Rhythm Pharmaceuticals Inc.’s Post

Kiora Pharmaceuticals, Inc. (NASDAQ:KPRX) CEO Brian Strem joined Steve Darling from Proactive to share news the company's plan to broaden the clinical development of KIO-301, a treatment designed for various inherited retinal diseases. The expansion includes initiating a Phase 2 trial which will be controlled, double-masked, randomized, and will involve an ascending-dose. This trial is set to include up to 20 patients suffering from Retinitis Pigmentosa (RP), who will receive doses monthly over a span of 90 days. Strem explained that this move is propelled by encouraging topline results from a Phase I/II clinical trial, which indicated that KIO-301 is a first-in-class small molecule with significant potential to improve vision for patients with RP, even those with extremely low vision or complete blindness. Moreover, Kiora Pharmaceuticals has plans to engage with the FDA in the fourth quarter of 2023. Watch at #Proactive #ProactiveInvestors #NASDAQ #KPRX http://ow.ly/2e8m1052gy1

Alcoholic Hepatitis Market Size in the 7MM is estimated to Increase by 2032 | Durect, Gilead Sciences, Novartis Pharmaceuticals, PharmaKing, Vital Therapies Inc, Intercept Pharmaceuticals Inc, Immuron Ltd, Mallinckrodt, Boehringer Ingelheim, and others. https://lnkd.in/ecEvEmst #marketanalysis #marketresearch #marketresearchreports #businessintelligence

I am excited to share that my article Why Oncology Global Safety Teams Should Develop the Safety Section of the Study’s Target Product Profile (TPP) was published online ahead of print on 27 February 2024 by the Springer Nature journal Pharmaceutical Medicine. This is my first sole author publication and the first publication for Focused Consulting, Staffing, & Drug Safety, LLC. Please see the article attached in this post.

BioPharmCatalyst currently has 56 upcoming events within the PDUFA, PDUFA Priority Review, and/or Advisory Committee categories over the next several years. Check out our PDUFA calendar to stay on top of all of these major regulatory decisions. #biotech #biotechinvesting #fdacalendar #regulatory #biotechstocks #pharmainvesting #pdufa #catalyst #betherewhenithappens #fasterscience

https://lnkd.in/e6mJGyHD Novartis is close to clinching an acquisition of Cytokinetics and its promising heart drug, as big pharmaceutical companies continue to snap up fast-growing biotechs to replenish their pipelines. A purchase by the Swiss drug company of South San Francisco-based Cytokinetics, which has a market value of more than $10 billion, could be completed as soon as this week, according to people familiar with the situation. Cytokinetics is focused on so-called muscle activators and inhibitors as potential treatments for people with debilitating diseases in which muscle performance is compromised, according to its website. Cytokinetics’ #cardiomyopathy therapy would complement drugs like the cholesterol treatment called #Leqvio that Novartis acquired as part of a nearly $10 billion deal for The Medicines Company in 2020. #BigPharma

Last March, the EFPIA (European Federation of Pharmaceutical Industries and Associations) published the results of a simulation exercise aiming at assessing the potential impact of the methods proposed by EUnetHTA21 guidelines for JCAs (Joint Clinical Assessment) applied to three currently approved and reimbursed oncology products, namely Products X, Y & Z. The simulated scoping process estimated a total of 16, 22 and 57 PICOs for Products X, Y & Z respectively, reduced down to 7, 6 & 23 when removing single country comparator requests. Following this simulation, the authors made a total of 5 recommendations aimed at the European Commission and Member States representatives to help supporting the implementation of the EU HTA regulation next January. Source: https://lnkd.in/emr3-6JP #hta #healthtechnologyassessment #euhta #heor #healtheconomics #pharma #access #marketaccess

Cough in Idiopathic Pulmonary Fibrosis Market Size in the 7MM is estimated to Increase by 2032 | Nerre Therapeutics Ltd., Pharm-Olam International, Trevi Therapeutics, Parexel, Afferent Pharmaceuticals Inc., and others. https://lnkd.in/eHZUnUyu #marketanalysis #marketresearch #marketresearchreports #businessintelligence

IgA Nephropathy Market to Exhibit Rapid Growth Rate | Chinook Therapeutics, RemeGen, Novartis, Ionis Pharma, Vera Therapeutics, Eledon Pharma, Guangdong Hengrui Pharma, companies are expected to change the IgA Nephropathy Market scenario by 2032, forecas https://lnkd.in/g_U6Fv3a #marketanalysis #marketresearch #marketresearchreports #businessintelligence

A Bear Bile Acid Goes Bust After Controversial Approval: Taurursodiol + Phenylbutyrate | https://lnkd.in/gx3MkAfN Relyvrio, developed by Amylyx Pharmaceuticals, is a combination treatment of 3 g sodium phenylbutyrate and 1 g taurursodiol, which was originally approved by the FDA in Sep. 2022 for amyotrophic lateral sclerosis (ALS) (“Lou Gehrig’s disease”) in adults. Now, Ph. III data have confirmed the drug is no better than placebo in ALS, turning this phenotypic drug discovery story from two undergrads into a post-mortem highlighting the importance of clearly defined molecular mechanisms of action. This article reviews what happened and what we can learn from the situation. Full article: https://lnkd.in/gx3MkAfN

New progress from Gan & Lee Pharmaceuticals & Lee - their glucagon-like peptide-1 receptor agonist GZR18 has reached the primary endpoint and all critical secondary endpoints in the Phase Ib/IIa clinical study. The complete data from the trial is set to be unveiled at this year's ADA and EASD annual meetings. #QMportfolio #QMhealthcare #ClinicalTrials #MedicalResearch #DiabetesCare #GanAndLee