Breakthrough therapy

Axsome Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update

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月曜日, 5月 6, 2024
Shift, Migraine, FDA, Agitation, Research, EMERGE, Webcast, CNS, Prescription, Reboxetine, Privacy, Medicine, BED, Obstructive sleep apnea, Fibromyalgia, Shift work, Deficit spending, Food, Sleep, System, ADHD, SWD, SYMPHONY, NT1, Therapy, Quality of life, Food and Drug Administration, MDD, Narcolepsy Network, Shirin David, Patient, Somnolence, FOCUS, Safety, Depression, CGRP, Binge eating disorder, Medicare, Cataplexy, Central nervous system, Government, DSM-IV codes, Clinical trial, EDS, NDA, NMDA, GPO, Psychiatry, MD, Idiopathic hypersomnia, Smoking, CMAI, Pivotal, Breakthrough therapy, Growth, Pharmaceutical industry

Auvelity net product sales were $53.4 million for the first quarter of 2024, representing 240% year-over-year growth.

Key Points: 
  • Auvelity net product sales were $53.4 million for the first quarter of 2024, representing 240% year-over-year growth.
  • Total cost of revenue was $6.3 million for the first quarter of 2024.
  • Research and development (R&D) expenses were $36.8 million for the first quarter of 2024, compared to $17.8 million for the comparable period in 2023, respectively.
  • Approximately 95,000 prescriptions were written for Auvelity in the first quarter of 2024, representing a 12% sequential increase versus the fourth quarter of 2023.

BioNTech Announces First Quarter 2024 Financial Results and Corporate Update

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月曜日, 5月 6, 2024
Drug discovery, CLDN6, Fast Track, Lung cancer, AI, CRC, Webcast, PDAC, BioNTech, Pfizer, Genentech, Prevalence, Corporation, Vaccination, Deutsche Bundesbank, Influenza, Food, Roche, Omicron, ADC, Toxicity, Tumor antigen, Messenger, NSCLC, Telephone, Combination therapy, Pfizer–BioNTech COVID-19 vaccine, Patient, Safety, HER3, Pembrolizumab, RNA, PD-1, Docetaxel, Digital, DNA, Observational study, Neoplasm, First, Investigational New Drug, EDT, Recurrence, PD-L1, Antigen, Bank statement, Breakthrough therapy, Uridine, COVID-19, Pharmaceutical industry

MAINZ, Germany, May 6, 2024 (GLOBE NEWSWIRE) -- BioNTech SE (Nasdaq: BNTX, “BioNTech” or “the Company”) today reported financial results for the three months ended March 31, 2024, and provided an update on its corporate progress.

Key Points: 
  • 1 Calculated applying the average foreign exchange rate for the three months ended March 31, 2024, as published by the German Central Bank (Deutsche Bundesbank).
  • BioNTech is now focused on preparing for variant strain vaccine adaptation to be ready for commercial launch ahead of the upcoming 2024/2025 vaccination season, pending approvals.
  • In March, BioNTech announced that Annemarie Hanekamp will be joining the Company’s Management Board as Chief Commercial Officer on July 1, 2024.
  • Second Quarter 2024 Financial Results and Corporate Update: August 5, 2024
    Innovation Series (Digital & AI Day): October 1, 2024
    BioNTech invites investors and the general public to join a conference call and webcast with investment analysts today, May 6, 2024, at 8:00 a.m. EDT (2:00 p.m. CEST) to report its financial results and provide a corporate update for three months ended March 31, 2024.

Galderma Receives Filing Acceptances for Nemolizumab in Prurigo Nodularis and Atopic Dermatitis in Four Additional Countries

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火曜日, 5月 7, 2024
Biotechnology, Pharmaceutical, Health, Other Health, FDA, Itch, Prurigo nodularis, Priority review, Safety, Food, Nemolizumab, Sleep, Disease, Patient, Atopic dermatitis, Breakthrough therapy, European Medicines Agency, Pharmaceutical industry

Galderma today announced that it has received filing acceptances for nemolizumab for the treatment of patients with prurigo nodularis and for adolescents and adults with moderate-to-severe atopic dermatitis in Australia, Singapore, Switzerland, and the United Kingdom, whose regulatory authorities are members of the Access Consortium.

