Press Release: TZIELD® Phase 3 data presented at ISPAD shows potential to slow the progression of Stage 3 type 1 diabetes in newly diagnosed children and adolescents; full data simultaneously published in The NEJM
TZIELD® Phase 3 data presented at ISPAD shows potential to slow the progression of Stage 3 type 1 diabetes in newly diagnosed children and adolescents; full data simultaneously published in The New England Journal of Medicine
- TZIELD met the study’s primary endpoint, significantly slowing the decline of C-peptide levels, compared to placebo
- Numerical trends favoring TZIELD were seen in key secondary endpoints, whilst statistical significance was not achieved
- PROTECT builds on the existing body of evidence on TZIELD’s potential to slow the progression of type 1 diabetes
- TZIELD fits at the intersection of Sanofi’s growth in immune-mediated diseases and disease modifying therapies, and the company’s expertise in diabetes
Paris, October 18, 2023. New data from TZIELD’s (teplizumab-mzwv) PROTECT Phase 3 trial were presented today at the 49th Annual ISPAD Conference, in Rotterdam, The Netherlands. PROTECT studied the efficacy and safety of TZIELD, compared to placebo, to slow the loss of beta cells and preserve beta cell function as measured by C-peptide, in children and adolescents aged 8-17 years diagnosed in the preceding 6 weeks with Stage 3 autoimmune type 1 diabetes (T1D). The full data set has been simultaneously published in The New England Journal of Medicine.
TZIELD met the study’s primary endpoint, demonstrating superior beta cell preservation assessed by significantly slowing the decrease in mean C-peptide levels (area under the curve [AUC] after a 4-hour mixed meal tolerance test) at trial completion, compared to placebo. C-peptide is a biomarker for beta cell function. This significant difference indicates the potential of TZIELD to slow the progression of Stage 3 type 1 diabetes in this population. While the study’s key secondary endpoints did not meet statistical significance, numerical trends favoring TZIELD were seen in relevant clinical parameters. On average, people on TZIELD required numerically fewer insulin units and had numerically higher time in range, compared to those on placebo. HbA1c reductions and the overall rates of clinically important low blood sugar (hypoglycemic) events were similar among both study groups.
Kevan Herold, MD
C.N.H. Long Professor of Immunobiology and of Medicine (Endocrinology), Yale School of Medicine and Primary Investigator of PROTECT.
“Type 1 diabetes is a chronic autoimmune disease, driven by the destruction of the insulin-producing beta cells, and as such, beta cell preservation remains a meaningful unmet need for all patients with diabetes. These new results build on the findings from multiple studies across different stages of the disease process, further supporting TZIELD’s potential to modulate the progression of T1D.”
Jose Eduardo Neves, M.D.
Senior Vice President, Global Head of Medical Affairs, General Medicines, Sanofi
“The PROTECT results are encouraging, as we believe they showcase the potential for TZIELD to slow down the progression of Stage 3 T1D in this population, as well as pointing towards favorable trends in relevant aspects for clinicians and people living with type 1 diabetes. We look forward to discussing this new data with the scientific community and regulatory authorities around the world.”
The availability of the PROTECT data represents a key early milestone for Sanofi on TZIELD, following the acquisition of Provention Bio (a Sanofi Company) in April 2023. TZIELD is a strategic fit for Sanofi at the intersection of our growth in immune-mediated diseases and disease modifying therapies, and our company’s expertise in diabetes.
The PROTECT clinical trial was a randomized, double blind, placebo-controlled, multi-national trial. From baseline and through the trial’s completion at 78 weeks, the following was observed for TZIELD vs placebo:
- Significantly less decrease in mean C-peptide levels (area under the concentration curve [AUC], following a 4-hour mixed-meal tolerance test [MMTT]): difference in least-squares means (LSM) of 0.13 pmol/mL; (95% CI: 0.09, 0.17; P<0.001).
