Shares of Sage Therapeutics Inc. (NASDAQ:SAGE) were trading at $10.54, down $2.54, or 19%, after the Cambridge, Mass.-based firm disclosed top-line results from the phase II Kinetic 2 dose-ranging study of oral SAGE-324 (also known as BIIB-124) for essential tremor (ET). The trial did not show a statistically significant dose-response relationship in change from baseline to day 91 based on the primary endpoint, The Essential Tremor Rating Assessment Scale (TETRAS) Performance Subscale (PS) Item 4 (upper limb) total score. No statistically significant differences turned up for any dose of SAGE-324 vs. placebo in the change from baseline to day 91, not only on the TETRAS PS Item 4 Total Score but also the TETRAS Activities of Daily Living composite measure. Given the results, Sage and partner Biogen Inc. will close the ongoing open label safety study of SAGE-324 in ET and do not plan to conduct further work. The drug is a neuroactive steroid designed to function as a positive allosteric modulator of gamma-aminobutyric acid A receptors

ODAC to consider whether phase matters in NSCLC trials

Tomorrow’s meeting of the U.S. FDA’s Oncologic Drugs Advisory Committee (ODAC) could impact the future development of immune checkpoint inhibitors, such as Astrazeneca plc’s Imfinzi (durvalumab), to treat patients with non-small-cell lung cancer (NSCLC) both before and after surgery. Although much of the discussion will focus on a proposed neoadjuvant indication for Imfinzi, the committee will be asked to vote on whether the FDA should require that new trial design proposals for perioperative regimens for resectable NSCLC include adequate within-trial assessment of the contribution of the treatment phase. That question arises from what the FDA sees as the inability of Astrazeneca’s two-arm Aegean trial to distinguish whether the efficacy of durvalumab is related to its use in the neoadjuvant phase, the adjuvant phase or both phases.

With Dren deal, Novartis has three of year’s top collaborations

A new collaboration with Dren Bio Inc. means Novartis Pharma AG has negotiated two of the biggest deals of 2024, with its parent company Novartis AG signing a third. Privately held Dren is getting $150 million up front and the chance to ultimately bring in $2.85 billion. The two plan to discover and develop bispecific antibodies for treating cancer. In May, Novartis Pharma and Peptidream Inc. said they would identify and optimize macrocyclic peptides against targets selected by Novartis for conjugation to radioligand therapies or other applications for therapeutic and diagnostic purposes. The collaboration could add up to $2.89 billion for Peptidream. Another deal between Novartis and Shanghai Argo Biopharmaceutical Co. Ltd. for cardiovascular disease programs is worth about $4.2 billion. Novartis Pharma is a subsidiary of Novartis AG.

Telix launches AU$650M bond offer, FDA accepts imaging NDA 

The U.S. FDA accepted Telix Pharmaceuticals Ltd.’s NDA drug application for TLX-007-CDx, a new cold kit for preparing prostate-specific membrane antigen-PET imaging for prostate cancer. The company is also raising AU$650 million (US$428 million) through a convertible bond offering that received strong support from eligible investors.

Biontech, Triastek ink $1.2B deal to 3D print oral RNA therapies

Triastek Inc., of Nanjing, China, scored a potential $1.2 billion collaboration and platform technology license deal with Biontech SE to manufacture oral RNA therapeutics with 3D printing technology. “Most, if not all, mRNA [therapeutics…] are injections… parenteral,” Triastek co-founder and chief scientific officer Xiaoling Li told BioWorld. “But if you could change it to an oral [drug… this would be] revolutionary.” Under the terms announced July 23, Triastek and Mainz, Germany-based Biontech will collaborate to develop oral RNA therapeutics with Triastek’s 3D printing Melt Extrusion Deposition (MED) platform technology. In turn, Triastek will receive an up-front payment of $10 million from Biontech and potential development, regulatory and commercial milestone payments summing up to $1.2 billion, along with tiered royalties on potential future product sales.

US PTO revisits subject matter eligibility for AI

The U.S. Patent and Trademark Office (PTO) provided updated guidance on the question of patent subject matter eligibility for inventions that rely on artificial intelligence (AI), stating that a patent claim that does little more than recite an abstract idea is not subject-matter eligible. However, a claim may be eligible if the AI algorithm calls for a specific treatment based on the patient’s risk of suffering from a particular condition, a crucial difference for developers of clinical decision support algorithms.

BioWorld Insider Podcast – The cost of delays in development and sales: It’s probably not what you think

Two costs of developing drug candidates have been upended by new research from the Tufts University School of Medicine’s Center for the Study of Drug Development. New data have produced some very different numbers than you might expect in the cost of a single day of a clinical trial and of missing a day to generate prescription drugs sales. The center’s director, Ken Getz, spoke to the BioWorld Insider podcast about updating the outdated numbers and what it means for companies and investors.

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