Fast track (FDA)

PRISM MarketView Highlights Work of Emerging Companies on Rare Disease Day

Retrieved on: 
Thursday, February 29, 2024

All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.

Key Points: 
  • All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.
  • Today on Rare Disease Day, PRISM MarketView highlights emerging companies working to deliver life changing treatments for those living with rare diseases.
  • RDEB is a severe rare disease characterized by extremely fragile, and extensive blistering and wounds.
  • Capricor is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases.

Imugene Highlights Recent Achievements and Looks Ahead to Key Upcoming Immuno-oncology Catalysts

Retrieved on: 
Thursday, February 22, 2024

Imugene Managing Director & CEO Leslie Chong said: “We are encouraged by the initial safety and efficacy signals seen to date.

Key Points: 
  • Imugene Managing Director & CEO Leslie Chong said: “We are encouraged by the initial safety and efficacy signals seen to date.
  • Notably, in our Phase 1 MAST CF33 oncolytic virus trial, we’ve seen encouraging response rates during dose escalation, including one complete response in a patient with cholangiocarcinoma, and two partial responses in melanoma as we continue to dose escalate with no safety issues.
  • Importantly, two out of three of these responses were achieved with CF33 monotherapy.
  • onCARlytics uses an antigen/target-armed CF33, followed by treatment with a CD19 targeting therapy directed against that antigen or target.

Seres Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Updates

Retrieved on: 
Tuesday, March 5, 2024

Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today reported fourth quarter and full year 2023 financial results and provided business updates.

Key Points: 
  • Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today reported fourth quarter and full year 2023 financial results and provided business updates.
  • From launch through year-end 2023, there were 2,833 completed prescription enrollment forms received for VOWST, of those 2,015 resulted in new patient starts by year-end 2023.
  • Strong adoption of VOWST since commercial launch in June 2023 with broad utilization, continued quarter over quarter growth, and significant progress achieving patient access.
  • General and administrative expenses for the fourth quarter of 2023 were $17.2 million, compared with $22.4 million for the same period in 2022.

Treadwell Announces Ocifisertib, a First-in-Class PLK4 Inhibitor, has Received Orphan Designation from U.S. FDA for the Treatment of Acute Myeloid Leukemia

Retrieved on: 
Tuesday, February 20, 2024

Ocifisertib is currently being evaluated in a Phase 1b/2 study in adults with relapsed/refractory AML following standard of care therapy.

Key Points: 
  • Ocifisertib is currently being evaluated in a Phase 1b/2 study in adults with relapsed/refractory AML following standard of care therapy.
  • Orphan drug designation is granted by the FDA to drugs intended for treatment, prevention or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation.
  • “The FDA’s decision to grant orphan drug designation, along with the previous FDA Fast Track designation for ocifisertib underscores Treadwell’s dedication to addressing this patient population with few treatment options.
  • Her leadership will be invaluable to Treadwell as we advance ocifisertib into potentially pivotal studies in 2025.”

FDA Grants Fast Track Designation to 9MW2821

Retrieved on: 
Tuesday, February 27, 2024

SHANGHAI, Feb. 27, 2024 /PRNewswire/ -- Mabwell (688062.SH), an innovative biopharmaceutical company with entire industry chain, announces that its self-developed novel ADC drug targeting Nectin-4 (R&D Code: 9MW2821) has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) for the treatment of advanced, recurrent, or metastatic esophageal squamous cell carcinoma (hereinafter referred to as "ESCC").

Key Points: 
  • SHANGHAI, Feb. 27, 2024 /PRNewswire/ -- Mabwell (688062.SH), an innovative biopharmaceutical company with entire industry chain, announces that its self-developed novel ADC drug targeting Nectin-4 (R&D Code: 9MW2821) has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) for the treatment of advanced, recurrent, or metastatic esophageal squamous cell carcinoma (hereinafter referred to as "ESCC").
  • 9MW2821 is the world's first Nectin-4-targeting drug to disclose clinical efficacy data for the indication of EC.
  • After injection, 9MW2821 can specifically bind to Nectin-4 on the cell membrane surface, be internalized and release cytotoxic drug, and induce the apoptosis of tumor cells.
  • 9MW2821 is also the first to disclose preliminary clinical efficacy data for the indication of CC among drugs with the same target in the world.

Kazia Therapeutics Reports Early Conclusion of Clinical Trial After Reaching Primary Endpoint

Retrieved on: 
Wednesday, February 21, 2024

SYDNEY, Feb. 21, 2024 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a biotechnology company specializing in oncology, today announced the early conclusion based on positive safety and promising clinical response findings observed to date of an important two-part Phase I trial. This investigator-initiated trial evaluated the use of paxalisib (an oral PI3K/mTOR dual inhibitor) with radiation therapy for the treatment of patients with PI3K pathway mutation brain metastases from solid tumors.

