Shots:
The US FDA has accepted the sNDA & granted priority review to mitapivat, under FDA’s accelerated approval pathway, for the treatment of Sickle Cell Disease (PDUFA: Nov 1, 2026)
sNDA was supported by the global P-II/III (RISE UP) trial assessing mitapivat (100mg, PO, BID; n=138) vs PBO (n=69) in 207 pts with SCD (≥16yrs.)…
Shots:
Splash secured exclusive global rights to develop & commercialize CannEpil for drug-resistant epilepsy, refractory epilepsy, seizure disorders, & related neurological conditions, incl. future product improvements & next-generation formulations
Splash secured a $1M strategic investment from C/M Capital Partners, while Mercer Street forgave ~$5M in Argent BioPharma debt and Splash issued $5.5M in preferred equity…
Shots:
Alcon has entered into a non-exclusive collaboration with RxSight to jointly develop adjustable presbyopia-correcting intraocular lenses (PCIOLs)
Under the collaboration, RxSight & Alcon will combine RxSight’s post-operative light-adjustable technology with Alcon’s PCIOL optical designs to co-develop adjustable PCIOLs for optimized cataract surgery outcomes
As per the deal, RxSight will lead development & manufacturing, receiving…
Shots:
Ionis has completed enrollment in Cohort 1 (n=136; 2 to <18yrs.) of the global P-III (REVEAL) study assessing obudanersen (ION582) in pts with AS with genetic confirmation of either a UBE3A deletion or UBE3A mutation
The Cohort 2 comprises adult pts (18 to ≤50yrs.) living with AS, with enrollment expected to be completed in…
Shots:
The EC has approved Tepkinly (epcoritamab) in combination with lenalidomide and rituximab (Tepkinly + R2) for the treatment of adults with R/R follicular lymphoma (FL)
Approval was based on the P-III (EPCORE FL-1) trial assessing fixed-duration Tepkinly + R2 (n=243) vs SoC R2 (n=245) in pts with R/R FL following ≥1 prior line of treatment
Trial…
Shots:
Vertex has entered into a definitive agreement to acquire Crinetics, incl. its assets Palsonify (paltusotine) & atumelnant, accelerating Vertex’s revenue growth and enhancing its long-term earnings profile
As per the deal, Vertex to acquire Crinetics for $85/share in cash, representing the total equity value of ~$10B, or ~$8.8B net of estimated cash acquired
Palsonify (QD, PO)…
Shots:
The US FDA has accepted NDA for deucrictibant immediate-release (IR) capsule (20 mg) for the on-demand treatment of Hereditary Angioedema (HAE) attacks (PDUFA: Apr 23, 2027)
NDA was backed by data from >1,300 HAE attacks. In P-III (RAPIDe-3) trial, deucrictibant IR met the 1EP & all eleven 2EPs, with median times to symptom relief,…
Shots:
Natera has reported the publication of data on Signatera, a personalized MRD test for resected colorectal liver metastases (CRLM), in JAMA Oncology, with findings also presented at the ESMO GI 2026
The analysis incl. 298 pts from the GALAXY trial, an observational arm of CIRCULATE-Japan, assessing outcomes by MRD status & adj. CT (ACT) use…
Shots:
Astex has entered into an exclusive, worldwide research collaboration & license agreement with Genentech to identify small molecules with selective inhibitory activity for the treatment for breast cancer
Astex will license compounds from its breast cancer discovery program to Genentech and jointly optimize lead compounds through preclinical candidate selection, after which Genentech will lead…
Shots:
Sanyou & Baiyunshan Xihe have entered into a strategic collaboration to co-develop novel radiopharmaceuticals & radiodiagnostic products by combining AI-driven drug discovery with nuclear medicine expertise
As per the deal, Sanyou Bio will contribute its AI-STAL libraries & AI-Drug Accelerator platform for target molecule discovery, while Baiyunshan Xihe will lead radionuclide production, radiolabeling, preclinical…

