Lancet

HUTCHMED Announces that Takeda Receives U.S. FDA Approval of FRUZAQLA™ (fruquintinib) for Previously Treated Metastatic Colorectal Cancer

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Thursday, November 9, 2023
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HONG KONG, SHANGHAI, China and FLORHAM PARK, N.J., Nov. 09, 2023 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (Nasdaq/AIM:​HCM, HKEX:​13) (“HUTCHMED”) today announced that its partner Takeda received approval from the U.S. Food and Drug Administration (“FDA”) for FRUZAQLA™ (fruquintinib), an oral targeted therapy for adults with metastatic colorectal cancer (“CRC”) who have been previously treated with fluoropyrimidine-, oxaliplatin‑, and irinotecan‑based chemotherapy, an anti‑vascular endothelial growth factor (“VEGF”) therapy, and, if RAS wild‑type and medically appropriate, an anti-epidermal growth factor receptor (EGFR) therapy. FRUZAQLA is the first and only selective inhibitor of all three VEGF receptor kinases approved in the U.S. for previously treated metastatic CRC regardless of biomarker status.1,2 This approval was received under Priority Review more than 20 days ahead of the scheduled Prescription Drug Users Fee Act (PDUFA) date of November 30, 2023.

Key Points: 
  • The FDA approval of FRUZAQLA triggers a US$35 million milestone payment from Takeda.
  • Fruquintinib is developed and marketed in China by HUTCHMED following approval in September 2018, under the brand name ELUNATE™, in partnership with Eli Lilly and Company.
  • The trials investigated FRUZAQLA plus best supportive care versus placebo plus best supportive care in patients with previously treated mCRC.
  • A submission to the Japan Pharmaceuticals and Medical Devices Agency (“PMDA”) also took place in September 2023 .

Takeda Receives U.S. FDA Approval of FRUZAQLA™ (fruquintinib) for Previously Treated Metastatic Colorectal Cancer

Retrieved on: 
Wednesday, November 8, 2023
Oncology, FDA, Health, Clinical Trials, Pharmaceutical, Biotechnology, Survival, VEGF, TAK, RAS, PDUFA, Food, Colorectal Cancer Alliance, MAA, Priority, JAMA, European Medicines Agency, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Medicines and Healthcare products Regulatory Agency, Priority review, PMDA, Colorectal cancer, CRC, EMA, FACP, Cancer, Metastasis, Patient, Diagnosis, Vanderbilt University Medical Center, Pharmaceutical industry, Takeda, EU, Lancet

"There is a pressing need for new treatments for individuals with metastatic colorectal cancer, who have had limited options and continue to face poor outcomes.

Key Points: 
  • "There is a pressing need for new treatments for individuals with metastatic colorectal cancer, who have had limited options and continue to face poor outcomes.
  • “For far too long, healthcare providers and patients have had limited options when selecting a therapy for metastatic colorectal cancer.
  • The trials investigated FRUZAQLA plus best supportive care versus placebo plus best supportive care in patients with previously treated mCRC.
  • “Colorectal cancer is a highly heterogeneous disease, making it difficult to bring advancements to patients whose cancer has metastasized.

VANFLYTA® Approved in the EU as the First FLT3 Inhibitor Specifically for Patients with Newly Diagnosed FLT3-ITD Positive AML

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Thursday, November 9, 2023
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VANFLYTA is the first FLT3 inhibitor approved in the EU specifically for the treatment of patients with newly diagnosed FLT3-ITD positive AML, which represents about 25 to 30% of all new AML cases.1,2

Key Points: 
  • VANFLYTA is the first FLT3 inhibitor approved in the EU specifically for the treatment of patients with newly diagnosed FLT3-ITD positive AML, which represents about 25 to 30% of all new AML cases.1,2
    The authorization by the European Commission (EC) follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on the results of the QuANTUM-First trial, which were published in The Lancet .
  • The most common grade 3 or 4 treatment emergent adverse events (occurring in ≥ 10% of patients) were febrile neutropenia (43%), hypokalemia (19%), neutropenia (18%) and pneumonia (11%).
  • QTcF > 500 ms occurred in 2.3% of patients receiving VANFLYTA and 0.8% of patients discontinued VANFLYTA due to QT prolongation.
  • Two (0.8%) patients receiving VANFLYTA experienced cardiac arrest with recorded ventricular fibrillation on ECG (one with fatal outcome), both in the setting of severe hypokalemia.

