Shots: The MHRA has granted marketing authorization to Ascendis’ Yorvipath (Palopegteriparatide) for the treatment of adults with chronic hypoparathyroidism, along with ODD (10 yrs. market exclusivity in Great Britain) MHRA approval is based on same dossier submitted for Yorvipath’s EU approval in Nov’23 Yorvipath (developed as TransCon PTH), a prodrug of parathyroid hormone (PTH 1-34) administered QD; also being developed in US, Japan and other countries for chronic hypoparathyroidism Ref: Ascendis Pharma | Image: Ascendis Pharma Related News:- Ascendis Enters into Collabo ..read more

Shots: An American investment firm, OrbiMed Advisor is a prominent name when it comes to healthcare investments. The company boasts a robust portfolio of more than 180 companies, including AbCellera, Acelyrin, Actus Medical, and Adaptive Biotechnologies, among others In 2023, OrbiMed invested in 38 funding rounds; 11, 6, 9 & 12 every quarter across 2023 respectively For a detailed report on all the investments, reach out to us at connect@pharmashots.com   OrbiMed Advisor Founded in 1989, Orbimed Advisor is a global healthcare investment firm, led by 21 partners. The company's investme ..read more

Shots: FILSPARI received conditional marketing authorization (CMA) from European Commission for treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (Urine Protein: Creatinine > 0.75g/g) The approval is based on PROTECT (P-III) study, evaluating safety and efficacy of sparsentan (400 mg) vs irbesartan (300 mg). Study met 1EPs: statistical significance at pre specified interim analysis; mean reduction in proteinuria with Sparsentan (49.8%) and Irbesartan (15.1%) after 36wks First EU market launch of FILSPARI, a non-immunosuppressive therapy is expected in H2’24 Ref ..read more

Shots: Neurocrine reports results from SAVITRI, a P-II study (n=183), evaluating safety and efficacy of NBI-1065845 (AMPA) in adults with MDD who had improper response to other MDD treatment Study met both 1EP and 2EP, showing significant reduction in MADRS score at D28 (Dose 1: -4.3; Other dose: -3) and D56 (Dose 1: -7.5; Other dose: -3.6), respectively. Common AE observed is headache, no deaths or SAEs; low discontinuation rate. Based on P-II data, company to discuss P-III studies path at FDA meeting Ref: Neurocrine Biosciences | Image: Neurocrine Biosciences Related News ..read more

Shots: Astellas received label extension approval of Xtandi as monothx or in combo with ADT to treat unsuitable salvage-radiotherapy, adult men with high-risk BCR nmHSPC The approval was based on EMBARK (P-III) study (n=1,068). Xtandi (160 mg) in combo with leuprolide (n=355) reduced cancer spreading risk or death by 57.6% and Xtandi alone (160 mg) (n=355) reduced risk by 36.9% vs PBO with 22.5 mg leuprolide (n=358) at every 12wks. Study met its both 1EP (MFS) and 2EP (reduction in risk of metastasis or death) Revised guidelines of EAU in Apr’24, recommends Xtandi in high-risk BCR nm ..read more

Shots: Incyte to acquire Escient Pharmaceuticals for $750 M plus Escient’s remaining net cash at closing of transaction (based on customary adjustments). Closing of acquisition is anticipated in Q3’24 Acquisition will enhance Incyte’s R&D activities in Inflammation and Autoimmunity (IAI) by addition of Escient's MRGPR antagonists, EP262 and EP547; possible launch begins in 2029 Moreover, EP262 in the P-I study showed safety and tolerability at all doses tested with no AEs & SAEs, vital signs, or ECG parameters, whereas TEAEs were mild having incidence lesser than PBO (33.3% vs. 62.5 ..read more

Shots: US FDA granted Fast Track designation to Medicure’s MC-1 for seizures associated with PNPO deficiency. Also received Rare Pediatric Disease Designation and ODD by US FDA; ODD by EMA Medicure is in launch phase of P-III study, daily dose of MC-1 will be administered to approx. 10 PNPO patients in US and Australia If FDA approves NDA for MC-1, Medicure will receive Priority Review voucher which can be redeemed or sold Ref: Medicure | Image: Medicure Related News:- Takeda’s Entyvio Receives the US FDA’s Approval for Subcutaneous Administration to Treat Moderately t ..read more

Shots: Krystal has begun first patient dosing of KYANITE-1 (P-I) study of Inhaled KB707 monotherapy in an open label trial to treat LA/M solid tumors of the lung.  Krystal Biotech received two US FDA FTDs for KB707 program: intratumoral use for anti-PD-1 r/r melanoma in Jul 2023 and inhaled use for solid tumors with pulmonary metastases in Feb 2024 Inhaled KB707 is a modified HSV-1 vector designed to enter into the lung and deliver genes encoding both human IL-12 and IL-2. Krystal Biotech is expecting readout data in later 2024 Ref: Krystal Biotech | Image: Krystal Bio ..read more

Shots:   Raviv Pryluk, CEO and Co-Founder of PhaseV, in a stimulating conversation with PharmaShots, sheds light on the company’s recent partnership with 9xchange Biopharma    The partnership aims to bring Phase V’s proprietary machine learning (ML) technology to ecosystem members, enabling them, to retrospectively analyze and optimally design and execute advanced clinical studies   Raviv highlights the importance of machine learning in clinical trial optimization and significantly reducing cost and time   Saurabh: Congratulations on your partnershi ..read more

Shots: The P-III trial (LUNA 3) evaluated the safety & efficacy of rilzabrutinib (400mg, BID) vs. PBO (12–24 wks. double blinded followed by 28 wks. open label), followed by a safety follow-up of extension period of 4wks. The LUNA 3 trial achieved its 1EP, showing a notably greater percentage of patients on rilzabrutinib vs. PBO, a durable platelet response (during the treatment period without rescue therapy, ≥50,000/μL for 8 of 12 wks. of the 24-wks. blinded). 2EPs highlight the possibility that rilzabrutinib will help individuals with persistent and chronic ITP in a clinically significan ..read more