FDA

Marker Therapeutics Announces Sustained Complete Response in First Lymphoma Patient Treated with MT-601 following CAR T Relapse

Retrieved on: 
Monday, December 11, 2023

The patient had diffuse large B cell lymphoma (DLBCL) and failed four prior lines of therapy, including anti-CD19 CAR T cell therapy.

Key Points: 
  • The patient had diffuse large B cell lymphoma (DLBCL) and failed four prior lines of therapy, including anti-CD19 CAR T cell therapy.
  • Marker reports today that six months following the initial treatment with MT-601 the study participant has maintained complete response to treatment.
  • “Relapse rates following CAR T cell therapy are high,” said Juan F. Vera, M.D., President and Chief Executive Officer of Marker Therapeutics.
  • “The ongoing complete response observed after MT-601 infusion in a CAR relapsed patient with lymphoma suggests superior durability of our therapy over CAR T cells in this patient.

BioXcel Therapeutics Strengthens Clinical Development Leadership to Advance Late-Stage Programs

Retrieved on: 
Monday, December 11, 2023

NEW HAVEN, Conn., Dec. 11, 2023 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience and immuno-oncology, today announced a key executive promotion and a clinical advisor appointment as part of the Company’s strategic reprioritization focused on the advancement of its late-stage clinical programs and emerging pipeline candidates.

Key Points: 
  • Vincent J. O’Neill, M.D., Senior Vice President and Chief Research & Development Officer of OnkosXcel Therapeutics, has been promoted to Executive Vice President, Chief of Product Development and Medical Officer of BioXcel Therapeutics.
  • Additionally, Rajiv Patni, M.D., has been appointed to the newly created position of Strategic Clinical Advisor to the BioXcel Therapeutics Chief Executive Officer and Board of Directors.
  • Prior to joining BioXcel Therapeutics, Dr. O’Neill held senior leadership roles at several leading global pharmaceutical companies, including Sanofi, Genentech, and GlaxoSmithKline.
  • “I look forward to working with the BioXcel leadership team in continuing to address the unmet medical needs of patients and caregivers and advance the Company’s product-development goals.”

SHAREHOLDER ALERT: The Gross Law Firm Notifies Shareholders of Outlook Therapeutics, Inc. of a Class Action Lawsuit and a Lead Plaintiff Deadline of January 2, 2024 – (NASDAQ: OTLK)

Retrieved on: 
Monday, December 11, 2023

Appointment as lead plaintiff is not required to partake in any recovery.

Key Points: 
  • Appointment as lead plaintiff is not required to partake in any recovery.
  • DEADLINE: January 2, 2024 Shareholders should not delay in registering for this class action.
  • The deadline to seek to be a lead plaintiff is January 2, 2024.
  • The Gross Law Firm is committed to ensuring that companies adhere to responsible business practices and engage in good corporate citizenship.

Umoja Biopharma Presents Preclinical Data at the 65th American Society of Hematology Annual Meeting Demonstrating In Vivo CAR T Cell Generation with Potent and Highly Durable Activity

Retrieved on: 
Monday, December 11, 2023

SEATTLE, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Umoja Biopharma, Inc., a transformative immunotherapy company creating off-the-shelf treatments for cancers and other indications of high unmet need, today announced proof-of-concept preclinical data evaluating the company’s VivoVec™ in vivo gene delivery technology to generate chimeric antigen receptor (CAR) T cells in vivo in an oral presentation at the 65th American Society of Hematology (ASH) Annual Meeting being held December 9-12, 2023, in San Diego, California.

Key Points: 
  • “At Umoja, we are focused on developing off-the-shelf approaches that overcome the limitations of approved ex vivo CAR T cell therapies,” said Andy Scharenberg, M.D., Co-Founder and Chief Executive Officer of Umoja.
  • CAR T cell expansion correlates with rapid and durable B cell aplasia in NHPs within our study which exceeds the current ex vivo CAR T cell benchmark​s.
  • Resulting CAR T cells demonstrate tumor cell killing, cytokine secretion, and proliferation in response to serial antigen exposure.
  • Anti-CD20 CAR T cell expansion correlates with rapid and durable B cell aplasia up to 76 days after VivoVec administration.

Basilea announces that FDA approves expanded use of antifungal Cresemba® (isavuconazole) in the United States in children with invasive aspergillosis and invasive mucormycosis

Retrieved on: 
Monday, December 11, 2023

Cresemba for injection is approved for adults and now for pediatric patients 1 year of age and older.

Key Points: 
  • Cresemba for injection is approved for adults and now for pediatric patients 1 year of age and older.
  • Cresemba capsules are approved for adults and now for pediatric patients 6 years of age and older, who weigh 16 kilograms and greater.
  • Dr. Marc Engelhardt, Chief Medical Officer of Basilea, said: “We congratulate Astellas on the approval of Cresemba for the use in children who suffer from invasive aspergillosis or mucormycosis.
  • In Europe, Basilea submitted a similar application for a pediatric label extension of Cresemba in August 2023.

