Fast Track

AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations

Retrieved on: 
Monday, April 15, 2024
Health, FDA, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, GRN, Brain, Personality, FTD, Fast Track, Traffic barrier, Mutation, Abnormal psychology, Hospital, Food, European Commission, Apathy, Death, Dementia, Frontotemporal dementia, Thalamus, Patient, Neurosurgery, Diagnosis, INC, Granulin, Pharmaceutical industry

AviadoBio , a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN).

Key Points: 
  • AviadoBio , a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN).
  • Clinical trial sites are currently open in Poland, Spain, and the Netherlands with additional sites expected to open in multiple countries, including the United States.
  • “AviadoBio is committed to bringing forward an innovative gene therapy treatment for FTD-GRN and this moment marks an important milestone for the FTD community and our company,” said Lisa Deschamps, CEO.
  • It has also received orphan drug designation by both the FDA and European Commission.

PureTech Receives FDA Fast Track Designation for LYT-200 in Head and Neck Cancers

Retrieved on: 
Thursday, April 11, 2024
Oncology, Health, FDA, Clinical Trials, Research, Science, Biotechnology, Cancer, Fast Track, Venetoclax, Food, Prognosis, Survival, AML, Bone marrow, Patient, Tislelizumab, Acute myeloid leukemia, PRTC, Head, Clinical trial, BeiGene, Pharmaceutical industry

PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Fast Track designation for LYT-200 in combination with anti-PD1 therapy for the treatment of recurrent/metastatic head and neck squamous cell carcinomas (“head and neck cancers”).

Key Points: 
  • PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Fast Track designation for LYT-200 in combination with anti-PD1 therapy for the treatment of recurrent/metastatic head and neck squamous cell carcinomas (“head and neck cancers”).
  • It is being evaluated in two ongoing clinical trials:
    a Phase 1/2 adaptive design trial in advanced/metastatic solid tumors, including head and neck cancers.
  • “By granting Fast Track designation to LYT-200 for head and neck cancers, the FDA continues to highlight areas of critical need within oncology as well as the potential for LYT-200,” said Aleksandra Filipovic, M.D., Ph.D., Head of Oncology at PureTech.
  • The FDA has also granted orphan drug designation to LYT-200 for the treatment of AML .

Recruitment milestone reached in Diamyd® Phase 3 trial

Retrieved on: 
Friday, April 12, 2024
FDA, HLA, Diamyd Medical, Fast Track, Medicine, Patient, Safety, Orphan drug, Type 1 diabetes, Pharmaceutical industry

STOCKHOLM, April 12, 2024 /PRNewswire/ -- Diamyd Medical's precision medicine Phase 3 trial for Type 1 Diabetes, DIAGNODE-3, has enrolled 100 patients.

Key Points: 
  • STOCKHOLM, April 12, 2024 /PRNewswire/ -- Diamyd Medical's precision medicine Phase 3 trial for Type 1 Diabetes, DIAGNODE-3, has enrolled 100 patients.
  • To date, no serious adverse events have been reported, and no patients have discontinued the trial.
  • "This is an important milestone in our Phase 3 trial and the notable zero dropout rate confirms our insights from previous trials regarding the safety and convinience of the treatment," says Ulf Hannelius, President & CEO of Diamyd Medical.
  • DIAGNODE-3, being the first ever precision medicine Phase 3 trial in type 1 diabetes, is ongoing in eight European countries and in the United States.

Recruitment milestone reached in Diamyd® Phase 3 trial

Retrieved on: 
Friday, April 12, 2024
FDA, HLA, Diamyd Medical, Fast Track, Medicine, Patient, Safety, Orphan drug, Type 1 diabetes, Pharmaceutical industry

STOCKHOLM, April 12, 2024 /PRNewswire/ -- Diamyd Medical's precision medicine Phase 3 trial for Type 1 Diabetes, DIAGNODE-3, has enrolled 100 patients.

Key Points: 
  • STOCKHOLM, April 12, 2024 /PRNewswire/ -- Diamyd Medical's precision medicine Phase 3 trial for Type 1 Diabetes, DIAGNODE-3, has enrolled 100 patients.
  • To date, no serious adverse events have been reported, and no patients have discontinued the trial.
  • "This is an important milestone in our Phase 3 trial and the notable zero dropout rate confirms our insights from previous trials regarding the safety and convinience of the treatment," says Ulf Hannelius, President & CEO of Diamyd Medical.
  • DIAGNODE-3, being the first ever precision medicine Phase 3 trial in type 1 diabetes, is ongoing in eight European countries and in the United States.

