Fast Track

Oncolytics Biotech® Recaps 2023 Accomplishments, Provides Outlook for 2024

Retrieved on: 
Thursday, January 4, 2024
Oncolytics Biotech, AB, PR, FDA, Breast, Roche, Progression-free survival, Health, AIO, Congress, Society, Pancreatic cancer, Cancer, Oncology, Partnering, Paclitaxel, Anal cancer, Conference, ASCO, ONC, Data, Annual general meeting, Patient, RNA, MBC, TIL, ESMO, Tumor microenvironment, Anal canal, Pancreatic Cancer Action Network, Physician, International, FOLFIRINOX, Survival, Christmas and holiday season, Neoplasm, Toxicity, Poster, Fast Track, Gemcitabine, SCCA, SAN, PDAC, Science Translational Medicine, TSX, Pancreas, Pharmaceutical industry

Overview: "2023 was an outstanding year for Oncolytics and the development of pelareorep as a potential cancer therapy.

Key Points: 
  • Overview: "2023 was an outstanding year for Oncolytics and the development of pelareorep as a potential cancer therapy.
  • "We believe our cash balance and strong data, supported by our active business development program, position us well for 2024."
  • Together, these data provide a strong foundation to support advancing pelareorep into registrational-track studies, starting in 2024," continued Dr Coffey.
  • 2024 Outlook: Dr. Coffey concluded by saying, "Looking ahead to 2024, we expect to initiate the first Phase 3 study for pelareorep in pancreatic cancer.

Oncolytics Biotech® Recaps 2023 Accomplishments, Provides Outlook for 2024

Retrieved on: 
Thursday, January 4, 2024
Oncolytics Biotech, AB, PR, FDA, Breast, Roche, Progression-free survival, Health, AIO, Congress, Society, Pancreatic cancer, Cancer, Oncology, Partnering, Paclitaxel, Anal cancer, Conference, ASCO, ONC, Data, Annual general meeting, Patient, RNA, MBC, TIL, ESMO, Tumor microenvironment, Anal canal, Pancreatic Cancer Action Network, Physician, International, FOLFIRINOX, Survival, Christmas and holiday season, Neoplasm, Toxicity, Poster, Fast Track, Gemcitabine, SCCA, SAN, PDAC, Science Translational Medicine, TSX, Pancreas, Pharmaceutical industry

Overview: "2023 was an outstanding year for Oncolytics and the development of pelareorep as a potential cancer therapy.

Key Points: 
  • Overview: "2023 was an outstanding year for Oncolytics and the development of pelareorep as a potential cancer therapy.
  • "We believe our cash balance and strong data, supported by our active business development program, position us well for 2024."
  • Together, these data provide a strong foundation to support advancing pelareorep into registrational-track studies, starting in 2024," continued Dr Coffey.
  • 2024 Outlook: Dr. Coffey concluded by saying, "Looking ahead to 2024, we expect to initiate the first Phase 3 study for pelareorep in pancreatic cancer.

Aqua-Tots Swim Schools Kicks Off the New Year With Opening of Newest Location in West Jordan, Utah

Retrieved on: 
Tuesday, January 2, 2024
Suite, Swimming, Risk, Student, Interest, School, Health, Certification, Bangladesh Technical Education Board, Sam's Club, Club, Fast Track, Tutoring

SALT LAKE CITY, Utah, Jan. 02, 2024 (GLOBE NEWSWIRE) -- Aqua-Tots Swim Schools , the largest international swim school franchise is excited to announce its second Utah location, and 146th worldwide, now open in West Jordan, Utah.

Key Points: 
  • SALT LAKE CITY, Utah, Jan. 02, 2024 (GLOBE NEWSWIRE) -- Aqua-Tots Swim Schools , the largest international swim school franchise is excited to announce its second Utah location, and 146th worldwide, now open in West Jordan, Utah.
  • Aqua-Tots Jordan Landing is owned by experienced franchisees, Mike Priest, Steve Harris and Craig Wright, along with Aqua-Tots co-founders, Ron Sciarro and Paul Preston.
  • The 6,300 square foot facility features a year-round 90° pool and premium amenities to make swim lessons convenient and comfortable for young, active families.
  • To learn more about Aqua-Tots Jordan Landing or to enroll in swim lessons, visit aqua-tots.com/jordan-landing or call 801-441-6141.

