Here's a roundup of top developments in the biotech space over the last 24 hours:

Stocks In Focus

FDA AdComm Gives Thumbs Down To Acadia's Pimavanserin

A panel of the FDA's outside experts voted 9-3 that Acadia Pharmaceuticals Inc's ACAD pimavanserin doesn't appear to be effective at treating patients with Alzheimer's-related psychosis.

The drug, Nuplazid, is already indicated for Parkinson's-related psychosis.

Acadia tried and failed last year to get another FDA approval for Alzheimer's psychosis. 

Shares are down 32.29% at $13.25 during the premarket session.

AstraZeneca - Ionis Eplontersen Achieves Positive Data In Rare Disease Trial

AstraZeneca plc AZN and partner Ionis Pharmaceuticals Inc IONS announced a positive interim analysis of eplontersen in hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).

In Phase 3 NEURO-TTRansform study, eplontersen met the main goals.

Eplontersen also showed significantly improved patient-reported quality of life versus the historical placebo group.

Pharma Giant Pfizer Buys 8% Stake In Valneva

Pfizer Inc PFE will invest €90.5 million ($95 million) for an 8.1% stake in Valneva SE VALN, as the companies updated the terms of agreement for Lyme disease vaccine candidate VLA15.

Under the revised deal, Valneva will now fund 40% of the remaining shared development costs, compared to 30% before.

Pfizer will pay Valneva tiered royalties ranging from 14% to 22%. In addition, the royalties will be complemented by up to $100 million in milestones payable to Valneva based on cumulative sales.

VALN shares are up 85% at $25.40 during the premarket session.

Recursion Pharma Starts Late-Stage Brain Cancer Trial Of REC-2282 

Recursion Pharmaceuticals Inc RXRX initiated Phase 2/3 POPLAR-NF2 trial of REC-2282 for progressive neurofibromatosis type 2-mutated meningiomas, tumors that form on the membrane that covers the brain and spinal cord.

The trial is designed to investigate the safety, efficacy, and pharmacokinetics of REC-2282. 

The study is expected to enroll approximately 90 participants.

Hepion Pharma's Liver Cancer Candidate Secures Orphan Drug Tag

The FDA has granted Orphan Drug Designation to Hepion Pharmaceuticals Inc's HEPA rencofilstat, a liver-targeting, orally-administered, novel cyclophilin inhibitor for hepatocellular carcinoma.

"In addition to two Phase 2 studies in patients with NASH, we remain on track to initiate patient enrollment in a Phase 2a study of rencofilstat in HCC in the third quarter of 2022," said Robert Foster, Hepion's CEO.

Iktos, Zealand Pharma Ink AI Technology Pact For Peptide Drug Design

Iktos, a specialized Artificial Intelligence company for new drug design, announced a research collaboration with Zealand Pharma A/S ZEAL to co-develop generative and predictive AI technologies for peptide drug design.

Iktos' AI technology, based on a comprehensive data-driven chemical structure generation technology, automatically designs virtual novel molecules with all the characteristics of a successful drug molecule. 

XBiotech Concludes Phase 1 Portion of Pancreatic Cancer Study

XBiotech Inc XBIT completed the Phase 1 portion of its 1-BETTER study, a Phase 1/2 to evaluate its anti-cancer drug Natrunix in combination with chemotherapy for pancreatic cancer. 

Enrollment in the Phase 2 portion is commencing immediately.

Key endpoints in the Phase 2 portion will be progression-free survival, overall survival, and time-to-treatment-failure. 

The Phase 2 portion is enrolling 60 subjects.

Midatech Pharma's Pediatric Brain Cancer Candidate Gets Orphan Drug Status In Europe

The European Medicines Agency has granted orphan drug designation to Midatech Pharma PLC's MTP MTX110 for glioma.

Midatech has been developing MTX110 for recurrent glioblastoma in adult patients and diffuse intrinsic pontine glioma and medulloblastoma in pediatric patients.

Edgewise's Dystrophy Candidate Lowers Muscle Damage Biomarkers

Edgewise Therapeutics Inc EWTX announced 2-month interim results from the ARCH study of EDG-5506 in adults with Becker Muscular Dystrophy. 

EDG-5506 at 10 mg showed a significant decrease in key biomarkers of muscle damage. Notably, creatine kinase and fast skeletal muscle troponin I were reduced by an average of 30% and 68%, respectively.

All patients have subsequently been dose-escalated to 15 mg daily as per protocol.

Clinical Readouts/Presentations

Krystal Biotech Inc KRYS: Phase 3 safety and immunogenicity results of beremagene geperpavec (B-VEC), topical gene therapy for dystrophic epidermolysis bullosa at the Society for Investigative Dermatology 2022 Annual Meeting.

Aura Biosciences Inc AURA: Final safety and efficacy data from phase 1b/2 trial of AU-011 for choroidal melanoma via intravitreal administration at the International Society of Ocular Oncology.

On The Radar

Earnings

Acasti Pharma Inc ACST: Before the market open.