Acer Starts Pivotal Phase 3 Trial in Vascular Condition

 Acer Therapeutics Inc. (Nasdaq: ACER), a clinical stage pharmaceutical company focused on the acquisition, development and commercialization of therapies for serious rare and life-threatening diseases with significant unmet medical needs, today announced the initiation of patient screening in its Phase 3 DiSCOVER (Decentralized Study of Celiprolol on vEDS-related Event Reduction) clinical trial of EDSIVO™ (celiprolol) for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS).

“With no approved treatments available for vEDS, ongoing clinical evaluation of EDSIVO™ (celiprolol) is an important step that we hope could lead to the first available treatment option for this fatal disorder,” said Adrian Quartel, MD, FFPM, Chief Medical Officer of Acer. “The innovative, decentralized study design will allow for greater access and ease of administration for vEDS patients, mobile nurses, remote clinical research coordinators and investigators. We'd like to thank all the patients, their family members, the advocacy groups and clinicians who have expressed interest in learning more about participating in the study, and look forward to rapidly enrolling the trial.”

The DiSCOVER trial is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy trial designed to evaluate EDSIVO™ (celiprolol) in patients with genetically confirmed COL3A1-positive vEDS using a decentralized clinical trial design and an independent adjudication committee. The primary objective of the trial is to determine whether EDSIVO™ (celiprolol) reduces the occurrence of vEDS-related clinical events requiring medical attention, including fatal and non-fatal cardiac or arterial events, uterine rupture, intestinal rupture, and/or unexplained sudden death, relative to placebo as measured by time to event. Acer plans to enroll approximately 150 COL3A1-positive vEDS patients, all in the U.S., randomized 2:1 to receive either EDSIVO™ (celiprolol) or placebo, respectively. Individuals seeking more information on the EDSIVO™ (celiprolol) pivotal clinical trial are invited to visit www.discoverceliprolol.com.

https://finance.yahoo.com/news/acer-therapeutics-announces-initiation-pivotal-123000936.html

FDA Presses Pause on Astellas' Pompe Disease Therapeutic

 The U.S. Food and Drug Administration has placed Astellas Pharma's Phase I/II FORTIS trial on clinical hold after one of its study participants experienced a serious adverse event (SAE).

The study, which is evaluating the investigational adeno-associated virus gene replacement therapy AT845 in adults with late-onset Pompe disease, reported peripheral sensory neuropathy in one of the patients. The SAE was classified as Grade 1, or mild in severity, but it was considered serious because it is medically significant.

AT845 is a gene replacement therapy that uses an AAV8 vector to deliver a functional copy of the acid alpha-glucosidase (GAA) gene to treat late-onset Pompe disease. The FDA said Astellas did not submit sufficient details to assess the risk of the drug to its participants, specifically regarding the SAE that surfaced. The regulator will send a detailed written explanation for its decision within the next 30 days.

"We remain committed to the safe and effective development of AT845 and will keep the scientific and patient communities informed with updates as we learn more. Patient safety is our top priority, and we are working closely with the FDA to determine appropriate next steps," Weston Miller, M.D., senior medical director of clinical development at Astellas Gene Therapies, said in a statement.

Astellas also said it is determining if the clinical hold will have a financial impact on its report for the fiscal year ending March 31, 2023.

Pompe disease is a rare autosomal recessive metabolic disorder characterized by progressive muscle degeneration and caused by alpha-glucosidase gene mutations, which prevent the production and kill the function of acid alpha-glucosidase (GAA). GAA metabolizes glycogen, and its absence leads to glycogen accumulation in the cardiac and skeletal muscles and, later, to tissue damage.

This isn't the first time Astellas has dealt with a serious adverse event in one of its trials. In September 2021, a patient that participated in Astellas' ASPIRO trial for AT132, an investigational drug for x-linked myotubular myopathy, died during the trial. The FDA placed the study on clinical hold after the company, alongside partner Audentes, reported multiple deaths of trial participants who received the therapy.

It appears that Astellas has managed to regain its footing after that incident, announcing new developments in the months that followed. The latest is a late-June announcement of a New Drug Application filing for fezolinetant, a selective neurokinin 3 (NK3) receptor antagonist for the treatment of moderate to severe vasomotor symptoms linked with menopause.

