Breakthrough Therapy Designation granted to zoldonrasib for the treatment of adult patients with KRAS G12D-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC) who have been previously treated with anti-PD-1/PD-L1 therapy and platinum-based chemotherapyFirst Breakthrough Therapy Designation for an investigational drug specifically targeting KRAS G12D mutation in NSCLCRevolution Medicines’ third RAS(ON) inhibitor to receive this designation REDWOOD CITY, Calif., Jan. 08, 2026 (GLOBE NEWS ...

Key Takeaways Alkermes said the FDA granted Breakthrough status to alixorexton for narcolepsy type 1.ALKS' Vibrance-1 phase II study met its primary endpoint, improving wakefulness versus placebo.Alkermes plans a global phase III program on alixorexton for narcolepsy in Q1 2026. Alkermes (ALKS) announced that the FDA has granted Breakthrough Therapy Designation to alixorexton, a novel, investigational, and oral orexin 2 receptor agonist (formerly ALKS 2680), for the treatment of patients with narcolepsy typ ...

Key Takeaways ALT received FDA Breakthrough Therapy Designation for pemvidutide in MASH, boosting shares by 16.52%.The tag is backed by IMPACT data, showing MASH resolution without worsening fibrosis & improved liver markers.ALT plans to begin a phase III MASH study in 2026.Altimmune (ALT) announced that the FDA has granted Breakthrough Therapy Designation to its lead pipeline candidate, pemvidutide, a novel, investigational GLP-1/glucagon dual receptor agonist, for the treatment of patients with metabolic ...

Core Insights - Oculis Holding AG announced that its neuroprotective drug, Privosegtor, has received Breakthrough Therapy Designation from the FDA for the treatment of optic neuritis, addressing significant unmet medical needs in this area [1][7]. Company Overview - Oculis is a global biotechnology company focused on innovation for treating eye diseases, with a pipeline that includes three key development drugs: Privosegtor, OCS-01, and Licaminlimab [15]. - The company is headquartered in Switzerland and has operations in the United States and Iceland, backed by leading global healthcare investment funds [15]. Drug Development and Market Opportunity - Privosegtor is a small molecule peptide mimic that can cross the blood-brain and retinal barriers, potentially becoming the first neuroprotective treatment for optic nerve disorders, with a combined market opportunity of over $7 billion in the U.S. alone [2][5]. - The ACUITY Phase 2 study demonstrated significant improvements in visual acuity with Privosegtor, showing an average increase of 18 letters on the ETDRS visual acuity scale compared to placebo after three months [4][7]. Clinical Trial Insights - The ACUITY study involved 36 eligible patients aged 18 to 60 with recent onset optic neuritis, comparing Privosegtor with placebo alongside intravenous steroids [12][13]. - Results indicated that Privosegtor not only improved visual acuity but also maintained the structural integrity of the retina and optic nerve, which typically suffer damage during acute optic neuritis [4][9]. Regulatory Status - The Breakthrough Therapy Designation is intended to expedite FDA review for drugs that show substantial improvement over existing treatments for serious conditions [14]. - Following a successful meeting with the FDA in 2025, Oculis initiated the PIONEER program, which includes three registration studies to support the approval of Privosegtor for optic neuritis and NAION [5][6].

TARA-002 selected for FDA manufacturing development and readiness pilot programNEW YORK, Jan. 05, 2026 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy and Fast Track designations for TARA-002, the Company’s investigational cell-based therapy, for the treatment of pediatric patients wit ...

Alignment on Phase 3 registrational trial parameters confirmed following receipt of minutes from end-of-phase 2 meeting with U.S. FDAGAITHERSBURG, Md., Jan. 05, 2026 (GLOBE NEWSWIRE) -- Altimmune, Inc. (Nasdaq: ALT), a late clinical-stage biopharmaceutical company developing therapies that address serious liver diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for pemvidutide, a balanced 1:1 glucagon/GLP-1 dual receptor agonist, for the ...

Core Insights - Praxis Precision Medicines received Breakthrough Therapy Designation (BTD) from the FDA for its experimental tremor treatment, ulixacaltamide, leading to a 13.25% increase in stock price [1][5] Company Overview - Praxis specializes in developing therapies for neurological disorders using genetic insights [3] - The company plans to submit a New Drug Application for ulixacaltamide in early 2026 [4] Clinical Development - The BTD was granted based on positive results from two recent phase 3 studies, which aim to accelerate the regulatory review process [4] - The CEO of Praxis highlighted the importance of the BTD in advancing the treatment to patients more quickly [4][7] Market Context - There is a significant unmet need for treatments for essential tremor, affecting approximately 7 million people in the U.S. [6] - Current treatment options for essential tremor are often ineffective and poorly tolerated [6]

U.S. stocks were lower, with the Nasdaq Composite falling more than 100 points on Monday.Shares of DigitalBridge Group Inc (NYSE:DBRG) rose sharply during Monday's session after the company announced it will be acquired by SoftBank Group for a total enterprise value of approximately $4.0 billion.DigitalBridge Group shares jumped 9.9% to $15.30 on Monday.Here are some other big stocks recording gains in today’s session.Eightco Holdings Inc (NASDAQ:ORBS) shares jumped 29.1% to $2.15 after the company announce ...

The Breakthrough Therapy Designation was granted based on the positive topline results from the Essential3 Phase 3 program in essential tremor Praxis remains on track to submit ulixacaltamide NDA in early 2026 based on recently completed pre-NDA meeting with the FDA BOSTON, Dec. 29, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders chara ...

Core Insights - GSK plc announced that the FDA has granted Orphan Drug Designation to its investigational drug GSK'227, now renamed risvutatug rezetecan, for treating small-cell lung cancer (SCLC) [1][7] - The Orphan Drug Designation was based on positive results from the phase I ARTEMIS-001 study, which demonstrated durable responses in patients with extensive-stage SCLC [2] Drug Development and Designations - A phase III study for risvutatug rezetecan in relapsed SCLC commenced in October 2025, with additional phase III studies evaluating the drug for osteosarcoma and early-stage studies for other cancers [3] - Risvutatug rezetecan has received multiple expedited designations, including Breakthrough Therapy Designation from the FDA and PRIME designation from the EMA for various cancer treatments [4] Market Context - Small-cell lung cancer accounts for approximately 13% of all lung cancers in the U.S. and has one of the poorest prognoses among major cancers, with most patients experiencing relapse after initial treatment [5] - GSK's shares have increased by 41.7% over the past year, outperforming the industry average rise of 11.6% [3]