BOSTON, May 09, 2024 (GLOBE NEWSWIRE) -- Monte Rosa Therapeutics, Inc. (Nasdaq: GLUE), a clinical-stage biotechnology company developing novel molecular glue degrader (MGD)-based medicines, today reported business highlights and financial results for the first quarter ending March 31, 2024.
“We’re excited by the significant advances made across our entire portfolio, including both our oncology and immunology/inflammation programs,” said Markus Warmuth, M.D., Chief Executive Officer of Monte Rosa Therapeutics. “Our Phase 1/2 clinical trial evaluating MRT-2359 for MYC-driven solid tumors is on track and we plan to announce the recommended Phase 2 dose later this quarter and to report clinical data from this program in the second half of the year. We eagerly anticipate the initiation of a Phase 1 study of MRT-6160, the first of our MGD drug candidates for immune-related diseases, in mid-2024, with results from the study expected in Q1 2025. Preclinical GLP toxicology data we’ve announced today, along with data in multiple disease models, suggest the potential for a highly differentiated profile across multiple autoimmune diseases. Additionally, MRT-8102, our NEK7-directed MGD targeting diseases driven by IL-1β and the NLRP3 inflammasome, is rapidly progressing toward clinical studies. We’ve recently demonstrated strong CNS exposure and NEK7 degradation in non-human primates, supporting the potential development of MRT-8102 in neurologic indications and obesity amongst others, in addition to its potential use in gout, pericarditis, and other peripheral inflammatory conditions. We’re also very pleased to announce our discovery program for CCNE1 (Cyclin E1), a well-validated oncology target generally considered undruggable by conventional modalities. We believe our ability to degrade CCNE1 potently and selectively and to elicit anti-tumor activity in in vivo models provides further validation for the differentiation of our QuEEN™ discovery engine and the potential of our MGDs against a broad spectrum of targets.”
Dr. Warmuth concluded, “The rapid and efficient progression of our pipeline clearly illustrates the power of our AI/ML-driven QuEEN™ discovery engine. Through our ongoing internal efforts as well as our research collaboration with Roche, we look forward to continuing this strong progress.”
RECENT HIGHLIGHTS
MRT-2359, GSPT1-directed MGD for MYC-driven solid tumors
MRT-6160, VAV1-directed MGD for systemic and neurological autoimmune/inflammatory diseases
MRT-8102, NEK7-directed MGD for inflammatory diseases driven by IL-1β and the NLRP3 inflammasome
CCNE1-directed MGD program for CCNE1-amplified tumors
Additional corporate updates
ANTICIPATED MILESTONES
UPCOMING PRESENTATIONS
FIRST QUARTER 2024 FINANCIAL RESULTS
Collaboration Revenue: Collaboration revenue for the first quarter of 2024 was $1.1 million, compared with $0 during the same period in 2023. Collaboration revenue represents revenue recorded under the Roche License and Collaboration agreement.
Research and Development (R&D) Expenses: R&D expenses for the first quarter of 2024 were $27.0 million, compared to $26.8 million during the same period in 2023. R&D expenses were driven by the successful achievement of key milestones in our R&D organization, including the continuation of the MRT-2359 clinical study, the progression and growth of our preclinical pipeline, the preparation of MRT-6160 to enter the clinic, and the continued development of the Company’s QuEEN™ discovery engine. Non-cash stock-based compensation constituted $2.7 million of R&D expenses for Q1 2024, compared to $2.1 million in the same period in 2023.
General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2024 were $9.0 million compared to $7.5 million during the same period in 2023. The increase in G&A expenses was a result of increased headcount, stock-based compensation expense, and fees paid to consultants in order to support our growth and operations. G&A expenses included non-cash stock-based compensation of $2.2 million for the first quarter of 2024, compared to $1.8 million for the same period in 2022.
Net Loss: Net loss for the first quarter of 2024 was $32.0 million, compared to $33.3 million for the fourth quarter of 2023.