Anavex Life Sciences Provides an Update on Rett Syndrome Program

Retrieved on: 
Tuesday, January 2, 2024
DSM-IV codes, Lethargy, Șaeș, AVATAR, Rett syndrome, Caregiver, Microsoft Access, Research, Fragile X syndrome, Marketing, FDA, EMA, Data analysis, Disease, Somnolence, MD, Doctor of Philosophy, Clinical trial, Seizure, Orphan drug, Clinical Global Impression, European Medicines Agency, CHMP, Expanded access, Anxiety, Committee, Safety, Patient, Placebo, Real, RTT, Therapy, OLE, Fast Track, RWE, Pharmaceutical industry

NEW YORK, Jan. 02, 2024 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders today reported topline results from the randomized, double-blind, placebo-controlled, Phase 2/3 EXCELLENCE clinical trial, which evaluated the clinical efficacy, safety, and tolerability of 30 mg ANAVEX®2-73 in 92 pediatric patients with Rett syndrome (RTT) between the ages of 5 through 17 years. Participants were randomized 2:1 (ANAVEX®2-73 [62 patients] to placebo [30 patients]) for 12 weeks, followed by a week 16 safety visit. As well, Anavex reported positive Real World Evidence (RWE) feedback from Rett syndrome patients under Compassionate Use Authorization.

Key Points: 
  • As well, Anavex reported positive Real World Evidence (RWE) feedback from Rett syndrome patients under Compassionate Use Authorization.
  • After 12 weeks, the study showed improvement on the key co-primary endpoint Rett Syndrome Behaviour Questionnaire (RSBQ), which is a detailed 45-item questionnaire for assessing multiple Rett syndrome characteristics by the patients’ caregivers.
  • As of today, some patients with Rett syndrome have been on ANAVEX®2-73-treatment for over 4 years, combined OLE and Compassionate Use Program.
  • In addition to Rett syndrome, Anavex is evaluating ANAVEX®2-73 in other neurodevelopmental disorders, including Fragile X syndrome, and in neurodegenerative disorders like Parkinson’s disease.

Lexeo Therapeutics Granted FDA Fast Track Designation and Orphan Drug Designation for LX2020, an AAV-Based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

Retrieved on: 
Monday, December 18, 2023
Food, FDA, PKP2, Therapy, ACM, Orphan drug, Cardiac muscle, Arrhythmogenic cardiomyopathy, Arrhythmia, Biomarker, Patient, Safety, Disorder, Fast Track, Pharmaceutical industry

“Receiving both Orphan Drug and Fast Track designations from the FDA for LX2020 further validates the importance of progressing a potential one-time treatment option for patients suffering from PKP2-ACM,” commented R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics.

Key Points: 
  • “Receiving both Orphan Drug and Fast Track designations from the FDA for LX2020 further validates the importance of progressing a potential one-time treatment option for patients suffering from PKP2-ACM,” commented R. Nolan Townsend, Chief Executive Officer of Lexeo Therapeutics.
  • Preliminary efficacy measures will evaluate myocardial protein expression, biomarkers measuring cardiac structure and function, and arrhythmia burden.
  • Long-term safety and efficacy will be evaluated for an additional four years following completion of the initial trial.
  • Fast Track designation allows more frequent FDA interactions to facilitate development and expedite the review process for novel drug candidates that treat serious or life-threatening diseases and address unmet medical needs.

Caribou Biosciences Provides Regulatory Update on CB-010 Pivotal Plan with Phase 3 Trial Initiation Expected by YE 2024

Retrieved on: 
Tuesday, December 12, 2023
ASCT, Regenerative Medicine Advanced Therapy, Food, FDA, CRISPR, Rachel Haurwitz, Reindeer, Doctor of Philosophy, Randomized controlled trial, Orphan drug, B-cell lymphoma, Patient, Fast Track, Pharmaceutical industry

BERKELEY, Calif., Dec. 12, 2023 (GLOBE NEWSWIRE) -- Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, today provided an update based on feedback from the U.S. Food and Drug Administration (FDA) following recent communications and a Type B clinical meeting regarding the CB-010 development program. The company has received the FDA’s feedback on a Phase 3 randomized controlled trial for CB-010, an allogeneic anti-CD19 CAR-T cell therapy in development for patients with second-line relapsed or refractory large B cell lymphoma (r/r LBCL). The FDA stated that Caribou’s proposed comparator arm of platinum-based immunochemotherapy followed by high dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT) is acceptable.

Key Points: 
  • The FDA stated that Caribou’s proposed comparator arm of platinum-based immunochemotherapy followed by high dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT) is acceptable.
  • “As our ongoing ANTLER trial progresses, we look forward to engaging again with the FDA ahead of initiating our planned Phase 3 pivotal trial.
  • In Q2 2024, the company plans to report initial dose expansion data from the ANTLER trial and share the recommended Phase 2 dose (RP2D) for CB-010.
  • Caribou plans to initiate a Phase 3 pivotal trial with this RP2D by year-end 2024.

Plus Therapeutics Partners With K2bio for Development of Novel Tests for Cerebrospinal Fluid (CSF) Tumor Cell and Molecular Biomarker Analyses

Retrieved on: 
Tuesday, December 12, 2023
CSF, Patient, Ecosystem, Partnership, Neoplasm, Cerebrospinal fluid, Learning, PSTV, Cancer prevention, Caregiver, Research, FDA, Doctor of Philosophy, Cancer, Therapy, CNS, Texas Medical Center, LM, Plus Therapeutics, Central nervous system, Diagnosis, Rhenium, Fast Track, Research institute

This trial is currently receiving grant funding through The Cancer Prevention and Research Institute of Texas (CPRIT).

