagulant treatment currently in a phase III program.

KYTHERA (KYTH - Snapshot Report) Acquires Ex-U.S. Rights to ATX-101: KYTHERA regained ex-U.S. rights to its double chin reduction candidate, ATX-101, from Bayer. Shares were up 4.7% as worldwide rights to the candidate returned to KYTHERA.

Amgen’s Nexavar Fails in Liver Cancer Study: Amgen and partner Bayer said that their cancer treatment, Nexavar, failed to achieve the primary endpoint in a phase III study evaluating Nexavar as an adjuvant treatment in liver cancer patients who had no detectable disease after surgical removal or local ablation.

Chimerix (CMRX - Snapshot Report) Makes Drug Available to Josh Hardy: Chimerix, which is developing a treatment for adenovirus infections in immunocompromised patients, has agreed to make its experimental drug available to 7 year old Josh Hardy. Josh will be the first patient to be enrolled in the open-label study.

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