Scholar Rock Reports Third Quarter 2024 Financial Results and Highlights Business Progress
Scholar Rock (SRRK) reported positive Phase 3 SAPPHIRE trial results for apitegromab in Spinal Muscular Atrophy (SMA), achieving its primary endpoint with statistically significant motor function improvement. The company plans to submit BLA and MAA applications in Q1 2025. The Phase 2 EMBRAZE obesity trial enrollment is complete with data expected in Q2 2025. Financial highlights include completion of a $345M public offering and Q3 2024 net loss of $64.5M ($0.66/share). Cash position stands at $139.1M, with pro forma balance of $463.5M including October offering, extending runway into Q4 2026.
Scholar Rock (SRRK) ha riportato risultati positivi della fase 3 dello studio SAPPHIRE per l'apitegromab nella Atrofia Muscolare Spinale (SMA), raggiungendo il parametro primario con un miglioramento statisticamente significativo della funzione motoria. L'azienda prevede di presentare le domande di BLA e MAA nel primo trimestre del 2025. L'arruolamento per lo studio di fase 2 EMBRAZE sull'obesità è completato e i dati sono attesi nel secondo trimestre del 2025. Tra i risultati finanziari, si segnala il completamento di un'offerta pubblica di 345 milioni di dollari e una perdita netta di 64,5 milioni di dollari nel terzo trimestre del 2024 (0,66 dollari/azione). La posizione di cassa è di 139,1 milioni di dollari, con un bilancio pro forma di 463,5 milioni di dollari che include l'offerta di ottobre, assicurando così liquidità fino al quarto trimestre del 2026.
Scholar Rock (SRRK) informó resultados positivos del ensayo de fase 3 SAPPHIRE para el apitegromab en la Atrofia Muscular Espinal (AME), alcanzando el objetivo principal con una mejora en la función motora que es estadísticamente significativa. La empresa planea presentar solicitudes de BLA y MAA en el primer trimestre de 2025. La inscripción del ensayo de obesidad EMBRAZE de fase 2 está completa y se esperan datos en el segundo trimestre de 2025. Los aspectos financieros incluyen la finalización de una oferta pública de 345 millones de dólares y una pérdida neta de 64.5 millones de dólares en el tercer trimestre de 2024 (0.66 dólares/acción). La posición de efectivo se sitúa en 139.1 millones de dólares, con un balance pro forma de 463.5 millones de dólares que incluye la oferta de octubre, extendiendo los recursos hasta el cuarto trimestre de 2026.
Scholar Rock (SRRK)는 척수성 근위축증(SMA) 치료를 위한 아피테그로맙의 3상 SAPPHIRE 시험에서 긍정적인 결과를 보고하였으며, 통계적으로 유의미한 운동 기능 개선을 통해 주요 목표를 달성했습니다. 이 회사는 2025년 1분기에 BLA 및 MAA 신청서를 제출할 계획입니다. 2상 EMBRAZE 비만 시험의 등록이 완료되었으며, 데이터는 2025년 2분기에 예상됩니다. 재무 하이라이트로는 3억 4천5백만 달러의 공개 공모가 완료되었고, 2024년 3분기 순손실은 6천4백50만 달러(주당 0.66달러)입니다. 현금 보유액은 1억 3천9백10만 달러이며, 10월 공모를 포함한 프로 포르마 잔액은 4억 6천3백50만 달러로, 2026년 4분기까지 자금이 연장되었습니다.
Scholar Rock (SRRK) a rapporté des résultats positifs de l'essai de phase 3 SAPPHIRE pour l'apitegromab dans l'atrophie musculaire spinale (AMS), atteignant son objectif principal avec une amélioration statistiquement significative de la fonction motrice. L'entreprise prévoit de soumettre des demandes de BLA et de MAA au cours du premier trimestre 2025. L'inscription à l'essai de phase 2 EMBRAZE sur l'obésité est complète et les données sont attendues au deuxième trimestre 2025. Les points saillants financiers comprennent l'achèvement d'une offre publique de 345 millions de dollars et une perte nette de 64,5 millions de dollars pour le troisième trimestre 2024 (0,66 dollar/action). La position de trésorerie est de 139,1 millions de dollars, avec un bilan pro forma de 463,5 millions de dollars incluant l'offre d'octobre, prolongeant ainsi la liquidité jusqu'au quatrième trimestre 2026.