- 94.9% of participants in the TZIELD group maintained peak C-peptide levels ≥0.2 pmol/mL, compared with 79.2% of those who received the placebo (P<0.001).
- Numerically lower mean insulin dose in favor of TZIELD at Week 78: the least-square mean (LSM) for insulin dose at week 78 was 0.46 U/Kg/day (TZIELD) and 0.59 U/kg/day (placebo), difference -0.13 U/kg/day (95% CI: -0.28, 0.02).
- Comparable change in mean HbA1c: LSM change of -1.98% (TZIELD) vs -1.89% (placebo), difference -0.09 (95% CI: -0.42, 0.24)
- Numerically higher mean time in range at week 78 in favor of TZIELD (>70 but ≤180 mg/dL): 68.7±19.6% (TZIELD) vs 64.6±22.4% (placebo). Difference of 4.71% (95% CI: -1.72, 11.15).
- Similar mean rates of overall clinically important hypoglycemic events: estimated rates of 4.68 (TZIELD) (95% CI: 3.70, 5.91) vs 4.24 (placebo) (95% CI: 3.06, 5.89) events/patient-year, with an estimated rate ratio of 1.10 (95% CI: 0.74, 1.64).
The safety results of the trial were consistent with previous data from TZIELD’s currently approved FDA indication to delay the onset of Stage 3 type 1 diabetes in adults and children 8 years and older diagnosed with Stage 2 T1D, as well as other prior clinical studies with TZIELD. No new safety signals were identified.
Adverse events of special interest (AESI) were prespecified and occurred in 29% of those on TZIELD vs 21.6% on placebo, the most frequent one being hypoglycemia (TZIELD: 13.4%; placebo: 16.2%). Other common adverse events (AEs) were headache, nausea, rash, lymphopenia and vomiting. Serious adverse events (SAEs) were reported by 5.5% of participant who received TZIELD vs 5.4% on placebo; the most common SAEs were cytokine release syndrome (TZIELD: 1.4%; placebo 0%) and infections (TZIELD: 0%; placebo: 2.7%).
The use of TZIELD in the PROTECT population is investigational, and its safety and efficacy in this population has not been evaluated by any regulatory authority.
PROTECT (NCT03875729) is a Phase 3, randomized, double blind, placebo-controlled, multi-national clinical trial. It enrolled 328 children and adolescents (TZIELD n=217, placebo n=111) aged 8-17 years diagnosed with clinical, Stage 3 T1D in the preceding 6 weeks; randomization ratio of TZIELD:placebo was 2:1. Participants received a first course of 12 daily infusions (of either TZIELD or placebo) at randomization, followed by a second course of 12 daily infusions after 26 weeks (approx. 6 months). All participants received standard-of-care as required.
The primary objective of PROTECT was to determine whether TZIELD can slow down beta cell loss and preserve beta cell function measured by C-peptide, compared to placebo. This was assessed via the trial’s primary endpoint, which measured the difference in mean change of C-peptide level (area under the time-concentration curve [AUC] measured after a 4-hour mixed meal tolerance test) from baseline to Week 78 between both groups.
Key secondary endpoints included HbA1c, time in range (TiR) as measured with a CGM, clinically important low blood sugar (hypoglycemia) events and exogenous insulin use. Time in range was defined as: >70 but ≤180 mg/d. Clinically relevant hypoglycemic events were defined: level 2 hypoglycemia (<54 mg/dL / 3.0 mmol/L) and level 3 hypoglycemia as episodes of severe cognitive impairment requiring external assistance for recovery, even in the absence of a blood glucose reading.
Other secondary endpoints were adverse events and overall safety aspects, as well as pharmacokinetics (PK) and immunogenicity of TZIELD. An observational extension study following participants for a further 42 months is ongoing.
TZIELD (teplizumab-mzwv) is a CD3-directed monoclonal antibody. TZIELD is the first and only disease modifying therapy in autoimmune type 1 diabetes (T1D); it was approved by the U.S. FDA in November 2022 to delay the onset of Stage 3 type 1 diabetes in adults and children 8 years and older diagnosed with Stage 2 T1D.
