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 quarter ended September 30, 2023.
"This has been a pivotal time for Monte Rosa as we announced
both our first clinical data and our first major strategic
collaboration,” said Markus Warmuth, M.D., Chief Executive Officer
of Monte Rosa Therapeutics. “We’re very excited about the clinical
data for MRT-2359, our GSPT1-directed molecular glue degrader, and
in particular the early signs of clinical activity in MYC-driven
solid tumors. We’re equally excited to partner with Roche to enable
and accelerate expansion of our QuEEN™ discovery engine to discover
and develop molecular glues against targets previously considered
impossible to drug for cancer and neurological diseases. We also
completed a registered direct offering that, together with our
ongoing pipeline prioritization efforts, is expected to extend our
cash runway into the first half of 2026. We look forward to
continuing to execute on our pipeline, including delivering Phase 2
interim data for MRT-2359 and Phase I healthy volunteer data for
our immunology and inflammation-directed VAV1 and NEK7
programs.”
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 $28.3 million,
compared to $21.3 million for the third quarter of 2022. These
increases were driven by the successful achievement of key
milestones in our R&D organization, including the advancement
of MRT-2359 in the clinic, the progression of our preclinical
pipeline, and the continued development of the Company’s QuEEN™
platform for discovery efforts. The increase in R&D expenses
was driven by increased headcount and laboratory-related expenses
to achieve these milestones. Non-cash stock-based compensation
constituted $2.3 million of R&D expenses for Q3 2023, compared
to $1.7 million in the same period in 2022.
General and Administrative (G&A) Expenses:
G&A expenses for the third quarter of 2023 were $8.7 million
compared to $7.0 million for the third quarter of 2022. The
increase in G&A expenses was a result of increased headcount
and expenses in support of the company’s growth and operations.
G&A expenses included non-cash stock-based compensation of $2.2
million for the third quarter of 2023, compared to $1.5 million for
the same period in 2022.
Net Loss: Net loss for the third quarter of
2023 was $34.9 million, compared to $27.3 million for the third
quarter of 2022.
Cash Position and Financial Guidance: Cash,
cash equivalents, restricted cash, and marketable securities as of
September 30, 2023, were $183.0 million, compared to cash, cash
equivalents, restricted cash, and marketable securities of $207.6
million as of June 30, 2023. The decrease of $24.6 million was
primarily related to cash used to fund operations. The September
30, 2023, cash and equivalents balance does not include the $50
million upfront payment from Roche or the approximately $25 million
proceeds from the registered direct offering, which were received
after the end of Q3 2023.
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-2359MRT-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-6160MRT-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 RosaMonte 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. Monta 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 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 December 31, 2023, filed with the
U.S. Securities and Exchange Commission on March 16, 2023, 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) |
|
September 30, |
December 31, |
|
|
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 endedSeptember
30, |
|
|
Nine months endedSeptember
30, |
|
|
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 |
) |
InvestorsAndrew Funderburk, Kendall
IRir@monterosatx.com
MediaCory Tromblee, Scient
PRmedia@monterosatx.com
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