Breakthrough therapy

PhaseBio Announces Publication of Interim Results from Pivotal REVERSE-IT Phase 3 Trial of Bentracimab in NEJM Evidence

Wednesday, December 1, 2021 - 10:01pm

We are pleased to see the publication of these highly positive interim Phase 3 results in a leading peer-reviewed scientific publication like NEJM Evidence, said John Lee, M.D., Ph.D., Chief Medical Officer of PhaseBio.

Key Points: 
  • We are pleased to see the publication of these highly positive interim Phase 3 results in a leading peer-reviewed scientific publication like NEJM Evidence, said John Lee, M.D., Ph.D., Chief Medical Officer of PhaseBio.
  • Additionally, the Phase 2a trial investigated a bentracimab regimen for the reversal of supratherapeutic doses of ticagrelor in healthy younger subjects.
  • These results are consistent with the results observed in healthy younger subjects treated with ticagrelor in the previously published Phase 1 trial.
  • PhaseBio initiated REVERSE-IT, a pivotal Phase 3 clinical trial of bentracimab, in March 2020 to support a potential Biologics License Application for bentracimab in both major bleeding and urgent surgery indications.

Zymeworks Announces Second Janssen Bispecific Antibody to Begin Clinical Development Utilizing Azymetric™ and EFECT™ Therapeutic Platforms

Wednesday, December 1, 2021 - 1:30pm

Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today announced that Janssen Biotech, Inc. (Janssen) dosed the first patient with JNJ-78306358, a bispecific antibody developed using Zymeworks Azymetric and EFECT therapeutic platforms.

Key Points: 
  • Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today announced that Janssen Biotech, Inc. (Janssen) dosed the first patient with JNJ-78306358, a bispecific antibody developed using Zymeworks Azymetric and EFECT therapeutic platforms.
  • This is the second Janssen bispecific program utilizing Zymeworks proprietary technology platforms to enter the clinic this year, following the announcement in August of Janssens dosing of the first patient with JNJ-78278343.
  • Zymeworks will receive a payment in connection with this milestone under Zymeworks 2017 licensing agreement with Janssen.
  • Under the terms of that agreement, Zymeworks provided Janssen with a worldwide, royalty-bearing license to research, develop and commercialize up to six bispecific antibodies directed to Janssen therapeutic targets using Zymeworks Azymetric and EFECT platforms.

BeyondSpring Pharmaceuticals Receives Complete Response Letter from the FDA for Plinabulin New Drug Application for Prevention of Chemotherapy-Induced Neutropenia (CIN)

Wednesday, December 1, 2021 - 1:00pm

The Company plans to request a meeting with the FDA and remains committed to its goal of bringing plinabulin to cancer patients in need globally.

Key Points: 
  • The Company plans to request a meeting with the FDA and remains committed to its goal of bringing plinabulin to cancer patients in need globally.
  • BeyondSpring remains confident in the efficacy and safety data for plinabulin in combination with G-CSF for the prevention of CIN.
  • Plinabulin, BeyondSprings lead asset, is a selective immunomodulating microtubule-binding agent (SIMBA), which is a potent antigen presenting cell (APC) inducer.
  • Plinabulin received Breakthrough Therapy designation and priority review from both U.S. and China FDA for the CIN prevention indication.

BioXcel Therapeutics Announces Extension of FDA Review Period of its NDA for BXCL501 for the Acute Treatment of Agitation Associated with Schizophrenia and Bipolar Disorders

Wednesday, December 1, 2021 - 12:00pm

NEW HAVEN, Conn., Dec. 01, 2021 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology, today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders I and II.

Key Points: 
  • As we prepare for anticipated FDA approval, we are confident in BXCL501s broad potential to treat the millions of patients suffering from agitation associated with schizophrenia and bipolar disorders.
  • BXCL501 is an investigational, proprietary, orally dissolving thin film formulation of dexmedetomidine, a selective alpha-2a receptor agonist for the treatment of agitation associated with neuropsychiatric disorders.
  • BXCL501 has been granted Breakthrough Therapy designation for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders and dementia.
  • Agitation is a common and difficult to manage symptom associated with multiple neuropsychiatric conditions, including schizophrenia and bipolar disorders I and II.

U.S. FDA Accepts for Priority Review the Supplemental Biologics License Application for Merck’s VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine) for Use in Infants and Children

Wednesday, December 1, 2021 - 11:45am

The FDA set a Prescription Drug User Fee Act (PDUFA), or target action date, of April 1, 2022.

Key Points: 
  • The FDA set a Prescription Drug User Fee Act (PDUFA), or target action date, of April 1, 2022.
  • The sBLA is supported by results from Phase 2 and Phase 3 clinical studies in pediatric populations including infants, children, and adolescents.
  • Invasive pneumococcal disease can cause serious and potentially life-threatening infections in infants and children.
  • Do not administer VAXNEUVANCE to individuals with a severe allergic reaction (e.g., anaphylaxis) to any component of VAXNEUVANCE or to diphtheria toxoid.

