Shots: Endeavor BioMedicines has dosed the first patient in its P-IIb (WHISTLE-PF) trial, evaluating taladegib (ENV-101), a Hedgehog pathway inhibitor, as a treatment for idiopathic pulmonary fibrosis (IPF). It also showed its effectiveness in its P-IIa study The P-IIb study assesses efficacy, safety & patient-reported outcomes of ENV-101 vs PBO as well as its effects on lung capacity and lung fibrosis, using HRCT imaging, in IPF patients across 14 countries incl. Australia for over 24wks. The P-IIa study results for ENV-101 demonstrated significantly improved lung function and total lung ..read more

Shots:    The EMA’s CHMP has granted positive opinions to 5 Biologics and 1 New Chemical Entity in October 2024, leading to treatments for patients and advances in the healthcare industry   The major highlighted drugs were Novo Nordisk’s Alhemo to treat Haemophilia A or B with inhibitors and AstraZeneca & Ionis’ Wainzua for Hereditary Transthyretin-Mediated Amyloidosis  PharmaShots has compiled a list of 4 drugs that have been granted positive opinion by the EMA’s CHMP    1. AstraZeneca and Ionis’ Wainzua (Eplontersen) Receives the CHM ..read more

Shots: Alloy Therapeutics has entered into a strategic collaboration and licensing agreement with Takeda to develop the latter’s induced pluripotent stem cell (iPSC) driven CAR-T cell platform (iCAR-T) and CAR-NK platform (iCAR-NK) As per the agreement, Alloy will advance therapies for solid and hematological cancers, securing co-exclusive rights to commercialize iCAR-T and iCAR-NK products for the same indication Alloy will enhance the iCAR-T/NK platform using synergies across its unique business model, enabling broader access for biotech and pharma partners to develop cancer therapies, while ..read more

Shots: The EMA has granted PRIME designation to NX-5948 for adults with r/r chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) treated with at least a BTK or a BCL-2 inhibitor NX-5948 is currently being assessed under P-I study for its safety & anti-cancer activity for treating r/r B cell malignancies, with further pivotal studies anticipated in 2025 NX-5948 (oral) is a small molecule that works by degrading BTK, responsible for growth signaling in B-cell. It has depicted the ability to cross blood-brain barrier in animal models, showing potential for CNS lymphoma & b ..read more

Shots: The US FDA has granted approval to Bimzelx (IL-17F & IL-17A inhibitor) for treating adults with moderate to severe hidradenitis suppurativa (HS), based on P-III (BE HEARD I and BE HEARD II) studies BE HEARD I and BE HEARD II assessed the safety & efficacy of Bimzelx vs PBO for treating moderate to severe hidradenitis suppurativa (HS) in 1,014 patients, combined Study depicted >50% improvement in HS signs and symptoms as assessed by HiSCR50 (1EP) & meaningful improvements in HiSCR75 (2EP) at wk.16, with responses maintained through wk.48; safety profile was consistent. Res ..read more

Shots: The US FDA’s accelerated approval of Ziihera (50mg/mL, IV) was based on P-IIb (HERIZON-BTC-01) study in patients (n=87) with HER2+, locally advanced unresectable or metastatic BTC across 2 arms, based on tumor IHC status Efficacy in 62 HER2+ BTC patients (arm 1 of HERIZON-BTC-01) showed a 52% ORR & 14.9mos. mDoR. Continued approval depends on P-III (HERIZON-BTC-302) confirmatory trial of zanidatamab + SoC vs SoC alone in 1L HER2+ BTC Zanidatamab is also being studied P-III (HERIZON-GEA-01) trial with CT & with/without tislelizumab for 1L HER2+ GEAs as well as P-III (EmpowHE ..read more

Shots: Pharmanovia has entered into a new licensing agreement with Lindis Biotech for the commercialization of catumaxomab to treat malignant ascites Pharmanovia secured exclusive rights to commercialize catumaxomab, a trifunctional bi-specific mAb for intraperitoneal treatment of malignant ascites in adults with EpCAM-positive carcinomas, ineligible for further systemic therapy Catumaxomab was granted with the CHMP’s positive opinion in Oct 2024, with the EC’s marketing authorization expected by YE’24 Ref: Businesswire  | Image: Pharmanovia & Lindis Biotech Related New ..read more

Shots: The US FDA has cleared IND application of ST-503 to initiate its P-I/II clinical evaluation to treat intractable pain due to idiopathic small fiber neuropathy The P-I/II trial will assess safety, tolerability & efficacy of ST-503 (intrathecal) to treat intractable pain from iSFN, a peripheral neuropathy causing severe pain symptoms, with the patient recruitment planned during mid-2025 ST-503 uses an AAV vector with a zinc finger repressor to target SCN9A, encoding the Nav1.7 sodium channel. It has selectively reduced Nav1.7 expression in sensory neurons, significantly reducing pain ..read more

Shots: The EC has approved Hympavzi (QW, SC) as a prophylactic treatment to prevent bleeding episodes in patients (≥12yrs., at least 35kg) with hemophilia A & B, without FVIII & FIX inhibitors, respectively. It is valid across the EU plus Iceland, Liechtenstein & Norway Approval was based on pivotal P-III (BASIS) study of Hympavzi in patients (12-75yrs.) with severe hemophilia A or mod. severe to severe hemophilia B with/without inhibitors Study showed a reduction in the annualized bleeding rate (ABR) by 35% (5.08 vs 7.85) during 12mos. with Hympavzi vs routine prophylaxis & on ..read more

Shots: The P-III (ESSENCE) study assessed semaglutide (2.4mg, QW) vs PBO in MASH adults (n=1,200) with liver fibrosis (stage 2/3), for 240wks., with part 1 aiming to show improved liver histology at 72wks. in first 800 randomized patients, while part 2 assessing long-term liver-related outcomes Study met its 1EP, depicting steatohepatitis resolution without fibrosis worsening in 62.9% vs 34.1% & improved fibrosis without steatohepatitis worsening in 37% vs 22.5%. 2EPs showed both steatohepatitis resolution & improved fibrosis in 32.8% vs 16.2% Trial also depicted histological benefits ..read more