Moderna Announces Recent Progress in Its Immuno-Oncology and Rare Disease Programs and Highlights Corporate Objectives
- IND amendment submitted to the
FDAfor a Phase 2 cohort of OX40L (mRNA-2416) to treat advanced ovarian carcinoma
- First patient dosed in the Phase 1 study of OX40L + IL23 + IL36γ (Triplet) (mRNA-2752) for advanced or metastatic solid tumor malignancies or lymphoma
- Planning for a Phase 2 study of personalized cancer vaccine (mRNA-4157) initiated together with strategic collaborator Merck
- IND submitted to the
FDAfor a Phase 1/2 study of methylmalonic acidemia (mRNA-3704)
“This year we are focused on making significant advances to our pipeline as we work to bring multiple programs into Phase 2 clinical trials, move programs within our rare disease portfolio toward the clinic and leverage our mRNA platform to create both new development candidates and potential new modalities where we believe there is an opportunity to develop therapies for a broad range of diseases,” said Stéphane Bancel, Moderna’s Chief Executive Officer. “I am pleased with the continued progress of our pipeline, our ability to now manufacture mRNA for clinical development at our new site in
Mr. Bancel will present a company overview and strategy update today at
Detailed program updates:
Intratumoral Immuno-oncology: These programs aim to drive anti-cancer T cell responses by injecting mRNA therapies directly into tumors.
- OX40L (mRNA-2416): Based on previously reported clinical observations in two patients with advanced ovarian carcinoma in its Phase 1 study,
Modernahas submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration( FDA) and to the study’s clinical research sites to commence a Phase 2 cohort of mRNA-2416 as a monotherapy in advanced ovarian carcinoma within its current Phase 1 study. Thus far, 28 patients have been dosed in the ongoing Phase 1 trial for mRNA-2416, an open-label, multicenter study of repeated intratumoral injections of mRNA-2416 in patients with advanced relapsed/refractory solid tumor malignancies and lymphomas. Initial data from the Phase 1 study were presented in a poster session at the Annual Meeting of the Society for Immunotherapyof Cancer in November 2018.
- OX40L + IL23 + IL36γ (Triplet) (mRNA-2752):
Modernahas dosed the first patient in the Phase 1 study of mRNA-2752, an intratumoral injection comprising three mRNAs encoding for OX40L + IL23 + IL36γ for the treatment of advanced or metastatic solid tumor malignancies or lymphoma. The open label, multi-center study is evaluating the safety and tolerability of mRNA-2752 as a single agent and in combination either with AstraZeneca’s durvalumab or tremelimumab, and will assess anti-tumor activity, protein expression in tumors and pharmacokinetics, and exploratory endpoints that include assessment of immunological response.
Cancer Vaccines: These programs focus on stimulating a patient’s immune system to tumor-related antigens to enable the immune system to elicit a more effective antitumor response.
- Personalized Cancer Vaccine (PCV) (mRNA-4157):
Modernaand Merck areplanning a randomized Phase 2 study comparing PCV and KEYTRUDA® against KEYTRUDA alone. To date, interim Phase 1 PCV study data from 24 patients showed no dose limiting toxicities up to 0.39 mg (the third of four dose levels). Interim Phase 1 immunogenicity data have also been collected in certain patients dosed with mRNA-4157 as a monotherapy, and potential antigen-specific T cell responses have been detected. The Phase 1 study continues in the dose-escalation phase of the protocol.
- KRAS vaccine (mRNA-5671): Merck will lead an open-label, multi-center, dose-escalation and dose-expansion Phase 1 study to evaluate the safety and tolerability of mRNA-5671 administered as an intramuscular injection both as a monotherapy and in combination with KEYTRUDA. KRAS is a frequently mutated oncogene in epithelial cancers, primarily in non-small cell lung, colorectal and pancreatic cancers. The IND for a KRAS vaccine was originally submitted by
Modernaand included an mRNA for the membrane protein STimulator of INterferon Gene (STING) to help promote antitumor activity. That IND was transferred to Merck which now will move the program forward under the same IND with KRAS as the sole mRNA. Merck may choose to include STING mRNA in later clinical development of the KRAS vaccine.
Systemic Intracellular Therapeutics: These programs aim to deliver mRNA into cells within target organs as a therapeutic approach for diseases caused by a missing or defective protein.
- Methylmalonic Acidemia (MMA) (mRNA-3704): An IND application has been submitted to the
FDAfor mRNA-3704, Moderna’s development candidate for MMA. If approved, this will be Moderna’s first rare disease program to advance into clinical trials. The Company plans to conduct an open-label, multi-center, dose escalation Phase 1/2 study of multiple ascending doses of mRNA-3704 in pediatric patients with isolated MMA due to MUT enzyme deficiency. The objectives of the study are to evaluate safety and tolerability. mRNA-3704 has received Rare Pediatric Disease Designation by the FDAand Orphan Drug Designation by both the FDAand the European Medicines Agency.
- Propionic Acidemia (PA) (mRNA-3927): mRNA-3927 was granted Orphan Drug Designation by the
FDAin December 2018and Rare Pediatric Disease Designation by the FDAin January 2019. PA is a rare, life-threatening, inherited metabolic disorder due to a defect in the mitochondrial enzyme propionyl-CoA carboxylase, or PCC. It primarily affects the pediatric population and there is no approved therapy. Modernais continuing to advance mRNA-3927 in pre-clinical studies. Modernaalso continues to enroll patients in a global natural history study of MMA and PA (MaP Study) designed to identify and correlate clinical and biomarker endpoints for these disorders. This is a global, multi-center, non-interventional study for patients with confirmed diagnosis of MMA due to methylmalonyl-CoA mutase (MUT) deficiency or PA.
More than 760 subjects have been dosed with a therapeutic or vaccine candidate developed with Moderna’s mRNA technology.
Information about each program in Moderna’s pipeline, including those discussed in this press release, can be found on our investor relations page of our website www.modernatx.com.
1. Generate human proof-of-concept data for multiple medicines
2. Execute on current development pipeline
3. Create new development candidates in existing modalities
4. Invent new modalities
Continued growth across organization:
Continued strong cash position: We expect our cash, cash equivalents, and investments in marketable securities as of
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended including, but not limited to, statements concerning: Moderna’s 2019-2020 corporate objectives; Moderna’s plans to move multiple programs into Phase 2 clinical trials and programs within its rare disease portfolio toward the clinic; Moderna’s plans to create new development candidates; Moderna’s plans to develop therapies in new modalities to treat a broad range of diseases; the potential of intratumoral immuno-oncology to drive anti-cancer T cell responses with mRNA medicines; the commencement of a Phase 2 cohort for mRNA-2416; the potential for Systemic Intracellular Therapeutic programs to deliver mRNA into cells within target organs as a therapeutic approach; the advancement of mRNA-3704 into clinical trials; and Moderna’s expectations regarding its cash, cash equivalents, and investments in marketable securities as of