Junshi Biosciences Announces Approval of Supplemental New Drug Application by NMPA for Toripalimab in Combination with Cisplatin and Gemcitabine as First-Line Treatment for Patients with Locally Recurrent or Metastatic Nasopharyngeal Carcinoma

Tuesday, November 30, 2021 - 1:00am

In December 2018, the NMPA granted a conditional approval to toripalimab for the second-line treatment of unresectable or metastatic melanoma.

Key Points: 
  • In December 2018, the NMPA granted a conditional approval to toripalimab for the second-line treatment of unresectable or metastatic melanoma.
  • In February 2021, the NMPA granted a conditional approval to toripalimab for the treatment of patients with recurrent or metastatic nasopharyngeal carcinoma (NPC) after failure of at least two lines of prior systemic therapy.
  • This study is the worlds largest Phase III clinical study for a checkpoint inhibitor in combination with chemotherapy for the first-line treatment of recurrent or metastatic NPC.
  • Junshi Biosciences has over 2,500 employees in the United States (San Francisco and Maryland) and China (Shanghai, Suzhou, Beijing and Guangzhou).

NRx Pharmaceuticals Identifies Significantly Higher Likelihood of Surviving and Recovering from Critical COVID-19 in ZYESAMI® (aviptadil) Treated Patients Previously Administered Remdesivir

Monday, November 29, 2021 - 11:31am

Dr. Schoenfeld analyzed the subgroup of patients in the COVID-AIV trial (NCT 04311697) that remained in respiratory failure despite treatment with remdesivir.

Key Points: 
  • Dr. Schoenfeld analyzed the subgroup of patients in the COVID-AIV trial (NCT 04311697) that remained in respiratory failure despite treatment with remdesivir.
  • Based on FDAs input, NRx has narrowed its requested BTD indication to Treatment of COVID-19 Respiratory Failure in patients who progress despite treatment with remdesivir and other approved therapies.
  • NRx Pharmaceuticals (NRx) draws upon more than 300 years of collective, scientific, and drug-development experience to bring improved health to patients.
  • The company assumes no obligation to revise any forward-looking statement, whether as a result of new information, future events, or otherwise.

NRx Pharmaceuticals Notes New Data on BriLife® COVID-19 Vaccine Effectiveness Against Delta Variant Posted by Israel Institute for Biological Research

Friday, November 26, 2021 - 3:06pm

A second group of blood samples (sera) were drawn from unvaccinated patients who contracted and recovered from Covid-19.

Key Points: 
  • A second group of blood samples (sera) were drawn from unvaccinated patients who contracted and recovered from Covid-19.
  • Those patients had antibodies against the wild-type Coronavirus but with a 3.8 fold reduction in immune response (neutralizing titers) to the Delta variant.
  • NRx Pharmaceuticals (NRx) draws upon more than 300 years of collective, scientific, and drug-development experience to bring improved health to patients.
  • The Company is developing the BriLife Covid vaccine, developed by the Israel Institute for Biological Research, under an exclusive license from the Israel Ministry of Defense.

Kura Oncology Provides Update on Phase 1b Study of KO-539 in Acute Myeloid Leukemia

Wednesday, November 24, 2021 - 11:45am

Patients currently enrolled in the Phase 1b study may continue to receive KO-539, although no additional patients may be enrolled until the partial clinical hold is resolved.

Key Points: 
  • Patients currently enrolled in the Phase 1b study may continue to receive KO-539, although no additional patients may be enrolled until the partial clinical hold is resolved.
  • Kura is working closely with the FDA and the site investigators to resolve the partial clinical hold as quickly as possible.
  • Until the partial clinical hold is resolved, and the Company has more clarity regarding the impact on timing, Kura is suspending guidance on the completion of enrollment in the KOMET-001 Phase 1b study and determination of the recommended Phase 2 dose of KO-539.
  • Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer.

FDA Approves First Treatment for Common Type of Post-Transplant Infection that is Resistant to Other Drugs

Tuesday, November 23, 2021 - 10:00pm

"Cytomegalovirus infections that are resistant or do not respond to available drugs are of even greater concern.

Key Points: 
  • "Cytomegalovirus infections that are resistant or do not respond to available drugs are of even greater concern.
  • Today's approval helps meet a significant unmet medical need by providing a treatment option for this patient population."
  • CMV is a type of herpes virus that commonly causes infection in patients after a stem cell or organ transplant.
  • CMV infection can lead to CMV disease and have a major negative impact on transplant recipients, including loss of the transplanted organ and death.