Sanofi : Sanofi presents R&D strategy and innovative pipeline

Press Release
Source: Sanofi (EURONEXT: SAN) (NYSE: SNY)

Sanofi presents R&D strategy and innovative pipeline

                  ·         Robust pipeline expected to support long-term growth

                  ·         9 planned regulatory submissions over next 18 months

                  ·         At least 10 pivotal phase 3 studies expected to begin over next 12 months

                  ·         R&D model leverages new proprietary technology platforms,
multi-targeting molecules and biologics

PARIS, France - 13 December 2017 - Sanofi will host an analyst meeting in Paris today to discuss the company's Research and Development strategy, development pipeline and milestones for 2018. The company will highlight the progress it has made against "Sustaining Innovation", a key pillar of its 2020 strategic roadmap, and advancing a differentiated portfolio addressing unmet needs.

The company's pipeline spans 71 R&D projects, which includes 37 new molecular entities and novel vaccines. At least 10 pivotal phase 3 studies are expected to start over the next 12 months and will evaluate new treatments for:

• chronic obstructive pulmonary disease and eosinophilic esophagitis (dupilumab^[1]);
• autosomal dominant polycystic kidney disease (ADPKD), a rare kidney disease (venglustat);
• type 2 diabetes (efpeglenatide, a once-weekly GLP-1 agonist);
• obesity (a GLP-1/GCG dual agonist);
• primary progressive multiple sclerosis (alemtuzumab), and;
• first line NSCLC^[2] (cemiplimab).

Regulatory filings expected in the next 12 months include two investigational cancer drugs (cemiplimab and isatuximab), a novel therapy for type 1 diabetes (sotagliflozin) and a potential treatment for uncontrolled, persistent asthma (dupilumab).

"We have seen significant advancement on our ambition to sustain innovation in R&D, with the development of leading technology platforms and proof of concept demonstrated in multiple high-potential projects in late stage trials. We are confident this portfolio will be the foundation for Sanofi's future long-term growth," said Olivier Brandicourt, MD, Chief Executive Officer at Sanofi.

As a key pillar of the 2020 Roadmap, the new Sanofi R&D model is based on three key strategic shifts:

• From small molecules to biologics;
• From mono-targeting to multi-targeting compounds; and
• From licensing to proprietary assets.

The company has continuously adapted its R&D model in recent years to deliver greater efficiency and excellence in development, resulting in a major uplift in productivity. Since 2016, consistent with the three key strategic shifts outlined above, Sanofi has placed increasing emphasis on developing proprietary technology platforms, including multi-specific antibodies (bi- & tri-specific), siRNA, trigonal peptides, dual and triple agonists, and PRR-Antibody conjugates. It has also leveraged external expertise in targeted platforms such as mRNA mixtures and Nanobodies^®.

"We aim to advance multi-targeting therapeutic approaches for core disease pathways that have the potential to attack more than one disease at a time or bring improved risk benefit in the treatment of a single disease," said Elias Zerhouni, MD, Global Head of R&D at Sanofi. "2018 will be an important year as we expect multiple milestones for Sanofi's late-stage pipeline, made possible through the prioritization principles we have consistently applied to our early-stage research programs."

Building a competitive position in Specialty Care

Immunology
Sanofi is strengthening its specialty care portfolio and has executed launches in its fast-growing immunology franchise. Dupilumab, which we are developing in collaboration with Regeneron, has potential across multiple indications. Phase 3 trials for uncontrolled, persistent asthma recently demonstrated a potentially clinically important profile among biologic treatments. Submission in this important indication is expected before the end of 2017. Clinical development is underway in nasal polyposis, eosinophilic esophagitis, food allergies and in pediatric populations in most of these indications. Additionally, phase 3 development for dupilumab is now planned in chronic obstructive pulmonary disease (COPD). Sanofi, in collaboration with Regeneron, also expects to bring SAR440340, an anti-IL-33 antibody, which has the potential for a broader spectrum of immune modulation, into phase 2 in atopic dermatitis, asthma and COPD in 2018, alone or in combination with dupilumab.

Oncology
Sanofi is committed to re-building its position in oncology and has made major progress in the past two years. This strategy is starting to deliver and we anticipate 14 new proof-of-concept studies to be initiated, four potential proof-of-concept readouts, six phase one starts and three BLA/ MAA submissions in 2018. Cemiplimab is an investigational PD-1 checkpoint inhibitor and the backbone of our checkpoint immuno-oncology strategy with our partner Regeneron. It is being studied in cutaneous squamous cell carcinoma (CSCC), for which it was granted "Breakthrough Therapy" designation by the U.S. Food and Drug Administration (FDA), with an expected regulatory submission in Q1 2018. The development program also includes large or untapped opportunities in immuno-oncology, such as basal cell carcinoma, cervical cancer, and first line lung cancer.

Isatuximab is a Sanofi investigational antiCD38 monoclonal antibody with a first regulatory submission expected in 2018 for relapsed refractory multiple myeloma (RRMM). Beyond multiple myeloma, and building on the emerging evidence that CD38 inhibition may reverse resistance to PD-L1, isatuximab will be studied in combination with cemiplimab or other immuno-oncology agents. Sanofi will also present early research programs for its Selective Estrogen Receptor Degrader (SERD) and TGF-beta program to overcome PD-1 resistance.

