Piper Sandler analyst Tyler Van Buren maintains an Overweight rating and $200 price target on shares of Sarepta after the company reported at the World Muscle Society virtual congress that it has seen sustained functional benefits for patients in both the SRP-9001 DMD and SRP-9003 LGMD gene therapy programs. For SRP-9001, the data enhance his confidence in the registrational study 102 readout and the potential for the treatment to achieve $3B+ in peak sales, said Van Buren. For SRP-9003, Van Buren argues that given these data the other two sarcoglycan programs “should also have a relatively high probability of success.”
Mizuho analyst Difei Yang says yesterday’s data update from Sarepta Therapeutics reinforces the stock’s investment thesis. The “continued positive data” show improvements in functional outcomes in Duchenne muscular dystrophy and Limb-girdle muscular dystrophy patients, Yang tells investors in a research note. The analyst continues to see SRP-9001 as the “best-in-class” gene therapy for DMD. Yang keeps a Buy rating on Sarepta with a $192 price target.
Biogen issued the following statement: “On September 29, 2020, it was published in the Federal Register that the U.S. Food and Drug Administration will convene a virtual advisory committee meeting of the Peripheral and Central Nervous System Drugs Advisory Committee to review data supporting the Biologics License Application – BLA – for aducanumab, an investigational product for Alzheimer’s disease. The advisory committee meeting is scheduled for November 6, 2020, and will be available for live streaming. The notice stated that the FDA intends to make the advisory committee meeting’s background materials and pre-recorded presentations available to the public no later than two business days before the meeting.
The notice also stated that, if the FDA is unable to post the background materials and/or pre-recorded presentations on its website prior to the advisory committee meeting, the background material and/or pre-recorded presentations will be made publicly available on the FDA’s website at the time of the advisory committee meeting. The notice stated that the advisory committee meeting will include brief summaries of the pre-recorded presentations, discussion, and oral presentations from the public.”
Biogen said, “In 2020, the cost of Alzheimer’s in the U.S. is estimated to be over $500 billion, impacting countless patient, family and caregiver lives. If approved, aducanumab may slow clinical decline and potentially help patients maintain independence longer. This could constitute an important breakthrough, after more than 100 clinical programs that have failed in this space. We look forward to engaging meaningfully with ICER and other stakeholders to help ensure that appropriate considerations and data representing the substantial cost and impact to patients, families, and society, as well as the benefits of aducanumab, are applied appropriately and transparently. The aducanumab Biologics License Application is the first for a product that aims to slow the clinical decline of Alzheimer’s disease, as well as one of its hallmarks, the buildup of amyloid plaque. The BLA is currently under priority review with the FDA. As pioneers in neuroscience, we aim to bring transformative treatments to patients and are deeply committed to making ongoing investments in the science and research required to create breakthrough therapies. We recognize that prices for these treatments – and the ability of patients to obtain access to them – are important concerns for patients, physicians, payers, and policymakers, and we are dedicated to engaging with all stakeholders in a constructive dialogue about innovation, price, and access.”
The biotech index has been up nearly 38 percent since its March lows. Big movers include TG Therapeutics and Arena Pharmaceuticals. Alethia Young, Cantor Fitzgerald biotech analyst, joins ‘Closing Bell’ to talk about the biotech stocks she’s excited about.