EQS-News: AdrenoMed Receives FDA Fast Track Designation for Enibarcimab for Treatment of Septic Shock

Retrieved on: 
Wednesday, April 10, 2024
Sepsis, Communication, Patient, Public, Fast Track, Adrenomedullin, Biomarker, Accelerated approval (FDA), Mortality, Septic shock, Death, DPP3, Septic, Medicine, Priority review, Organ dysfunction, Intensive care medicine, CMO, Collection, FDA, Infection, RCI, Food, Pharmaceutical industry

AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.

Key Points: 
  • AdrenoMed is now preparing a confirmatory Phase IIb/III clinical trial to confirm the reduced septic shock mortality under enibarcimab treatment employing a precision medicine approach.
  • AdrenoMed’s CEO Dr. Richard Jones commented: “We are very pleased that enibarcimab has received Fast Track designation from the FDA, recognizing its potential as an innovative biomarker-guided treatment against septic shock to fill the unmet medical need in this very serious condition with a high death toll.
  • With a mortality rate of 20-30% for sepsis [1] and 30%-50% for septic shock in developed countries, [2] sepsis represents an enormous public health burden and is responsible for almost 20% of all deaths worldwide.
  • Dr. Stephan Witte, CMO of AdrenoMed, said: “We are very confident that the use of enibarcimab in combination with two biomarkers, Adrenomedullin (bio-ADM) and circulating dipeptidyl peptidase 3 (cDPP3), holds the promise to become the first effective targeted treatment against septic shock.

Cardiol Therapeutics Announces Year-End 2023 Update on Operations

Retrieved on: 
Tuesday, April 2, 2024
Hypertrophy, Heart, CRDL, Patient, Mayo Clinic, PPAR, Agent handling, Cleveland Clinic, Fibrosis, European, Therapy, Myocarditis, Analysis, American Heart Association, Human, Massachusetts General Hospital, Pericarditis, MMT, Heart failure, American College, Safety, ODD, Cardiology, Clinical professor, Disease, Pericardium, Research, World Heart Federation, Management, University, Death, Pericardial effusion, Maverick, Reactive oxygen species, TSX, Priority review, FDA, Conversation, Financial statement, Inflammation, Fast Track, Calcium, Wound healing, API, Pharmaceutical industry

"Cardiol Therapeutics made important progress in 2023 and early 2024 as we pursued our primary objective of providing new therapeutic options to patients with poorly served heart diseases," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics.

Key Points: 
  • "Cardiol Therapeutics made important progress in 2023 and early 2024 as we pursued our primary objective of providing new therapeutic options to patients with poorly served heart diseases," said David Elsley, President and Chief Executive Officer of Cardiol Therapeutics.
  • In January 2023, Cardiol announced the first patient had been enrolled in the Company's Phase II open-label pilot study ("MAvERIC-Pilot") investigating the tolerance, safety, and efficacy of CardiolRx™ in patients with recurrent pericarditis.
  • In November 2023, Cardiol announced that it had exceeded 50% of the patient enrollment target for the MAvERIC-Pilot study.
  • In January 2024, Cardiol announced that ARCHER had exceeded 50% patient enrollment and was progressing ahead of the original study timeline.

Longeveron Issues Letter to Shareholders Highlighting Corporate Strategy, Clinical Pipeline and 2024 Key Priorities and Goals

Retrieved on: 
Monday, April 8, 2024
SOC, CDMO, Orphan drug, Letter, Cell, Science, Bone marrow, Disease, Partnership, Caregiver, Hypoplastic left heart syndrome, Patient, Fast Track, Research, Microsoft Exchange Server, Standard of care, Security (finance), CLEAR, Surgeon, Regenerative medicine, FDA, BLA, Development, Pharmaceutical industry

MIAMI, April 08, 2024 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing regenerative medicines, today announced that the Company’s CEO, Wa’el Hashad, issued the following letter to Longeveron shareholders.