Multiple Sclerosis
In multiple sclerosis (MS), Sanofi plans to build on the proven long-term clinical profile of Lemtrada^® (alemtuzumab) by initiating a Phase 3 study in 2018 for alemtuzumab in patients with primary progressive multiple sclerosis (PPMS). Consistent with Sanofi's rigorous prioritization methodology, the company will deprioritize GLD-52 in this indication in favor of  alemtuzumab.  In addition, Sanofi, in collaboration with Principia, will be developing a novel Bruton's tyrosine kinase (BTK) inhibitor, designed to access the brain and spinal cord by crossing the blood-brain barrier and impact immune cell and brain cell signaling. It is currently being studied in MS with potential applications in other central nervous system diseases^[3].

Sustaining leadership in Rare Disease, Diabetes & Cardiovascular and Vaccines

Rare Disease
Sanofi's Rare Disease pipeline is structured with the goal of sustaining innovation in lysosomal storage disorders, while also expanding strategically into related conditions. Clinical development programs include venglustat, an oral inhibitor of glucosylceramide synthase, in Fabry Disease, Gaucher Disease Type 3, GBA Parkinson's Disease and autosomal dominant polycystic kidney disease (ADKPD). Late-stage/pivotal programs include olipudase, a first-in-class enzyme replacement therapy (ERT) for the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), and avalglucosidase alfa, a novel ERT for Pompe disease. Finally, through a strategic collaboration with Alnylam, we are advancing the development of patisiran for hATTR^[4] amyloidosis and fitusiran for hemophilia A and B, with and without inhibitors.

Diabetes & Cardiovascular
Sanofi is committed to sustaining a leadership position in diabetes and expanding into adjacent co-morbidities. Its late-stage diabetes pipeline includes sotagliflozin, an investigational SGLT-1/2 inhibitor being developed in collaboration with Lexicon, and efpeglenatide, a once-weekly GLP-1 being developed in collaboration with Hanmi. Both of which potentially offer unique patient advantages. Additionally, Sanofi is leveraging its novel peptide incretin platform to develop breakthrough assets for diabetes, obesity and non-alcoholic steatohepatitis (NASH). The lead compound is an oral dual agonist of GLP-1/GCG which has shown highly competitive weight loss in the clinic and is expected to enter phase 3 in obesity in 2018. A phase 2 study in NASH is also due to start in 2018.

In cardiovascular, Sanofi continues to work in collaboration with Myokardia on therapeutic options for genetic forms of cardiomyopathy. The lead compound is mavacamten, an oral modulator of cardiac myosin, which is in phase 2 for HCM^[5] and is expected to start a registrational phase 2b/3 study in 2018. 

Vaccines
Sanofi has six key vaccine projects currently in development, and priority disease areas include influenza, meningitis and respiratory syncytial virus (RSV). RSV is the leading cause of infant viral mortality and represents a new potential category for Sanofi. The company is taking a complementary dual approach to RSV with a monoclonal antibody in phase 2, in collaboration with MedImmune, and a vaccine in phase 1.

Webcast details
The event will be webcast live on Sanofi's website at 8:30 am CET/2:30 am EST. The webcast details and full presentation will be made available on Sanofi's Investor Relations webpage and an Appendix compiling all Sanofi studies registered on clinicaltrials.gov will also be published.

Appendix 1: R&D Pipeline - New Molecular Entities ^(*)

R : Registration Study (other than Phase 3)

O : Opt-in rights products for which rights have not been exercised yet

Phase 3

Phase 2

Phase 1

1. Regeneron product for which Sanofi has opt-in right
2. Alnylam product for which Sanofi has opt-in right
3. Also known as MYK491
4. Also known as SAR439684 and REGN2810
5. Also known as Niemann Pick type B
6. Regulus product for which Sanofi has opt-in right
    
7. Also known as SAR439152 and as MYK461
8. Also known as MEDI8897
9. Currently on clinical hold pending outcome of FDA discussion - Expected to resume around year-end

(*) data related to all studies published in clinicaltrials.gov

(**) Partnered and/or in collaboration - Sanofi may have limited or shared rights on some of these products

Appendix 2: R&D Pipeline - Additional Indications (*)

R : Registration Study (other than Phase 3)

O : Opt-in rights products for which rights have not been exercised yet

Registration

Phase 3

Phase 2

Phase 1

1. Also known as SAR439684 and REGN2810
2. Cyclophosmamide + bortezomib (Velcade) + dexamethasone
3. Regeneron product for which Sanofi has opt-in right
4. Also known as SAR439152 and as MYK461

 (*) data related to all studies published in clinicaltrials.gov

(**) Partnered and/or in collaboration - Sanofi may have limited or shared rights on some of these products

^[1] Partnered products: cemiplimab, dupilumab, anti-IL33 mAb (Regeneron); sotagliflozin (Lexicon); efpeglenatide (Hanmi); fitusiran, patisiran (Alnylam); mavacamten, MYK-491 (Myokardia).

^[2] Non-Small Cell Lung Cancer

^[3] The Principia transaction remains subject to customary regulatory approvals and has not yet closed.

^[4]hATTR = Hereditary Transthyretin-Mediated Amyloidosis

^[5] HCM= Hypertrophic cardiomyopathy

Attachments:

http://www.globenewswire.com/NewsRoom/AttachmentNg/27cd8267-f6e1-4c3b-bb6a-79e295409d2a