Sanofi said today it plans nine regulatory submissions over the next 18 months, and at least 10 pivotal trials for pipeline candidates over the next 12 months—parts of an R&D strategy update of its “2020 Roadmap” offered to analysts.

“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,” Elias Zerhouni, M.D., Sanofi’s global head of R&D, said in a statement.

One of the nine submissions, announced separately today by the pharma giant and development partner Regeneron, will be for cemiplimab, a programmed cell death protein 1 (PD-1) checkpoint inhibitor developed for advanced cutaneous squamous cell carcinoma (CSCC).

The companies released positive topline results from the study showing an overall response rate (ORR) of 46.3% for cemiplimab, which Sanofi declared was “the backbone of our checkpoint immuno-oncology strategy” with Regeneron.

Sanofi said the companies have launched a rolling Biologics License Application (BLA) submission to the FDA based on positive data from a pivotal Phase II clinical study of cemiplimab in 82 patients with advanced CSCC. Cemiplimab has the FDA’s “Breakthrough Therapy” designation, and the regulatory submission is expected to be completed during the first quarter of 2018.

Sanofi and Regeneron also said they plan to complete a submission to the European Medicines Agency (EMA) in the first quarter of 2018 for cemiplimab—which will also be studied in a Phase III trial in another indication, first-line treatment of non-small-cell lung cancer (NSCLC).

Submissions for Two Cancer Candidates

Cemiplimab is one of two cancer drugs for which Sanofi said it will pursue approvals over the next 18 months. The other is isatuximab, a Phase III anti-CD38 monoclonal antibody for which Sanofi said a first regulatory submission will be filed in 2018 in relapsed or refractory multiple myeloma (RRMM).

Additionally, Sanofi said, it will build on emerging evidence that CD38 inhibition may reverse resistance to programmed death-ligand 1 (PD-L1) by planning to study isatuximab in combination with cemiplimab or other immuno-oncology agents.

Also in oncology, Sanofi said, it anticipates launching 14 new proof-of-concept studies, four potential proof-of-concept readouts, and six Phase I starts, in addition to the planned submissions to the FDA and EMA.

In another key specialty care area of therapeutic focus, Sanofi said it expects to submit by year’s end a regulatory filing seeking approval for an additional indication for Dupixent® (dupilumab), also being co-developed with Regeneron—namely uncontrolled, persistent asthma, in which the drug showed positive Phase III results in September of reduced severe asthma attacks (exacerbations) and improved lung function when added to standard therapies.

Dupixent is now indicated for adults with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

Dupixent will also be assessed in Phase III studies in chronic obstructive pulmonary disease (COPD) and eosinophilic esophagitis, Sanofi said, with clinical development underway in eosinophilic esophagitis as well as nasal polyposis, food allergies, and in pediatric populations in most of these indications.

Another immunology candidate being co-developed with Regeneron, the anti-interleukin-33 (anti-IL33) antibody SAR440340, will be advanced into phase II studies in atopic dermatitis, asthma, and COPD in 2018, alone or in combination with dupilumab. The companies reason that SAR440340 has the potential for a broader spectrum of immune modulation, Sanofi said.

Other R&D updates

Sanofi also offered other R&D updates:

  • Multiple sclerosis—Sanofi said it will initiate a Phase III study in 2018 for Lemtrada® (alemtuzumab) in patients with primary progressive multiple sclerosis (MS), and deprioritize development of GLD-52 in that indication. Sanofi also plans to partner with Principia in developing a novel Bruton's tyrosine kinase inhibitor, now under study in MS with potential applications in other central nervous system diseases.
  • Rare diseases—Sanofi said it is partnering with Alnylam to advance development of patisiran for hereditary transthyretin-mediated amyloidosis (hATTR) and fitusiran for hemophilia A and B, with and without inhibitors. Late-stage/pivotal programs include olipudase, a first-in-class enzyme replacement therapy (ERT) for the nonneurological manifestations of acid sphingomyelinase deficiency, and alglucosidase alfa, a novel enzyme replacement therapy (ERT) for Pompe disease.
  • Diabetes—Late-stage candidates include sotagliflozin, a sodium–glucose cotransporters 1 and 2 (SGLT-1/2) inhibitor being developed with Lexicon; and efpeglenatide, a once-weekly glucagon-like peptide-1 (GLP-1) being developed in collaboration with Hanmi. Sanofi said it is developing breakthrough candidates for diabetes, obesity, and nonalcoholic steatohepatitis (NASH) using its novel peptide incretin platform. The lead compound—an oral dual agonist of GLP-1/GCG is expected to enter phase III in obesity in 2018.
  • Cardiovascular—Sanofi said it continues to work with Myokardia on candidates for genetic forms of cardiomyopathy. The company’s lead compound is mavacamten, a Phase II oral modulator of cardiac myosin for hypertrophic cardiomyopathy, which is expected to start a registrational phase IIb/III study in 2018. 
  • Vaccines—Six key vaccine projects are currently in development, Sanofi said, with priority disease areas including influenza, meningitis, and respiratory syncytial virus (RSV).

Sanofi said its pipeline spans 71 R&D projects, including 37 new molecular entities and novel vaccines.  The company’s R&D strategy rests on three pillars that undergird its 2020 Roadmap—namely, concurrent shifts from small molecules to biologics, from monotargeting to multitargeting compounds, and from licensing to developing proprietary assets.

“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,” stated Sanofi CEO Olivier Brandicourt, M.D.