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The regulatory path for companies developing drugs for rare diseases is often fraught with challenges. KemPharm CEO Travis Mickle discussed just some of these with BioSpace.

Curis announced that the FDA has allowed patient enrollment to resume in the monotherapy phase of its TakeAim Leukemia Phase I/II trial studying emavusertib.

Cassava Sciences has been informed by the Journal of Prevention of Alzheimer’s Disease that there is no evidence to support the charges, the company said Thursday.

This week’s Movers & Shakers include Arvinas, Inspire and Cambridge Isotope Laboratories all announcing new VP roles, and several others tap new C-suite executives.

A fat hormone that slows liver tumor growth, a genetic map to track tumorigenesis, study that shows we inherit more than expected epigenetic information from our mothers, Alzheimer linked APOE gene protects against glaucoma.

A trial evaluating generic drugs against severe COVID-19 provided data showing their overall ineffectiveness, and COVID-19-related lawsuits continue to pile up.

CDC director Dr. Rochelle Walensky announced plans to launch a “reset” of the agency amid criticism over its handling of the COVID-19 pandemic and other public health threats.

South Korea-based Genuv Inc. announced the publication of a preclinical study showing the potential of Mekinist, approved by the FDA for melanoma, in Alzheimer’s disease.

Jazz Pharma is breaking new ground with its cannabidiol epilepsy treatment Epidiolex, taking the drug into a Phase III study in children and adolescents.

Genentech has partnered with Jemincare to develop and commercialize an asset that may provide new treatment options for people with advanced-stage prostate cancer.

Cases of monkeypox are growing at an alarming rate and Bavarian Nordic, the company that owns one of the two approved vaccines, may no longer be able to keep up.

The approval, which was made under Priority Review, is a watershed moment in gene therapy as it marks the first FDA green light for a lentiviral vector.

Expanded sweetener molecule development program aims to broaden access to plant-based sweetening solutions tailored to diverse customer food and beverage categories.

• $5.5 million financing that closed on April 2, 2026 together with $5.7 million in capital raised in Q1-2026, expected to provide Company with cash runway into 2027 • $4.5 million of accrued VAT refunds and R&D refundable tax credits from UK subsidiary begin maturing, including $1.9 million VAT cash refund expected in June 2026 • Successful EMA and Australia TGA (ATGA) meetings aligned around use of existing biomarker and pending release of 3-year overall survival data to support Conditional Marketing Authorisations (CMAs) for OST-HER2 in prevention or delay of recurrence in fully-resected, pulmonary metastatic osteosarcoma (“Metastatic Osteosarcoma”) • 2.5-year overall survival data expected to be released during ASCO 2026 • EMA and ATGA have aligned on key design aspects of confirmatory Phase 3 Metastatic Osteosarcoma protocol required to have commenced prior to grant of CMAs or FDA BLA, including alignment on 3-year overall survival as a primary clinical efficacy endpoint • Upcoming U.S. FDA Type C meeting and U.K. MHRA SAM meeting to align with EMA & ATGA prior to Australia Clinical Trial Notification submission for Phase 3, including alignment on 3-year overall survival as a primary clinical efficacy endpoint. U.S. FDA (“FDA”) Pre-BLA meeting to confirm alignment on surrogate and primary clinical efficacy endpoints with EMA/ATGA, including alignment on 3-year overall survival as a primary clinical efficacy endpoint, and seek decisions on rolling review acceptance and RMAT designation following the EMA and U.K. MHRA granting ATMP designation (EMA/MHRA RMAT equivalent) for OST-HER2 in Metastatic Osteosarcoma • OS Animal Health S-1 filing for proposed ‘Go-Public’ transaction and receipt of SEC comments