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Sling Therapeutics Initiates Global Phase 3 Trial for Oral Thyroid Eye Disease Treatment

Sling Therapeutics Initiates Global Phase 3 Trial for Oral Thyroid Eye Disease Treatment

Sling Therapeutics, Inc., a clinical-stage biopharmaceutical company, has announced that the first patients have been dosed in its global Phase 3 ORBIT pivotal trial. This trial is evaluating linsitinib, an oral small molecule inhibitor of the insulin-like growth factor-1 receptor (IGF-1R), for the treatment of moderate to severe active Thyroid Eye Disease (TED) in adults. The ORBIT trial is a global, randomized, double-masked, placebo-controlled study designed to assess the efficacy and safety of linsitinib. TED is a serious, progressive, and vision-threatening rare autoimmune disease affecting over 200,000 people in the U.S., often occurring in individuals with Graves’ disease and hyperthyroidism. The disease is characterized by fibrous tissue growth behind the eyes, leading to symptoms like pain, eye bulging (proptosis), and potential vision loss. Linsitinib is the first and only oral IGF-1R inhibitor in Phase 3 development, aiming to provide a convenient oral treatment option for TED patients.

Cell and Gene Therapy Industry Faces Scale-Up Challenges, Lifecycle Approach Proposed

Cell and Gene Therapy Industry Faces Scale-Up Challenges, Lifecycle Approach Proposed

Cell and Gene Therapy Industry Faces Scale-Up Challenges, Lifecycle Approach Proposed

Ultragenyx sets $3.95 million price for rare disease gene therapy

Ultragenyx sets $3.95 million price for rare disease gene therapy

By Sriparna Roy and Kamal Choudhury Sept 17 (Reuters) - Ultragenyx Pharmaceutical said on Thursday it has set a U.S. list price of $3.95 million for its newly approved gene therapy Fayuvi, launching one of the world's most expensive drugs as a one-time treatment for a rare, fatal childhood disease.

Exclusive-US weighs allowing most pharma licensing deals with China, sources say

Exclusive-US weighs allowing most pharma licensing deals with China, sources say

By Michael Erman NEW YORK, Sept 18 (Reuters) - The US is working on rules for pharmaceutical companies investing in China that would likely preserve their ability to strike most licensing deals for Chinese drugs, according to three people who have been briefed on the process.

Experts urge FDA to set clear safety and access standards for psychedelic drugs (September 14)

Experts urge FDA to set clear safety and access standards for psychedelic drugs (September 14)

(Corrects quote attribution in paragraph 8 of September 14 story from Lynn Watkins, a U.S.

Detroit Free Press

Homeland Security questioned Chinese researcher before his suicide

Homeland Security questioned Chinese researcher before his suicide

Danhao Wang's suicide at the University of Michigan came amid beefed up scrutiny by the Trump administration of foreign citizens on U.S. college campuses.

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics Advances Gene Therapies for Inherited Retinal Diseases, Secures FDA Alignment for Phase 3 Trial

Opus Genetics is making significant strides in developing gene therapies for inherited retinal diseases (IRDs), specifically OPGx-LCA5 for LCA5-associated IRD and OPGx-BEST1 for BEST1-associated IRD. According to Dr. George Magrath, CEO of Opus Genetics, the OPGx-LCA5 program, targeting an ultra-rare disease affecting approximately 200 U.S. children, has shown promising results. All six participants treated to date (three pediatric and three adult) demonstrated improvement across various measures, including full-field stimulus threshold (FST) testing, best-corrected visual acuity (BCVA), virtual-reality maze tests, and microperimetry. Retinal sensitivity improved by about 1.5 log units at both blue and red wavelengths. The company has also secured FDA alignment for an eight-participant Phase 3 registrational trial for OPGx-LCA5, utilizing a 6-month pretreatment period where each participant serves as their own control. The OPGx-BEST1 program aims to restore bestrophin-1 channel function in the retinal pigm...