Marinus Pharmaceuticals Initiates Global Access Program for ZTALMY® (ganaxolone) Oral Suspension CV

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Tuesday, November 7, 2023
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Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced the initiation of the Marinus Access Program, a global managed access program for ZTALMY® (ganaxolone) oral suspension CV.

Key Points: 
  • Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced the initiation of the Marinus Access Program, a global managed access program for ZTALMY® (ganaxolone) oral suspension CV.
  • The new program enables physicians to request ZTALMY for eligible patients with seizures associated with CDKL5 deficiency disorder (CDD) in geographies where the product is not commercially available and as supported by local regulatory requirements.
  • The Marinus Access Program will be managed by Durbin, a leader in the international distribution of specialized pharmaceuticals.
  • “Consistent with our company mission, we are committing appropriate resources to help facilitate broader access to ZTALMY for patients with critical unmet medical needs,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.

Calliditas Interim Report January - September 2023

Retrieved on: 
Tuesday, November 7, 2023
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Operating loss amounted to SEK 159.6 million and SEK 36.2 million for the three months ended September 30, 2023, and 2022, respectively.

Key Points: 
  • Operating loss amounted to SEK 159.6 million and SEK 36.2 million for the three months ended September 30, 2023, and 2022, respectively.
  • Significant Events in Q3 2023, in Summary
    On July 13 Calliditas announced supportive interim data from Phase 2 head and neck cancer trial with lead NOX inhibitor candidate, setanaxib.
  • Calliditas invites investors, analysts and press to a presentation of the Quarterly Report 2023 at 14:30 pm.
  • Calliditas' CEO Renee Aguiar-Lucander will present the report together with CFO Fredrik Johansson, CMO Richard Philipson and President North America Andrew Udell.

Calliditas Interim Report January - September 2023

Retrieved on: 
Tuesday, November 7, 2023
International Symposium on Endovascular Therapy, FDA, Operating cost, PDUFA, SEK, Nephrology, Magazine, ASN, NOX, USD, CMO, IgA nephropathy, Alport syndrome, Rare-earth element, Cryptocurrency, Pharmaceutical industry, Alport, EU, Lancet

Operating loss amounted to SEK 159.6 million and SEK 36.2 million for the three months ended September 30, 2023, and 2022, respectively.

Key Points: 
  • Operating loss amounted to SEK 159.6 million and SEK 36.2 million for the three months ended September 30, 2023, and 2022, respectively.
  • Significant Events in Q3 2023, in Summary
    On July 13 Calliditas announced supportive interim data from Phase 2 head and neck cancer trial with lead NOX inhibitor candidate, setanaxib.
  • Calliditas invites investors, analysts and press to a presentation of the Quarterly Report 2023 at 14:30 pm.
  • Calliditas' CEO Renee Aguiar-Lucander will present the report together with CFO Fredrik Johansson, CMO Richard Philipson and President North America Andrew Udell.

Travere Therapeutics Announces Late-Breaking Data from Phase 3 Studies of Sparsentan in IgAN and FSGS Published in The Lancet and The NEJM Respectively and Presented at the American Society of Nephrology (ASN) Kidney Week 2023

Retrieved on: 
Friday, November 3, 2023
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SAN DIEGO, Nov. 03, 2023 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (Nasdaq: TVTX) today announced additional data from two pivotal clinical studies demonstrating sparsentan has the potential to preserve kidney function and significantly delay time to kidney failure compared to an active comparator, suggesting long-term benefits in IgA nephropathy (IgAN) and focal segmental glomerulosclerosis (FSGS). Data from the Phase 3 PROTECT and DUPLEX Studies were presented as late-breaking oral presentations at the American Society of Nephrology (ASN) Kidney Week 2023 and simultaneously published in The Lancet (PROTECT) and The New England Journal of Medicine (DUPLEX).

