Telix Pharmaceuticals Ltd. received an FDA complete response letter (CRL) a day after the Aug. 27 PDUFA date for its BLA for radiopharmaceutical renal cancer imaging agent, Zircaix (TLX250-CDx, 89Zr-DFO-girentuximab). Zircaix is an 89Zr-radiolabeled version of girentuximab, the monoclonal antibody-drug conjugate that targets the tumor antigen carbonic anhydrase IX, as a positron emission tomography imaging agent for the diagnosis and characterization of clear cell renal cell carcinoma. The CRL cites deficiencies relating to the chemistry, manufacturing and controls package, and the FDA has requested additional data to establish comparability between the product used in the Zircon phase III trial and the scaled-up manufacturing process intended for commercial use. The FDA also issued a Form 483 to the two third-party manufacturing and supply chain partners that will require remediation prior to resubmission.

CRL for wet AMD prospect divides Outlook shares by half

Shares of Iselin, N.J.-based Outlook Therapeutics Inc. (NASDAQ:OTLK) were trading at $1.17, down $1.21, or 50%, on word of a complete response letter (CRL) regarding its BLA resubmission for ONS-5010, or Lytenava (bevacizumab-vikg) to treat wet age-related macular degeneration (AMD). In the CRL, the FDA advised that, because ONS-5010 fell short of the primary efficacy endpoint in Norse Eight, regulators need more evidence of efficacy. Another study, Norse Two, had a better outcome, hitting the primary endpoint in AMD. Outlook said a near-term meeting with the FDA will determine whether a new trial will be needed.

Monarez out at CDC as FDA restricts uses in COVID vaccines

The U.S. Department of Health and Human Services announced Aug. 27 that Susan Monarez no longer occupies the post of director for the Centers for Disease Control and Prevention (CDC), a discharge that has been confirmed by the White House. The news arrived within hours of a more restrictive FDA approval of the supplemental application for both the Moderna and the Pfizer/Biontech COVID-19 vaccines, a development that seems to have been the reason for Monarez’s departure from CDC.

Bioarctic's new era: Leqembi takes off, Novartis signs $802M deal

Twenty-two years since its formation, Bioarctic AB expects to become profitable in 2025, as milestones for marketing approvals and royalties on sales of the Alzheimer’s drug Leqembi (lecanemab) roll in, and partners sign up to use its proprietary Braintransporter drug delivery technology. The company has received $124 million in up-front payments and milestones in 2025, with a recent payment of $24 million triggered on the EMA’s approval of Leqembi in Europe. And the 9% royalty on Leqembi from partner Eisai Co. Ltd. is mounting up, with take-up of the anti-beta amyloid antibody gathering pace. Adding to the total, Stockholm-based Bioarctic is in line for a $30 million up-front payment from Basel, Switzerland-based Novartis AG, plus a possible $772 million in milestones, under the terms of an agreement announced earlier this week, in which the Braintransporter technology will be applied to optimize a Novartis antibody for delivery to the brain.

Novo to use Replicate’s srRNA platform in $550M deal

To strengthen its development efforts, Novo Nordisk A/S will collaborate in a deal that could bring Replicate Bioscience Inc. about $550 million. The two are aiming at targets in cardiometabolic diseases that include treatments for obesity and type 2 diabetes. Novo is getting an exclusive, worldwide license to use Replicate’s self-replicating RNA platform to develop the candidates. The $550 million that could come Replicate’s way includes an undisclosed up-front payout and milestone payments. Replicate is developing vaccines for rabies and infectious mononucleosis, plus therapeutics for inflammatory and autoimmune diseases, diabetes and obesity, and estrogen receptor-positive breast cancer.

Fosun out-licenses immunology candidate to Sitala in $675M deal

Shanghai Fosun Pharmaceutical (Group) Co. Ltd.’s subsidiary, Shanghai Fosun Pharma Industrial, signed off ex-China rights to a phase II small-molecule inhibitor, FXS-6837, to Sitala Bio Ltd. in a potential $675 million deal. Announced Aug. 27, the deal includes $190 million tied to up-front, development and launch milestone payments and up to $480 million in sales milestone payments based on annual net sales upon successful commercialization. Fosun will also receive $5 million worth of Sitala shares as part of the agreement. In exchange, Sitala will gain exclusive rights to develop, manufacture and commercialize FXS-6837 and any products containing it as an active ingredient globally excluding China, Hong Kong and Macau, where Fosun will retain rights.

Leal secures $30M to advance neuro-metabolic therapies

Having raised $80 million over multiple seed funding rounds, Leal Therapeutics Inc. has gone back to the well with a $30 million series A round to support a clinical trial for its lead drug, LTX-001, in patients with schizophrenia and an initial clinical trial for LTX-002 in patients with amyotrophic lateral sclerosis. The programs target enzymes in metabolic pathways, thus reducing high levels of glutamate and sphingolipids, respectively, which are believed to be involved in psychosis and neurodegeneration, making it possible that the drugs could be used for other psychiatric and neurodegenerative diseases.

BioWorld Insider podcast: Bioxcel closer to sNDA for Igalmi

Bioxcel Therapeutics Inc.’s phase III results in bipolar disorders or schizophrenia could bring Igalmi out of the clinic and into patients’ homes for easier treatment. Vimal Mehta, Bioxcel CEO, talks about the potential with BioWorld’s Lee Landenberger.

Also in the news

Antiva, Bayer, Beone, Bioversys, Bioxcel, Catalyst, Clock.bio, Cosmo, Eli Lilly, Excellthera, Grace, Hope, Inovio, Leal, Lineage, Lupin, Mannkind, Maxion, Moleculin, Quell, Remegen, Serb, Treos, United, Vor, Wugen, Zentalis