Ipsen Inks Global Deal for Day One’s Tovorafenib, First-Ever Targeted Therapy for Pediatric Low-Grade Glioma, and Raises 2024 Guidance
Ipsen
Biopharmaceutical Manufacturer
丨Medicine Hunter Club Research Team
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Business Progress of the Company Since the First Quarter of 2024
Ipsen's Sales Performance in the First Half of 2024:
· Total sales increased by 9.5% in CER.
· Core operating profit margin was 32.4%, a decrease of 1.6 percentage points year-on-year, mainly due to increased R&D investment.
· International Financial Reporting Standards, operating profit margin was 19.2%, unchanged from last year.
Note: At a fixed exchange rate (CER) calculation, which means recalculating the performance of the relevant period by applying the exchange rate used in the previous period, excluding any foreign exchange impact.
2024H1 growth was mainly driven by Cabometyx® (Cabozantinib), Dysport® (Botulinum Toxin Type A), and Odevixibat.at (Product Name:Bylvay) and the sales contribution of Onivyde, which was just approved in the first quarter.
Raise 2024 Financial Guidance
Ipsen CEO David Loew commented:"The strong performance in the first half of the year demonstrates our strategic planning, and we are well-positioned to continue achieving significant growth in the medium term. In the fields of oncology, rare diseases, and neuroscience, we now possess a comprehensive portfolio of drugs, and this year we will launch more products targeting key indications.OnivydeAndIqirvo It is currently underway.We are confident in unlocking their potential. I am also pleased that our product portfolio is further expanded, including the introduction of late-stage assets in the field of pediatric oncology. tovorafenib。”
Onivyde （irinotecan）：
Onivyde received FDA approval in February this year for a supplemental new drug application as a first-line therapy in combination with the NALIRIFOX chemotherapy regimen (including 5-fluorouracil/leucovorin, and oxaliplatin) for adult patients with metastatic pancreatic ductal adenocarcinoma (mPDAC), showing promising potential for increased utilization.
Iqirvo(elafibranor)：
In June 2024, the U.S. FDA granted accelerated approval to Ipsen'sIqirvo (Elafibranor) 80mg TabletsAndUrsodeoxycholic Acid (UDCA)Combined use for the treatment of UDCAInadequate ResponseAdult Primary Biliary Cholangitis (PBC),Or as a monotherapy for patients intolerant to UDCA. Shortly thereafter, physicians will be able to prescribe and dispense Iqirvo. In addition to the United States, the company has submitted regulatory applications for Iqirvo to two other regulatory agencies, including the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA). Both are expected to make final regulatory decisions in the second half of this year, potentially becoming another blockbuster drug driving Ipsen's performance.
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Ipsen Introduces DayOne'sTovorafenib
Today, Ipsen and Day One Biopharmaceuticals entered into an exclusive non-U.S. licensing agreement to co-commercialize tovorafenib, a drug used for treating the most common type of pediatric brain tumor—pediatric low-grade glioma (pLGG). Under the terms of the agreement, Day One will receive approximately$111 millionCash and equity investments, as well asUp to $350 millionMilestone payments and double-digit sales royalties,Ipsen will be responsible for the regulatory and commercial activities of tovorafenib in all regions outside the United States.
Ojemda（Tovorafenib）
Tovorafenib isAOral, once-weekly type II RAF kinase inhibitor。On April 23, 2024, the U.S. Food and Drug Administration (FDA) announcedAccelerated Approval of Tovorafenib (Brand Name: Ojemda)For the treatment of pediatric low-grade glioma (LGG) patients aged 6 months and above, especially those whose tumors harbor BRAF fusions/rearrangements or BRAF V600 mutations, and is FDAFor the first timeSystemic Treatment Approval for Pediatric LGG Patients with BRAF Rearrangement.
Pediatric low-grade glioma (pLGG) is a common type of tumor in the pediatric central nervous system (CNS).Its incidence accounts for up to 30% of pediatric brain tumors.. Although this type of tumor is often considered slow-growing in many cases, if it is located in a critical area of the brain, complete surgical removal may not be possible in certain situations. In these cases, the tumor may cause functional damage or exhibit more aggressive biological behavior, necessitating additional treatments to control the condition and prevent further functional loss. Currently, the primary treatment option is chemotherapy.
TovorafenibAccelerated approval was based on the performance of Tovorafenib in the Phase II FIREFLY-1 study:
FIREFLY-1 is a multicenter, open-label, single-arm Phase II clinical trial designed to evaluate the efficacy and safety of Tovorafenib in treating recurrent/refractory pediatric LGG with BRAF gene alterations.
The study enrolled 76 patients, all of whom carried BRAF-activating mutations and had previously received at least one line of systemic therapy. Tovorafenib was administered once weekly based on the patient's body surface area, with a dosage range of 290-476 mg/m² and a maximum dose of 600 mg. The primary efficacy endpoint of the study was ORR, determined by blinded independent central review (BICR).
The research results show that,ORR was 51%, with a median duration of response (DoR) of 13.8 months.
In terms of safety assessment, Tovorafenib treatmentThe most common adverse reactions include rash, changes in hair color, fatigue, etc., while grade 3 or 4 laboratory abnormalities included decreased phosphate, decreased hemoglobin, etc. These data provide an important reference for the safety of Tovorafenib.
Ipsen Aggressively Acquires "BIC Pipeline, Promising Future in Oncology and CNS Fields
Ipsen has carried out multiple early-stage external innovation deals in the fields of oncology and neuroscience, reaching five inbound agreements this year with a total value of nearly $54 billion:
Ipsen's Pipeline Introduction from 2022 to 2024
· April 22, 2024 - Ipsen and Skyhawk Therapeutics announced the signing of a global exclusive cooperation agreement to discover and develop modulatorsNovel Small Molecules for Rare Neurological Disorders RNAThe agreement includes an option under which Ipsen will obtain an exclusive global license for the successful candidate development project (DC). Skyhawk's unique platform accelerates the construction of small molecules targeting RNA across multiple therapeutic areas, including rare neurological diseases.
· April 2, 2024 - Ipsen and Sutro Biopharma announced a global exclusive licensing agreement for STRO-003. STRO-003 is currently in the final stages of preclinical development.Antibody-Drug Conjugate (ADC), targeting the ROR1 tumor antigen, which is known to be overexpressed in many different cancer types, including solid tumors and hematologic malignancies.
· In June 2024, based on the positive results of the CABINET Phase 3 trial, Ipsen confirmed the expansion of its collaboration and licensing agreement with Exelixis for the development of Cabometyx® in advanced neuroendocrine tumors, allowing Ipsen to pursue opportunities outside the United States and Japan.Cabometyx in the Treatment of Advanced Pancreatic and Extra-Pancreatic Neuroendocrine TumorsPotential marketing authorization in China.
· In June 2024, Ipsen and Marengo Therapeutics announced the expansion of their ongoing early-stage oncology research collaboration, which includesExpand two additional collaborative pipelines, and use Marengo'sNext-Generation Precision T Cell Engager (TCE) Technology TriSTAR Platform, with a potential total transaction value of $1.2 billion.
· In July 2024, the company announced a global exclusive licensing agreement with Foreseen Biotechnology for FS001.FS001 is an antibody-drug conjugate with first-in-class potential.According to the terms of the agreement, the company will be responsible for the preparation activities of clinical Phase I.
Data SourceSource:
2.Oncology Drug News: The World's First! FDA Approves Tovorafenib (Ojemda) for Marketing: Treatment of Pediatric Low-Grade Glioma Patients with BRAF Mutations
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