In early June, the company celebrated the opening of a new manufacturing facility in Sanford, North Carolina. The 135,000 square-foot standalone structure is intended to broaden Astellas' clinical and commercial capabilities, particularly for its pipeline of adeno-associated virus products. The site also houses quality control testing facilities. The company is reportedly hiring over 200 people between now and 2026.

https://www.biospace.com/article/fda-hits-the-hold-button-on-another-astellas-gene-therapy-/

Relay: Virtual Analyst and Investor Event Eyes Cholangiocarcinoma, Breast Cancer

 End-of-phase 1 meeting with the U.S. Food and Drug Administration (FDA) resulted in alignment on the design of a single arm trial for pan-FGFR (FGFRi) treatment-naïve FGFR2-fusion cholangiocarcinoma (CCA) to potentially support accelerated approval

Interim data from the once daily (QD) dosing schedule shared with the FDA demonstrated confirmed partial responses in eight out of thirteen (62%) FGFRi-naïve FGFR2-fusion CCA patients, including all four of the patients treated at the registrational trial dose of 70 mg QD

Relay Therapeutics discloses three new programs as part of a growing HR+/HER2- breast cancer franchise: selective CDK2 inhibitor, ERα degrader, and chemically distinct pan mutant-PI3Kα (RLY-5836)

Relay Therapeutics continues to expect its current cash, cash equivalents and investments will be sufficient to fund its current operating plan into 2025

https://finance.yahoo.com/news/relay-therapeutics-discloses-anticipated-registrational-100000172.html

NeuroSense: Positive Results from Stage III ALS Biomarker Study

 

• Preliminary results show levels of disease-related biomarkers remain unchanged in people living with ALS who are treated with standard of care, in contrast to the statistically significant decline in these biomarkers in patients treated in NeuroSense's Phase IIa study with PrimeC

• New biomarker data in people with ALS validates NeuroSense's clinical strategy

https://finance.yahoo.com/news/neurosense-therapeutics-reports-positive-results-123000570.html

Capricor: Statistically Significant Clinical Benefits in Duchenne

-Met Primary Endpoint of Performance of the Upper Limb PUL 2.0 (p=0.02)-

-Results Suggest Disease Modification in DMD and Long-term Safety of CAP-1002-

-Results were Presented at Late Breaking Session at PPMD’s Annual Conference on Saturday June 25, 2022-

-Principal Investigator Dr. Craig McDonald and Capricor Management Will Host Conference Call and Webcast Today at 8:00 a.m. ET-

Capricor will host a conference call and webcast with slides today, June 27, 2022, at 8:00 a.m. ET to discuss the data findings. To participate in the conference call, please dial 877-451-6152 (domestic) or 201-389-0879 (international) and reference the access code: 13730622

To participate via a webcast and view the slides, please click here. The webcast will be archived for approximately 30 days and will be available at http://capricor.com/news/events/.

https://finance.yahoo.com/news/capricor-therapeutics-announces-statistically-significant-110000987.html

BridgeBio: Positive Phase 1 Data and Phase 2/3 Trial Design for Kidney Stones

 – BBP-711 led to near complete inhibition of glycolate oxidase throughout the dosing period and greater than 10-fold increases in plasma glycolate, suggesting it has the potential to be both a best-in-class therapy and the first oral therapy for PH1 and recurrent kidney stone formers

– Based on the tolerability and potency of the oral therapy, BridgeBio has met with regulators and intends to initiate a Phase 2/3 pivotal study by the end of 2022

– At the end of 2022, BridgeBio also intends to launch a Phase 2 study of BBP-711 in adult recurrent kidney stone formers, which affects an estimated 1.5 million individuals in the United States and European Union

https://finance.yahoo.com/news/bridgebio-pharma-announces-positive-phase-113000372.html

Sellas Update for GFH009 Ongoing Phase 1 Clinical Trial

  SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today provided a clinical update on the ongoing Phase 1 dose-escalating clinical trial of GFH009, its novel and highly selective CDK9 inhibitor, in advanced relapsed and refractory lymphoma and acute myeloid leukemia (AML).

In the AML group, patients treated at the 22.5 mg dose level experienced no dose limiting toxicities, including no grade 3/4 neutropenias (an abnormally low count of neutrophils, a type of white blood cell). The AML group has entered the last planned dose level of 30 mg. As previously reported, significant anti-leukemic effects (i.e., greater or equal to 50 percent decrease in bone marrow blasts following GFH009 monotherapy) have been observed in AML patients treated sufficiently long enough to assess efficacy at previous dose levels.

In the lymphoma group, the 15 mg dose level cohort has completed enrollment. Safety assessments for this cohort are currently underway. In the previous 9 mg dose level cohort, one patient, with peripheral T-cell lymphoma, an aggressive type of lymphoma that develops from mature-stage T-cells and natural killer (NK) cells who was refractory to three prior lines of therapy, demonstrated a partial response as seen on a computerized tomography (CT) scan.

https://finance.yahoo.com/news/sellas-life-sciences-provides-clinical-123000447.html