Key Points: 
  • SYDNEY, Feb. 21, 2024 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA), a biotechnology company specializing in oncology, today announced the early conclusion based on positive safety and promising clinical response findings observed to date of an important two-part Phase I trial.
  • After reviewing the Part II patient data generated to date, the three lead investigators have determined that the primary endpoint of the study has been reached.
  • In addition, the investigators continued to observe encouraging signs of clinical response in patients in the expansion cohort.
  • The increasing incidence of brain metastasis and the low response rates to existing treatments underscores the need for new treatment options.

Cancer Immunotherapy Stocks - Tapping into Immune Systems for Next Generation Treatments

Retrieved on: 
Thursday, February 15, 2024

According to the American Association for Cancer Research "Cancer immunotherapeutics work by unleashing the power of a patient's immune system to fight cancer and, over the last decade, have emerged as one of the most exciting new approaches to cancer treatment."

Key Points: 
  • According to the American Association for Cancer Research "Cancer immunotherapeutics work by unleashing the power of a patient's immune system to fight cancer and, over the last decade, have emerged as one of the most exciting new approaches to cancer treatment."
  • Oncolytics Biotech® Inc. (NASDAQ: ONCY) (TSX: ONC) is developing pelareorep, a unique immunotherapy with the power to awaken the immune system and unlock its antitumor potential.
  • It is committed to delivering the power of T cells and to unlocking new avenues for patients in their fight against cancer.
  • Bristol-Myers Squibb and Fate Therapeutics are some of the other key patent filers in cancer immunotherapy."

CAN-3110 Receives FDA Fast Track Designation for Treatment of Recurrent High-Grade Glioma

Retrieved on: 
Tuesday, February 13, 2024

“Receiving FDA Fast Track Designation for CAN-3110 reinforces the critical need to find effective treatment options for patients with recurrent HGG and further supports the potential of CAN-3110 to address the challenges that the standard of care and conventional therapies have failed to meet,” said Paul Peter Tak, MD, PhD, FMedSci, President and CEO of Candel.

Key Points: 
  • “Receiving FDA Fast Track Designation for CAN-3110 reinforces the critical need to find effective treatment options for patients with recurrent HGG and further supports the potential of CAN-3110 to address the challenges that the standard of care and conventional therapies have failed to meet,” said Paul Peter Tak, MD, PhD, FMedSci, President and CEO of Candel.
  • “As recently published in Nature, a strong local and systemic anti-tumoral response and improved survival in patients with recurrent HGG was observed following a single injection of CAN-3110.
  • Positive HSV-1 serology was a predictor of response and was associated with improved survival (mOS in this population reached 14 months).
  • “With Fast Track Designation, I look forward to the potential of accelerating the development of CAN-3110 and the possibility of bringing this differentiated therapy to patients with recurrent high-grade glioma as we strive to improve outcomes and provide hope for patients and their families.”

Candel Therapeutics Sets Path to Success: Recent Achievements Pave the Way for a Promising 2024 Propelled by Key Value Drivers and Catalysts

Retrieved on: 
Monday, February 5, 2024

“Candel is focused on delivering key value drivers to maximize the impact of its assets and to create substantial value to patients and other stakeholders.

Key Points: 
  • “Candel is focused on delivering key value drivers to maximize the impact of its assets and to create substantial value to patients and other stakeholders.
  • In 2023, we received Fast Track Designation from the FDA for CAN-2409, our most advanced product candidate, for both NSCLC and pancreatic cancer.
  • This platform is open for collaborations with external partners.”
    The Company's strategic prioritization of programs highlights Candel Therapeutics' commitment to operational excellence and efficiency.
  • This approach positions the Company for sustained success in the current dynamic market for new cancer therapeutics.

BioNTech and DualityBio Receive FDA Fast Track Designation for Next-Generation Antibody-Drug Conjugate Candidate BNT325/DB-1305

Retrieved on: 
Wednesday, January 31, 2024

BNT325/DB-1305 is a next-generation antibody-drug conjugate (“ADC”) candidate targeting the trophoblast cell-surface antigen 2 (“TROP2”), a protein which is overexpressed on a range of tumor types.

Key Points: 
  • BNT325/DB-1305 is a next-generation antibody-drug conjugate (“ADC”) candidate targeting the trophoblast cell-surface antigen 2 (“TROP2”), a protein which is overexpressed on a range of tumor types.
  • The candidate is currently being evaluated in an ongoing Phase 1/2 study ( NCT05438329 ) in patients with TROP2-expressing advanced solid tumors.
  • Platinum-based chemotherapy is the backbone of treatment for ovarian epithelial cancer and related subtypes that form in the epithelial tissue.
  • “Recent studies have indicated the role of TROP2 in aggressive tumor growth and progression in patients with chemotherapy-resistant ovarian tumors.