Press Launch: Pivotal information show once-weekly

Press Launch: Pivotal information show once-weekly
Press Release: Pivotal data demonstrate once-weekly

Pivotal information demonstrate once-weekly efanesoctocog alfa offers superior bleed safety in comparison with prior issue prophylaxis

  • Investigational onceweekly efanesoctocog alfa prophylaxis met the first efficacy endpoint offering clinically significant bleed safety for folks with severe hemophilia A
  • Results underscore the capacity of efanesoctocog alfa to maintain regular to near-normal issue ranges and the potential to remodel prophylactic therapy, offering folks with hemophilia A with larger safety for longer
  • Further information confirmed efanesoctocog alfa prophylaxis resulted in statistically important and clinically significant enhancements in bodily well being, ache depth and joint well being in sufferers on prior issue VIII prophylaxis

Paris and Stockholm – July 10, 2022 – Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi®) (STO:SOBI) introduced for the primary time at present, in a late-breaking session on the 30th Worldwide Society on Thrombosis and Haemostasis (ISTH) Congress, constructive outcomes from the XTEND-1 pivotal Part 3 research evaluating the security, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001), an investigational issue VIII alternative remedy, in beforehand handled adults and adolescents ≥12 years with extreme hemophilia A.

The research met the first efficacy endpoint, with once-weekly efanesoctocog alfa prophylaxis offering clinically significant bleed safety for folks with extreme hemophilia A. The median and imply annualized bleeding charges (ABR) have been 0.00 (IQR: 0.00-1.04) and 0.71 (SD: 1.43) respectively. The research additionally met the important thing secondary endpoint, demonstrating superior bleed safety (p<0.0001) over prior issue VIII prophylaxis with an estimated ABR discount of 77% and a imply ABR of 0.69 in comparison with 2.96 on prior prophylaxis, based mostly on an intra-patient comparability (n=78). In a subset of individuals (n=17) studied at baseline and week 26, imply issue VIII ranges remained within the regular to near-normal vary (>40 IU/dL) for almost all of the week, and at 15 IU/dL at Day seven post-dose, offering elevated issue exercise degree safety for sufferers with once-weekly prophylaxis.

Annette von Drygalski, MD, PharmD
Investigator, Professor and Director, Hemophilia and Thrombosis Remedy Middle, UC San Diego
The section 3 information show as soon asweekly efanesoctocog alfa’s potential to offer superior bleed safety, resulting in substantial enhancements in bodily well being, ache and joint well being, by sustaining excessive issue ranges for almost all of the week. These unprecedented outcomes might supply folks with hemophilia A the chance to redefine their therapy expectations.”

Information present adults and adolescents handled with once-weekly efanesoctocog alfa skilled statistically important and clinically significant enhancements in bodily well being (p=0.0001), ache depth (p=0.0276), and joint well being (p=0.0101) when evaluating week 52 and baseline measurements.i Furthermore, efanesoctocog alfa was efficient at treating bleeds, together with in goal joints; 96.7% of bleeds have been resolved with a single 50 IU/kg dose. Efanesoctocog alfa was effectively tolerated and inhibitor improvement to issue VIII was not detected. The most typical treatment-emergent hostile occasions (>5% of individuals total) have been headache, arthralgia, fall, and again ache.

Dietmar Berger, MD, PhD
International Head of Improvement and Chief Medical Officer, Sanofi
We’re dedicated to advancing innovative medicines that disrupt the status-quo and handle the unmet wants that persist for folks with uncommon circumstances like hemophilia. These strong information illustrate the promise of efanesoctocog alfa’s efficacy with as soon asweekly dosing and underscore its potential as a remedy with best-in-disease efficacy.”

Anders Ullman, MD, PhD
Head of R&D and Chief Medical Officer, Sobi
“We consider reworking the therapy paradigm for hemophilia A can solely be achieved by means of elevating requirements of care in direction of regular hemostasis. These information show the profile of efanesoctocog alfa in important scientific phrases, and additional strengthen its potential to in the end enhance the lives of many residing with this situation.”

The united statesFood and Drug Administration (FDA) granted efanesoctocog alfa Breakthrough Remedy Designation in Could 2022, Quick Monitor designation in February 2021 and Orphan Drug designation in August 2017. The European Fee additionally granted efanesoctocog alfa Orphan Drug designation in June 2019. Regulatory submission of the Biologics License Software to the U.S. FDA occurred in June 2022 and submission within the EU will observe availability of information from the continued XTEND-Youngsters pediatric research, anticipated in 2023.

About Part 3 XTEND-1 Examine (NCT04161495)
The Part 3 XTEND-1 research (NCT04161495) was an open-label, non-randomized interventional research assessing the security, efficacy and pharmacokinetics of once-weekly efanesoctocog alfa in folks 12 years of age or older (n=159) with extreme hemophilia A who have been beforehand handled with issue VIII alternative remedy. The research consists of two parallel therapies arms — the prophylaxis Arm A (n=133), during which sufferers who had obtained prior issue VIII prophylaxis started receiving once-weekly intravenous efanesoctocog alfa prophylaxis (50 IU/kg) for 52 weeks, and the on-demand Arm B (n=26), during which sufferers who had obtained prior on-demand issue VIII remedy started 26 weeks of on-demand efanesoctocog alfa (50 IU/kg), then switched to once-weekly prophylaxis (50 IU/kg) for a further 26 weeks.

