Media Update: New data at ERS showed Xenpozyme® (olipudase alfa) improved respiratory functions in adults with ASMD
New data at ERS showed Xenpozyme® (olipudase alfa) improved respiratory functions in adults with ASMD
Paris, September 14, 2023. New data presented in Milan at the European Respiratory Society (ERS) International Congress demonstrated that Xenpozyme® (olipudase alfa) significantly improved pulmonary function in adults with acid sphingomyelinase deficiency (ASMD), providing positive disease modification across four critical domains of interstitial lung disease – a group of disorders that can progressively scar lung tissue and eventually affect a person’s ability to breathe.
Additional data at the congress highlight the role that artificial intelligence could play in decreasing diagnosis delay of ASMD. Many ASMD patients endure years of suffering from disease complications before receiving an accurate diagnosis. Using electronic health records of patients with unexplained interstitial lung disease, researchers applied a machine learning derived algorithm to identify high-risk patients for ASMD diagnosis.
Francesco Bonella, MD, PhD
Head of the Center for Interstitial and Rare Lung Diseases and Professor of Medicine at the Ruhrlandklinik University Hospital, Essen, Germany
“For patients with ASMD, respiratory disease most commonly manifests as interstitial lung disease, which is associated with respiratory failure and higher infection risk – a major contributor to patient mortality. In this analysis, we evaluated the effect of olipudase alfa on respiratory outcomes, and our findings showed that treatment with olipudase alfa provided sustained and progressive improvement in lung function, substantially modifying respiratory deterioration by addressing the underlying enzyme deficiency in ASMD.”
In individuals with ASMD, a deficiency in acid sphingomyelinase—an enzyme that allows for the breakdown of a fatty substance called sphingomyelin—leads to lifelong accumulation of sphingomyelin in cells throughout the body which can damage vital organs, including the lungs. According to newly developed consensus clinical guidelines for ASMD, pulmonary dysfunction is a key clinical characteristic of ASMD caused by the accumulation of sphingomyelin throughout the respiratory system.
Alaa Hamed, MD, MPH, MBA
Global Head of Medical Affairs, Rare Diseases, Sanofi
“Our data at the congress illustrate the many difficulties people with ASMD face – from the exhaustive search for answers and diagnosis to the physical symptoms that impact daily life, including shortness of breath, fatigue, and respiratory infections. We believe the data are encouraging and demonstrate our ongoing commitment to improving diagnosis and long-term care for those living with this rare genetic disease.”
Xenpozyme is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase. In 2022, Xenpozyme became the first and only approved therapy for ASMD when approved in Japan, soon followed by a string of approvals by other regulatory agencies, including the European Medicines Agency (EMA) and the US Food & Drug Administration (FDA).
Xenpozyme significantly improved lung function in adult patients with ASMD
The Phase 2 ASCEND trial evaluated 36 adults with ASMD, excluding those with neurological disease, to receive either Xenpozyme or placebo. Following the primary analysis period which spanned 52 weeks, patients continued for up to two years in the open-label extension trial to assess the long-term efficacy and safety of Xenpozyme.
These data from a post-hoc analysis of the primary analysis and open-label extension showed that Xenpozyme significantly improved respiratory function across four critical domains of interstitial lung disease, including pulmonary imaging as measured by clearance of ground glass opacities, gas exchange as measured by diffusion capacity of the lungs for carbon monoxide (DLco), lung mechanics as measured by forced vital capacity (FVC), and exercise capacity as measured by the maximum rate of oxygen (VO2) used during exercise.
Lung high-resolution computed tomography (HRCT) ground glass appearance score
- Patients who received Xenpozyme in the primary analysis experienced a -0.51 decrease in mean value HRCT score. Of those, patients continuing Xenpozyme for up to two years in the extension trial experienced a further mean decrease of -0.04.
Percent predicted DLco
- The percent predicted DLco mean increase was 10.69% for patients receiving Xenpozyme in the primary analysis. Of those, patients continuing Xenpozyme for up to two years in the open-label extension experienced an additional mean increase of 4.94%.
Percent predicted FVC
- The percent predicted FVC mean increase was 5.78% for patients receiving Xenpozyme in the primary analysis. Of those, patients continuing Xenpozyme for up to two years in the extension trial experienced an additional mean increase of 1.15%.
Percent predicted VO2
- The percent predicted VO2 mean increase was 4.65% for patients receiving Xenpozyme in the primary analysis. Of those, patients continuing Xenpozyme for up to two years in the open-label extension experienced an additional mean increase of 1.81%.
Patients who switched from placebo to Xenpozyme following the primary analysis period also experienced improvements in HRCT score, percent predicted DLco, percent predicted FVC, and percent predicted VO2.
Overall, nearly all treatment-related adverse events (99%) were mild or moderate, with one serious treatment-related adverse event, which was extrasystoles in a patient with previously documented cardiomyopathy. The most frequently reported adverse events were headache and transient transaminase elevations (i.e., increased levels of liver enzymes). No patient discontinued treatment due to adverse events.
About ASMD
ASMD, historically known as Niemann-Pick disease types A, A/B, and B, is an extremely rare, progressive genetic disease with significant morbidity and mortality, especially among infants and children. ASMD represents a spectrum of disease, with two types that may represent opposite ends of a continuum referred to as ASMD type A and ASMD type B. ASMD type A/B is an intermediate form that includes varying degrees of central nervous system (CNS) involvement. Many pediatric patients will not survive to adulthood. Signs and symptoms of ASMD may include enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding, among other disease manifestations.
About Xenpozyme (olipudase alfa)
Xenpozyme® (olipudase alfa) is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase. Xenpozyme has been evaluated in pediatric and adult patients to treat non-CNS manifestations of ASMD type A/B and ASMD type B. Xenpozyme has not been studied in patients with ASMD type A.
About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
Media Relations
Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com
Sally Bain | + 1 617 834 6026 | sally.bain@sanofi.com
Victor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com
Investor Relations
Eva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.com
Arnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.com
Corentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.com
Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com
Tarik Elgoutni| + 1 617 710 3587 | tarik.elgoutni@sanofi.com
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com
Sanofi Forward-Looking Statements
This media update contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that pandemics or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2022. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.
Attachment
