Scholar Rock Reports Full Year 2023 Financial Results and Highlights Business Progress
- Scholar Rock's Phase 3 SAPPHIRE trial for SMA is on track to report topline data in Q4 2024.
- FDA clearance received for a Phase 2 trial with apitegromab for obesity, set to commence in mid-2024.
- Preclinical data supporting SRK-439's potential to preserve lean muscle mass presented at Keystone Symposia.
- Company ended 2023 with approximately $280 million in cash, cash equivalents, and marketable securities.
- Scholar Rock made significant progress in its antimyostatin pipeline in 2023, positioning for a strong future.
- Apitegromab, a muscle-targeted therapy for SMA, showed clinical proof-of-concept, with Phase 3 trial data expected in 2024.
- SRK-439, a myostatin inhibitor for obesity, received FDA clearance for a Phase 2 trial in mid-2024.
- Katie Peng appointed to the Board of Directors, bringing extensive commercial experience.
- Mo Qatanani promoted to Chief Scientific Officer, enhancing leadership in research and development.
- Company to host an Investor & Analyst Day event in May 2024 to showcase its programs and development plans.
- Financially, Scholar Rock ended 2023 with $280 million in cash, sufficient for operations into 2025.
- None.
Insights
The financial results of Scholar Rock, with a reported net loss of $165.8 million for the year ended December 31, 2023, reflect a typical pattern in the biopharmaceutical industry where companies invest heavily in R&D prior to product commercialization. The lack of revenue in 2023, compared to the $33.2 million in 2022, is indicative of the volatility in earnings associated with milestone-based collaboration agreements. The company's cash reserves of approximately $280 million, which are expected to fund operations into the second half of 2025, provide a buffer for the company's continued investment in their clinical trials.
Investors should note the potential inflection points, such as the SAPPHIRE trial data read-out and the anticipated commercial product launch in 2025, which could significantly impact the company's valuation. However, the inherent risks of clinical trials and regulatory hurdles remain and success is not guaranteed. The stock's performance will likely be closely tied to the outcomes of these trials and the subsequent FDA review process.
From a medical research perspective, the progress of Scholar Rock's SAPPHIRE trial and the initiation of the Phase 2 proof-of-concept trial for obesity are significant. Apitegromab, if successful, could be a novel muscle-targeted therapy for SMA, a disease with high unmet medical needs despite existing treatments. The company's focus on myostatin inhibition, a well-researched pathway in muscle growth, presents a promising therapeutic strategy. The extension data from the Phase 2 TOPAZ trial, demonstrating sustained improvements in motor function, bolsters the scientific rationale behind pursuing a Phase 3 trial.
Moreover, the exploration of apitegromab in obesity, in combination with GLP-1 RA, represents an innovative approach to preserving lean muscle mass during weight loss. This could address a significant gap in obesity management, where muscle preservation is often overlooked. The potential for SRK-439 to be filed for an IND in 2025 further demonstrates the company's commitment to expanding its pipeline within the cardiometabolic space.
From a market perspective, Scholar Rock's strategic focus on SMA and obesity, both areas with substantial market potential, positions the company to capture significant market share if their therapies prove to be successful. SMA, while a rare disease, commands high drug prices and a dedicated patient population. The company's anticipation of a commercial launch in 2025 for apitegromab, following a successful trial, could lead to rapid revenue growth.
Obesity, on the other hand, represents a larger but highly competitive market. The company's approach of combining myostatin inhibition with GLP-1 RAs could differentiate their product and target a niche segment within this space. The clinical data expected in mid-2025 will be important in determining the viability of SRK-439 in the obesity market.
- Pivotal Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy on track to report topline data in 4Q 2024
- Announced FDA clearance of IND application to initiate Phase 2 proof-of-concept trial with apitegromab to treat obesity; expected to commence in mid-2024
- Presented preclinical data supporting potential benefit of SRK-439 to preserve lean muscle mass as part of healthier weight management at Keystone Symposia
- Ended 2023 with approximately
“In 2023, Scholar Rock made significant progress across our industry-leading antimyostatin pipeline, and we are poised for a transformational year ahead. We remain on track to release topline data for our Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy in the fourth quarter of 2024, which if successful, will be the foundation of our neuromuscular franchise. Additionally, we remain on track to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 RA in mid-2024, as we continue to move SRK-439, our novel preclinical myostatin inhibitor for obesity, toward IND,” said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock. “As the only company to show clinical proof of concept in SMA with our selective antimyostatin approach, we are well positioned to further extend our leadership with a potential launch in SMA and clinical data in obesity anticipated in 2025."
