On February 22, 2024 Morphic Therapeutic (Nasdaq: MORF), a biopharmaceutical company developing a new generation of oral integrin therapies for the treatment of serious chronic diseases, reported corporate highlights and financial results for the full year 2023 (Filing, 3 mnth, DEC 31, Morphic Therapeutic, 2024, FEB 22, 2024, View Source [SID1234640378]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Today, our conviction in MORF-057 as a potential oral, well tolerated, and efficacious treatment for inflammatory bowel disease (IBD) is stronger than ever, based on the clear success of the EMERALD-1 trial in UC. Looking forward in 2024, we will work to translate this momentum into further progress with the GARNET phase 2 study in patients with moderately to severely active Crohn’s disease running in parallel with the EMERALD-2 phase 2b study in UC," commented Praveen Tipirneni, Chief Executive Officer of Morphic. "Further, we are working to expand the pipeline of candidates generated by Morphic’s MInT Platform, most notably with our α5β1 program for pulmonary hypertensive diseases and additional new projects against both integrin and non-integrin targets
"On a personal note, I am thankful to return to Morphic after an acute medical event and grateful for the immense support from friends and peers within the biotechnology community and especially the Morphic team," Tipirneni continued. "This experience has crystallized, for me, that Morphic’s opportunity to fundamentally improve patients’ lives is immense and that there is no time to waste."
2023 and Recent Corporate Highlights
EMERALD-1 Phase 2a trial of MORF-057 in UC:
•In the EMERALD-1 phase 2a trial of MORF-057 in ulcerative colitis (UC), topline data and additional data presented at UEGW 2023 indicated that in a moderately-to-severely-active UC population with severe disease burden, MORF-057:
◦Was generally well tolerated with no safety signal observed
◦Achieved the study’s primary endpoint with statistical significance in reduction of Robarts Histopathology Index (RHI) Score from baseline to week 12 of 6.4 points (p=0.002)
◦Showed consistent clinical improvement across key measures at week 12, including modified Mayo Clinic Score (mMCS) remission of 25.7% and mMCS response of 45.7%
◦Demonstrated RHI change ≥7 points in 48.6% of patients and RHI remission in 22.9% of patients
◦Led to clinical improvement in mMCS within the 12-week induction period for 77% of patients
◦Pharmacokinetic and pharmacodynamic results confirmed the results seen in healthy volunteer studies
▪Median α4β7 RO >99% and sustained saturation at week 12
▪α4β1 inhibition below the limit of quantification, in line with the design of MORF-057
▪Predicted lymphocyte subset changes observed, consistent with engagement of α4β7
◦Demonstrated deepening of clinical effect beyond the 12-week induction period, with symptomatic remission rates continuing to increase out to 44 weeks in both advanced treatment-naïve and advanced treatment-experienced patients
•Announced completion of enrollment in the exploratory cohort of the EMERALD-1 study comprised of UC patients who have previously failed treatment with vedolizumab
•Continued the 40-week maintenance phase of the EMERALD-1 study as planned

EMERALD-2 Phase 2b trial of MORF-057 in UC:
•Continued to enroll the EMERALD-2 phase 2b study of MORF-057 in patients with moderately-to-severely active UC
◦EMERALD-2 is a global phase 2b randomized, double-blind, placebo-controlled trial of MORF-057 in patients with moderate-to-severe UC
◦The primary endpoint of EMERALD-2 is clinical remission rate as measured by mMCS at 12 weeks and is expected to report in the first half of 2025
GARNET Phase 2 trial of MORF-057 in Crohn’s Disease:
•Announced that launch activities are underway for the randomized placebo-controlled GARNET Phase 2 study of MORF-057 in CD and that the study is anticipated to enroll its first patients in the first half of 2024
◦The GARNET study will evaluate 210 patients across three cohorts, each comprising 70 patients: 70 patients receiving MORF-057 200 mg BID (twice daily), 70 patients receiving MORF-057 100 mg BID and 70 patients receiving placebo
◦The primary endpoint of the GARNET study is the proportion of participants in endoscopic response (>=50% reduction) at week 14 as determined using Simple Endoscopic Score for Crohn’s Disease (SES-CD)
MORF-057 Preclinical and Phase 1 Studies:
