-Orally administered MORF-057 achieved primary endpoint and demonstrates clinically meaningful improvements across secondary and exploratory measures-
-Clinical improvement consistently seen across key measures at week 12, mMCS response of 45.7% and endoscopic improvement of 25.7%-
-MORF-057 well tolerated with no safety signals observed-
-PK/PD confirm results seen in healthy volunteer studies, with median RO >99% and saturation achieved early and sustained at week 12-
WALTHAM, Mass., Sept. 22, 2023 (GLOBE NEWSWIRE) -- Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, today announced the publication of an abstract discussing new EMERALD-1 phase 2a primary results. The data is included in an abstract for a moderated poster presentation that will be presented by Bruce Sands, M.D., M.S., at United European Gastroenterology Week (UEGW) 2023 in Copenhagen, Denmark.
The data to be presented provide Morphic with conviction in the profile of MORF-057 as it advances towards our goal of providing a safe and effective oral α4β7 inhibitor treatment option in pill form for inflammatory bowel disease.
Abstract data and new results from the EMERALD-1 Study of MORF-057 in ulcerative colitis
In EMERALD-1 an open-label, single-arm Phase 2a trial of MORF-057 that enrolled 35 patients with moderate to severe UC, MORF-057 achieved the primary endpoint and demonstrated clinically meaningful improvements across secondary and exploratory measures. The mean Robarts Histopathology Index (RHI) score from baseline to week 12 was -6.4 (p=0.0019). Patients also demonstrated a reduction in modified Mayo Clinic Score (mMCS) from baseline to week 12 of -2.3 and 25.7% of patients achieved mMCS clinical remission.
The proportion of patients achieving a mMCS clinical response was 45.7% and endoscopic improvement was achieved in 25.7% of patients at week 12 in EMERALD-1. In the context of α4β7 inhibitors studied in ulcerative colitis, endoscopic improvement at a much longer timepoint, 52 weeks, was observed in 39.7% of patients receiving vedolizumab in the VARSITY trial, a double-blind, double dummy trial, which enrolled a patient population with less severe and less refractory disease. However comparing the results from different trials may be unreliable due to different protocol designs, trial designs, patient selection and populations, number of patients, trial endpoints, trial objectives and other parameters that may not be the same between trials. Therefore, cross-study comparisons provide very limited information about the efficacy or safety of a drug. Results of a head-to-head comparison may differ significantly from different trial comparisons.
Pharmacokinetic (PK)/Pharmacodynamic (PD) evaluations confirmed results seen in healthy volunteer studies, showing trough drug concentrations leading to a median α4β7 receptor occupancy (RO) >99%. This level of saturation was achieved early and sustained at week 12.
MORF-057 was well tolerated in EMERALD-1 with no safety signals observed. There were no serious treatment related adverse events (TEAEs) and the only two grade 3 TEAEs were exacerbation of UC, which is consistent with the disease. The most common TEAEs were UC exacerbation (11.4%) and anemia (8.6%). Anemia occurs in a third of IBD patients and all anemia occurred in EMERALD-1 in patients who had anemia at baseline, and they continued on study with iron supplements.
Moderated Poster Presentation Information
Clinical proof of principle and favorable tolerability profile shown with orally dosed MORF-057 as induction therapy for moderately to severely active UC: Phase 2a from EMERALD-1 study results
Presenter : Bruce Sands, M.D., M.S., the Dr. Burrill B. Crohn Professor of Medicine at the Icahn School of Medicine at Mount Sinai, and Chief of the Dr. Henry Janowitz Division of Gastroenterology at Mount Sinai Health System
Authors: Bruce Sands, Stefan Schreiber, Silvio Danese, Jaroslaw Kierkus, Brihad Abhyankar, Michael Choi, Carolyn Soo, Yujun Wu, Fangui Sun, Dooyoung Lee, Dan Cui, Cuyue Tang, Maloy Mangada, Ali Hussain, Peter Linde, Adrian Ray, Oladele Babalola, Sharon Brown, Sarah Hammer, Kerry McConie, Bruce Rogers, Laurent Peyrin-Biroulet, Brian Feagan
Morphic is developing MORF-057 as a selective, oral small molecule inhibitor of the α4β7 integrin for patients with inflammatory bowel disease (IBD). α4β7 has been clinically validated as a target for the treatment of IBD by the success of the approved injectable antibody therapeutic vedolizumab. MORF-057, like vedolizumab, is designed to block the interactions between α4β7 on the surface of lymphocytes and the mucosal endothelial cell ligand MAdCAM-1, substantially reducing lymphocyte migration from the bloodstream into intestinal mucosal tissues and avoiding inflammation that is associated with IBD.
