Cold Agglutinin Disease

The decision marks a significant step in Sanofi’s ongoing effort to sharpen its focus on its core areas of innovative medicines and vaccines. Credit: jd8/Shutterstock. Sanofi has announced negotiations with Clayton Dubilier & Rice (CD&R), a US-based investment company, regarding the potential sale of a 50% controlling stake in its consumer healthcare business, Opella. The move marks a significant step in the company’s ongoing effort to sharpen its focus on its core areas of innovative medicines and vaccines. The French finance ministry, as reported by Reuters , has acknowledged CD&R as a “serious investment fund” with favourable prospects for Opella’s growth and its operations in the country. The ministry has stipulated economic commitments from both Sanofi and CD&R. These include ensuring that Opella’s decision-making centres remain in France and protecting the company’s industrial footprint within the country. The ministry has also emphasised that the proposed sale will not affect the production of Doliprane or other essential medicines by Opella in France, nor the supply of these medicines to the market. See Also: Evergreen Theragnostics Radiopharmaceutical Manufacturing Facility, New Jersey, US Merck Biotech Development Center, Corsier-sur-Vevey, Switzerland The reported valuation of the deal by Bloomberg stands at €15bn ($16.4bn). The fact that CD&R has reportedly outbid PAI Partners suggests a competitive process that reflects the high value and potential of Opella within the broader consumer health market. Opella, with its headquarters in France, employs more than 11,000 people, has operations in 100 countries and manages 13 manufacturing sites and four research and innovation centres. It has a portfolio of 100 brands, including Doliprane, Allegra, Novanight, Icy Hot and Dulcolax. Operating independently within Sanofi, Opella has resources allocated for research and development, production, and digital operations, along with a sustainability roadmap. It reached a 6.3% sales growth at constant exchange rates in 2023, demonstrating a focus on brands and consumer orientation. In October 2024, Recordati reached an agreement with Sanofi to acquire the worldwide rights to Enjaymo, the only approved treatment specifically targeting cold agglutinin disease, a rare B-cell lymphoproliferative disorder.

Sanofi is offloading its rare autoimmune disease medicine Enjaymo to an Italian pharmaceutical company for $825 million upfront. Recordati, based in Milan, said Friday it will pay up to an additional $250 million over time for the monoclonal antibody, which was greenlit in the US , Europe and Japan in 2022 for hemolysis in adults with cold agglutinin disease (CAD). In patients with the rare condition, their immune system prematurely demolishes red blood cells, and it can lead to severe anemia. Sanofi recorded €55 million ($60 million) in Enjaymo sales in the first six months of 2024, it said in July. Recordati predicts the drug could reach annual peak sales of €250 million to €300 million (about $275 million to $330 million). It marks a winding road for Enjaymo, which has gone through the full biotech-to-pharma journey. A Bay Area biotech called True North Therapeutics developed the drug and then was bought by Bioverativ for $400 million upfront in 2017. Less than 12 months later, Sanofi acquired Bioverativ for almost $11.6 billion. Two years after that, Sanofi was handed a rejection for the drug before it was approved in 2022. For Recordati, the deal pushes it further into the rare disease market. The company bought EUSA Pharma and its four rare disease drugs for nearly $850 million in December 2021. It’s also struck deals with GSK , Almirall and others in recent years. With “a strong clinical profile and as the only product approved for the treatment of CAD, Enjaymo addresses a serious unmet medical need for patients living with this debilitating disease,” Recordati CEO Rob Koremans said in a press release. Earlier this year, Sobi and Apellis Pharmaceuticals stopped a Phase 3 in CAD, citing “a decreased medical need in CAD and therefore a limited number of patients eligible for the study.” CAD affects about one person per million every year, according to the National Organization for Rare Disorders. The Recordati deal comes as Sanofi adjusts its portfolio. It now plans to hone in on immunology and inflammation under new R&D head Houman Ashrafian and new CSO Mike Quigley , who joined Sept. 30. It’s also planning to divest its consumer health group Opella. Earlier this year, Sanofi sold some early-stage cancer projects to Vir Biotechnology and shuttered a cell therapy unit, though it’s still making a few bets in oncology, including its first foray into radiopharma . The French drugmaker also has invested in several companies, including Ventyx, Vicore and Graviton.

