According to their data, Sarepta Therapeutics' drug for Duchenne muscular dystrophy (DMD) “Eteplirsen” has been effective in a Phase III clinical study. Because DMD is both rare and serious, Sarepta has been a candidate for a sped up review process from the FDA, however as Robert Weissman from the Boston Globe recently reported an “influential advisory committee” has recommended “to reject its experimental treatment”. Sarepta’s March press release explains: “The FDA has granted Eteplirsen Priority Review status, which is designated for drugs which provide a treatment where no adequate therapy exists. The FDA also granted Rare Pediatric Disease Designation to Eteplirsen, as well Orphan Drug Designation and Fast Track Status.” What does Eteplirsen do? Sarepta explains: “The underlying cause of DMD is a mutation or error in the gene for dystrophin, an essential protein involved in muscle fiber function. Our investigational therapies for DMD are designed to skip an exon in the dystrophin pre-m RNA to enable the synthesis of a functional shorter form of the dystrophin protein.” UPDATE: Boston Business Journal reported the morning of September 19, 2016: "More than six months after its original deadline to make a decision, the U.S. Food and Drug Administration has granted accelerated approval to the first drug in the U.S. that can slow the progression of Duchenne muscular dystrophy."
The action date for FDA review was late May, but the FDA requested more data from Sarepta in June. Sarepta explained the dire need for a Duchenne cure, and how they could potentially meet that need, in their March press release: “It is estimated that Duchenne muscular dystrophy affects approximately one in every 3,500 – 5,000 boys born worldwide, with 13% of people with the disease having mutations addressable by Eteplirsen/exon 51 skipping.” This potential for a cure gives new hope for those affected by this disease, a hope which has been increasingly channeled into activism. Fierce Biotech reported last month that: “Hundreds of patient advocates, patients and families turned out to give their raucous support for an approval during the (FDA) panel review and vote.” In addition, the Washington Post reported that families whose sons have taken Eteplirsen have documented the “apparent halt in their sons' decline” on Facebook and YouTube. Note: since this article was written, biotech Santhera was set back by potentially three years by the FDA decision for them to run another Phase III study for their Duchenne’s drug. The jury is out on how this decision will affect Sarepta’s chances. Matthew Herper of Forbes had this to say on Twitter right after that decision: “I don’t think there is any read-through from Santhera to Sarepta.”
Back in 2013, Robert Weissman reported on this devastating Boston biotech setback: “It took Aveo Pharmaceuticals Inc. seven years to develop a much anticipated kidney cancer drug (Tivozanib). Federal regulators needed only about four hours to crush the company’s hopes.” Three years later, after a corporate restructuring (the SEC has threatened to ban three of its former executives), loss of Biogen and Astellas as research partners, a rebrand as Aveo Oncology and then gain of $17 million in funding, Aveo has given the go ahead from the FDA to begin a Phase III trial of Tivozanib for renal cell carcinoma. What does Tivozanib do? Aveo’s website describes it as: “a potent, selective, long half-life inhibitor of all three vascular endothelial growth factor (VEGF) receptors that is designed to optimize VEGF blockade while minimizing off-target toxicities.” Why blockade VEGF? Aveo explains: “vascular endothelial growth factor (VEGF) pathway plays a significant role in angiogenesis, which is critical in cancer”. With perhaps lessons learned from how they work with the FDA, success may be on the horizon for this drug, eagerly awaited by patients and patient advocates.
In partnership with NSGO (Nordic Society of Gynaecological Oncology), Tesaro just had the first successful Phase III trial of a PARP inhibitor Niraparib (NOVA) for ovarian cancer. Tesaro’s NDA and MDA submissions “are planned for Q4 2016”. Niraparib (BRAVO) is also in a Phase III trial for the treatment of breast cancer. One of the youngest companies of the five on our list, founded in 2010, it is also one of the most successful, with a commercial drug for chemotherapy side effects called Varubi, a $500 million deal with Janssen for commercial use of Niraparib for prostate cancer, and plans to add 100 employees by the end of the year.
Paratek Pharmaceuticals’ website describes their efforts as “working to change how bacterial infections are treated.” Having just received positive results in Phase III trials for Omadacycline, they are poised to fulfill that promise. Paratek believes that Omadacycline will solve a great deal of the issues with the bacterial resistance doctors are increasingly dealing with. Paratek says that Omadacycline is “Active against drug resistant pathogens, including methicillin-resistant Staphylococcus aureus, penicillin-resistant and multi-drug resistant Streptococcus pneumonia da, and vancomycin-resistant Enterococcus species.”
ArQule is currently in two Phase III trials for Tivantinib to treat hepatocellular carcinoma, the most common type of liver cancer, in partnership with Daicchi Sankyo and Kyowa Hakko Kirin. The National Cancer Institute gives a great explanation of what exactly Tivantinib does: ”Tivantinib binds to the c-Met protein and disrupts c-Met signal transduction pathways, which may induce cell death in tumor cells overexpressing c-Met protein or expressing constitutively activated c-Met protein.” In more layman’s terms, it’s a “kinase inhibitor” a type of research in which ArQule specializes, having “eight kinase inhibitors into human clinical trials with a ninth about to enter the clinic.” In late June ArQule was featured by Zack’s Investment Research Firm with the call to action “Forget Valeant, Invest in These Attractive Stocks Instead.”
Two Boston biotechs that may not have an impending breakthrough, but are looking fairly certain (if anything is certain in this biotech world) to have success by 2020, are Alnylam and Boston Biomedical. Andrew McConaghie raved about Alnylam this June in Pharma Phorum, saying it was: “one biotech company which is tipped to deliver on huge expectations, thanks to a rigorous approach to validating its science.” Alynlam proposes to have three drugs approved and another ten in the pipeline by 2020. Alynylam’s core focus is RNAi therapeutics, and Pharma Phorum describes their pipeline as: ” based on targets in the liver, but covering a broad range of diseases; rare genetic diseases, cardiometabolic conditions and hepatic infectious diseases, such as hepatitis B.”
Boston Biomedical’s Napabucasin was just granted Orphan Drug Designation from the FDA in the treatment of gastric cancer this June. Orphan Drug status is given to: “Drugs that are not developed by the pharmaceutical industry for economic reasons but which respond to public health need.” Boston Biomedical is currently in Phase III trials for this drug, which inhibits cancer cell pathways by targeting the STAT3 pathway.
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