Regenxbio Product RGX-121 Achieves Primary Endpoint From Regenxbio (RGNX) we learned that results from the Phase I/II/III CAMPSIITE® trial of the firm’s product RGX-121 for the treatment of 5 years old patients diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, demonstrated that the pivotal phase of the trial met its primary . . . This content is for paid subscribers. Please …

Madrigal Pharmaceuticals Publication in the New England Journal of Medicine Resmetirom is the first investigational medicine for NASH to achieve fibrosis improvement and NASH resolution primary endpoints in a Phase 3 trial Detailed analyses reinforce the safety profile of resmetirom Resmetirom has the potential to become the first and only medicine approved for NASH; PDUFA date is March 14, 2024 Today, February 8, 2024, Madrigal …

MiNK Therapeutics in the NEWS  Today, February 6, 2024, MiNK Therapeutics (INKT) announced the publication of the results in Nature Communications from phase 1/2 study of agenT-797 . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Vera Therapeutics to Participate in Guggenheim Healthcare Talks February 2, 2024, Vera Therapeutics, Inc. (VERA) announced that the Company’s management team will participate in the 6th Annual Guggenheim Biotechnology Conference, which is taking place in New York in February 7and Feb 8. Fireside Chat Details: Date: Wednesday, February 7th at 2:00 PM ET The . . . This content is for paid subscribers. Please click here …

Intellia Therapeutics in the NEWS Yesterday, January 31, Intellia Therapeutics (NTLA) announced that the results from Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published online in the New England Journal of Medicine (

Abbott Laboratories New PROTALITY™ for Maintaining Muscle Mass During Weight Loss Abbott Laboratories (ABT) announced the launch of its new PROTALITY™ brand. The high-protein nutrition shake is the first product to support the adults interested in pursuing weight loss while maintaining muscle mass and good nutrition. Half of U.S. adults attempted to lose weight in the past year.  Whether pursuing weight loss by taking GLP-1 medications, …

Vertex Pharmaceuticals Positive Results for VX-548 Today, Vertex Pharmaceuticals (VRTX) announced positive results from its Phase 3 program for the treatment of moderate-to-severe acute pain. The Phase 3 program included two randomized, double- blind, placebo-controlled, pivotal trials, one following abdominoplasty . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Apellis Pharmaceuticals Negative CHMP Opinion Apellis Pharmaceuticals (APLS) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a negative opinion on the marketing authorization application (MAA) of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). This opinion was expected based . . . This content is for paid subscribers. …

Vera Therapeutics Positive Data Announcement Vera Therapeutics (VERA) announced positive 72-week data from the open label extension (OLE) period of its Phase 2b ORIGIN clinical trial of atacicept in participants with IgA nephropathy (IgAN). In aggregate, the 72-week data with atacicept are consistent with a profile of true disease modification in IgAN. From Vera . . . This content is for paid subscribers. Please click here …

Akous Inc to Present at the Association for Research in Otolaryngology Akouos, Inc. - a wholly owned subsidiary of Eli Lilly and Company (LLY) announced positive initial clinical results from the Phase 1/2 AK-OTOF-101 study, which restored the hearing within 30 days of AK-OTOF administration in the first participant, with more than a decade history of deep . . . This content is for paid …

Sanofi to Acquire Inhibrx Inc Paris, January 23, 2024, Sanofi (SNY) and Inhibrx, Inc. (INBX) entered into a definitive agreement under which Sanofi agreed to acquire Inhibrx following the spin-off of non-INBRX-101 assets into New Inhibrx. INBRX-101 is a human recombinant protein that holds the promise of allowing Alpha-1 Antitrypsin Deficiency (AATD) patients to achieve normalization of serum AAT levels with less frequent (monthly vs. …

Ionis Pharmaceuticals in the NEWS Ionis Pharmaceuticals (IONS) today announced positive topline results for the Phase 3 OASIS-HAE study of donidalorsen in people with hereditary angioedema (HAE). The trial met its primary endpoint of reduction in the rate of angioedema attacks in patients treated with donidalorsen (80mg) via subcutaneous injection dosed every 4 weeks) or every 8 . . . This content is for paid …

Lexaria Bioscience Extensive Program Designed to Support Commercial Discussions. January 16, 2024, Lexaria Bioscience (LEXX) announced a comprehensive planned applied research program to thoroughly evaluate DehydraTECH for the improved delivery of GLP-1 drugs, designed to support prospective commercial partnering with the global pharmaceutical companies. Lexaria Bioscience patented drug delivery technology DehydraTECHTM . . . This content is for paid subscribers. Please click here to subscribe or …

Improving eyesight is speeding towards treating devastating eye diseases from the Retina in the back of the eye to the cornea on the front surface of the eye. As we wrote in Prohost Letter #457, the current biotechnology revolution capabilities have never existed before in the history of the diagnosis and treatment of diseases. In the near past, drugs were designed to target the symptoms …

Vertex Pharmaceuticals Updates Vertex Pharmaceuticals (VRTX) announced multiple program updates ahead of the upcoming investor meetings in January, including the 42 Annual J.P. Morgan Healthcare Conference, which took place on January 8, 2024. From Vertex Pharmaceuticals Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex said,  “2023 was marked by continued strong performance in the cystic fibrosis business and acceleration of our . . …

Cognition Therapeutics CEO Letter to Shareholders Cognition Therapeutics (CGTX) today announced that CEO Lisa Ricciardi has issued a Letter to Shareholders to provide an update on recent pipeline developments and a preview of the company’s strategy in 2024. The full text of the letter: A MESSAGE FROM OUR CHIEF EXECUTIVE OFFICER To my fellow shareholders, The year ahead holds great promise for Cognition Therapeutics. We …

CymaBay Therapeutics in the NEWS CymaBay Therapeutics (CBAY) today announced the first published findings demonstrating the impact of seladelpar on serum interleukin-31 (IL-31) levels and its correlation with pruritus improvement in people with primary biliary cholangitis (PBC). Seladelpar is a first-in-class oral, selective PPARδ agonist being investigated for the treatment of patients with primary . . . This content is for paid subscribers. Please click here …

Compugen's Soaring Stock Many recent events  led to Compugen's (CGEN) soaring stock after a huge decline in its price. In spite of the tremendous stock decline, Prohost never doubted the fact that Compugen has solid scientific fundamentals and great scientists. The firm has done a great  job utilizing its broadly applicable predictive computational discovery capabilities to identify new drug targets and biological . . . …

Cytokinetics We recently became enthusiastic about Cytokinetics’ (CYTK) current clinical trial results. A few days ago, Cytokinetics announced positive results from SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Why the Great Enthusiasm? The following has been behind our enthusiasm . . . This …

Sarepta Therapeutics in the NEWS On 12, 22, 2023, Sarepta Therapeutics (SRPT) - a precision genetic medicine for rare diseases, announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows: “ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed . . . This content is for paid …