About the EMERALD-1 Study
EMERALD-1 (MORF-057-201) is an open-label multi-center phase 2a trial designed to evaluate the efficacy, safety, and tolerability of MORF-057 in adults with moderate to severe ulcerative colitis. The 35 patients enrolled in the main cohort of the EMERALD-1 study have been treated with 100 mg BID (twice daily) at sites in the United States and Poland. The primary endpoint of the trial was the change in Robarts Histopathology Index (RHI), a validated instrument that measures histological disease activity in ulcerative colitis at 12 weeks compared to baseline. Patients will continue for an additional 40 weeks of maintenance therapy followed by a 52-week assessment. Secondary and additional pre-specified measures in the EMERALD-1 study include change in the modified Mayo clinic score, safety, pharmacokinetic parameters and key pharmacodynamic measures including α4β7 receptor occupancy and lymphocyte subset trafficking.
About the EMERALD-2 Study
EMERALD-2 (MORF-057-202) is a global phase 2b randomized, double-blind, placebo-controlled trial of MORF-057 that is currently enrolling patients with moderate-to-severe ulcerative colitis. The primary endpoint of EMERALD-2 is clinical remission rate as measured by the Modified Mayo Clinic Score (mMCS) at 12 weeks. EMERALD-2 will also measure several secondary and exploratory endpoints based on the mMCS as well as histologic, pharmacokinetic and pharmacodynamic measures, and safety parameters. Patients in the EMERALD-2 study will be randomized to receive either 200 mg BID MORF-057, 100 mg BID MORF-057, a QD (once daily) dose of MORF-057, or a placebo dose. Following the 12-week induction phase, all patients will receive MORF-057 for 40 weeks of maintenance dosing. For more information about the EMERALD clinical trials of MORF-057, please click here .
About Morphic Therapeutic
Morphic Therapeutic is a biopharmaceutical company developing a portfolio of oral integrin therapies for the treatment of serious chronic diseases, including autoimmune, cardiovascular, and metabolic diseases, fibrosis, and cancer. Morphic is also advancing its pipeline and discovery activities in collaboration with Schrödinger using its proprietary MInT technology platform which leverages the Company’s unique understanding of integrin structure and biology. For more information, visit www.morphictx.com .
Cautionary Note Regarding Forward-Looking Statements
This press release contains “forward-looking” statements within the meaning of the Securities Act of 1933, as amended, the Securities Exchange Act of 1934, as amended, and of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the MInT technology platform’s ability to discover drug candidates; Morphic’s plans to develop and commercialize oral small-molecule integrin therapeutics and any proposed timing thereof; the execution, timing and completion of the EMERALD-1 and EMERALD-2 clinical trials; any expectations about safety, efficacy, timing and ability to commence or complete clinical studies and/or trials and to obtain regulatory approvals for MORF-057, MORF-088 and other candidates in development; the timing of further data presentation; the ability of MORF-057 to treat IBD, including ulcerative colitis, or related indications; the ability of αvβ8 small molecule inhibitors, including MORF-088, to treat myelofibrosis; the ability for additional integrin targets to treat pulmonary hypertensive diseases; the company’s cash position and anticipated runway. Statements including words such as “believe,” “plan,” “continue,” “expect,” “will be,” “develop,” “signal,” “potential,” “anticipate” or “ongoing” and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Forward-looking statements are subject to risks and uncertainties that may cause Morphic’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties disclosed in this press release and other risks set forth in our filings with the Securities and Exchange Commission, including Morphic’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022 filed with the SEC on February 23, 2023 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2023 filed with the SEC on August 23, 2023. These forward-looking statements speak only as of the date hereof and Morphic specifically disclaims any obligation to update these forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.