IPSEN, OOO
🇫🇷France
- Country
- 🇫🇷France
- Ownership
- -
- Established
- 1929-01-01
- Employees
- 5.7K
- Market Cap
- $9.8B
- Website
- https://www.ipsen.com/
US Orphan Drug Market Set to Exceed $190 Billion by 2030 as FDA Designations Accelerate
• The US orphan drug market is projected to surpass $190 billion by 2030, with over 7,300 molecules receiving FDA Orphan Drug Designation to date, of which approximately 17.9% have gained approval.
• Since 2020, more than half of all new drug approvals by the FDA's Center for Drug Evaluation and Research have been granted orphan status, highlighting the growing importance of rare disease treatments in pharmaceutical development.
• Despite criticism over high pricing, exemplified by Abeona Therapeutics' Zevaskyn at $3.1 million per treatment, the orphan drug model has evolved into a sound business strategy offering fewer competitors, faster approvals, and seven-year market exclusivity.
Pharma Leadership Shuffle: Key Executive Moves in Early 2025 Reshape Industry Landscape
• The pharmaceutical industry has experienced significant leadership changes in early 2025, with major transitions at companies including Pfizer, Boehringer Ingelheim, and Takeda.
• Former FDA CDER Director Patricia Cavazzoni's move to Pfizer as Chief Medical Officer has sparked "revolving door" criticisms, highlighting ongoing concerns about regulatory-industry transitions.
• Several biotechnology firms have strengthened their executive teams with specialized expertise in clinical development, particularly in areas like stroke treatment, antibody-drug conjugates, and neuropsychiatric therapies.
GENFIT Reports Strong 2024 Financial Results with First Commercial Revenues from Iqirvo®
• GENFIT reported a net profit of €1.5 million for 2024, driven by €67.0 million in revenues including a €48.7 million milestone payment and royalties from Iqirvo® (elafibranor) sales in PBC.
• The company secured a transformative non-dilutive royalty financing agreement worth up to €185 million, significantly extending its cash runway beyond 2027.
• GENFIT is advancing five complementary programs targeting Acute-on-Chronic Liver Failure (ACLF), with key clinical data readouts expected by the end of 2025.
Ipsen's Elafibranor Shows Promise in Phase II Trial for Primary Sclerosing Cholangitis
• Elafibranor demonstrated a favorable safety profile and significant dose-dependent efficacy in the Phase II ELMWOOD trial for primary sclerosing cholangitis (PSC), a rare liver disease with no currently approved treatments.
• Patients treated with elafibranor showed significant improvements in liver biochemical parameters, including alkaline phosphatase, with stabilization of non-invasive markers of liver fibrosis compared to placebo.
• The 120mg dose of elafibranor significantly improved pruritus symptoms, offering potential relief for a common and distressing symptom experienced by PSC patients.
Robust Pipeline of 200+ Therapies Targets Colorectal Cancer Treatment Landscape
• DelveInsight's latest report reveals a robust pipeline with over 195 companies developing 200+ therapies for colorectal cancer, highlighting significant innovation in targeted treatments and immunotherapies.
• Key drug candidates include XL092 (Exelixis), Adagrasib (Mirati Therapeutics), Olaparib (Merck/AstraZeneca), and novel approaches like LYL845, an autologous tumor-infiltrating lymphocyte therapy from Lyell Immunopharma.
• The metastatic colorectal cancer segment shows particular promise with 150+ companies advancing 180+ pipeline therapies, including innovative treatments targeting specific mutations and immune pathways.
China's NMPA Grants Conditional Approval to Tazemetostat for EZH2-Mutant Follicular Lymphoma
• China's National Medical Products Administration (NMPA) has granted conditional approval to tazemetostat (Tazverik) for adult patients with relapsed or refractory EZH2-mutated follicular lymphoma who have received at least two prior systemic therapies.
• Tazemetostat is the first and only EZH2 inhibitor approved in China, representing HUTCHMED's fourth approved product and its first in hematological malignancies, following previous approvals in the US and Japan.
• The approval was supported by a Phase II bridging study in China and international clinical trials, with the ongoing SYMPHONY-1 trial serving as the confirmatory study to validate clinical benefits.