RoslinCT to manufacture first-ever US FDA approved CRISPR-based gene therapy CASGEVY™ (exagamglogene autotemcel)

Retrieved on: 
Monday, December 11, 2023

EDINBURGH, UK, 11 December 2023 – RoslinCT, a cell and gene therapy Contract Development and Manufacturing Organisation (‘CDMO’) dedicated to developing therapies for patients who live with serious and life-threatening diseases, will manufacture the first ever U.S. Food and Drug Administration (FDA) approved CRISPR-based gene therapy.

Key Points: 
  • EDINBURGH, UK, 11 December 2023 – RoslinCT, a cell and gene therapy Contract Development and Manufacturing Organisation (‘CDMO’) dedicated to developing therapies for patients who live with serious and life-threatening diseases, will manufacture the first ever U.S. Food and Drug Administration (FDA) approved CRISPR-based gene therapy.
  • This builds on the world’s first authorisation of the treatment by UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on 15 November 2023.
  • The FDA is also undertaking a standard review of Exa-cel for transfusion-dependent beta thalassemia due by 20 March 2024.
  • This approval by the FDA further demonstrates our world leading capabilities in development, support and manufacture cutting-edge cell and gene therapies such as CASGEVY.

Nyxoah Announces Real World Case Series Demonstrating Positive Results in Treating CCC Patients with Genio®

Retrieved on: 
Monday, December 11, 2023

The investigator-sponsored case series was presented by Dr. Christian Plettenberg from the Universitätsklinikum Düsseldorf on December 8, 2023.

Key Points: 
  • The investigator-sponsored case series was presented by Dr. Christian Plettenberg from the Universitätsklinikum Düsseldorf on December 8, 2023.
  • Results showed an average apnea-hypopnea index (AHI) decrease of 73% and Epworth Sleepiness Scale (ESS) decrease of 58%.
  • “These data further validate Genio’s bilateral stimulation approach in treating CCC patients, who represent approximately 30% of HGNS eligible-to-treat OSA patients and are contraindicated to commercially available HGNS therapy in the US.
  • “I want to thank Dr. Plettenberg and his colleagues for their important work which reinforces Genio as a treatment solution for both non-CCC and CCC patients.”

Novartis presents new 48-week results from Phase III APPLY-PNH trial showing sustained efficacy and long-term safety of Fabhalta® (iptacopan) in adults with paroxysmal nocturnal hemoglobinuria (PNH)

Retrieved on: 
Monday, December 11, 2023

Data will be presented at the 65th American Society of Hematology Annual Meeting & Exposition (ASH).

Key Points: 
  • Data will be presented at the 65th American Society of Hematology Annual Meeting & Exposition (ASH).
  • “Additionally, the new data confirm that these benefits may occur within weeks after switching from anti-C5s.
  • There were no serious infections caused by N. meningitidis, S. pneumoniae, or H. influenzae and no treatment discontinuations because of TEAEs1,2.
  • Full 48-week results from the Phase III APPOINT-PNH trial in treatment-naïve PNH patients will be presented at a congress in 2024.

ACADIA SHAREHOLDER ALERT: Kaskela Law LLC Announces Investigation of Acadia Pharmaceuticals, Inc. (NASDAQ: ACAD) and Encourages Long-Term ACAD Investors to Contact the Firm

Retrieved on: 
Sunday, December 10, 2023

PHILADELPHIA, Dec. 10, 2023 (GLOBE NEWSWIRE) -- Kaskela Law LLC announces that it is investigating Acadia Pharmaceuticals Inc. (“Acadia”) (NASDAQ: ACAD) on behalf the company’s long-term investors.

Key Points: 
  • PHILADELPHIA, Dec. 10, 2023 (GLOBE NEWSWIRE) -- Kaskela Law LLC announces that it is investigating Acadia Pharmaceuticals Inc. (“Acadia”) (NASDAQ: ACAD) on behalf the company’s long-term investors.
  • According to the complaint, Acadia’s most valuable drug from a commercial perspective is pimavanserin, which Acadia touts as a treatment for dementia-related psychosis (“DRP”).
  • Current Acadia stockholders who purchased or acquired shares of the company’s common stock prior to September 9, 2019 are encouraged to contact Kaskela Law LLC (D. Seamus Kaskela, Esq.
  • Kaskela Law LLC exclusively represents investors in securities fraud, corporate governance, and merger & acquisition litigation on a contingent basis.

Autolus Therapeutics Presents Clinical Data Updates at the American Society of Hematology (ASH) Annual Meeting 2023

Retrieved on: 
Sunday, December 10, 2023

Finally, in an oral presentation pre-clinical and Phase I clinical data from AUTO8, a BCMA/CD19 co-targeting CAR T cell candidate, evaluated in patients with refractory multiple myeloma.

Key Points: 
  • Finally, in an oral presentation pre-clinical and Phase I clinical data from AUTO8, a BCMA/CD19 co-targeting CAR T cell candidate, evaluated in patients with refractory multiple myeloma.
  • “The FELIX study, with 127 patients, is one of the largest CAR T cell studies in adults with r/r B-ALL.
  • A high proportion of patients responded, with many responses sustained, particularly in patients with low or intermediate disease-burden at lymphodepletion.
  • Cellular kinetic data shows high expansion and long-term persistence of CAR T cells in most responders.