About autoimmune, type 1 diabetes (T1D)
T1D is a chronic autoimmune condition where the body’s ability to regulate blood sugar levels is impacted due to the gradual destruction of insulin producing beta cells by one’s own immune system.
There are 3 stages to the progression of T1D:
- In Stage 1, the autoimmune attack to the beta cells has started, and this can be detected by the presence of 2 or more T1D-related autoantibodies in the blood. During Stage 1, blood sugar levels are in a normal range. At this stage, T1D is asymptomatic.
- In Stage 2 (also asymptomatic), in addition to the presence of 2 or more T1D-related autoantibodies, blood sugar levels are now abnormal (dysglycemia) due to the progressive loss of beta cells / beta cell function. People diagnosed with Stage 2 T1D have a near 100% lifetime chance of progression to Stage 3 T1D, with 75% of them progressing to it within five years.
- Stage 3 (also known as clinical stage) comes once a significant portion of the beta cells have been destroyed. At this point, rising blood sugar levels reach the point of clinical hyperglycemia (which defines diabetes), and many people will experience the classic symptoms that come with the onset of Stage 3 T1D: increased thirst, frequent urination, unexplained weight loss, blurred vision and generalized fatigue. Management of Stage 3 T1D requires daily and burdensome insulin replacement therapy.
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
Sandrine Guendoul | + 33 6 25 09 14 25 | email@example.com
Evan Berland | + 1 215 432 0234 | firstname.lastname@example.org
Victor Rouault | + 33 6 70 93 71 40 | email@example.com
Eva Schaefer-Jansen | + 33 7 86 80 56 39 | firstname.lastname@example.org
Arnaud Delépine | + 33 6 73 69 36 93 | email@example.com
Corentine Driancourt | + 33 6 40 56 92 21 | firstname.lastname@example.org
Felix Lauscher | + 1 908 612 7239 | email@example.com
Tarik Elgoutni| + 1 617 710 3587 | firstname.lastname@example.org
Nathalie Pham | + 33 7 85 93 30 17 | email@example.com
This press release 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 regarding the marketing and other potential of the product, or regarding potential future revenues from the product. 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, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, 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.
AttachmentTo view this piece of content from www.globenewswire.com, please give your consent at the top of this page.
To view this piece of content from ml-eu.globenewswire.com, please give your consent at the top of this page.
About GlobeNewswire by notified
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire by notified is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Subscribe to releases from GlobeNewswire by notified
Subscribe to all the latest releases from GlobeNewswire by notified by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from GlobeNewswire by notified
Exhibition World Bahrain Secures "World’s Leading New Exhibition and Convention Centre 2023" Award at World Travel Awards 20233.12.2023 11:00:00 CET | Press release
DUBAI, United Arab Emirates, Dec. 03, 2023 (GLOBE NEWSWIRE) -- Exhibition World Bahrain (EWB), the Middle East’s newest and largest exhibition and convention centre, is proud to announce securing the “World’s Leading New Exhibition and Convention Centre 2023” at the World Travel Awards 2023. The award accolade was presented to Dr. Nasser Qaedi, the CEO of Bahrain Tourism and Exhibitions Authority (BTEA), during the World Travel Awards 2023 Grand Final Gala Ceremony, which took place at Burj Al Arab in Dubai, UAE, on December 1, 2023, in the presence of HE Philip Joseph Pierre, the prime minister of Saint Lucia, tourism ministers, and travel elites across the globe. Exhibition World Bahrain captured the highest number of votes, clinching the internationally recognised award from Bharat Mandapam, India, and Tākina Wellington Convention and Exhibition centre, New Zealand. With this unrivalled win, the Kingdom of Bahrain has further cemented its reputation as the leading global hub for the
Merus Presents Interim Data on MCLA-129 at ESMO Asia Congress 20233.12.2023 02:40:00 CET | Press release
