Regeneron ditches higher dose of pain drug fasinumab in PhIII, triggering safety scare; In a small win for Alnylam, FDA delays review for Ionis' inotersen
→ Regeneron $REGN has canned its high dose arm of fasinumab, an osteoarthritis and back pain drug being tested in Phase III trials, following recommendations from an independent data monitoring committee. A risk-benefit assessment was done on the higher dose of the drug, and the committee found it wanting.
Regeneron hasn’t yet released any details about the assessment, but this isn’t the first time fasinumab has come under fire. In 2016, the FDA slapped a clinical hold on the company’s Phase IIb study of the drug in lower back pain. The hold was triggered after a patient in the study suffering from advanced osteoarthritis developed a case of adjudicated arthropathy – joint disease.
Dropping the high dose is surfacing concerns about drugs like fasinumab, which target nerve growth factor (NGF) to silence pain. NGF drugs were all the rage until a few years ago, when some of the patients in clinical studies began to blow out their joints with meds designed to silence nerve growth factors. The FDA hit J&J, Regeneron, and Pfizer with clinical holds, which were followed by reports of joint replacements in patients taking the drugs. Some speculate that NGF drugs work so well at silencing pain, that people become more active — accelerating the pace of cartilage deterioration at the joints.
The safety issues sidelined the drugs, once tapped as likely blockbusters, but developers slowly worked out a plan to protect patients, and back into the clinic. Regeneron’s investors will be interested to see how the lower dose of fasinumab moves forward.
→ As Alnylam $ALNY prepares to hear back from the FDA on its leading drug patisiran, it can now be sure that it’s in the lead. The FDA has extended the review period for inotersen — a rival hereditary TTR amyloidosis drug developed by Ionis $IONS — and pushed the PDUFA date back to October 6. That’s 90 days later than the original date, and two months after Alnylam’s drug will likely have been approved. “The FDA determined that they need additional time to review our responses to their standard information requests and we are working closely with them to advance the review of our filing as quickly as possible,” said Sarah Boyce, president of Akcea $AKCA, the Ionis spinoff now handling inotersen. That slight edge can be key in the intense rivalry between the antisense experts at Alnylam and Ionis over a blockbuster market.
→ Under the spotlight for an expected surge of biotech listings, Hong Kong Stock Exchange unveiled a panel of biotech industry veterans it’s assembled to review listing applications. Given the high hopes and serious skepticism surrounding the market’s ability to evaluate pre-revenue biotech companies — which before this week weren’t allowed to list on the exchange — HKEX has assembled a star-studded group. The 13-strong panel features Zai Lab founder Samantha Du as well as prominent VCs Jonathan Wang (of OrbiMed Asia) and Ling Su (of Lilly Asia Ventures). Other members include biotech execs, analysts and regulatory professionals, all of whom have been appointed for an initial term of two years. In addition to advising on questions about prospectus disclosure, the panel will also serve as a sounding board for policy recommendations.
With contribution by Amber Tong