Dilanubicel

Treg cells have been getting more and more attention recently among autoimmune specialists. There’s been Jeff Bluestone’s Sonoma, the $157 million launch of GentiBio this summer and Egle Therapeutics — which launched just last week — to name a few. Now, there’s a new Treg player jumping in that wants to distinguish itself in the market: Mozart Therapeutics. Today, the biotech is emerging from stealth in its official debut with a $55 million Series A — with a bunch of A-list Big Pharma names on board a syndicate co-led by ARCH and Sofinnova. Playing the company’s name off as a metaphor to how the biotech wants to “orchestrate” regulatory T cell networks — as CEO Katie Fanning put it — Mozart is looking to branch itself out in the world of Treg cells by looking at CD8+ Tregs, she told Endpoints News in an interview. In her words, most of the existing biotechs looking at Treg cells for autoimmune diseases are looking at the CD4 type of cells. “We’re capitalizing on the work that came out of Mark Davis’ lab at Stanford, where he identified this novel pathway or crosstalk between CD8 regulatory T cells and CD4 cells,” Fanning said. “And he found that when those CD8 Treg cells are mobilized, that they had a cytolytic and specific killing to pathogenic CD4 cells.” Davis’ work on CD4+ and CD8+ T cells was published in Nature in 2019. That technology was then licensed to Mozart, which quietly started in July 2020 — and stayed in stealth until now. What are they focusing on? As a company, they say autoimmune and inflammatory diseases — and their first program will focus on Celiac disease. And while that program is still in lead optimization, Fanning told us that based on their own timeline, it will most likely be early 2024 until we see the candidate in the clinic. The backers Mozart has are well-known — and well-funded. Those names include Bayer through its venture division Leaps, Eli Lilly, ARCH Capital Ventures and Merck’s early-stage fund MRL Ventures Fund. One of these key investors has worked before with Fanning, a biotech veteran who had been a part of VentiRx. That investor had made the same kind of deal for the now-defunct Nohla: gather university research, make it into a company, organize a syndicate of investors to back it, and then repeat. That would be the handiwork of Steven Gillis, managing director of Arch Venture Partners, who is now chairman of the board at Mozart. Before Mozart, Arch helped raise $43.5 million in a Series A for Nohla in 2016. The biotech then quietly sold its assets in 2019 after its lead candidate, dilanubicel, failed to meet primary endpoints in clinical trials. Lucio Iannone — a new member of Mozart’s board of directors and a vice president of Leaps — has worked with Fanning before; both are board members for MIT-based biotech eGenesis. From Iannone’s perspective, Mozart’s potential technology goes hand-in-hand with one of Leaps’ main investment goals — to “reverse autoimmune diseases and chronic inflammation.” “The idea is that Leaps is always invested in transformative technologies and whatever helps to switch … from treatment to cure is something we look at,” Iannone said. “And Mozart came up as a new way to regulate CD8 regulatory T cell networks.” The biotech will use the new funds to progress its lead CD8 Treg modulator — the Celiac program — into the clinic while also advancing additional programs generated from its platform.

SEATTLE, June 15, 2018 (GLOBE NEWSWIRE) -- Nohla Therapeutics, a leading developer of universal, off-the-shelf cell therapies for patients with hematologic malignancies and other critical diseases, today announced the presentation of dilanubicel (NLA101) data demonstrating rapid immune recovery and improved long-term outcomes in Hematopoietic Stem Cell Transplant (HSCT) patients at the 23rd Congress of European Hematology Association (EHA) Annual Conference in Stockholm. Rapid Immune Recovery The data presented at EHA demonstrates that Nohla’s dilanubicel cell therapy, when added to a myeloablative cord blood transplant, significantly improved hematopoietic recovery with no incidence of transplant-related mortality (0% vs. 16% in a concurrent control group) or severe acute Graft-Versus-Host Disease, or GVHD (0% vs. 29% in a concurrent control group). Improved Long-term Outcomes In addition, data presented showed that dilanubicel treatment substantially improved long-term overall survival in patients undergoing a cord blood transplant compared to a concurrent control group (87% vs 66%), with a median follow up of 6.5 years. Of the 13 evaluable patients in the dilanubicel cohort, 9 patients (69%) no longer required immunosuppression within 2 years following transplant. GVHD Relapse-Free Survival (GRFS) is a novel composite endpoint developed by the Blood and Marrow Transplant Clinical Trials Network to measure recovery and long-term survival without ongoing major morbidity or relapse after HSCT. In addition to improved immune recovery and long-term survival, data were also presented measuring GRFS in patients who received dilanubicel. Patients who received dilanubicel in combination with a cord blood transplant were shown to have significantly improved GRFS compared to the concurrent control group (67% vs. 28%). “Evaluation of allogeneic hematopoietic cell transplant success is commonly assessed as incidence of individual complications and treatment failures,” said Colleen Delaney, MD, MSc, Chief Medical Officer and Co-founder of Nohla Therapeutics. “The GRFS endpoint provides a more complete measurement of transplant success and long-term recovery that, along with survival, also accounts for complications such as GVHD and disease relapse. When evaluating dilanubicel data using this composite benchmark, we observed a compelling improvement for patients receiving dilanubicel compared to the concurrent control group. Our ongoing randomized phase 2b study will also evaluate this important endpoint, and we look forward to these results later this year.” About Dilanubicel The company’s lead product candidate, dilanubicel, is currently under evaluation in two ongoing Phase 2 trials. A Phase 2b randomized trial (NCT01690520), has completed enrollment of 160 patients with hematologic malignancies undergoing a myeloablative allogeneic cord blood transplant with or without dilanubicel. In addition, the global Phase 2 LAUNCH trial (NCT03301597) is currently enrolling AML patients at risk for chemotherapy-induced myelosuppression. Dilanubicel is a universal donor, off-the-shelf, ex vivo expanded hematopoietic stem and progenitor cell investigational product intended to provide rapid, transient hematopoiesis with long-term immunologic benefits. Unlike autologous or patient-specific allogeneic cell therapies, dilanubicel does not require Human Leukocyte Antigen (HLA) tissue matching. Intentionally developed to provide short-term, temporary bone marrow function until a patient’s immune system recovers, dilanubicel may also induce long-term immunologic benefits with the potential for improved survival. Dilanubicel is manufactured ahead of time, cryopreserved, and intended for immediate off-the-shelf use. About Nohla Therapeutics Nohla Therapeutics is a leading developer of off-the shelf cell therapies for patients with cancer and other critical diseases. Nohla’s proprietary notch ligand technology platform serves as the foundation for its ongoing clinical, preclinical and discovery programs. The company’s lead product candidate, dilanubicel, is currently under evaluation in two ongoing Phase 2 trials. Nohla is also pursuing multiple preclinical and discovery programs in the areas of immune tolerance and other diseases. More information is available at . Nohla Contact: Jim DeNike Senior Director, Corporate Development & Investor Relations 206.519.5294