MILAN, Italy I October 04, 2024 I Recordati today announces an agreement with Sanofi to acquire the global rights to Enjaymo® (sutimlimab), a biologic which is the only approved targeted product for the treatment of cold agglutinin disease (CAD), a rare B-cell lymphoproliferative disorder. Enjaymo® (sutimlimab) is a humanized monoclonal antibody indicated for the treatment of hemolysis in adults with CAD. In 2022, it was granted approval by the U.S. Food and Drug Administration (FDA), the European Commission (EC) and the Japanese Ministry of Health, Labor and Welfare. Administered as chronic IV treatment, Enjaymo® addresses a serious unmet medical need in patients with CAD. Financial highlights Enjaymo® generated approximately € 100 million in revenue over the last 12 months as of August 2024 and is expected to generate revenue in excess of € 150 million in FY 2025, with peak sales potential of € 250-300 million, more than double current levels. Subject to the closing date, Recordati expects minimal revenue contribution in 2024. The transaction is expected to be immediately accretive at the EBITDA level, with margin above the current Rare Diseases average as of 2025. Transaction details Under the terms of the agreement, Recordati will make an upfront payment of US$ 825 million and additional commercial milestone payments of up to US$ 250 million, if net sales reach certain thresholds at or above the top end of peak year sales expectations. The transaction is expected to close by the end of 2024, subject to regulatory clearances. The deal will be funded by existing cash and new committed bank debt facilities. Net debt is expected to be approximately 2.4 – 2.5x EBITDA (pro-forma) at the end of 2024, de-leveraging to less than 2.0x EBITDA at the end of 2025, assuming no additional business development transactions. The Group’s dividend and capital allocation policy remains unchanged. Rob Koremans, Chief Executive Officer of Recordati, commented: “This transaction is in-line with our broader strategy, reaffirms our commitment to the Rare Diseases space and is complementary to our Oncology portfolio, specifically Sylvant®. Enjaymo® further expands our Rare Diseases footprint in the U.S., Japan and Europe, and will contribute positively to both our top and bottom lines. Most importantly, with a strong clinical profile and as the only product approved for the treatment of CAD, Enjaymo® addresses a serious unmet medical need for patients living with this debilitating disease.” About Cold agglutinin disease (CAD) Cold agglutinin disease (CAD) is a rare B-cell lymphoproliferative disorder, a subgroup of autoimmune hemolytic anemia (AIHA), caused by autoantibodies secreted by B-cells that bind to erythrocytes (temp ≤ 37°C) leading to erythrocyte destruction. CAD symptoms include severe, debilitating fatigue and other anemic manifestations (e.g. arthralgia, muscle weakness), that can significantly impact patients’ quality of life. Disease prevalence in the U.S., Japan and Europe is approximately 11K patients, and while median age of onset is approximately 60 years, CAD has been diagnosed in patients as young as 30. About Enjaymo®(sutimlimab) Enjaymo® is a humanized monoclonal antibody that is designed to selectively target and inhibit C1s in the classical complement pathway, which is part of the innate immune system. By blocking C1s, Enjaymo® inhibits the activation of the complement cascade in the immune system and inhibits C1-activated hemolysis in CAD to prevent the abnormal destruction of healthy red blood cells. Enjaymo® does not inhibit the lectin and alternative pathways. Enjaymo® was approved by the US Food and Drug Administration (FDA) in February 2022 as the first and only treatment indicated to decrease the need for red blood cell transfusion due to hemolysis in adults with CAD. The Japanese Ministry of Health, Labor and Welfare approved Enjaymo® in June 2022. The European Medicines Agency (EMA) also made the decision to maintain orphan designation. Conference Call Recordati will host a conference call today, 4 th October , at 13:00 CEST ( 12:00 p.m. GMT ) to discuss the agreement to acquire Enjaymo®. The dial-in numbers for the conference call service are: Italy + 39 02 802 09 11, toll free 800 231 525 UK + 44 1 212818004, toll free (44) 0 800 0156371 USA +1 718 7058796, toll free (1) 1 855 2656958 France +33 1 70918704 Germany +49 6917415712 Participants are invited to dial in 10 minutes before conference time. If conference operator assistance is required to connect, please dial *0. The slides that will be referenced during the call will be available at www.recordati.com under Investors/Company Presentations. The audio conference live webcast will also be available at the following link Recordati (REC.MI) is an international pharmaceutical group listed on the Italian Stock Exchange (ISIN IT 0003828271) uniquely structured to bring treatment across specialty and primary care and rare diseases. We believe that health, and the opportunity to live life to the fullest, is a right, not a privilege. We want to support people in unlocking the full potential of their lives. We have fully integrated operations across research & development, chemical and finished product manufacturing through to commercialization and licensing. Established in 1926, Recordati operates in approximately 150 countries across EMEA, Americas and APAC regions. At the end of 2023, Recordati employed over 4,450 people and consolidated revenue of € 2,082.3 million. For more information, please visit www.recordati.com SOURCE: Recordati