Cash Position and Financial Guidance: Cash, cash equivalents, restricted cash, and marketable securities as of March 31, 2024, were $197.8 million, compared to cash, cash equivalents, restricted cash, and marketable securities of $237.0 million as of December 31, 2023. The decrease of $39.2 million was primarily due to operational use of cash.
The Company expects its cash and cash equivalents to be sufficient to fund planned operations and capital expenditures into the first half of 2026.
About MRT-2359
MRT-2359 is a potent, highly selective, and orally bioavailable investigational molecular glue degrader (MGD) that induces the interaction between the E3 ubiquitin ligase component cereblon and the translation termination factor GSPT1, leading to the targeted degradation of GSPT1 protein. The MYC transcription factors (c‑MYC, L-MYC and N-MYC) are well-established drivers of human cancers that maintain high levels of protein translation, which is critical for uncontrolled cell proliferation and tumor growth. Preclinical studies have shown this addiction to MYC-induced protein translation creates a dependency on GSPT1. By inducing degradation of GSPT1, MRT-2359 is designed to exploit this vulnerability, disrupting the protein synthesis machinery, leading to anti-tumor activity in MYC-driven tumors.
About MRT-6160
MRT-6160 is a potent, highly selective, and orally bioavailable investigational molecular glue degrader of VAV1, which in our in vitro studies has shown deep degradation of its target with no detectable effects on other proteins. VAV1, a Rho-family guanine nucleotide exchange factor, is a key signaling protein downstream of both the T- and B-cell receptors. VAV1 expression is restricted to blood and immune cells, including T and B cells. Preclinical studies have shown that targeted degradation of VAV1 protein via an MGD modulates both T- and B-cell receptor-mediated activity. This modulation is evident both in vitro and in vivo, demonstrated by a significant decrease in cytokine secretion, proteins vital for maintaining autoimmune diseases. Moreover, VAV1-directed MGDs have shown promising activity in preclinical models of autoimmune diseases and thus have the potential to provide therapeutic benefits in multiple systemic and neurological autoimmune indications, such as multiple sclerosis, rheumatoid arthritis, inflammatory bowel disease, and dermatological disorders. Preclinical studies have demonstrated that MRT-6160 can inhibit disease progression in in vivo autoimmunity models.
About MRT-8102
MRT-8102 is a potent, highly selective, and orally bioavailable investigational molecular glue degrader (MGD) that targets NEK7 for the treatment of inflammatory diseases driven by IL-1β and the NLRP3 inflammasome. NEK7 has been shown to be required for NLRP3 inflammasome assembly, activation and IL-1β release both in vitro and in vivo. Aberrant NLRP3 inflammasome activation and the subsequent release of active IL-1β and interleukin-18 (IL-18) has been implicated in multiple inflammatory disorders, including gout, cardiovascular disease, neurologic disorders including Parkinson’s disease and Alzheimer’s disease, ocular disease, diabetes, obesity, and liver disease. In a non-human primate model, MRT-8102 was shown to potently, selectively, and durably degrade NEK7, and resulted in near-complete reductions of IL-1β models following ex vivo stimulation of whole blood. MRT-8102 has shown a favorable profile in non-GLP toxicology studies.
About Monte Rosa
Monte Rosa Therapeutics is a clinical-stage biotechnology company developing highly selective molecular glue degrader (MGD) medicines for patients living with serious diseases in the areas of oncology, autoimmune and inflammatory diseases, and more. MGDs are small molecule protein degraders that have the potential to treat many diseases that other modalities, including other degraders, cannot. Monte Rosa’s QuEEN™ (Quantitative and Engineered Elimination of Neosubstrates) discovery engine combines AI-guided chemistry, diverse chemical libraries, structural biology, and proteomics to identify degradable protein targets and rationally design MGDs with unprecedented selectivity. The QuEEN discovery engine enables access to a wide-ranging and differentiated target space of well-validated biology across multiple therapeutic areas. Monte Rosa has developed the industry’s leading pipeline of MGDs, which spans oncology, autoimmune and inflammatory disease and beyond, and has a strategic collaboration with Roche to discover and develop MGDs against targets in cancer and neurological diseases previously considered impossible to drug. For more information, visit www.monterosatx.com.