Key Points: 
  • Galderma today announced that it has received filing acceptances for nemolizumab for the treatment of patients with prurigo nodularis and for adolescents and adults with moderate-to-severe atopic dermatitis in Australia, Singapore, Switzerland, and the United Kingdom, whose regulatory authorities are members of the Access Consortium.
  • Nemolizumab was also granted Breakthrough Therapy designation by the U.S. FDA in December 2019 for the treatment of pruritus associated with prurigo nodularis, a status reconfirmed in March 2023.
  • The U.S. FDA subsequently granted nemolizumab Priority Review for the treatment of prurigo nodularis; its decisions on prurigo nodularis and atopic dermatitis are anticipated this year.
  • “These four new filing acceptances once again reinforce the potential blockbuster status of nemolizumab, which has shown great promise in both prurigo nodularis and atopic dermatitis.

Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001) for Treatment of Myotonic Dystrophy Type 1

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水曜日, 5月 8, 2024
FDA, Food, RNA, QMT, Initiation, Facial muscles, Natural history, AOC, Therapy, Myopathy, European Medicines Agency, EMA, DM1, Avidity, Breakthrough therapy, SAN, Pharmaceutical industry

SAN DIEGO, May 8, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1 (DM1). Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.

Key Points: 
  • Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.
  • "We are pleased that the FDA has granted Breakthrough Therapy designation to del-desiran for myotonic dystrophy type 1, underscoring the potential of del-desiran to be an effective treatment and the urgency of bringing this treatment to people living with DM1," said Sarah Boyce, president and chief executive officer at Avidity.
  • Avidity is initiating the global pivotal HARBOR™ study of del-desiran this quarter.
  • In addition to receiving FDA Breakthrough Therapy designation, del-desiran has previously been granted Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Medicines Agency (EMA) for the treatment of DM1.

Kura Oncology Reports First Quarter 2024 Financial Results

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木曜日, 5月 2, 2024
FDA, Cancer, KURA, Investment, Venetoclax, HNSCC, Food, Prognosis, Research, Renal cell carcinoma, FTI, AML, Webcast, Disease, LDAC, Patient, Azacitidine, QT interval, BTD, Acute myeloid leukemia, FLT3, Head, Initiate, ID, Breakthrough therapy, Pharmaceutical industry

ET –

Key Points: 
  • ET –
    SAN DIEGO, May 02, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today reported first quarter 2024 financial results and provided a corporate update.
  • Research and development expenses for the first quarter of 2024 were $36.3 million, compared to $25.2 million for the first quarter of 2023.
  • General and administrative expenses for the first quarter of 2024 were $18.2 million, compared to $11.4 million for the first quarter of 2023.
  • ET / 1:30 p.m. PT today, May 2, 2024, to discuss the financial results for the first quarter 2024 and to provide a corporate update.

Tonix Pharmaceuticals Announces Presentation at Planet MicroCap Showcase

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水曜日, 4月 24, 2024
FDA, CD40L, Cancer, Sumatriptan, NDA, Long, Food, Risk, Planet, Disease, Annual report, Security (finance), Cocaine, Research, CNS, Therapy, Failure, Infection, Central nervous system, Government, Breakthrough therapy, Fibromyalgia, Autoimmunity, Pharmaceutical industry

The presentation will include data from the recently reported positive Phase 3 RESILIENT trial of Tonmya™ (also known as TNX-102 SL) for the management of fibromyalgia.

Key Points: 
  • The presentation will include data from the recently reported positive Phase 3 RESILIENT trial of Tonmya™ (also known as TNX-102 SL) for the management of fibromyalgia.
  • A copy of the Company’s presentation will be available under the Presentations tab of the Tonix website at www.tonixpharma.com following the conference.
  • Additional meeting information can be found on the showcase website .
  • Tonix is a biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering.

Kura Oncology Receives Breakthrough Therapy Designation for Ziftomenib in NPM1-Mutant AML

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月曜日, 4月 22, 2024
FDA, Kura, Cancer, KURA, Food, Daunorubicin, AML, LDAC, Patient, BTD, Acute myeloid leukemia, Breakthrough therapy, SAN, Pharmaceutical industry

SAN DIEGO, April 22, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced that its investigational drug, ziftomenib, has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed/refractory (R/R) NPM1-mutant acute myeloid leukemia (AML).

Key Points: 
  • SAN DIEGO, April 22, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced that its investigational drug, ziftomenib, has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of patients with relapsed/refractory (R/R) NPM1-mutant acute myeloid leukemia (AML).
  • FDA granted BTD for ziftomenib based on data from Kura’s ongoing KOMET-001 clinical trial in patients with R/R NPM1-mutant AML.
  • “NPM1-mutant AML represents approximately 30% of new AML cases annually, and this designation reflects that NPM1-mutant AML is a disease of significant unmet need for which there is no approved targeted therapy as well as the fact that ziftomenib offers potential to demonstrate substantial improvement over available therapies.
  • Ziftomenib is also being evaluated in combination with current standards of care, including venetoclax/azacitidine or cytarabine plus daunorubicin (7+3) in NPM1-mutant and KMT2A-rearranged AML (KOMET-007) and with gilteritinib, FLAG-IDA or LDAC in NPM1-mutant and KMT2A-rearranged AML (KOMET-008).

