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June 17, 2016 By Mark Terry , BioSpace.com Breaking News Staff Rumors are flying that Paris-based Sanofi is developing a backup plan just in case its hostile takeover bid for San Francisco-based Medivation falls through. The possible target? Novato, California-based BioMarin Pharmaceutical , best known for its failed drug for Duchenne Muscular Dystrophy. In late March and early April, Olivier Brandicourt , Sanofi’s chief executive officer, contacted Medivation’s founder, president, and chief executive officer, David Hung , regarding a potential acquisition. Both Hung and the Medivation board rejected the offer. On April 15, Sanofi then sent a written proposal, which Medivation ignored. Turning up the heat, on April 28, Sanofi went public with its offer in hopes of pressuring the board and shareholders into at least discussing the deal. In the proposal, Sanofi offered $52.50 per share, which was a 50 percent premium to Medivation’s average share price the two months prior to the offer. Medivation took measures to hold off a takeover, including adding a provision to its bylaws that would make it more difficult for stockholders to overturn the board of directors. In May, Sanofi escalated the attempt, sending a public letter to the company that, in a reasonable tone, spelled out the threat of a hostile takeover. Medivation’s appeal is its prostate cancer drug, Xtandi. With its marketing partner, Astellas Pharma (ALPMY) , sales of Xtandi grew 73 percent in the U.S. in 2015 and 116 percent globally. It has at least two additional cancer drugs in its pipeline, pidilizumab for B-cell lymphoma and other blood cancers, and talazoparib for breast cancer. Now, writing on his blog Betaville, Ben Harrington says, “Top sources have told me the French pharmaceutical giant has begun looking at U.S.-based rare disease specialist BioMarin Pharmaceutical as a potential alternative takeover deal after growing frustrated in pursuit of Medivation, which has resisted the French company’s advances.” On June 1, BioMarin announced that it was abandoning its Kyndrisa (drisapersen) program for DMD. The U.S. Food and Drug Administration (FDA) rejected the company’s application for the drug in January. DMD is a muscle wasting disease caused by mutations in the dystrophin gene. The disease is progressive and typically causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 to 5,000 male children. BioMarin also canceled its European application for the drug, as well as three other first-generation follow-on products, BMN 044, BMN 045 and BMN 053, which were all in Phase II trials for specific types of DMD. If Sanofi is interested in BioMarin, it’s probably not related to its DMD programs, but for its focus on rare genetic diseases. It has five products on the market, Vimizim for Morquio A Syndrome (MPS IVA), Kuvan for PKU, Naglazyme for MPS VI, Aldurazyme for MPS I, and Firdapse for LEMS. It also has several products in the pipeline for achondroplasia, CLN2 disease, Hemophilia A and Sanfilippo Syndrome. “Now, clearly the very idea of Sanofi—which is being advised by Morgan Stanley and Goldman Sachs—looking at an alternative deal to Medivation might just give the U.S. company’s shareholders that extra bit of encouragement to work with the French giant in replacing Medivation’s board,” writes Harrington. “But this story really has come from excellent sources, so I’m taking Sanofi’s interest in BioMarin seriously.” BioMarin is currently trading for $83.63. Medivation is trading for $59. Sanofi is trading for $38.04.

June 7, 2016 By Mark Terry , BioSpace.com Breaking News Staff Cambridge, Massachusetts-based Sarepta Therapeutics jumped after the U.S. Food and Drug Administration (FDA) requested additional data about the company’s Promovi (eteplirsen) for Duchenne muscular dystrophy (DMD). Sarepta has been on quite the roller-coaster ride this year. In January, the FDA was scheduled to review the New Drug Application (NDA) for eteplirsen, but postponed it because of a pending snowstorm on the east coast. After several dramatic public hearings by the FDA, 109 members of Congress sending a letter to the FDA urging it accelerate approval of any DMD drug, and a public letter urging approval signed by 36 DMD experts, the FDA delayed final approval . On May 25, the FDA reported that it would miss the May 26 deadline because “they are continuing their review and internal discussions related to our pending NDA for eteplirsen.” Yesterday’s announcement indicated that the FDA “has requested that Sarepta provide dystrophin data, as measured by western blot, from biopsies already obtained from the ongoing confirmatory study of eteplirsen (PROMOVI), as part of its ongoing evaluation of the eteplirsen New Drug Application (NDA).” DMD is a muscle wasting disease caused by mutations in the dystrophin gene, which results in insufficient levels of the dystrophin protein. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children. To add even a little more urgency to the situation, the other company that was fairly well along in the development of a DMD drug, San Rafael, California-based BioMarin Pharmaceutical , announced on June 1 that it was abandoning its Kyndrisa (drisapersen) program for DMD. The drug had been rejected by the FDA in January. It was terminating that program, pulling its Marketing Authorization Application (MAA) in Europe, and folding three other first-generation follow-on products, BMN 044, BMN 045 and BMN 053, which were in Phase II trials for DMD. Although there are other companies working on DMD drugs, Sarepta and BioMarin were furthest along in the development process. Many analysts and investors believe that the FDA’s most recent request is an indication they are inclined to approve eteplirsen. Alexander Maxwell , writing for Seeking Alpha, says , “The request for data would suggest that the FDA is open to approving a drug that is modestly effective, even if it is just a first step. Especially in a condition like Duchenne muscular dystrophy, where the patients have no treatment options, approving a modestly effective drug could be important to help create even a marginally better life for patients. The recent decision to delay the approval decision until there is more information provided by Sarepta suggests that this is the mindset for the FDA.” Sarepta has indicated it will submit data from 13 patient biopsy samples, at both baseline and Week 48, in the next couple weeks. Then the FDA would likely take several weeks or even a few months to analyze and evaluate the data. Maxwell writes, “While some delays in approval decisions could be a cause for concern, this delay is different. It helps to evaluate the thesis that the FDA is at the very least considering approving Sarepta’s drug, and that Sarepta’s drug is unlikely to meet the same fate as BioMarin’s. The potential to have the only approved product for DMD could be very lucrative for Sarepta shareholders, and should help to command a premium valuation for the company.” Christopher Marai , an analyst with Oppenheimer , essentially agrees, and reiterated the company’s “outperform” rating with a $60 price target.