Shots: US FDA granted approval to Pfizer’s BEQVEZ, based on BENEGENE-2 (P-III) study (n=45) to treat of Haemophilia B adult patient using factor IX (FIX) prophylaxis therapy, with life-threatening hemorrhage, spontaneous bleeding episodes, no FDA-approved test detected neutralizing antibodies to AAVRh74var capsid; met 1EP of non-inferiority in the ABR of total bleeds Based on durability response of patients to BEQVEZ, Pfizer launching a warranty program to provide greater certainty to payers, maximize access for eligible patients, and financial protection against risk of efficacy failure BEQVE ..read more

Shots: Moderna and OpenAI entered into a collaboration to co-innovate mRNA medicine by integrating generative AI in the future of business and healthcare In early 2023, companies started working together with launch of Moderna's mChat, a ChatGPT, built on top of OpenAI's API Moderna plan to launch multiple products, and AI based collaborations to maximize the impact on patients in next few years Ref: Moderna | Image: Moderna Related News:- Moderna and Merck Report Results from the P-IIb (KEYNOTE-942) Study of mRNA-4157 (V940) + Keytruda (pembrolizumab) for Resected Hig ..read more

This week PharmaShots’ news was all about the updates on Regulatory, Clinical Trials, Pharma, Animal Health & MedTech. Check out our full report below:    Health Canada Accepts Filling and Priority Review of Acadia’s TROFINETIDE for Rett Syndrome Read More: Health Canada                                                                                        &n ..read more

Shots: Vistagen reports P-IIa study (n=10) results, PH15 (1.6µg) shown statistically significant improvement in reaction time vs PBO & caffeine (p<0.001) in sleep-deprived participants at midnight and 3:00 a.m. (most fatigued) with no SAEs P-IIa study evaluating safety, efficacy & tolerability of intranasal PH15(1.6 µg multiple doses or 9.6 µg total dose) vs PBO (nasal spray or oral), or caffeine (single 400 mg oral dose 1 hr before session) in consecutive sleep deprivation sessions set apart 1wk, scheduled for 6:00 p.m., 9:00 p.m., midnight, and 3:00 a.m. Company to further explore ..read more

Shots: Regeneron and Mammoth signed a collaboration to develop and commercialize in vivo CRISPR-based gene editing therapies by using Regeneron’s AAV and antibody engineering and Mammoth’s ultracompact gene editing systems Under agreement, Mammoth will receive $100M includes $95M upfront cash and equity, $370M R&D and commercial milestones per target, plus single digits to mid-teens royalties, with an option to receive co-funding and profit-sharing on majority of collaborative initiatives in exchange of milestones and royalties Regeneron get access to Mammoth’s editing technologies, other ..read more

Shots: Onconic’s JAQBO (zastaprazan citrate) received Korea’s MFDS approval, based on comprehensive data of P-III study from 28 sites of Korea presented at UEG in Oct’23. Data shown effectiveness on erosive esophagitis with 97.9% healing rate, after 8wks Launch of JAQBO is expected in 2024, following its Korean National Healthcare Insurance Reimbursement listing. Jeil Pharmaceutical (Onconic’s parent company) will be responsible for JAQBO's domestic sales In Mar’23, Livzon Pharmaceutical acquired a license to use JAQBO in China for total transaction value of $127.5 million  Ref: Onco ..read more

Shots:  RNAi therapeutics are innovative medications that enable rapid identification of specific inhibitors of disease targets across a wide range of molecule classes. Several biopharma companies are investing in RNAi therapeutics extravagantly to maintain a healthy foothold in innovative medicines  In 2023, the global RNAi drug delivery market size was valued at $84.1B and is anticipated to register a market size of $410.3B by 2032 with a CAGR of 18.17 percent. Like the previous year, Roche retains the topmost position with an R&D expenditure of $15.73B followed by Novartis and ..read more

Shots: US FDA granted ODD and RPDD to Medivir’s MIV-711 for treatment of Legg-Calvé-Perthes Disease (LCPD) Animal model of LCPD shown the ability to prevent femoral head deformity and positive impact on bone degradation biomarker without affecting bone formation. Common consequences of LCPD in children includes Obesity, Depression, and Osteoarthritis With RPDD, MIV-711 is eligible for fast-track review and Priority review voucher post-FDA approval for LCPD Ref: Medivir | Image: Medivir Related News:- Medivir AB Reports the First Patient Dosing of Fostrox + Lenvima in P ..read more

Shots: Labcorp won Invitae’s auction bid, in the sale process under US Bankruptcy Code Section 363 Following the auction, LabCorp will acquire all assets for $239M in cash, plus other non-cash consideration The current hearing date of sale approval is May 6, 2024. Deal closing is expected by Q3’24, with court approval, customary regulatory clearances and closing conditions Ref: Labcorp  | Image: Labcorp  Related News:- Labcorp Launched Neurofilament Light Chain Blood Test to Identify Neurodegenerative Disease PharmaShots! Your go-to media platform for customi ..read more

Shots: Atom Bioscience initiated patient enrolment in the US for a P-IIb/III study of ABP-671 in oral formulation to treat Chronic Gout P-IIb/III study (n=580) evaluates ABP-671 in two parts; Part 1 for safety and efficacy at different doses and regimens vs PBO or allopurinol (SOC) in the US; Part 2 for comparing dose regimens selected from Part 1 with PBO in patients who were not enrolled in Part 1; identified 36 sites in 17 states ABP-671, an orally administered URAT1 inhibitor; preclinical data showed significant efficacy and good safety, whereas existing drugs showed poor efficacy and SAEs ..read more