Amlitelimab

Taylor Tieden for BioSpace Blueprint has a next-generation systemic mastocytosis treatment, called elenestinib, that Sanofi CEO Paul Hudson told analysts provides an “opportunity to grow through the ‘30s.” Sanofi has struck a $9.5 billion deal to buy Blueprint Medicines for an approved rare disease drug and an early-stage immunology pipeline. The deal, $9.1 billion of which is upfront, will give Sanofi ownership of the systemic mastocytosis therapy Ayvakit. Blueprint posted net product revenues of $479 million in 2024 and, after achieving 61% growth in the first quarter, recently raised its guidance for this year to between $700 million and $720 million. Brian Foard, Sanofi’s head of specialty care, discussed the product’s longer-term prospects on a call with analysts Monday. The launch is “just getting started,” he said. Sanofi’s valuation of Blueprint reflects “extremely low advanced therapy penetration” so far, the executive said, and the company’s belief in the potential of Ayvakit over a long period of time. Leerink analyst Andy Berens expects that Blueprint’s revenue will hit $1.7 billion in 2030. Other observers have higher hopes, putting the consensus estimate at $2.1 billion. Leerink analyst David Risinger said in a note to investors that Sanofi “will need to deliver on its plans to find and treat more patients.” The ability of Blueprint’s pipeline to deliver blockbuster sales over the long term will be critical, too, Risinger said. There are unanswered questions about Ayvakit’s growth trajectory. Analysts asked Foard whether he expects the product to lose exclusivity in 2034 or 2040, two years in which different patents on the drug expire. Foard declined to comment beyond saying the company is “extremely comfortable with the IP.” Blueprint has a next-generation systemic mastocytosis treatment, called elenestinib, that Sanofi CEO Paul Hudson told analysts provides an “opportunity to grow through the ‘30s.” Hudson said elenestinib’s potential is “a big deal.” Elenestinib is one of two Blueprint pipeline programs that Sanofi named as drivers of the deal. The other candidate is BLU-808, a KIT inhibitor that has applications in a range of inflammatory diseases. The deal features contingent value rights tied to development and regulatory milestones for BLU-808. Hitting the milestones will bring the total value of the deal up to $9.5 billion. Hudson said Blueprint’s “established presence among key specialist physicians” was another driver of the deal. Under Hudson, Sanofi has invested in a range of immunology candidates. Blueprint’s commercial infrastructure maps on to the capabilities Sanofi expects to need to launch its immunology drugs. “If you think about where they are today, their call points, uniquely, fit us perfectly,” Foard said. “Think about our pipeline that is being built right now with amlitelimab, with lunsekimig, with TL1A. The call points for those assets are going to be dermatologists, allergists, GIs, which is exactly where Blueprint is today. So, it is going to be a natural build around these individuals.” Sanofi, which is selling a controlling stake in its consumer-healthcare arm, has struck three takeovers this year. The Blueprint deal joins agreements to buy Dren Bio and Vigil Neuroscience for $600 million and $470 million upfront, respectively. The buyouts have added to a pipeline that Hudson said has endured “a bumpy year in terms of some of the readouts.” The Blueprint takeover is at the upper end of the valuation range that Hudson has told investors Sanofi will look at. Yet, the buyout, which Sanofi will finance through cash and new debt, has not sated the company’s appetite. Sanofi CFO François-Xavier Roger told analysts the company retains “significant capacity for further business development, particularly in early-stage medicines and vaccines.”

Sanofi said it will take itepekimab data to regulators to \"evaluate our path forward.\"\n Sanofi’s plans to submit itepekimab to regulators for approval this year have been thrown into doubt after the Regeneron-partnered drug failed one of a pair of phase 3 chronic obstructive pulmonary disease (COPD) trials.Both of the studies, dubbed Aerify-1 and Aerify-2, evaluated administering the IL-33 drug itepekimab subcutaneously every two weeks or four weeks across a combined total of more than 2,000 former smokers with moderate to severe COPD. Both trials were designed to demonstrate a statistically significant reduction in moderate or severe exacerbations of COPD compared to placebo after 52 weeks.Aerify-1 hit this endpoint by demonstrating a 27% relative reduction in exacerbations among patients who received itepekimab every two weeks and a 21% reduction among those who received the drug every four weeks.But Aerify-2 was less successful, producing just a 2% reduction at two-week dosing and a 12% reduction at four-week dosing.Sanofi partly blamed the results on the lower total number of exacerbations “than prospectively anticipated,” which the company said had “decreas[ed] the power of both trials.”“Enrollment largely occurred during the time of the global COVID pandemic, which could have contributed to the overall lower exacerbation rates,” the pharma explained.Adverse events were “generally comparable” between the itepekimab and placebo cohorts, Sanofi said in its May 30 release. In the failed Aerify-2 trial, adverse events that led to death were 3% for each of the itepekimab cohorts compared to 2% for the placebo group.The failure of that trial is a significant setback for a drug that was listed in Sanofi’s most recent earnings presentation in April as being lined up for a regulatory filing in the second half of this year. At the time, Houman Ashrafian, Ph.D., Sanofi’s head of R&D, described the COPD readouts as “significant” and told analysts they could potentially lead to itepekimab’s market launch in 2026.In a statement attached to this morning’s data, Ashrafian sounded more muted on the drug’s near-term plans.“While we are encouraged by the results of AERIFY-1, the results of both studies merit further exploration to have a full understanding of the data and the role that IL-33 plays in this complex disease,” Ashrafian said. “Certain people with COPD are in desperate need of new treatment options, especially those who continue to experience exacerbations despite being on maximal therapy, and we remain committed to discussing these data with regulatory agencies to evaluate our path forward.”Sanofi declined to confirm to Fierce Biotech that its previous FDA timeline remains on track. The French Big Pharma had been developing itepekimab in collaboration with Regeneron. The two companies have seen blockbuster success with their immunology drug Dupixent and had previously evaluated the two therapies in combination as an asthma treatment in a phase 2 trial before halting that work in 2021. Separate phase 2 and 3 trials of itepekimab in COPD remain ongoing. Even if today’s unimpressive results mark the end of the road for itepekimab in that respiratory indication, the drug is still being evaluated in trials for rhinosinusitis with nasal polyps and bronchiectasis.“We are encouraged by the initial results from AERIFY-1 and are carefully reviewing the results from both itepekimab trials to inform next steps,” Regeneron Chief Scientific Officer George Yancopoulos, M.D., Ph.D., said in today’s release.“We remain committed to our broader itepekimab development program,” Yancopoulos added. “The learnings will be invaluable as we continue to advance itepekimab in respiratory diseases with unmet need.”The Aerify-2 fail continues a run of bad luck for Sanofi this year. The Paris-based pharma has also seen its psoriasis drug balinatunfib, its asthma hopeful amlitelimab and its Johnson & Johnson-sourced E. coli vaccine all underwhelm in the clinic in 2025.