Company Highlights and Upcoming Milestones
Spinal Muscular Atrophy (SMA) Program
Apitegromab is an investigational, fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle and is being developed as a potential first muscle-targeted therapy for the treatment of SMA. Apitegromab is the only muscle-targeted therapy candidate to show clinical proof-of-concept in SMA.
- On track to announce topline data from the Phase 3 SAPPHIRE clinical trial in 4Q 2024. In September 2023, the Company announced that it completed enrollment of SAPPHIRE. Scholar Rock remains on track to report topline data from the Phase 3 trial in the fourth quarter of 2024. If the trial is successful and apitegromab is approved, the Company expects to initiate a commercial product launch in 2025.
- Presented two posters at the Muscular Dystrophy Association Conference 2024 (MDA) in March 2024. Earlier this month, Scholar Rock presented 36-month extension data from the Phase 2 TOPAZ trial at MDA showing long-term substantial and sustained improvements in motor function and improvements in patient- and caregiver-reported outcomes in patients with nonambulatory types 2 and 3 SMA receiving survival motor neuron (SMN) therapy. Scholar Rock also presented a literature review that describes the ongoing burden and significant unmet needs in SMA despite advancements in treatment. In addition, the Company hosted a “Muscle Matters in SMA” lunch symposium highlighting the experiences of a person living with SMA, a neurologist, and a physical therapist.
- 12-month data from Phase 2 TOPAZ trial published in Neurology. These data demonstrated that apitegromab led to improved motor function in participants with types 2 and 3 SMA, supporting further study of apitegromab in the pivotal Phase 3 SAPPHIRE trial. The full manuscript titled, “Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3” appeared in the March 12 issue of Neurology.
- ONYX long-term extension study for patients from both the TOPAZ and SAPPHIRE studies is ongoing.
Cardiometabolic Program
SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity.
- Announced FDA clearance of IND application to initiate a Phase 2 proof-of-concept trial of apitegromab to treat obesity in patients taking a GLP-1 receptor agonist (GLP-1 RA). The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the effect of apitegromab, a highly selective myostatin inhibitor, to safely preserve lean muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. The clinical study is expected to commence in mid-2024 with data expected in mid-2025 and will be used to guide clinical development of SRK-439. The Company plans to file an IND for SRK-439 for the treatment of obesity in 2025.
- Presented preclinical SRK-439 data at the Keystone Symposia’s Obesity: Causes and Consequences meeting in February 2024. Preclinical data evaluating SRK-439 in combination with GLP-1 RAs supports the potential of SRK-439 to preserve lean muscle mass as part of healthy weight loss management. The Company intends to present additional preclinical SRK-439 data at upcoming conferences.
Corporate
- Appointed Katie Peng to the Board of Directors. In February 2024, Ms. Peng joined Scholar Rock’s Board of Directors. As an accomplished biotechnology executive, she brings over three decades of deep commercial experience in neurology and rare diseases, including with Evrydsi® for SMA, as the Company prepares for the potential commercial launch of apitegromab. Ms. Peng is currently the Chief Commercial Officer at Denali Therapeutics.
- Promoted Mo Qatanani, Ph.D. to Chief Scientific Officer. In February 2024, Dr. Qatanani was promoted to Chief Scientific Officer of Scholar Rock. Dr. Qatanani joined Scholar Rock in 2021 and has been leading all research functions since 2022 and driving Scholar Rock’s early-stage pipeline, including SRK-439. He brings over 15 years of discovery and translational research experience in developing and advancing multiple therapeutic modalities in the neuromuscular and cardiometabolic disease areas.
-
Scholar Rock to host an Investor & Analyst Day event on May 22, 2024 in
New York City . Event to highlight the Company’s myostatin inhibition programs in SMA and obesity, including commercial readiness activities for apitegromab and the SRK-439 development plan.