Forward-Looking Statements
This communication includes express and implied “forward-looking statements,” including forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that are not historical facts and in some cases, can be identified by terms such as “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue,” “ongoing,” or the negative of these terms, or other comparable terminology intended to identify statements about the future. Forward-looking statements contained herein include, but are not limited to, statements about our ability to grow our product pipeline, statements around the Company’s QuEEN™ discovery engine and the Company’s view of its potential to identify degradable protein targets and rationally design MGDs with unprecedented selectivity, statements around the power and differentiation of the QuEEN discovery engine and the potential of the Company’s MGDs against a broad spectrum of targets, statements about the advancement and timeline of our preclinical and clinical programs, pipeline and the various products therein, including (i) our product development activities, our ongoing clinical development of MRT-2359, our expectations to announce the recommended Phase 2 dose later in the second quarter of 2024, the timing for our disclosure of any initial data from our Phase 1 clinical trial of MRT-2359 in the second half of 2024, and our plans to initiate the Phase 2 portion of the study before year-end, (ii) the ongoing development of MRT-6160, and the planned submission of an IND to the FDA for MRT-6160 in the second quarter of 2024, our expectations of timing for initiation of a Phase 1 SAD/MAD study mid-2024 and the timing for our disclosure of Phase 1 clinical data of MRT-6160 in the first quarter of 2025, as well as our expectation to present additional preclinical data in models of autoimmune and inflammatory diseases at the upcoming DDW and EULAR medical meetings and our expectation to initiate POC studies for MRT-6160 in autoimmune/inflammatory diseases including ulcerative colitis and rheumatoid arthritis, with additional potential POC studies, dermatology, rheumatology, and neurology indications in mid-2024, (iii) our ongoing development of MRT-8102 and our expectations around its potential across neurologic indications and obesity amongst others, as well as potential use in gout, pericarditis, and other peripheral inflammatory conditions, including our expectations to submit an IND to the FDA in the first quarter of 2025, and (iv) our expectations to nominate a development candidate for the CDK2 preclinical program in 2024, statements around the advancement and application of our platform, and statements concerning our expectations regarding our ability to nominate and the timing of our nominations of additional targets, product candidates, and development candidates, statements regarding regulatory filings for our development programs, including the planned timing of such regulatory filings, such as IND applications, and potential review by regulatory authorities as well as our expectations of success for our programs and the strength of our financial position, our use of capital, expenses and other financial results in the future, availability of funding for existing programs, ability to fund operations into the first half of 2026, among others. By their nature, these statements are subject to numerous risks and uncertainties, including those risks and uncertainties set forth in our most recent Annual Report on Form 10-K for the year ended December 31, 2023, filed with the U.S. Securities and Exchange Commission on March 14, 2024, and any subsequent filings, that could cause actual results, performance or achievement to differ materially and adversely from those anticipated or implied in the statements. You should not rely upon forward-looking statements as predictions of future events. Although our management believes that the expectations reflected in our statements are reasonable, we cannot guarantee that the future results, performance, or events and circumstances described in the forward-looking statements will be achieved or occur. Recipients are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date such statements are made and should not be construed as statements of fact. We undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, any future presentations, or otherwise, except as required by applicable law. Certain information contained in these materials and any statements made orally during any presentation of these materials that relate to the materials or are based on studies, publications, surveys and other data obtained from third-party sources and our own internal estimates and research. While we believe these third-party studies, publications, surveys and other data to be reliable as of the date of these materials, we have not independently verified, and make no representations as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, no independent source has evaluated the reasonableness or accuracy of our internal estimates or research and no reliance should be made on any information or statements made in these materials relating to or based on such internal estimates and research.