Scholar Rock (SRRK) berichtete über positive Ergebnisse der Phase 3 der SAPPHIRE-Studie zu Apitegromab bei spinaler Muskelatrophie (SMA) und erreichte dabei das primäre Ziel mit einer statistisch signifikanten Verbesserung der motorischen Funktion. Das Unternehmen plant, im 1. Quartal 2025 Anträge auf BLA und MAA einzureichen. Die Rekrutierung der Phase 2 EMBRAZE-Studie zur Adipositas ist abgeschlossen, und die Daten werden im 2. Quartal 2025 erwartet. Finanzielle Höhepunkte umfassen den Abschluss eines öffentlichen Angebotes über 345 Millionen USD und einen Nettoverlust von 64,5 Millionen USD im 3. Quartal 2024 (0,66 USD/Aktie). Die Liquiditätsposition beläuft sich auf 139,1 Millionen USD, mit einer pro forma Bilanz von 463,5 Millionen USD, die das Angebot im Oktober umfasst und somit die finanziellen Ressourcen bis zum 4. Quartal 2026 sichert.
- Positive Phase 3 SAPPHIRE trial results showing statistically significant improvement in motor function
- Successful completion of $345M upsized public offering
- Strong cash position of $463.5M (pro forma) extending runway into Q4 2026
- 98% patient retention rate in ONYX open-label expansion study (185/188 patients)
- Completed enrollment for Phase 2 EMBRAZE obesity trial
- Increased net loss to $64.5M in Q3 2024 vs $42.4M in Q3 2023
- Research and development expenses increased to $48.7M from $30.3M year-over-year
- General and administrative expenses rose to $16.1M from $13.3M year-over-year
- No revenue recorded for Q3 2024
Insights
The Q3 results and recent developments present a significant positive catalyst for Scholar Rock. Key financial highlights include:
- Successful completion of a
$345 million upsized public offering strengthening the balance sheet - Extended cash runway into Q4 2026 with pro forma cash position of
$463.5 million - Q3 net loss of
$64.5 million ($0.66 per share) vs$42.4 million in Q3 2023
The positive Phase 3 SAPPHIRE trial results for apitegromab in SMA validate the company's technology platform and set up potential commercialization in late 2025. The increased R&D spend of
The SAPPHIRE trial results represent a major clinical milestone with statistically significant improvements in motor function for SMA patients. Key findings include:
- Primary endpoint achieved with 1.8 point mean improvement in HFMSE scores vs placebo (p=0.0192)
- Strong efficacy signal at 10 mg/kg dose with 2.2 point improvement (p=0.0121)
- 98% patient retention in ONYX extension study indicates favorable safety profile
The data validates apitegromab as potentially the first muscle-targeted therapy for SMA. The successful completion of EMBRAZE trial enrollment in obesity also demonstrates broader platform potential in cardiometabolic disorders. The planned OPAL trial expansion into patients under 2 years old could further expand the addressable market.
- Reported positive topline data from pivotal Phase 3 SAPPHIRE trial evaluating apitegromab in patients with Spinal Muscular Atrophy (SMA), achieving primary endpoint
- On track to submit a
- Phase 2 EMBRAZE trial enrollment completed for apitegromab in obesity with topline data expected in 2Q 2025
- Successful completion of upsized
- Management to host update call today at 8:15 a.m. ET
“We were thrilled to announce positive and statistically significant topline data from the pivotal Phase 3 SAPPHIRE trial, a major achievement that underscores apitegromab’s potential to redefine the standard of care for patients with SMA,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “The positive SAPPHIRE trial validates our approach to targeting pro-and latent myostatin and demonstrates apitegromab’s potentially transformative benefit. Based on these results, we plan to submit a BLA and MAA in the first quarter of 2025 and are working diligently to prepare for the commercial launch of apitegromab, and if approved, to deliver the potentially transformative benefits of apitegromab to children and adults living with SMA.”