Ipsen Secures €500 Million Inaugural Rated Public Bond Following Investment Grade Ratings
• Ipsen has successfully completed its first Rated Public Bond of €500 million with a 3.875% coupon maturing in March 2032, following Investment Grade ratings from S&P (BBB-) and Moody's (Baa3).
• The transaction was heavily oversubscribed by institutional investors, demonstrating market confidence in Ipsen's financial strategy and long-term growth prospects.
• This bond issuance is part of Ipsen's broader refinancing plan, which recently included the renewal of a €1.5 billion Revolving Credit Facility, significantly extending the company's debt maturity profile.
XOMA Royalty Expands Portfolio to Over 120 Assets, Reports Financial Results for 2024
• XOMA Royalty doubled its portfolio to over 120 royalty assets through five strategic transactions in 2024, strengthening its position as a biotech royalty aggregator.
• The company completed two whole company acquisitions and celebrated FDA approvals for Day One's OJEMDA™ (tovorafenib) and Zevra's MIPLYFFA™ (arimoclomol), generating significant milestone payments.
• Despite reporting a net loss of $13.8 million for 2024, XOMA Royalty received $46.3 million in cash receipts and maintains over $100 million in cash, positioning it for sustainable cashflow from royalties.
Bluebird Bio Goes Private in $29M Deal with Carlyle and SK Capital Partners
• Gene therapy pioneer Bluebird Bio agrees to private acquisition by Carlyle and SK Capital Partners for $3 per share, with potential additional payments of up to $6.84 per share through CVR.
• Despite having three FDA-approved gene therapies, Bluebird Bio has faced significant financial challenges, including European market withdrawal and workforce reductions of 55% since 2022.
• The company's struggles intensified after failing to secure an expected $103M FDA priority review voucher for Lyfgenia in 2023, leading to critical cash flow issues.
Ipsen's Sohonos Becomes First Approved Treatment for FOP in Canada
• Health Canada has approved Sohonos (palovarotene), making it the first-ever treatment for fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder that turns soft tissue into bone.
• The approval follows regulatory challenges in the US, where Ipsen withdrew its marketing application in 2022 after the FDA requested additional phase 3 trial data.
• The drug is approved for both chronic use and flare-ups in patients aged 8+ (females) and 10+ (males), marking a significant milestone following Ipsen's $1.3 billion acquisition of Clementia Pharma.
FDA Grants Accelerated Approval to Ipsen's Iqirvo, First Dual PPAR Agonist for Primary Biliary Cholangitis
• Ipsen's Iqirvo (elafibranor) receives FDA accelerated approval as the first dual PPAR alpha/delta agonist and first new therapy in over a decade for primary biliary cholangitis treatment.
• In the ELATIVE trial, Iqirvo demonstrated significant efficacy with 51% of patients achieving cholestasis response compared to 4% on placebo, marking a substantial therapeutic advancement.
• The drug will be available at $11,500 per month list price, entering a market estimated at $1.5 billion annually for UDCA-refractory PBC treatments.
UGN-102 Demonstrates Durable Responses in Low-Grade Non-Muscle Invasive Bladder Cancer
• UGN-102 achieved a 79.6% complete response rate at 3 months in patients with low-grade, intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC) in the ENVISION trial.
• The Kaplan-Meier estimate showed an 82.3% 12-month duration of response in patients who achieved complete response after initial UGN-102 treatment.
• The ENVISION trial's safety profile of UGN-102 was favorable, supporting its potential as a non-surgical alternative to TURBT for this patient population.
• An FDA decision on UGN-102 for LG-IR-NMIBC is expected by June 13, 2025, potentially transforming the treatment landscape.
J&J Seeks FDA Approval for TAR-200 in BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
• Johnson & Johnson has initiated a New Drug Application (NDA) submission to the FDA for TAR-200.
• TAR-200 is intended for patients with Bacillus Calmette-Guérin (BCG)-unresponsive high-risk non-muscle-invasive bladder cancer (HR-NMIBC).
• Phase 2b SunRISe-1 study data showed an 83.5% complete response rate with TAR-200 monotherapy.
• The FDA is reviewing the application under the Real-Time Oncology Review (RTOR) program to expedite potential approval.
Ariceum's 225Ac-Satoreotide Receives FDA Orphan Drug Designation and IND Clearance for SCLC and Merkel Cell Carcinoma
• The FDA granted Orphan Drug Designation to Ariceum Therapeutics' 225Ac-satoreotide for small cell lung cancer (SCLC), offering development incentives and potential market exclusivity.