Key Points: 
  • This trial is currently receiving grant funding through The Cancer Prevention and Research Institute of Texas (CPRIT).
  • K2bio is based in Houston and is part of the Texas Medical Center life sciences ecosystem specializing in all aspects of translational cancer diagnostic and therapeutic research and development.
  • “K2bio is a leader in enabling rapid diagnostic and therapeutic progress for innovative companies such as Plus,” said Colby Suire, PhD, acting President and CEO of K2bio.
  • Patients interested in learning more about the ReSPECT-LM trial can visit ClinicalTrials.gov ( NCT05034497 ).

Lexicon Enrolls First Patient in Phase 2b Progress Study of LX9211 in Diabetic Peripheral Neuropathic Pain (DPNP)

Retrieved on: 
Thursday, November 30, 2023
T1DM, Food, FDA, AAK1, Type, Lexicon Pharmaceuticals, Patient, Dose-ranging study, Neuropathic pain, Extension, Trial of the century, Diagnosis, PROGRESS, Safety, T2DM, Fast Track, Pharmaceutical industry

THE WOODLANDS, Texas, Nov. 30, 2023 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced that it has enrolled the first patient in PROGRESS (A Phase 2b, Dose-ranging, Randomized, Double-blind, PlacebO-controlled, Parallel-GRoup, MulticEnter Study with an Open-label Extension in PatientS with Diabetic Peripheral Neuropathic Pain) studying LX9211, a potent, orally-delivered, selective, investigational small molecule inhibitor of adaptor-associated kinase 1 (AAK1).

Key Points: 
  • PROGRESS will enroll adult patients with a diagnosis of type 1 diabetes mellitus (T1DM) or type 2 diabetes mellitus (T2DM) with moderate to severe DPNP.
  • The primary endpoint of PROGRESS is a reduction of Average Daily Pain Score (ADPS), consistent with the primary endpoint achieved in the successful RELIEF-DPN-1 Phase 2a proof-of-concept study and, informed by regulatory feedback, the primary endpoint planned for Phase 3 studies of LX9211 in DPNP.
  • The placebo-controlled treatment period of PROGRESS is intended to identify the optimal dosing for Phase 3 studies.
  • The open-label extension is expected to run in parallel with planned next-stage Phase 3 studies, supporting long-term safety requirements for regulatory approval.

Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH

Retrieved on: 
Thursday, November 30, 2023
Quality of life, Glycosaminoglycan, Food, ESGCT, Health, Antibody, FDA, Cell, Disease, Mortality, Tissue, Congress, Gene, HSC, POC, Therapy, RPD, Heart, Hurler syndrome, Eye, San Raffaele Hospital, Death, San Raffaele, ERT, European Medicines Agency, Cognitive development, IDUA, Growth, HSCT, Patient, Safety, Diagnosis, Hurler, Fast Track, Pharmaceutical industry

BOSTON and LONDON, Nov. 30, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs. Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.

Key Points: 
  • Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs.
  • Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.
  • “We are encouraged that OTL-203 has been granted Fast Track designation because new treatment options are urgently needed for children with MPS-IH, as the current standard of care is associated with significant morbidity and mortality.
  • Throughout the PoC study, treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen.

Oxford BioTherapeutics Announces Partner Boehringer Ingelheim Received U.S. FDA Fast Track Designation for BI 764532 for the Potential Treatment of Advanced or Metastatic Large-Cell Neuroendocrine Carcinoma of the Lung

Retrieved on: 
Wednesday, November 29, 2023
Partnership, Food, Drug discovery, Boehringer Ingelheim, FDA, Lung, Disease, Boehringer, News, Orphan drug, NEC, Carcinoma, OBT, SCLC, Patient, ADC, DLL3, Fast Track, Pharmaceutical industry

BI 764532 is an investigational DLL3/CD3 IgG-like T-cell engager for potential treatment of patients with LCNEC-Lung that is being developed by Boehringer Ingelheim.

Key Points: 
  • BI 764532 is an investigational DLL3/CD3 IgG-like T-cell engager for potential treatment of patients with LCNEC-Lung that is being developed by Boehringer Ingelheim.
  • The discovery of BI 764532 (OBT620) was enabled through a successful partnership initiated in 2013, leveraging OBT’s proprietary OGAP® drug discovery platform for identification of the DLL3 antigen and Boehringer Ingelheim’s longstanding expertise in oncology and development of biotherapeutics.
  • BI 764532 was also granted Orphan Drug designation by the FDA for the treatment of SCLC, and is currently being investigated in a Phase 2 study, ‘DAREON™-5’ ( NCT05882058 ), in patients with relapsed/refractory extensive-stage SCLC and other relapsed/refractory NEC.
  • Christian Rohlff, Chief Executive Officer of OBT, commented: “We are delighted about the clinical development to help address unmet needs for people living with small cell lung cancer and other neuroendocrine carcinomas.