•Presented new biomarker data at DDW 2023, demonstrating increases in circulating plasmablasts, consistent with the increased antibody activity expected with anti-inflammatory mechanism of α4β7 inhibition, supporting MORF-057 program in UC
•Presented preclinical data on rational selection of combination therapy for IBD treatment using an established clinical mode at UEGW 2023
◦This study explored preclinical combination models in UC and preliminarily examined the potential utility and rationale of combining anti-inflammatory mechanisms with α4β7 integrin inhibition in IBD
Pipeline Programs:
•Announced α5β1 as the integrin target of Morphic’s small molecule integrin inhibitor program in pulmonary hypertensive diseases
◦The inhibition of fibronectin integrins, including α5β1, suppresses pulmonary arterial smooth muscle cell proliferation and data to date indicate that inhibition of α5β1 contributes to improved cardiac output and the reversal of vascular remodeling
•Initiated discovery efforts that expand the Company’s scope in the immunology space with oral programs targeting the IL-23 and TL1A pathways, among others
Financial Results for the Full Year 2023
•Net loss for the year ended December 31, 2023, was $152.1 million or $3.59 per share compared to a net loss of $59.0 million or $1.55 per share for the year ended December 31, 2022
•Revenue was $0.5 million for the year ended December 31, 2023, compared to $70.8 million for the year ended December 31, 2022. The change was primarily due to recognition of revenue due to the conclusion of the AbbVie collaboration in 2022 and to the amounts due at the conclusion of the Janssen collaboration in 2023
•Research and development expenses were $140.4 million for the year ended December 31, 2023, as compared to $102.1 million for the year ended December 31, 2022. The increase was primarily attributable to higher development costs along with increased clinical trial costs to support phase 2 clinical studies and development activities for MORF-057, as well as other research costs to support early development candidates
•General and administrative expenses were $38.8 million for the year ended December 31, 2023, compared to $32.1 million for the year ended December 31, 2022. The increase was due to increased personnel related costs and non-cash equity-based compensation, partially offset by decreases in consulting and insurance expenses
•Morphic raised approximately $444 million, net, through equity financings in 2023 comprised of approximately $100 million in proceeds from a PIPE offering, approximately $259 million in a public offering and approximately $85 million though use of the ATM facility
•As of December 31, 2023, Morphic had cash, cash equivalents and marketable securities of $704.3 million, compared to $348.2 million as of December 31, 2022. We believe that our cash, cash equivalents and marketable securities of $704.3 million as of December 31, 2023, will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2027
About MORF-057
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 primary endpoint of EMERALD-1, change in Robarts Histopathology Index (RHI) from baseline at twelve weeks, was achieved with statistical significance. RHI is a validated instrument that measures histological disease activity in ulcerative colitis. Patients were eligible to continue for an additional 40 weeks of maintenance therapy followed by a 52-week assessment as well as an open-label extension period. Secondary and additional outcome 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 (twice daily) 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 the GARNET Study
GARNET (MORF-057-203) is a global Phase 2b randomized, double-blind, placebo-controlled trial of MORF-057 in Crohn’s disease. The primary endpoint of GARNET is the proportion of participants in endoscopic response (>=50% reduction) at week 14 as determined using Simple Endoscopic Score for Crohn’s Disease, or SES-CD. The secondary endpoints will include the change in Crohn’s Disease Activity Index, or CDAI, measures, as well as safety parameters. Patients enrolled in the GARNET study will be randomized to receive one of two active doses or a placebo: 200 mg BID (twice daily), 100 mg BID or a placebo that will cross over to MORF-057 after the 14-week induction phase. Following the 14-week induction phase, patients will move to a 38-week maintenance phase.