On January 6, 2025 Norgine reported that it completed its marketing authorisation application filing to European Medicines Agency (EMA) for eflornithine in high-risk neuroblastoma (HRNB) (Press release, Norgine, JAN 6, 2025, View Source [SID1234649445]). This follows the submissions in April 2024, via Project Orbis, in Australia, Switzerland and the United Kingdom.

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This milestone further supports Norgine’s efforts to give patients access to eflornithine and bring a further treatment option in the field of paediatric oncology.

Norgine and USWM, LLC (dba US WorldMeds), a Kentucky-based specialty pharmaceutical company, have an exclusive licensing agreement by which Norgine will register and commercialise eflornithine, also referred to as DFMO, in Europe, Australia and New Zealand.

On 13 December 2023, the US Food and Drug Administration (FDA) approved eflornithine as the first oral maintenance therapy for HRNB, indicated to reduce the risk of relapse in adult and paediatric patients who have received certain prior therapies.[1] The approval decision was based on findings from a trial comparing outcomes from patients treated with eflornithine in Study 3b (NCT02395666)[2],[4] to control patients derived from Study ANBL0032 (NCT00026312; clinical-trial-derived external control arm)[3],[4] . The study with eflornithine treated patients showed improved event-free survival and overall survival when compared to outcomes for patients with HRNB treated with the standard of care (SoC).[1]

Dr David Gillen, Chief Medical Officer at Norgine, added, "This submission via the EU Centralised Procedure represents another important step in the regulatory process for eflornithine and further emphasises Norgine’s passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need."

Janneke van der Kamp, CEO of Norgine added "Submitting this marketing authorisation to the EMA marks a pivotal step for patients facing this challenging cancer. We are committed to advancing innovative therapies that address the unmet needs of young patients and their families, and this milestone brings us closer to offering hope where it’s most needed".

On January 6, 2025 Leap Therapeutics, Inc. (Nasdaq:LPTX), a biotechnology company focused on developing targeted and immuno-oncology therapeutics, reported that Douglas E. Onsi, President and Chief Executive Officer, will present a corporate overview at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco, CA (Press release, Leap Therapeutics, JAN 6, 2025, View Sourcenews-releases/news-release-details/leap-therapeutics-participate-43rd-annual-jp-morgan-healthcare" target="_blank" title="View Sourcenews-releases/news-release-details/leap-therapeutics-participate-43rd-annual-jp-morgan-healthcare" rel="nofollow">View Source [SID1234649442]).

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Leap Presentation Details:

43rd Annual J.P. Morgan Healthcare Conference
Date: Wednesday, January 15, 2025
Time: 2:15 p.m. Pacific Time

A live webcast of the presentation may be accessed on the Investors page of the company’s website at View Source, where a replay of the events will also be available for a limited time.

On January 6, 2025 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, reported that Emil D. Kakkis, M.D., Ph.D., the company’s chief executive officer and president, will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 AM PT (Press release, Ultragenyx Pharmaceutical, JAN 6, 2025, View Source [SID1234649441]).

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The live and archived webcast of the presentation will be accessible from the company’s website at View Source The replay of the webcast will be available for 30 days.

On January 6, 2025 Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and immune-mediated therapeutics to fight cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to amezalpat (TPST-1120), an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with hepatocellular carcinoma (HCC) (Press release, Tempest Therapeutics, JAN 6, 2025, View Source [SID1234649440]).

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"Receiving orphan drug designation for amezalpat to treat HCC underscores the critical need for new treatment options for patients suffering from this historically hard to treat disease," said Sam Whiting, M.D., Ph.D., chief medical officer and head of R&D of Tempest. "Tempest is dedicated to developing groundbreaking cancer treatments that will improve patients’ lives, and with broad agreement in hand from both the FDA and EMA, the team continues to prepare for a pivotal phase 3 study for amezalpat in first-line HCC patients."

This important regulatory designation follows positive data across multiple key study efficacy and safety endpoints in a global randomized Phase 1b/2 clinical study evaluating amezalpat plus standard-of-care atezolizumab and bevacizumab versus atezolizumab and bevacizumab alone in the first-line treatment of patients with unresectable or metastatic HCC. Notable positive outcomes of the randomized comparison include a six-month improvement in median overall survival (OS) with a hazard ratio (HR) of 0.65 for patients receiving the amezalpat combination therapy and an objective response rate (ORR) of 30% vs 13% favoring the amezalpat arm. In addition, survival benefit from the addition of amezalpat was preserved in key sub-populations including PD-L1 negative disease and b-catenin mutated disease, which is consistent with amezalpat’s proposed mechanism of action to target both the tumor cells directly and the patient’s immune system.

About Hepatocellular Carcinoma

HCC is an aggressive cancer with rising mortality and is projected to become the third leading cause of cancer death by 2030.2 Every year, more than 900,000 people worldwide are diagnosed with HCC.3 Incidence and mortality are highest in East Asia and are increasing in parts of Europe and the US. In the US, HCC represents the fastest-rising cause of cancer-related death.

Nine out of ten cases of HCC are caused by chronic liver disease, which includes chronic hepatitis B and C infection, non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), alcohol-related liver disease (ALD) and cirrhosis resulting from these conditions.

Even if diagnosed in the early stage, an estimated 70-80% of people with early-stage HCC experience disease recurrence following surgery.6 Early recurrence is associated with poorer prognosis and shorter survival.5,7 Tumor size, number of tumors, and portal vein invasion are associated with an increased risk of recurrence.

About Amezalpat

Amezalpat is an oral, small molecule, selective PPAR⍺ antagonist. Data suggest that amezalpat treats cancer by targeting tumor cells directly and by modulating immune suppressive cells and angiogenesis in the tumor microenvironment. In a global randomized phase 1b/2 study of amezalpat in combination with atezolizumab and bevacizumab in first-line patients with advanced HCC, the amezalpat arm showed clinical superiority across multiple study endpoints, including overall survival in both the entire population and key subpopulations, when compared to atezolizumab and bevacizumab alone, the standard of care. These randomized data were supported by additional positive results observed in the Phase 1 clinical trial in patients with heavily pretreated advanced solid tumors, including renal cell carcinoma and cholangiocarcinoma.

About Orphan Drug Designation

The FDA’s Orphan Drug Designation program provides orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved.

On January 6, 2025 Revolution Medicines, Inc. (Nasdaq: RVMD), a clinical-stage oncology company developing novel targeted therapies for patients with RAS-addicted cancers, reported that Mark A. Goldsmith, M.D., Ph.D., the company’s chief executive officer and chairman, will deliver a corporate presentation as part of the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 10:30 a.m. PT (Press release, Revolution Medicines, JAN 6, 2025, View Source [SID1234649439]).

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To access the live webcast of the presentation, please visit the "Events & Presentations" page of Revolution Medicines’ website at View Source Additionally, a replay of the webcast will be available on the "Events & Presentations" page of the Revolution Medicines website for at least 14 days following the conference.