MCLA-129 in combination with chemotherapy in 2L+ EGFRm NSCLC planned to initiate 1Q24 UTRECHT, The Netherlands and CAMBRIDGE, Mass., Dec. 02, 2023 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS) (“Merus”, “the Company”, “we”, or “our”), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics® and Triclonics®), today announced updated interim clinical data on MCLA-129 from ongoing expansion cohorts in non-small cell lung cancer (NSCLC) and in previously treated head and neck squamous cell carcinoma (HNSCC) were presented at the European Society for Medical Oncology (ESMO) Asia Congress 2023. “MCLA-129 is a very active drug in EGFRm NSCLC and we’re planning a focused investment to evaluate MCLA-129 in combination with chemotherapy, which we expect to start early in 2024,” said Bill Lundberg M.D., President, Chief Executive Officer of Merus. “We are in a fortunate position to have a strong balance sheet. We also recognize the importance of be
CASGEVY Gets Bahrain approval for treatment, marking it second country in the world2.12.2023 18:59:21 CET | Press release
MANAMA, Bahrain, Dec. 02, 2023 (GLOBE NEWSWIRE) -- Bahrain has become the second country in the world and the first in the Middle East to approve the use of CASGEVY (exagamglogene autotemcel) for the treatment of patients with sickle cell disease and transfusion-dependent beta-thalassemia. The treatment was developed by Vertex Pharmaceuticals and CRISPR Therapeutics, after conducting a series of successful clinical trials in the UK, US, France, Italy, and Germany, among others. Following the UK MHRA’s authorisation to use CASGEVY, Bahrain is the second country in the world and the first in the region to approve this treatment. This extraordinary development opens up new possibilities for medical advancements and reinforces Bahrain's commitment to pioneering healthcare solutions, offering a better quality of life for patients with these blood disorders. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene-editing technique that holds immense potenti
Scatec at COP28: Accelerating renewable energy transition from Egypt to South Africa2.12.2023 14:01:22 CET | Press release
Dubai, 2 December 2023: This week, Scatec ASA, a leading renewable energy company, is actively engaging in the UN Climate Change Conference in Dubai (COP28). Our focus is to underscore renewable energy as an unequivocal solution to climate change and emphasise the critical need for increased green energy investments in emerging markets. COP28 serves as a pivotal platform for global stakeholders to reinforce commitments aimed at averting catastrophic consequences of climate change. Scatec aligns with climate experts in stressing the urgency of the situation and the imperative to continue advancing towards the objectives outlined in the Paris Agreement. There's no time to waste, and collective progress is crucial for safeguarding our planet's future. “We are at COP28 to highlight the key role renewable energy must play in our energy future, "At COP28, our focus is on underscoring the crucial role renewable energy must assume in shaping our energy future, particularly in emerging markets,
Unplugged Performance Introduces UP INVINCIBLE®: A Game-Changer for Tesla Cybertruck Enthusiasts2.12.2023 05:06:20 CET | Press release
HAWTHORNE, Calif., Dec. 01, 2023 (GLOBE NEWSWIRE) -- Unplugged Performance, the industry leader in performance upgrades for Tesla vehicles, debuts a new chapter in Tesla tuning with the arrival of their UP INVINCIBLE® product line. UP INVINCIBLE® is tailored exclusively for Tesla Cybertruck owners who want to push the limits of on-road and off-road performance. The unveiling took place at Unplugged Performance’s celebratory afterparty for Tesla's highly anticipated Cybertruck delivery event on November 30th. Hosted at the company's headquarters in Hawthorne, California, neighboring Tesla Design Studio, SpaceX, and Starlink, the event drew a capacity crowd, reflecting the immense enthusiasm for this innovative product range. UP INVINCIBLE® represents a concerted effort to harness and amplify the rugged DNA of the Cybertruck, making it even more robust and capable in a variety of challenging environments. During the event, Ben Schaffer, CEO of Unplugged Performance, presented an extensiv