Dr. Backstrom continued, “The robust results from SAPPHIRE also reinforce the broader potential of our selective myostatin inhibition strategy across our pipeline, with potential readthrough to our cardiometabolic program as we evaluate apitegromab in obesity in our Phase 2 EMBRAZE proof-of-concept trial. We were pleased to announce in September that the EMBRAZE trial is fully enrolled, and we expect to report initial data in the second quarter of next year.”
Company Highlights and Upcoming Milestones
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 first muscle-targeted therapy to show clinical proof-of-concept in SMA.
-
Reported positive topline data from Phase 3 SAPPHIRE clinical trial. The study achieved its primary endpoint demonstrating a statistically significant and clinically meaningful improvement for apitegromab versus placebo in motor function as measured by the gold standard HFMSE in SMA patients on chronic dosing of standard of care therapies (either nusinersen or risdiplam).
- In the main efficacy population (ages 2-12), the mean difference in change from baseline in HFMSE was 1.8 points (p=0.0192) for all patients receiving apitegromab 10 mg/kg and 20 mg/kg (n=106) compared to placebo (n=50). Patients receiving 20 mg/kg of apitegromab (n=53) showed a 1.4 point mean difference compared to placebo (p=0.1149).
- The prespecified analysis of the 10 mg/kg dose showed that patients receiving 10 mg/kg of apitegromab (n=53) showed a mean difference in change from baseline in HFMSE of 2.2 points compared to placebo (nominal p=0.0121).
- Based upon PK/PD data from the SAPPHIRE trial, similar levels of target engagement were observed for the 10 mg/kg and 20 mg/kg dose groups.
-
Scholar Rock plans to submit a
U.S. BLA and a European Union MAA in 1Q 2025.
-
Presented preliminary baseline characteristics from SAPPHIRE during a poster presentation at the 29th Annual Congress of the World Muscle Society in October in
Prague, Czech Republic . Analyses of the full Phase 3 SAPPHIRE data are ongoing, and Scholar Rock plans to present detailed results at an upcoming medical conference in early 2025. - The ONYX open-label, multi-center extension study remains ongoing. The study is evaluating the long-term safety and efficacy of apitegromab in patients who completed the TOPAZ or SAPPHIRE trials. More than 90 percent of patients on combination therapy in the TOPAZ trial have completed 4 years of apitegromab treatment and enrolled into ONYX. Following trial completion, 98 percent of SAPPHIRE patients (185/188) enrolled in the ongoing ONYX open-label expansion study.
- On track to initiate OPAL clinical trial in SMA patients under two years old in mid-2025.
Cardiometabolic Program
SRK-439 is a novel, preclinical, investigational myostatin inhibitor that binds to pro- and latent myostatin with high affinity and is selective for myostatin (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 during weight loss.
- Completed enrollment of Phase 2 EMBRAZE proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in September. The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of apitegromab, a highly selective investigational myostatin inhibitor, to preserve muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. Data are expected in the second quarter of 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.
Immuno-Oncology Program
SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy, such as anti-PD-(L)1 antibodies, in patients with advanced cancer.
-
New SRK-181 data from the Phase 1 DRAGON proof-of-concept trial presented at the SITC 39th Annual Meeting in November. The presentation, “DRAGON Trial: Durable remission rate with the latent TGFβ1 inhibitor linavonkibart (SRK-181) and pembrolizumab in patients with immune checkpoint inhibitor resistant advanced cancers,” highlighted new clinical and biomarker results. Highlights since the Company’s last update on the DRAGON trial include one additional complete response, seen in the clear cell renal cell (ccRCC) cohort (ORR=
23.3% ) and durable responses in multiple cohorts. Additionally, biomarker results continue to support proof of mechanism; new data shows suppressed granulocytic myeloid-derived suppressor cells (gMDSC) levels in the tumor microenvironment of responders. Notably new biomarker data in the ccRCC cohort supports a potential patient selection strategy with positive correlations between clinical response and baseline CD8+ infiltration status, elevated baseline Treg levels, and elevated baseline TGFβ1 levels in the tumor compartment. Enrollment of the DRAGON trial was completed in December 2023, and patients who remain on the study continue to be treated.
