EP-301

Sarah Silbiger/Getty Images The U.S. Food and Drug Administration had a busy week leading up to the Labor Day holiday. Here’s a look at the agency’s recent activities. Sept 1 Immusoft’s IND for a Phase I trial of ISP-001, being developed to treat mucopolysaccharidosis type I, was granted the FDA’s green light. It is the first-ever engineered B cell therapy to enter the clinic, the company reported. eCential Robotics received FDA 501(k) clearance for its 3D imaging, navigation and robotics guidance system. Aston Sci. obtained FDA approval to initiate a Phase II trial of AST-301, a therapeutic cancer vaccine, in patients with triple-negative breast cancer. BioCryst Pharmaceuticals received Orphan Drug Designation for BCX9250, which is being developed to treat fibrodysplasia ossificans progressiva (FOP). Intellia Therapeutics was granted Orphan Drug Designation for its in vivo CRISPR/Cas9 genome editing candidate NTLA-2002 for hereditary angioedema (HAE). Revelle Aesthetics received extended FDA clearance for its precision cellulite release device Aveli. It is now indicated for long-term reduction in the appearance of cellulite in the buttocks and thigh areas of adult women. GenBody America announced the FDA had amended its EUA for the GenBody COVID-19 Ag kit, which was updated to include prefilled individual reagent tubes for each device. Aug 31 Longeveron received Fast Track Designation for Lomecel-B for Hypoplastic Left Heart Syndrome (HLHS). Sanofi reported the FDA-approved Xenpozyme (olipudase alfa-rpcp) for adult and pediatric patients with acid sphingomyelinase deficiency (ASMD), also called Niemann-Pick disease. The company also received Priority Review for its BLA for efanesoctocog alfa for the treatment of hemophilia A. The PDUFA date is February 28, 2023. United BioPharma received the go-ahead for a Phase II trial. The IND was submitted by NIAID to evaluate the antiviral activity of UB-421 in combination with optimized background ART in HIV-1 patients with multi-drug resistance. UB-421 is an anti-CD4 antibody. Scilex Holding Company, a subsidiary of Sorrento Therapeutics, received Fast Track Designation for SP-103, a drug and device product candidate to treat acute and chronic pain. The drug is being developed to be a non-opioid triple-strength, non-aqueous lidocaine topical system for treatment of acute LBD. Maze Therapeutics’ MZE001 received Orphan Drug Designation from the FDA for Pompe disease. The drug is an oral glycogen synthase (GYS1) inhibitor. Pfizer and BioNTech’s bivalent and Omicron BA.4/BA.5 COVID-19 vaccines for COVID-19 were granted Emergency Use Authorization for people 12 years and older as a single booster dose. Moderna reported the FDA granted EUA for its bivalent COVID-19 vaccine for people 18 years and older as a single booster. Aquestive Therapeutics reported the FDA granted tentative approval for Libervant (diazepam) Buccal Film for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity. Baxter International announced the FDA granted 510(k) clearance for its new Novum IQ syringe infusion pump with Dose IQ Safety Software. Aug. 29 Neuronetics received 510(k) clearance for its D-Tect MT Accessory, which helps doctors by visually reporting the magnitude of finger movements during motor threshold (MT) mapping for treatment of major depressive disorder. Serpex Medical received 510(k) clearance for its Recon Steerable Sheath, a steerable endobronchial tool to enable physicians to access difficult-to-reach areas of the lung. TRACON Pharmaceuticals received the go-ahead for a Phase I/II trial of YH001 in combination with envafolimab and doxorubicin for sarcoma patients, including treatment-naïve patients. It will evaluate patients with the combination of YH001 and envafolimab in patients with the rare sarcoma subtypes of alveolar soft part sarcoma and chondrosarcoma. It will also evaluate YH001, envafolimab and doxorubicin in the more prevalent subtypes of leiomyosarcoma and dedifferentiated liposarcoma. YH001 is an IgG1 antibody against CTLA-4. BrainsWay received 510(k) clearance from the FDA for its Deep TMS H7 Coil for use in treating adults with major depressive disorder and depression including those with comorbid anxiety symptoms (anxious depression). Deep TMS is Deep Transcranial Magnetic Stimulation.

SEOUL, South Korea, Sept. 1, 2022 /PRNewswire/ -- Aston Sci. Inc., a clinical stage biopharmaceutical company specializing in clinical development of therapeutic cancer vaccines, announced that it has obtained IND approval from the U.S. Food and Drug Administration(FDA) for Phase 2 clinical trial of its main product, AST-301(HER2-hICD vaccine). Accordingly, CornerStone-001(NCT05163223), a Phase 2 clinical trial of AST-301(pNGVL3-hICD) for treating HER2- 1+/2+ breast cancer, is anticipated to proceed smoothly in the U.S. following Australia and the Taiwan region. Aston Sci. has already confirmed the safety/tolerability, immunogenicity, and survival rate of AST-301 by presenting the Phase 1 results(PI: Mary L. (Nora) Disis, M.D., University of Washington) including a follow-up study of more than 10 years on breast cancer patients with HER2 expression, at the American Society of Clinical Oncology(ASCO) last year. The therapeutic cancer vaccine is expected to play a major role in preventing cancer recurrence after surgery and treating advanced cancer as it causes few adverse reactions and maintains long-term immunological memory compared to existing anticancer treatments. The Phase 2 clinical trial of AST-301 adopts a placebo-controlled randomized design and is a multiregional clinical trial(MRCT) in which AST-301(study drug) is administered in combination with standard treatments, such as Capecitabine or Pembrolizumab, in patients with a high risk of recurrence following a curative surgery. A total of 146 patients would be enrolled in the study and get 3 immunizations(every 3 weeks, intradermal injection), fully aligning with a standard treatment schedule, followed by a booster immunization based on the investigator's discretion. It is also emphasized that the Phase 2 clinical trial adopts the same patient group and design as in Phase 3 clinical trials. Depending on the early results, pivotal cohort expansion to generate corroborative clinical data in Phase 2 clinical trial is expected. Aston Sci. expects faster patient recruitment, subsequent to IND clearance in the U.S., from the first site(University of Washington, PI: Mary L. (Nora) Disis, M.D.) opened up in the U.S. Dr. Choi, Regulatory Affairs Director of Aston Sci. said, "It is a remarkable milestone that is definitely a leap forward for us in the cancer vaccine clinical development field. Based on the significant regulatory value of the Phase 1 clinical trial, including more than 10 years of long-term survival data, we have reinforced regulatory-required data which complies to the latest guidelines of therapeutic cancer vaccine, and the U.S. FDA has fully reviewed and approved it." Eun Kyo Joung, M.D., Chief Medical Officer of Aston Sci., pointed out the 'cancer survivorship'. Dr. Joung said, "The number of cancer survivors is rapidly increasing thanks to early cancer detection and advances in medical technology. According to 2018 statistical data, the number of fully recovered and surviving cancer patients in the past 5 years reached about 44 million worldwide. Nevertheless, cancer survivors still suffer from physical and psychological pain during the period of survival and even after full recovery due to the side effects of chemotherapy, fear of recurrence, and social problems that must be faced throughout their lives. We are confident that our therapeutic cancer vaccines will contribute to disease management, prevention of recurrence, and recovery of the overall quality of life for cancer survivors." SOURCE Aston Sci. Inc.