• Ariceum's IND application for 225Ac-SSO110 was cleared by the FDA, enabling a Phase I/II trial for SCLC and Merkel Cell Carcinoma (MCC) patients.
• The SANTANA-225 trial, set to begin in Q1 2025, will assess the safety, tolerability, and preliminary efficacy of 225Ac-SSO110 in patients with advanced SCLC or MCC.
• Preclinical data showed 225Ac-satoreotide's potential to outperform SSTR2 targeting agonists, demonstrating complete responses and 100% survival in models of aggressive cancers.
FDA Foregoes Advisory Committee for Cabozantinib's Neuroendocrine Tumor Application
• The FDA has canceled the Oncologic Drugs Advisory Committee meeting for Exelixis' cabozantinib sNDA, streamlining the review process.
• The sNDA seeks approval for cabozantinib to treat advanced pancreatic and extra-pancreatic neuroendocrine tumors in previously treated adults.
• The FDA's decision is based on Phase III CABINET trial results, which showed significant progression-free survival improvements with cabozantinib.
• The Prescription Drug User Fee Act (PDUFA) target action date for the FDA's decision on cabozantinib remains April 3, 2025.
Breakthroughs in Clinical Trials: A Week of Promising Developments
This week's clinical trials round-up highlights significant advancements in treatments for various conditions, including idiopathic pulmonary fibrosis, diabetes, COPD, and advanced solid tumours. Key developments include positive Phase 2 results for Faron Pharmaceuticals' BEXMAB trial, AstraZeneca's Phase 2a COURSE trial for COPD, and Johnson & Johnson's Phase 3 QUASAR maintenance study for ulcerative colitis.
Kymriah Receives EU Approval as First CAR-T Therapy for Follicular Lymphoma
• Novartis' Kymriah is the first CAR-T therapy approved in the EU for relapsed/refractory follicular lymphoma, offering a new option for patients after multiple treatments.
• The approval was based on the ELARA trial, demonstrating an 86% response rate and 69% complete response rate in patients treated with Kymriah.
• Kymriah provides durable treatment effects, with 87% of complete responders remaining in remission for at least nine months after initial response.
• This approval expands Kymriah's indications in the EU, adding to its existing approvals for diffuse large B-cell lymphoma and acute lymphoblastic leukemia.
Keytruda Approved in China for Early-Stage NSCLC Treatment
• Keytruda gains approval in China for resectable stage II, IIIA, or IIIB NSCLC in combination with platinum-containing chemotherapy.
• The approval is based on the KEYNOTE-671 trial, demonstrating a significant improvement in overall survival and event-free survival.
• This marks Keytruda's fourth indication for NSCLC in China, extending its use to earlier stages of the disease.
• The treatment involves neoadjuvant Keytruda plus chemotherapy, followed by adjuvant Keytruda monotherapy post-surgery.
Gilead's Seladelpar Receives European Commission Approval for Primary Biliary Cholangitis
• The European Commission has granted conditional marketing authorization for Gilead's seladelpar for primary biliary cholangitis (PBC).
• Seladelpar is approved for use in combination with UDCA for those with inadequate response, or as a monotherapy for those who cannot tolerate UDCA.
• The approval is based on Phase 3 RESPONSE trial data, showing significant improvements in biochemical response and pruritus reduction.
• This decision provides a new treatment option for PBC patients in Europe, addressing a critical unmet need.
GSK's Linerixibat Shows Positive Phase III Results for Cholestatic Pruritus in Primary Biliary Cholangitis
• GSK's linerixibat met its primary endpoint in the GLISTEN Phase III trial, demonstrating a statistically significant reduction in itch for PBC patients with moderate to severe pruritus.
• The trial evaluated linerixibat in PBC patients already receiving guideline-suggested therapies, treatment-naïve patients, and previously treated patients, showing potential as a targeted therapy.
• Linerixibat, an ileal bile acid transporter (IBAT) inhibitor, could be the first global therapy specifically developed to treat itch in PBC, addressing a significant unmet need.
• Preliminary safety results were consistent with prior studies, and full results from the GLISTEN trial will be presented at a future scientific congress.
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