Interim data from Phase 1/2 clinical trial of MRT-2359 demonstrated tumor reductions in patients with biomarker-positive cancers; recommended Phase 2 dose expected in Q2 2024
Announced strategic collaboration with Roche to discover novel molecular glue degraders targeting cancer and neurological diseases;
Presented preclinical data demonstrating that MRT-6160, a VAV1-targeting MGD, attenuates autoimmune disease progression; IND submission expected 1H 2024
Completed
Strong cash position expected to fund operations into H1 2026 and enable advancement of GSPT1, VAV1 and NEK7 pipeline programs through clinical milestones
"This has been a pivotal time for Monte Rosa as we announced both our first clinical data and our first major strategic collaboration,” said
THIRD QUARTER 2023 AND RECENT HIGHLIGHTS
- Announced interim clinical data from the ongoing MRT-2359 Phase 1/2 study demonstrating favorable pharmacokinetic (PK), pharmacodynamic (PD), and tolerability profiles in heavily pretreated patients with MYC-driven solid tumors, including lung cancers and high-grade neuroendocrine tumors. MRT-2359 showed evidence of tumor size reductions, including partial responses, in heavily pretreated patients with biomarker-positive tumors. Optimal PD modulation of GSPT1 in peripheral blood mononuclear cells and tumor tissue biopsies was observed at all dose levels, consistent with its designed activity based on preclinical studies.
- Entered into a strategic collaboration and licensing agreement with global healthcare leader Roche to discover and develop MGDs against targets in cancer and neurological diseases. Under the terms of the agreement,
Monte Rosa Therapeutics will receive an upfront payment of$50 million , and is eligible to receive future preclinical, clinical, commercial and sales milestone payments that could exceed$2 billion , as well as tiered royalties. - Completed a
$25 million registered direct offering, priced at-the-market under Nasdaq rules, with a life sciences-dedicated investor. - Presented preclinical data at the
American College of Rheumatology (ACR) Convergence Annual Meeting demonstrating the potential of MRT-6160, a VAV1-targeting MGD, to treat immunological and inflammatory diseases. The data show that MRT-6160 attenuated disease progression in a murine collagen-induced arthritis (CIA) model. - Appointed
Anthony M. Manning , Ph.D., to the Company’s Board of Directors.Dr. Manning is a highly accomplished drug discovery leader in the field of autoimmune and inflammatory diseases. - Discontinued sickle cell disease program to prioritize internal efforts on ongoing clinical-, Investigational New Drug (IND)- and lead optimization-stage programs.
UPCOMING MILESTONES
- The Company now expects to release the recommended Phase 2 dose for the MRT-2359 Phase 1/2 study in Q2 2024.
- On track for planned IND submission for MRT-6160 in the first half of 2024.
- The Company expects to nominate a development candidate for its NEK7 preclinical program in Q1 2024.
- The Company expects to nominate a development candidate for its CDK2 preclinical program in 2024.
THIRD QUARTER 2023 FINANCIAL RESULTS
Research and Development (R&D) Expenses: R&D expenses for the third quarter of 2023 were
General and Administrative (G&A) Expenses: G&A expenses for the third quarter of 2023 were
Net Loss: Net loss for the third quarter of 2023 was
Cash Position and Financial Guidance: Cash, cash equivalents, restricted cash, and marketable securities as of
The company expects its cash and cash equivalents, including proceeds from the Roche collaboration, will be sufficient to fund planned operations and capital expenditures into the first half of 2026.
About MRT-2359
MRT-2359 is a potent, 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 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 autoimmune indications, such as multiple sclerosis, rheumatoid arthritis, and dermatological disorders. Preclinical studies demonstrate MRT-6160 inhibits disease progression in in vivo autoimmunity models.
