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

Shots: Vertex will own exclusive license for Treefrog’s cell manufacturing technology, C-Stem As per the agreement, TreeFrog will receive $25M as upfront payment, ~$540M for clinical, regulatory and commercial milestones on up to two products, ~215M as milestone payment on development of fully differentiated Islet cells, an equity investment from Vertex and a tiered single digit Royalties; Vertex to also fund future R&D cost related to collaboration C-Stem imitates natural microenvironment allowing cell to grow exponentially in 3D and Vertex plans to use C-Stem to scale up production of In ..read more

Shots: GENETIKA+ enters into a multiphasic collaboration with NeuroSense for its ongoing P-II study in patients with Alzheimer's in this Genetika will use its technology to quantify neuronal plasticity from frontal cortex neurons derived from patient's blood  NeuroSense’s PrimeC therapy addresses multiple disease related pathologies like Aβ aggregation and TDP-43 simultaneously making it different, unique, more potent approach for treatment outcomes from conventional method that just targets amyloid-beta (Aβ) Currently enrolling at the Stroke and Cognition Institute (Israel) for P-II  ..read more

Shots: In this multi-year, multi-target collaboration, Ochre Bio’s discovery platform combines machine learning with imaging and deep genomic phenotyping, RNA chemistry, and ex-vivo human-organ perfusion models to discover multiple novel regenerative targets for chronic liver diseases (CLDs) BI aims to develop a treatment that is capable of increasing the liver’s self-repair capability which could eventually reduce disease progression by modifying regenerative targets Ochre Bio will receive total deal value >$1B, includes $35M upfront and R&D milestone, and an undisclosed clinical, regu ..read more

Shots: Health Canada accepted New Drug Submission (NDS) for Acadia’s Trofinetide to treat Rett Syndrome; also granted priority review P-III LAVENDER study (n=187) evaluated safety and efficacy of Trofinetide in Rett Syndrome girls & young women; At wk12, co-1EP’s RSBQ score, and CGI-I score, were statistically significant; 2EP: statistically significant measuring improvements in communication Trofinetide is currently not permitted for sale in Canada, but has been approved in US for adults and pediatric patients aged >2yrs Ref: Health Canada | Image: Health Canada Rel ..read more