Key Points: 
  • MIAMI, April 08, 2024 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing regenerative medicines, today announced that the Company’s CEO, Wa’el Hashad, issued the following letter to Longeveron shareholders.
  • Today, I am excited to update you on our overall strategy, business objectives, approach to capital allocation, and 2024 key priorities and goals.
  • Our focus remains steadfast on raising the funds necessary to continue our operations and delivering transformative solutions to patients and creating sustainable value for our shareholders.
  • In 2024, we are focusing our efforts on two of our most promising programs: Hypoplastic Left Heart Syndrome (HLHS); and Alzheimer’s Disease.

Genmab to Broaden and Strengthen Oncology Portfolio with Acquisition of ProfoundBio

Retrieved on: 
Wednesday, April 3, 2024
Ovarian cancer, BofA Securities, PIN, U.S. FDA, Shearman, Webcast, Patient, Fast Track, Goldman Sachs, Morgan Stanley, LLP, Acquisition, Cancer, Spacecraft, Cooley LLP, DKK, Shearman & Sterling, ADC, USD, Law, Food, Pharmaceutical industry

Genmab will acquire ProfoundBio for USD 1.8 billion in cash, payable at closing (subject to adjustment for ProfoundBio’s closing net debt and transaction expenses).

Key Points: 
  • Genmab will acquire ProfoundBio for USD 1.8 billion in cash, payable at closing (subject to adjustment for ProfoundBio’s closing net debt and transaction expenses).
  • The transaction will further broaden Genmab’s mid- to late-stage clinical pipeline and strengthen and complement Genmab’s already validated suite of proprietary technology platforms.
  • The addition of Rina-S to Genmab’s portfolio will enable Genmab to deepen its presence in the gynecologic oncology space and establish a firm foundation in solid tumors.
  • Based on the data from the ongoing Phase 1/2 clinical trial Genmab intends to broaden the development plans for Rina-S within ovarian cancer and other FRα-expressing solid tumors.

SELLAS Life Sciences Reports Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Thursday, March 28, 2024
Ovarian cancer, SLS, Survival, Lymphoma, Fast track (FDA), PTCL, National Cancer Institute, CMC, Clinical trial, Committee, Research, NCI, European School, Bone marrow, AML, GPS, Pembrolizumab, Doctor of Science, Nivolumab, Safety, MRD, CDK9, BLA, Food, CR2, Zanubrutinib, Mesothelioma, BIW, MD, Completeness, WT1, FDA, DLBCL, IDMC, Therapeutic index, Type, Fast Track, Biomarker, BTK, Pharmaceutical industry

The SLS009 aza-ven treatment was well-tolerated and evoked anti-leukemic effects in 67% of patients across all levels dosed.

Key Points: 
  • The SLS009 aza-ven treatment was well-tolerated and evoked anti-leukemic effects in 67% of patients across all levels dosed.
  • The first patient who achieved a complete response continues on the study and remains leukemia-free 9 months post-enrollment.
  • The net proceeds from the offering strengthen the Company’s financial position and will be used for research and development activities, working capital, and general corporate purposes.
  • Cash Position: As of December 31, 2023, cash and cash equivalents totaled approximately $2.5 million.

Cellectar Biosciences Reports Financial Results for Year Ended 2023 and Provides a Corporate Update

Retrieved on: 
Wednesday, March 27, 2024
Research, NCI, AON, Development, Sarcoma, Disease, Neuroblastoma, Shareholder, Patient, CNS, Fast Track, Cash, SBIR, NDA, Syndrome, Cancer, MRR, CLR, BNS, University, Head, External beam radiotherapy, Cure, Wisconsin Alumni Research Foundation, WAM, HERE, Completeness, Safety, Tat, Intellectual property, Patent, PLE, WARF, Symantec Endpoint Protection, Tranche, UW, Pharmaceutical industry

FLORHAM PARK, N.J., March 27, 2024 (GLOBE NEWSWIRE) -- Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development, and commercialization of drugs for the treatment of cancer, today announced financial results for the year ended December 31, 2023, and provided a corporate update.

Key Points: 
  • FLORHAM PARK, N.J., March 27, 2024 (GLOBE NEWSWIRE) -- Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development, and commercialization of drugs for the treatment of cancer, today announced financial results for the year ended December 31, 2023, and provided a corporate update.
  • Net cash used in operating activities during the twelve months ended December 31, 2023, was approximately $32.4 million.
  • General and Administrative Expense: G&A expense for the year ended December 31, 2023, was $10.7 million, compared to $9.6 million for the year ended December 31, 2022.
  • Cellectar management will host a conference call for investors today, March 14, 2024, beginning at 8:30 am Eastern Time to discuss these results and answer questions.