Full Year 2023 Financial Results
For the full year ended December 31, 2023, net loss was
-
The Company did not record any revenue for the year ended December 31, 2023. The Company recorded
$33.2 million -
Research and development expense was
$121.9 million $124.4 million -
General and administrative expense was
$49.4 million $43.1 million -
As of December 31, 2023, Scholar Rock had cash, cash equivalents, and marketable securities of approximately
$280 million
“We executed across our portfolio in 2023 and leveraged our world-leading expertise in myostatin inhibition with the formal launch of our cardiometabolic program. Additionally, we managed spend carefully throughout the year, and the capital raise in the fourth quarter extended our cash runway,” said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. “We are off to a strong start in 2024 and are well positioned for a pivotal year, looking to the SAPPHIRE data read-out as a critical inflection point for the company.”
About the Phase 3 SAPPHIRE Trial
SAPPHIRE is an ongoing randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving SMN-targeted therapy (either nusinersen or risdiplam). SAPPHIRE targeted enrolling approximately 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that targeted enrolling up to 48 patients aged 13-21 years old will also separately be evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo. For more information about SAPPHIRE, visit www.clinicaltrials.gov. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established.
About SRK-439
SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass. The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency.
About Scholar Rock
Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily of cell proteins and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role.
Scholar Rock is the only company to show clinical proof of concept for a muscle-targeted treatment in spinal muscular atrophy (SMA). This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at ScholarRock.com and follow @ScholarRock and on LinkedIn.
Availability of Other Information About Scholar Rock
Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on Twitter and LinkedIn. The information that we post on our website or on Twitter or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.
Scholar Rock® is a registered trademark of Scholar Rock, Inc.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab and SRK-181 and its preclinical programs, including SRK-439, and indication selection and development timing, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, its cash runway, expectations regarding the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 2 clinical trial of apitegromab, or Part A or Part B of the Phase 1 clinical trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates, including, without limitation, the Phase 3 clinical trial of apitegromab in SMA or Part B of the Phase 1 clinical trial of SRK-181; Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; and Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives; as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Annual Report on Form 10-K for the year ended December 31, 2023, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.
| Scholar Rock Holding Corporation | ||||||||
| Condensed Consolidated Statements of Operations | ||||||||
| (unaudited) | ||||||||
| (in thousands, except share and per share data) | ||||||||
| Years Ended December 31 | ||||||||
2023 |
2022 |
|||||||
| Revenue | $ |
— |
|
$ |
33,193 |
|
||
| Operating expenses | ||||||||
| Research and development |
|
121,900 |
|
|
124,444 |
|
||
| General and administrative |
|
49,395 |
|
|
43,119 |
|
||
| Total operating expenses |
|
171,295 |
|
|
167,563 |
|
||
| Loss from operations |
|
(171,295 |
) |
|
(134,370 |
) |
||
| Other income (expense), net |
|
5,506 |
|
|
(132 |
) |
||
| Net loss | $ |
(165,789 |
) |
$ |
(134,502 |
) |
||
| Net loss per share, basic and diluted | $ |
(1.99 |
) |
$ |
(2.26 |
) |
||
| Weighted average common shares outstanding, basic and diluted |
|
83,347,086 |
|
|
59,611,656 |
|
||
| Scholar Rock Holding Corporation | ||||||
| Condensed Consolidated Balance Sheets | ||||||
| (unaudited) | ||||||
| (in thousands) | ||||||
| December 31, 2023 | December 31, 2022 | |||||
| Assets | ||||||
| Cash, cash equivalents and marketable securities | $ |
279,938 |
$ |
315,361 |
||
| Other current assets |
|
8,256 |
|
12,663 |
||
| Total current assets |
|
288,194 |
|
328,024 |
||
| Other assets |
|
22,841 |
|
30,144 |
||
| Total assets | $ |
311,035 |
$ |
358,168 |
||
| Liabilities and Stockholders' Equity | ||||||
| Current liabilities | $ |
32,741 |
$ |
36,389 |
||
| Long-term liabilities |
|
53,076 |
|
61,544 |
||
| Total liabilities |
|
85,817 |
|
97,933 |
||
| Total stockholders' equity |
|
225,218 |
|
260,235 |
||
| Total liabilities and stockholders' equity | $ |
311,035 |
$ |
358,168 |
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20240319344594/en/
Scholar Rock:
Investors & Media
Rushmie Nofsinger
Scholar Rock
rnofsinger@scholarrock.com
ir@scholarrock.com
857-259-5573
Source: Scholar Rock
FAQ
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