| Consolidated Balance Sheets | ||||||||
| (in thousands, except share amounts) | ||||||||
| (Unaudited) | ||||||||
| March 31, | December 31, | |||||||
| 2024 | 2023 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 99,752 | $ | 128,101 | ||||
| Marketable securities | 93,140 | 104,312 | ||||||
| Other receivables | 601 | 505 | ||||||
| Prepaid expenses and other current assets | 5,543 | 3,294 | ||||||
| Total current assets | 199,036 | 236,212 | ||||||
| Property and equipment, net | 34,036 | 33,803 | ||||||
| Operating lease right-of-use assets | 28,422 | 28,808 | ||||||
| Restricted cash | 4,863 | 4,580 | ||||||
| Other long-term assets | 389 | 352 | ||||||
| Total assets | $ | 266,746 | $ | 303,755 | ||||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 6,828 | $ | 11,152 | ||||
| Accrued expenses and other current liabilities | 10,713 | 14,600 | ||||||
| Current deferred revenue | 20,407 | 17,678 | ||||||
| Current portion of operating lease liability | 3,345 | 3,162 | ||||||
| Total current liabilities | 41,293 | 46,592 | ||||||
| Deferred revenue, net of current | 28,529 | 32,323 | ||||||
| Defined benefit plan liability | 2,568 | 2,713 | ||||||
| Operating lease liability | 41,837 | 42,877 | ||||||
| Total liabilities | 114,227 | 124,505 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity | ||||||||
| Common stock, $0.0001 par value; 500,000,000 shares authorized, 50,210,309 shares issued and 50,200,304 shares outstanding as of March 31, 2024; and 50,154,929 shares issued and 50,140,233 shares outstanding as of December 31, 2023 | 5 | 5 | ||||||
| Additional paid-in capital | 553,063 | 547,857 | ||||||
| Accumulated other comprehensive loss | (2,693 | ) | (2,724 | ) | ||||
| Accumulated deficit | (397,856 | ) | (365,888 | ) | ||||
| Total stockholders’ equity | 152,519 | 179,250 | ||||||
| Total liabilities and stockholders’ equity | $ | 266,746 | $ | 303,755 | ||||
| Consolidated Statement of Operations and Comprehensive Loss | ||||||||
| (in thousands, except share and per share amounts) | ||||||||
| (unaudited) | ||||||||
| Three months ended March 31, | ||||||||
| 2024 | 2023 | |||||||
| Collaboration revenue | $ | 1,064 | $ | — | ||||
| Operating expenses: | ||||||||
| Research and development | 27,026 | 26,755 | ||||||
| General and administrative | 8,985 | 7,504 | ||||||
| Total operating expenses | 36,011 | 34,259 | ||||||
| Loss from operations | (34,947 | ) | (34,259 | ) | ||||
| Other income (expense): | ||||||||
| Interest income, net | 2,442 | 2,437 | ||||||
| Foreign currency exchange (loss) gain, net | 620 | (85 | ) | |||||
| Loss on sale of marketable securities | — | (131 | ) | |||||
| Total other income | 3,062 | 2,221 | ||||||
| Net loss before income taxes | (31,885 | ) | (32,038 | ) | ||||
| Provision for income taxes | (83 | ) | — | |||||
| Net loss | $ | (31,968 | ) | $ | (32,038 | ) | ||
| Net loss per share—basic and diluted | $ | (0.53 | ) | $ | (0.65 | ) | ||
| Weighted-average number of shares outstanding used in computing net loss per common share—basic and diluted | 60,156,187 | 49,347,473 | ||||||
| Comprehensive loss: | ||||||||
| Net loss | $ | (31,968 | ) | $ | (32,038 | ) | ||
| Other comprehensive loss: | ||||||||
| Provision for pension benefit obligation | 35 | 14 | ||||||
| Unrealized gain (loss) on available-for-sale securities | (4 | ) | 345 | |||||
| Comprehensive loss | $ | (31,937 | ) | $ | (31,679 | ) | ||
Investors
Andrew Funderburk
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Media
Cory Tromblee, Scient PR
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| Last Trade: | US$9.92 |
| Daily Change: | 0.81 8.89 |
| Daily Volume: | 259,183 |
| Market Cap: | US$608.790M |
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