uniQure Reports the US FDA BLA Submission for Ifezuntirgene Inilparvovec to Treat Huntington’s Disease

Shots: The US FDA has received a BLA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130) for the treatment of Huntington’s disease, plus a request for Priority Review BLA was supported by the 3yr. data analysis from the P-I/II trial of AMT-130 vs a propensity score-matched external control derived from the Enroll-HD natural history database;…

ByRidhi Rastogi16 hours ago

NEWS

Daiichi Sankyo Doses First Patient in the P-I Trial with DS1025 for Advanced Solid Tumors

Shots: Daiichi Sankyo has dosed the first patient in the P-I trial with DS1025 for the treatment of advanced or metastatic solid tumors with disease progression on or after ≥1 prior line of standard therapy The P-I trial of DS1025 will evaluate safety, tolerability, PK & preliminary efficacy above-mentioned pts (n=~45), assessing safety & tolerability,…

ByRidhi RastogiAugust 28, 2026

NEWS

Dyne Therapeutics Reports IND Clearance to Initiate P-I Trial of DYNE-302 for FSHD

Shots: The US FDA has granted IND clearance to initiate a P-I multiple ascending dose trial assessing DYNE-302 against PBO in ambulatory adults with facioscapulohumeral muscular dystrophy (FSHD) In the first cohort, 9 pts will receive DYNE-302 (1.5 mg/kg, Q4W) or PBO, with higher doses & less frequent dosing planned. Pts completing the PBO-controlled phase…

ByRidhi RastogiJuly 28, 2026

NEWS

Kyowa Kirin Reports the EC Approval for Crysvita for X-Linked Hypophosphataemia

Shots: The EC has approved an expansion of the indication for Crysvita (burosumab) for the treatment of X-linked hypophosphataemia (XLH), to incl. infants from 1mos. to 1yr. of age Approval was supported by data from P-I/II (BUR-CL207) study assessing safety, tolerability, PK & efficacy of burosumab in pts with XLH from birth to 1yr. Trial showed…

ByRidhi RastogiJuly 21, 2026

NEWS

Diagonal Therapeutics Reports First Patient Dosed in P-I/II (DIAMOND) Trial of DIAG723 for HHT

Shots: Diagonal has dosed the first patient with DIAG723, a clustering agonist antibody, in P-I/II (DIAMOND) trial (n=~93) for people living with hereditary hemorrhagic telangiectasia (HHT) Trial will evaluate DIAG723 (SC) vs PBO in 3 parts: Part A (P-I) will assess single ascending doses; Part B (P-II) will evaluate multiple doses over 14wks. for safety…

ByRidhi RastogiJuly 16, 2026

NEWS

Samsung Bioepis Reports Clinical Data on SB27 (Biosimilar, Keytruda)

Shots: Samsung Bioepis has reported the P-I & P-III trial results on SB27, a proposed biosimilar to Keytruda (pembrolizumab), which met their 1EP, with trial completion expected within 2026 The P-I trial (n=163) evaluated PK, efficacy, safety, & immunogenicity of SB27 vs EU & US-sourced Keytruda in resected stage II/IIIA NSCLC pts after adjuvant Pt-based CT,…

ByRidhi RastogiJune 29, 2026

NEWS

Eli Lilly Reports P-I (AJX-101) Trial Data on AJ1-11095 in Previously Treated Myelofibrosis

Shots: Eli Lilly reported the P-I (AJX-101) trial, assessing AJ1-11095 (25, 50, 75, 100, & 125mg, QD) in 23 pts with myelofibrosis who have been failed by a type I JAK2 inhibitor, at EHA 2026 AJ1-11095 achieved a 70% SVR35 & 70% TSS50 rate at Wk. 12, with driver mutation VAF reductions observed in 21 pts;…

ByRidhi RastogiJune 15, 2026

NEWS

Bayer’s Hyrnuo (Sevabertinib) Gains the US FDA Priority Review for 1L Treatment of HER2-Mutated NSCLC

Shots: The US FDA has granted Priority Review to Bayer’s Hyrnuo (sevabertinib) for the 1L treatment of adults with locally advanced or metastatic NSCLC harboring HER2 (ERBB2) TKD-activating mutations & no prior systemic therapy sNDA was supported by preliminary data from Cohort F of the P-I/II (SOHO-01) trial evaluating the efficacy & safety of Hyrnuo…

ByRidhi RastogiMay 19, 2026

NEWS

BeOne Medicines Receives US FDA Accelerated Approval for Beqalzi (Sonrotoclax) to Treat R/R Mantle Cell Lymphoma

Shots: The US FDA has granted accelerated approval to BeOne's Beqalzi for the treatment of adults with r/r mantle cell lymphoma, after ≥2L of systemic therapy, incl. a BTK inhibitor; regulatory review is ongoing in the EU Approval was supported by the global P-I/II (BGB-11417-201) assessing Beqalzi in r/r MCL pts (n=125), which showed 52%…

ByRidhi RastogiMay 14, 2026

NEWS

PulseSight Therapeutics Reports P-I (PST-611-CT1) Trial Data on PST-611 in Dry AMD/Geographic Atrophy

Shots: PulseSight has presented the P-I (PST-611-CT1) trial data assessing two dose levels (low and high) of PST-611 in two successive dose groups involving 6 pts with late-stage dry AMD/Geographic Atrophy, with a 16wk. follow-up, at ARVO 2026 Trial met its 1 & 2EPs, showing favorable safety & tolerability, along with stable BCVA, no SAEs/SUSARs…

ByRidhi RastogiMay 11, 2026

uniQure Reports the US FDA BLA Submission for Ifezuntirgene Inilparvovec to Treat Huntington’s Disease

Daiichi Sankyo Doses First Patient in the P-I Trial with DS1025 for Advanced Solid Tumors

Dyne Therapeutics Reports IND Clearance to Initiate P-I Trial of DYNE-302 for FSHD

Kyowa Kirin Reports the EC Approval for Crysvita for X-Linked Hypophosphataemia

Diagonal Therapeutics Reports First Patient Dosed in P-I/II (DIAMOND) Trial of DIAG723 for HHT

Samsung Bioepis Reports Clinical Data on SB27 (Biosimilar, Keytruda)

Eli Lilly Reports P-I (AJX-101) Trial Data on AJ1-11095 in Previously Treated Myelofibrosis

Bayer’s Hyrnuo (Sevabertinib) Gains the US FDA Priority Review for 1L Treatment of HER2-Mutated NSCLC

BeOne Medicines Receives US FDA Accelerated Approval for Beqalzi (Sonrotoclax) to Treat R/R Mantle Cell Lymphoma

PulseSight Therapeutics Reports P-I (PST-611-CT1) Trial Data on PST-611 in Dry AMD/Geographic Atrophy

Contact US

FOLLOW US