The first efficacy endpoint was the ABR in Arm A, and the important thing secondary endpoint was an intra-patient comparability of ABR through the efanesoctocog alfa weekly prophylaxis therapy interval versus the prior issue VIII prophylaxis ABR for individuals in Arm A who had participated in a earlier observational research (Examine 242HA201/OBS16221).

About hemophilia A
Hemophilia A is a uncommon, genetic dysfunction during which the flexibility of an individual’s blood to clot is impaired on account of an absence of issue VIII. Hemophilia A happens in about one in 5,000 male births yearly, and extra not often in females. Folks with hemophilia can expertise bleeding episodes that may trigger ache, irreversible joint injury and life-threatening hemorrhages. Issue alternative remedy stays a cornerstone of care and can be utilized throughout a number of therapy eventualities.

About efanesoctocog alfa
Efanesoctocog alfa, previously BIVV001, is a novel and investigational recombinant issue VIII remedy that’s designed to increase safety from bleeds with once-weekly prophylactic dosing for folks with hemophilia A. It builds on the progressive Fc fusion know-how by including a area of von Willebrand issue and XTEN® polypeptides to increase its time in circulation. It’s the first investigational issue VIII remedy that has been proven to interrupt by means of the von Willebrand issue ceiling, which imposes a half-life limitation on present issue VIII therapies. Efanesoctocog
alfa is presently underneath scientific investigation and its security and efficacy haven’t been evaluated by any regulatory authority.

In regards to the Sanofi and Sobi collaboration
Sobi and Sanofi collaborate on the event and commercialization of Alprolix® and Elocta®/Eloctate®.The businesses additionally collaborate on the event and commercialization of efanesoctocog alfa, an investigational issue VIII remedy with the potential to offer excessive sustained issue exercise ranges with once-weekly dosing for folks with hemophilia A. Sobi has remaining improvement and commercialization rights within the Sobi territory (basically Europe, North Africa, Russia and most Center Japanese markets). Sanofi has remaining improvement and commercialization rights in North America and all different areas on the planet excluding the Sobi territory.

About Sobi®
Sobi is a specialised worldwide biopharmaceutical firm reworking the lives of individuals with uncommon ailments. Offering sustainable entry to progressive medicines within the areas of haematology, immunology and specialty care, Sobi has roughly 1,600 workers throughout Europe, North America, the Center East and Asia. In 2021, income amounted to SEK 15.5 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. Extra about Sobi at, LinkedIn and YouTube.

About Sanofi
We’re an progressive world healthcare firm, pushed by one function: we chase the miracles of science to enhance folks’s lives. Our group, throughout some 100 nations, is devoted to remodeling the apply of medication by working to show the unattainable into the potential. We offer doubtlessly life-changing therapy choices and life-saving vaccine safety to tens of millions of individuals globally, whereas placing sustainability and social duty on the heart of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

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Sanofi Ahead-Trying Statements
This press launch accommodates forward-looking statements as outlined within the Personal Securities Litigation Reform Act of 1995, as amended. Ahead-looking statements are statements that aren’t historic details. These statements embody projections and estimates and their underlying assumptions, statements concerning plans, goals, intentions and expectations with respect to future monetary outcomes, occasions, operations, companies, product improvement and potential, and statements concerning future efficiency. Ahead-looking statements are typically recognized by the phrases “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and related expressions. Though Sanofi’s administration believes that the expectations mirrored in such forward-looking statements are affordable, buyers are cautioned that forward-looking info and statements are topic to numerous dangers and uncertainties, lots of that are tough to foretell and customarily past the management of Sanofi, that would trigger precise outcomes and developments to vary materially from these expressed in, or implied or projected by, the forward-looking info and statements. These dangers and uncertainties embody amongst different issues, the uncertainties inherent in analysis and improvement, future scientific information and evaluation, together with put up advertising and marketing, selections by regulatory authorities, such because the FDA or the EMA, concerning whether or not and when to approve any drug, system or organic utility that could be filed for any such product candidates in addition to their selections concerning labelling and different issues that would have an effect on the supply or business potential of such product candidates, the truth that product candidates if accredited will not be commercially profitable, the long run approval and business success of therapeutic options, Sanofi’s capacity to profit from exterior development alternatives, to finish associated transactions and/or receive regulatory clearances, dangers related to mental property and any associated pending or future litigation and the final word end result of such litigation, traits in alternate charges and prevailing rates of interest, unstable financial and market circumstances, price containment initiatives and subsequent adjustments thereto, and the affect that COVID-19 may have on us, our prospects, suppliers, distributors, and different enterprise companions, and the monetary situation of any one in all them, in addition to on our workers and on the worldwide economic system as an entire. Any materials impact of COVID-19 on any of the foregoing may additionally adversely affect us. This example is altering quickly and extra impacts might come up of which we’re not presently conscious and will exacerbate different beforehand recognized dangers. The dangers and uncertainties additionally embody the uncertainties mentioned or recognized within the public filings with the SEC and the AMF made by Sanofi, together with these listed underneath “Danger Components” and “Cautionary Assertion Relating to Ahead-Trying Statements” in Sanofi’s annual report on Type 20-F for the yr ended December 31, 2021. Aside from as required by relevant legislation, Sanofi doesn’t undertake any obligation to replace or revise any forward-looking info or statements.

i Bodily well being was assessed with the Haem-A-QoL Bodily Well being rating. Ache depth was assessed utilizing the PROMIS Ache Depth 3a previous 7 days depth of ache at its worst rating. Joint well being was assessed utilizing the Hemophilia Joint Well being rating.

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