Q1 2024 Revenue and Business Update

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水曜日, 4月 17, 2024
FDA, Drug discovery, Fast Track, Physics, TLX, NDA, Food, ASX, Research, Biologics license application, IND, Prostate cancer, Science, Chemistry, Acquisition, BLA, Breakthrough therapy, Growth, Radiochemistry, Medical imaging

MELBOURNE, Australia, April 17, 2024 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today provides an update on its revenue and operational performance for the quarter ended 31 March 2024 (Q1 2024).

Key Points: 
  • MELBOURNE, Australia, April 17, 2024 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (ASX: TLX, Telix, the Company) today provides an update on its revenue and operational performance for the quarter ended 31 March 2024 (Q1 2024).
  • U.S. revenue grew by 18% to US$111.8M (US$95.1M in Q4 2023), compared to 11% growth between Q3 2023 and Q4 2023.
  • Dr Christian Behrenbruch, Managing Director and Group CEO of Telix, commented, “The continued, consistent growth of our precision diagnostics business is further evidence of an effective market growth strategy for our prostate cancer franchise.
  • Telix reaffirms guidance provided on 22 February 2024 for full year revenue expected to be in the range of US$445M to $465M (AU$675M to $705M at current exchange rates), representing an approximate 35-40% increase versus 2023.

Kiromic BioPharma Reports Consistent Favorable Safety, Tolerability, and Efficacy in Deltacel-01 Clinical Trial First Three Patients

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水曜日, 4月 24, 2024
Oncology, Health, Technology, Clinical Trials, Artificial Intelligence, Pharmaceutical, Biotechnology, FDA, FTD, Priority review, Safety, Food, OTCQB, GDT, Communication, Patient, Accelerated approval (FDA), BHCC, NSCLC, Breakthrough therapy, Lung cancer, Pharmaceutical industry

Kiromic BioPharma, Inc. (OTCQB: KRBP) (“Kiromic” or the “Company”) announces consistent favorable safety, tolerability, and efficacy from follow-up visits of the first cohort of three patients enrolled in the Company’s Deltacel-01 Phase 1 clinical trial.

Key Points: 
  • Kiromic BioPharma, Inc. (OTCQB: KRBP) (“Kiromic” or the “Company”) announces consistent favorable safety, tolerability, and efficacy from follow-up visits of the first cohort of three patients enrolled in the Company’s Deltacel-01 Phase 1 clinical trial.
  • Imaging scans from Patient 2 also showed no brain metastases, confirming findings from that patient’s six-week follow-up visit.
  • The four-month follow-up for the second and third patients on trial is scheduled for June.
  • "We are highly encouraged by the initial outcomes from the first three patients in our Deltacel-01 Phase 1 clinical trial.

ImmunityBio Announces FDA Approval of ANKTIVA®, First-in-Class IL-15 Receptor Agonist for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer

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火曜日, 4月 23, 2024
Oncology, Health, Hospitals, General Health, Pharmaceutical, Biotechnology, UCLA, FDA, Cystoscopy, Safety, Food, Immunotherapy, Recurrence, Carcinoma in situ, Cystectomy, BCG, CIS, Patient, Medicine, NMIBC, Bacillus, Multimedia, Memory, Biology, Associate, Quality of life, Breakthrough therapy, NK, Pharmaceutical industry

View the full release here: https://www.businesswire.com/news/home/20240422820209/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20240422820209/en/
    ImmunityBio’s ANKTIVA is approved for non-muscle invasive bladder cancer (Photo: Business Wire)
    “The FDA’s approval of ANKTIVA marks our launch of a next-generation immunotherapy beyond checkpoint inhibitors,” said Patrick Soon-Shiong, M.D., Executive Chairman and Global Chief Scientific and Medical Officer at ImmunityBio.
  • This novel mechanism of action, which mimics the biology of the dendritic cell, begins the evolution of immunotherapy beyond T cells alone.
  • The combination of the proliferation of key cancer-killing immune cells, together with the activation of T cells with memory, results in durable complete responses.
  • The 77 evaluable patients in this single-arm, multicenter trial received ANKTIVA with BCG maintenance therapy for up to 37 months.