About Monte Rosa
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 product development activities, our ongoing clinical development of our GSPT1 degrader referred to as MRT-2359, including our expectations for the nature, significance, and timing for our disclosure of any initial data from our Phase 1/2 clinical trial of MRT-2359 in MYC-driven solid tumors, timing for our identification and any disclosure of a recommended phase 2 dose for MRT-2359, statements about the advancement of our preclinical programs, pipeline and the various products therein, including the ongoing development of our VAV1-directed degrader, referred to as MRT-6160, and the planned submission of an IND to the FDA for MRT-6160 in the first half of 2024, our expectations regarding the potential clinical benefit for this program and our expectations of timings for the program, statements around the advancement and application of our pipeline, including identification and the timing thereof of a development candidate for NEK7 and a development candidate for CDK2, 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, as well as our expectations of success for our programs and the strength of our financial position, 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
Consolidated Balance Sheets | |||||||
(in thousands, except share amounts) | |||||||
2023 | 2022 | ||||||
Assets | |||||||
Current assets: | |||||||
Cash and cash equivalents | $ | 59,055 | $ | 54,912 | |||
Marketable securities | 119,422 | 207,914 | |||||
Other receivables | 294 | 7,656 | |||||
Prepaid expenses and other current assets | 4,140 | 4,444 | |||||
Current restricted cash | — | 960 | |||||
Total current assets | 182,911 | 275,886 | |||||
Property and equipment, net | 34,992 | 27,075 | |||||
Operating lease right-of-use assets | 29,408 | 34,832 | |||||
Restricted cash, net of current | 4,522 | 4,318 | |||||
Other long-term assets | 270 | 278 | |||||
Total assets | $ | 252,103 | $ | 342,389 | |||
Liabilities and stockholders’ equity | |||||||
Current liabilities: | |||||||
Accounts payable | $ | 5,500 | $ | 7,862 | |||
Accrued expenses and other current liabilities | 14,228 | 14,580 | |||||
Current portion of operating lease liability | 2,881 | 3,127 | |||||
Total current liabilities | 22,609 | 25,569 | |||||
Defined benefit plan liability | 1,453 | 1,533 | |||||
Operating lease liability | 43,517 | 43,874 | |||||
Total liabilities | 67,579 | 70,976 | |||||
Commitments and contingencies | |||||||
Stockholders’ equity | |||||||
Common stock | 5 | 5 | |||||
Additional paid-in capital | 518,610 | 503,696 | |||||
Accumulated other comprehensive loss | (1,455 | ) | (1,752 | ) | |||
Accumulated deficit | (332,636 | ) | (230,536 | ) | |||
Total stockholders’ equity | 184,524 | 271,413 | |||||
Total liabilities and stockholders’ equity | $ | 252,103 | $ | 342,389 |
Consolidated Statements of Operations and Comprehensive Income (Loss) | |||||||||||||||
(In thousands, except share and per share amounts) | |||||||||||||||
Three months ended | Nine months ended | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Operating expenses: | |||||||||||||||
Research and development | $ | 28,306 | $ | 21,342 | $ | 84,137 | $ | 60,193 | |||||||
General and administrative | 8,662 | 7,020 | 24,311 | 19,702 | |||||||||||
Total operating expenses | 36,968 | 28,362 | 108,448 | 79,895 | |||||||||||
Loss from operations | (36,968 | ) | (28,362 | ) | (108,448 | ) | (79,895 | ) | |||||||
Other income: | |||||||||||||||
Interest income | 2,227 | 997 | 6,966 | 1,774 | |||||||||||
Foreign currency exchange gain (loss), net | 27 | 63 | (151 | ) | 293 | ||||||||||
Gain on disposal of fixed assets | — | (16 | ) | 24 | 109 | ||||||||||
Loss on sale of marketable securities | — | — | (131 | ) | — | ||||||||||
Total other income | 2,254 | 1,044 | 6,708 | 2,176 | |||||||||||
Net loss before income taxes | (34,714 | ) | (27,318 | ) | (101,740 | ) | (77,719 | ) | |||||||
Provision for income taxes | (170 | ) | — | (360 | ) | — | |||||||||
Net loss | $ | (34,884 | ) | $ | (27,318 | ) | $ | (102,100 | ) | $ | (77,719 | ) | |||
Net loss per share attributable to common stockholders—basic and diluted | $ | (0.70 | ) | $ | (0.58 | ) | $ | (2.06 | ) | $ | (1.67 | ) | |||
Weighted-average number of shares outstanding used in computing net loss per common share—basic and diluted | 49,814,903 | 46,732,353 | 49,533,143 | 46,666,000 | |||||||||||
Net loss | $ | (34,884 | ) | $ | (27,318 | ) | $ | (102,100 | ) | $ | (77,719 | ) | |||
Other comprehensive income (loss): | |||||||||||||||
Provision for pension benefit obligation | 14 | 32 | 42 | 99 | |||||||||||
Unrealized gain (loss) on available-for-sale securities | 171 | (176 | ) | 255 | (680 | ) | |||||||||
Comprehensive loss | $ | (34,699 | ) | $ | (27,462 | ) | $ | (101,803 | ) | $ | (78,300 | ) |
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