Corbus Pharmaceuticals Announces New Obesity Drug CRB-913 with Fewer Side Effects Than GLP-1s

Corbus Pharmaceuticals Announces New Obesity Drug CRB-913 with Fewer Side Effects Than GLP-1s

Corbus Pharmaceuticals has announced promising results for its new anti-obesity drug, CRB-913, claiming it can achieve similar weight loss to GLP-1s with fewer gastrointestinal side effects. In a recent drug trial, CRB-913 demonstrated a 5% weight loss at a 60mg dose after 12 weeks. The drug is a CB1 inverse agonist, which works by reducing the influence of the CB1 receptor, thereby decreasing hunger signals. This mechanism differs from GLP-1 receptor agonists, which mimic the GLP-1 hormone. Previous attempts to target CB1 receptors for obesity treatment faced challenges due to psychiatric side effects, but Corbus Pharmaceuticals states that CRB-913 has minimal brain penetration, with psychiatric adverse events broadly in line with those experienced by GLP-1 users.

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Rocket Pharmaceuticals Aligns with FDA on Pivotal Phase 2 Trial for Danon Disease Treatment

Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics Reaches 100th Participant Enrollment in ALS Trial for COYA 302

Coya Therapeutics, Inc., a clinical-stage biotechnology company, has announced the enrollment of its 100th participant in the Phase 2/3 ALSTARS Trial (NCT07161999) for COYA 302. This investigational drug, a combination of proprietary low-dose IL-2 and CTLA-4 Ig, is being evaluated for the treatment of amyotrophic lateral sclerosis (ALS). The company anticipates completing the full enrollment of 120 participants in the coming weeks, with topline data expected in early Q2 2027. COYA 302 has received FDA Fast Track designation for ALS treatment. The ALSTARS Trial is a randomized, multi-center, double-blind, placebo-controlled study designed to assess the efficacy and safety of COYA 302. Participants are randomized to receive one of two dosing regimens or a placebo over a 24-week double-blind treatment phase, followed by a 24-week blinded active-treatment extension phase. The primary endpoint focuses on changes in disease severity as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised...

Cullinan and Taiho Plan FDA Filing for Lung Cancer Pill Zipalertinib Despite Patient Deaths

Cullinan and Taiho Plan FDA Filing for Lung Cancer Pill Zipalertinib Despite Patient Deaths

Taiho Oncology and Cullinan Therapeutics are moving forward with plans to file for FDA approval of their oral drug candidate, zipalertinib, for non-small cell lung cancer (NSCLC), despite three patient deaths reported during a late-stage study. The Phase 3 REZILIENT3 trial demonstrated that zipalertinib, when combined with chemotherapy, significantly improved progression-free survival (PFS) in patients with first-line advanced non-squamous NSCLC harboring exon 20 mutations to the EGFR gene. Patients receiving the zipalertinib regimen experienced a 50% reduction in the risk of death or disease progression, with a median PFS of 14.5 months compared to 8.5 months in the control group. While adverse events of grade 3 or higher were more frequent in the zipalertinib arm (87% vs. 54.4%), analysts believe the robust efficacy and potential mitigation strategies will outweigh the safety concerns.

Detroit City Council Explores Alternatives to DTE Energy After Widespread Power Outages

Detroit City Council Explores Alternatives to DTE Energy After Widespread Power Outages

The Detroit City Council is debating the city's continued reliance on DTE Energy for its power supply. This discussion follows severe storms, including a tornado, that caused power outages for over 450,000 people, with thousands affected within Detroit. Last week, the City Council voted to direct its Legislative Policy Division to research alternative energy options. This initiative will also involve examining energy strategies adopted by other Michigan cities, such as Wyandotte and potentially Ann Arbor. The vote, which passed without objection, reflects significant frustration among council members and residents regarding the recent power disruptions. DTE Energy, however, is reportedly pushing back against these efforts to explore alternative power sources. Council members Gabriela Santiago-Romero, Denzel McCambell, and Mary Waters have publicly discussed the issue.