Key Points: 
  • Data from the Phase 3 PROTECT and DUPLEX Studies were presented as late-breaking oral presentations at the American Society of Nephrology (ASN) Kidney Week 2023 and simultaneously published in The Lancet (PROTECT ) and The New England Journal of Medicine (DUPLEX) .
  • The absolute overall change in kidney function from baseline to the end of the study for patients treated with FILSPARI was -5.8 mL/min/1.73m2 compared to -9.5 mL/min/1.73m2 with irbesartan.
  • Treatment with FILSPARI demonstrated lower rates of the composite endpoint of 40% decline in eGFR, kidney failure or death compared to irbesartan.
  • Sparsentan showed a consistent and sustained achievement of complete remission of proteinuria in 18.5% of patients on sparsentan vs. 7.5% for irbesartan.

Cue Biopharma Presents New Positive Data from Phase 1 Trials of CUE-101 in Head and Neck Cancer and CUE-102 in Wilms’ Tumor 1 Positive Cancers at SITC 2023

Retrieved on: 
Friday, November 3, 2023
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New clinical data will also be reported from the company’s ongoing Phase 1 trial evaluating its second candidate, CUE-102, for the treatment of Wilms’ Tumor 1 positive (WT1+) recurrent/metastatic cancers.

Key Points: 
  • New clinical data will also be reported from the company’s ongoing Phase 1 trial evaluating its second candidate, CUE-102, for the treatment of Wilms’ Tumor 1 positive (WT1+) recurrent/metastatic cancers.
  • I look forward to evaluating the trial results as they continue to mature and remain highly encouraged by the observations to date.
  • Two patients at the 2mg/kg dose, one with gastric cancer and one with ovarian cancer have demonstrated reduction in tumor burden.
  • Pembrolizumab With or Without Chemotherapy in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma: Updated Results of the Phase III KEYNOTE-048 Study.

New Phase 3 data with aprocitentan for patients with resistant hypertension has been presented at the American Society of Nephrology Kidney Week 2023

Retrieved on: 
Friday, November 3, 2023
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Patients with hypertension can often successfully control their blood pressure by combining a healthier lifestyle with effective medication.

Key Points: 
  • Patients with hypertension can often successfully control their blood pressure by combining a healthier lifestyle with effective medication.
  • However, approximately 10% of patients have resistant hypertension where the blood pressure remains high despite receiving at least three antihypertensive medications of different pharmacological classes, including a diuretic, at optimal doses.
  • Detailed results were published in The Lancet and presented as a Late-Breaking Science presentation during the American Heart Association (AHA) Scientific Sessions in November 2022.
  • More details and commentary can be found in the dedicated press release and an investor webcast featuring Prof. Markus Schlaich, an investigator in PRECISION.

Apellis Pharmaceuticals Reports Third Quarter 2023 Financial Results

Retrieved on: 
Wednesday, November 1, 2023
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Delivered approximately 37,000 commercial vials and 10,000 samples of SYFOVRE to physician practices in the third quarter 2023.

Key Points: 
  • Delivered approximately 37,000 commercial vials and 10,000 samples of SYFOVRE to physician practices in the third quarter 2023.
  • EMPAVELI for the treatment of PNH:
    Generated $23.9 million in EMPAVELI U.S. net product revenue in the third quarter 2023.
  • In August 2023, Apellis announced a corporate restructuring to drive growth of SYFOVRE and EMPAVELI, positioning Apellis for long-term success.
  • Apellis will host a conference call and webcast to discuss its third quarter 2023 financial results and business highlights today, November 1, 2023, at 8:00 a.m.