The Daily Biotech Pulse: Merck Releases Twin Dose Of Positive Tidings, Astellas Voluntarily Pauses Gene Therapy Study, Ascendis Offering

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

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs Aug. 31)

• AC Immune SA ACIU (announced mid-stage Alzheimer's drug data)
• Adagio Therapeutics, Inc. ADGI (IPOed Aug. 6)
• Alkermes plc ALKS
• Axonics, Inc. AXNX
• BeyondSpring Inc. BYSI
• Bicycle Therapeutics plc BCYC
• BioLife Solutions, Inc. BLFS
• Bio-Rad Laboratories, Inc. BIO
• Biohaven Pharmaceutical Holding Company Ltd. BHVN
• Celldex Therapeutics, Inc. CLDX
• Cerevel Therapeutics Holdings, Inc. CERE
• DexCom, Inc. DXCM
• Dynavax Technologies Corporation DVAX
• Eagle Pharmaceuticals, Inc. EGRX (reacted to favorable patent ruling)
• Enanta Pharmaceuticals, Inc. ENTA
• Erasca, Inc. ERAS
• Fulcrum Therapeutics, Inc. FULC
• Kezar Life Sciences, Inc. KZR
• MiMedx Group, Inc. MDXG
• Nuvalent, Inc. NUVL
• PDS Biotechnology Corporation PDSB
• Pieris Pharmaceuticals, Inc. PIRS
• Prothena Corporation plc PRTA
• Regeneron Pharmaceuticals, Inc. REGN
• Repligen Corporation RGEN
• SOPHiA GENETICS SA SOPH
• Surmodics, Inc. SRDX
• Syneos Health, Inc. SYNH
• West Pharmaceutical Services, Inc. WST
• Zentalis Pharmaceuticals, Inc. ZNTL

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Aug. 31)

• Burning Rock Biotech Limited BNR (announced its quarterly results)
• Esperion Therapeutics, Inc. ESPR
• Mesoblast Limited MESO (reacted to FDA requesting additional clinical trial for its stem cell therapy in ventilator-dependent patients with moderate or severe acute respiratory distress syndrome results from COVID-19)

Related Link: Rani Therapeutics, RxSight: 2 Bullish Health Care Stock Picks From BofA

Stocks In Focus

Merck's Keytruda Label In Bladder Cancer Expanded; Company Announces Start of Phase 3 Study of Preventative COVID-19 Antiviral Treatment Candidate

Merck & Co., Inc. MRK announced a label update for Keytruda for its indication in first-line advanced urothelial carcinoma in the U.S. The Food and Drug Administration has converted this indication from an accelerated to a full approval. In addition, as part of the label update, this indication has been revised to be for the treatment of patients with locally advanced or metastatic urothelial carcinoma who are not eligible for any platinum-containing chemotherapy.

Previously, Keytruda was indicated for the treatment of patients with locally advanced or mUC who were not eligible for cisplatin-containing chemotherapy and whose tumors expressed PD-L1, as determined by an FDA-approved test, or in patients who were not eligible for any platinum-containing chemotherapy regardless of PD-L1 status.

Separately Merck and Ridgeback Biotherapeutics announced the initiation of the Phase 3 MOVe-AHEAD clinical trial to evaluate molnupiravir, an investigational oral antiviral therapeutic, for the prevention of COVID-19 infection.

The global study is enrolling individuals who are at least 18 years of age and reside in the same household as someone with laboratory-confirmed SARS-CoV-2 infection with symptoms.

The stock was up 0.73% at $76.85 in premarket trading.

Teva's In-licensed Schizophrenia Drug Accepted For Review By FDA

Teva Pharmaceutical Industries Ltd. TEVA and licensing partner Medincell said the FDA accepted its new drug application for TV-46000/mdc-IRM (risperidone extended-release injectable suspension for subcutaneous use) for the treatment of schizophrenia.

Teva will continue to lead the clinical development and regulatory process and be responsible for commercialization of this candidate treatment, with MedinCell eligible for development milestones, royalties on net sales and future commercial milestones.

Teva shares were gaining 1.06% to $9.52 in premarket trading.

Dermata Appoints New CFO To Replace The Retiring Incumbent

Dermata Therapeutics, Inc. DRMA announced the appointment of Kyri Van Hoose as SVP, chief financial officer.
Van Hoose is replacing Thomas Insley, who is retiring as full time CFO after six years, but will continue to work with the company as a financial consultant, the company added.

The stock was down 6.90% at $5.40 in premarket trading.

Astellas Voluntarily Pauses Neuromuscular Disease Gene Therapy Study Due to Safety Concerns

Astellas Pharma Inc. ALPMY announced it has voluntarily paused screening and dosing of additional participants in its ASPIRO clinical trial evaluating AT132 in patients with X-linked Myotubular Myopathy, a rare neuromuscular disease .

This decision follows the reporting of a recent serious adverse event in a study participant due to abnormal liver function tests observed in the weeks following dosing of the AT132 investigational gene therapy product at a lower dose.

Astellas voluntarily halted screening and dosing, reported the SAE to regulatory agencies, and is engaged in dialogue with regulators about this SAE.

Brickell In-licenses Phase 1-ready Program Targeting Autoimmune and Inflammatory Diseases

Brickell Biotech, Inc. BBI said it has entered into a definitive agreement with Voronoi Inc., a platform-based drug discovery company in South Korea, which grants Brickell exclusive, worldwide rights to research, develop and commercialize novel therapeutics generated from a proprietary DYRK1A inhibitor platform.

These novel DYRK1A inhibitors aim to restore immune balance in patients whose immune system has become dysregulated, thus offering large potential across a wide array of autoimmune and inflammatory diseases.

Under the terms of the license agreement, Brickell will make a one-time payment to Voronoi of $2.5 million in cash and $2.5 million in shares of Brickell common stock. Additionally, the company will pay success-based development, regulatory and sales milestone payments as well as tiered royalty payments ranging from low single digits up to 10% of net sales.

Neuronetics Gains On Insider Buying

Neuronetics, Inc. STIM disclosed in a filing that Robert Cascella, a director of the company, purchased 15,000 shares in the company at $6.7233 apiece.

The stock was up 8.93% to $7.32 in premarket trading.

Offerings

Ascendis Pharma A/S ASND, which recently received regulatory approval for its Skytrofa for treating pediatric growth hormone deficiency, said it has commenced an underwritten public offering of $400 million of ADSs, each of which represents one ordinary share of Ascendis.

All of the ADSs are being offered by Ascendis.

In premarket trading, the stock was down 4.07% at $150.35.