Pharmaceuticals

SHERIDAN, WYOMING – July 8, 2025 – BrainStorm Cell Therapeutics is once again at the center of the amyotrophic lateral sclerosis (ALS) treatment debate as patients formally petition the FDA to reconsider the approval of its investigational stem cell therapy, NurOwn. Citing new survival data and expanded access outcomes, the Citizens’ Petition seeks regulatory reassessment of the once-rejected therapy, underscoring the shifting landscape of ALS drug development and patient advocacy.

Renewed Push for Regulatory Reevaluation

The Citizens’ Petition, submitted last week by several individuals living with ALS, requests that the agency approve the stem cell therapy based on “new evidence and totality of evidence.”

SHERIDAN, WYOMING – July 8, 2025 – Regeneron has received accelerated approval from the U.S. Food and Drug Administration (FDA) for its bispecific antibody linvoseltamab, now branded as Lynozyfic, for the treatment of patients with relapsed or refractory multiple myeloma—unlocking a market potential estimated at $600 million.

While the therapy enters a space already led by Johnson & Johnson’s Tecvayli, approved in 2022, Regeneron is seeking to distinguish Lynozyfic through its efficacy profile and flexible dosing advantages. According to analysts at BMO Capital Markets, the approval is a “small win” for Regeneron, noting that Lynozyfic will be a “minor contributor to the company’s broader product portfolio.” The firm estimates around $600 million in potential revenues from the drug.

SHERIDAN, WYOMING – July 8, 2025 – In a landmark agreement that underscores the growing convergence of artificial intelligence and drug discovery, Tokyo-based Chugai Pharmaceutical has entered into a multi-phase research collaboration with Singapore-headquartered biotech Gero to target age-related diseases using AI-driven insights. The deal could exceed $1 billion in total value, positioning both companies at the forefront of longevity-focused pharmaceutical innovation.

AI-Powered Discovery for High-Impact Biologics

Chugai, majority-owned by Roche, will leverage Gero’s proprietary AI target discovery platform to identify novel biological targets linked to aging-related conditions. Based on these targets, Chugai will engineer antibody-based drug candidates using its advanced internal capabilities.

SHERIDAN, WYOMING – July 8, 2025 – Cogent Biosciences is accelerating its strategic trajectory with plans to submit its first New Drug Application (NDA) to the FDA by year-end, following strong Phase II data for its investigational tyrosine kinase inhibitor, bezuclastinib. The move positions Cogent as a serious contender in the systemic mastocytosis treatment landscape, directly challenging Blueprint Medicines' market presence.

Strategic Differentiation in a Competitive Field

SHERIDAN, WYOMING – July 8, 2025 – KalVista Pharmaceuticals has received U.S. FDA approval for its oral treatment Ekterly (sebetralstat), marking a major milestone as the first and only on-demand tablet therapy for hereditary angioedema (HAE) in patients aged 12 and older.

The company confirmed that the drug will be available in the U.S. “immediately” under the brand name Ekterly. According to KalVista’s press release, it is “the first and so far only oral and on-demand treatment for hereditary angioedema (HAE).”

Regulatory delays and internal agency tensions

Ekterly’s road to approval was not smooth. Last month, the FDA informed KalVista that it would not meet its original target action date of June 17, citing “heavy workload and limited resources,” according to the biotech’s announcement at the time.

SHERIDAN, WYOMING – July 8, 2025 – The TIGIT immunotherapy landscape has thinned dramatically amid a string of late-stage failures and corporate exits, yet several biopharma players remain committed to unlocking its clinical promise—driven by differentiated approaches and early signals of efficacy. AstraZeneca, Gilead, Agenus, and Mereo BioPharma are now at the forefront of this once-promising immuno-oncology frontier, recalibrating strategies in a space shaped by volatility and unmet potential.

AstraZeneca Bets Big on Dual Checkpoint Innovation

AstraZeneca’s rilvegostomig is one of the most advanced TIGIT assets in development today. The bispecific antibody is currently undergoing evaluation in ten Phase III trials across non-small cell lung cancer (NSCLC), gastric cancer, biliary tract cancer, and other solid tumors.

SHERIDAN, WYOMING – July 8, 2025 – A confluence of artificial intelligence and genomics is creating a critical inflection point for the pharmaceutical industry—offering a long-awaited opportunity to reduce adverse drug reactions, improve clinical outcomes, and restore public trust in a sector burdened by scrutiny and skepticism.

As adverse drug reactions remain among the top five causes of death in the U.S.—with more than 150,000 fatalities annually and billions in associated healthcare costs—pharmacogenetics is emerging as a strategic imperative rather than a research niche. But until recently, scaling this precision approach to drug prescription had remained elusive due to technical limitations. Today, AI-enabled analysis of vast, real-world genomic datasets is transforming that landscape.

SHERIDAN, WYOMING – July 1, 2025 – The U.S. Food and Drug Administration’s recent decision to remove Risk Evaluation and Mitigation Strategies (REMS) from approved CAR T cell therapies marks a pivotal shift that could reshape access to these potentially curative treatments and drive significant market expansion across the cell and gene therapy industry.

SHERIDAN, WYOMING – July 1, 2025 – Neurogene has reached alignment with the FDA regarding the design of a registrational study for the investigational gene therapy NGN-401 for Rett Syndrome, enabling the company to convert its current Phase I/II study into a pivotal trial—a milestone that analysts describe as the “best-case scenario.”

The FDA has allowed Neurogene to run “a single-arm and baseline-controlled study with female patients aged three years and up,” according to the company’s announcement. NGN-401 will be administered at a single dose, with the trial assessing treatment responders as measured by the Clinical Global Impression-Improvement (CGI-I) scale and the achievement of developmental milestones or skills.

Analysts Call Agreement the ‘Best-Case Scenario’

SHERIDAN, WYOMING – July 1, 2025 – Argenx has taken a decisive step to diversify its pipeline beyond its flagship FcRn blocker, signing a multi-target research agreement with Unnatural Products, Inc. (UNP) that could exceed $1.5 billion. The deal aims to harness UNP’s proprietary macrocyclic peptide technology to develop oral therapies for disease targets historically deemed “undruggable,” marking the largest licensing transaction to date in the macrocyclic peptide space.

Strategic Expansion Beyond FcRn Blockade