Other Corporate Updates
- Appointed Beth Shafer, Ph.D., to the newly created role of Chief Business Officer. Ms. Shafer brings over 20 years of biopharmaceutical industry leadership and expertise to Scholar Rock, where she will drive the Company’s long-term corporate and business development strategy.
-
Raised
$345 million $345 million
Third Quarter 2024 Financial Results
For the quarter ended September 30, 2024, net loss was
- The Company did not record any revenue for the quarter ended September 30, 2024, or for the quarter ended September 30, 2023.
-
Research and development expense was
$48.7 million $30.3 million -
General and administrative expenses were
$16.1 million $13.3 million -
As of September 30, 2024, Scholar Rock had cash, cash equivalents and marketable securities of approximately
$139.1 million $463.5 million $324.4 million
“Supported by our recent upsized public offering on the heels of successful SAPPHIRE data, Scholar Rock is advancing our mission from a position of strength as we move forward with preparations for our first commercial launch in the fourth quarter of 2025,” said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. “We are working tirelessly to obtain approval for apitegromab and deliver it to patients with SMA as soon as possible, as well as continuing to advance our portfolio of highly selective myostatin inhibition programs.”
Conference Call Information
Management will provide an update on the Company and discuss third quarter 2024 results via conference call on Tuesday, November 12 at 8:15 am ET. To access the live conference call, participants may register here. The live audio webcast of the call will be available under “Events and Presentations” in the Investor Relations section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. An archived replay of the webcast will be available on the Company’s website for approximately 90 days.
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.
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.
The efficacy and safety of apitegromab, SRK-181, and SRK-439 have not been established and apitegromab, SRK-181, and SRK-439 have not been approved for any use by the FDA or any other regulatory agency.
Scholar Rock® is a registered trademark of Scholar Rock, Inc.
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 we post on our website, Twitter or LinkedIn could be deemed material information. As a result, we encourage investors, the media and others interested to review the information we post there regularly. 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.
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 timing of any regulatory submissions, the therapeutic potential, clinical benefits and safety of any product candidates, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, its cash runway, expectations relating to commercial launch in the US in the fourth quarter of 2025, 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 3 SAPPHIRE trial will be sufficient to support regulatory approval, that the full results from the Phase 3 SAPPHIRE trial may differ from the topline data, that preclinical and clinical data, including the results from the Phase 2 or Phase 3 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; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials; 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, and as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, 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) | ||||||||||||||||
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
|
|
|
2024 |
|
|
|
2023 |
|
|
|
2024 |
|
|
|
2023 |
|
| Operating expenses | ||||||||||||||||
| Research and development | $ |
48,719 |
|
$ |
30,337 |
|
$ |
134,185 |
|
$ |
86,939 |
|
||||
| General and administrative |
|
16,061 |
|
|
13,335 |
|
|
48,512 |
|
|
36,324 |
|
||||
| Total operating expenses |
|
64,780 |
|
|
43,672 |
|
|
182,697 |
|
|
123,263 |
|
||||
| Loss from operations |
|
(64,780 |
) |
|
(43,672 |
) |
|
(182,697 |
) |
|
(123,263 |
) |
||||
| Other income (expense), net |
|
301 |
|
|
1,313 |
|
|
2,857 |
|
|
3,600 |
|
||||
| Net loss | $ |
(64,479 |
) |
$ |
(42,359 |
) |
$ |
(179,840 |
) |
$ |
(119,663 |
) |
||||
| Net loss per share, basic and diluted | $ |
(0,66 |
) |
$ |
(0,53 |
) |
$ |
(1,86 |
) |
$ |
(1,49 |
) |
||||
| Weighted average common shares outstanding, basic and diluted |
|
97,050,637 |
|
|
80,606,438 |
|
|
96,587,149 |
|
|
80,115,143 |
|
||||
| Scholar Rock Holding Corporation | ||||||
| Condensed Consolidated Balance Sheets | ||||||
| (unaudited) | ||||||
| (in thousands) | ||||||
| September 30, 2024 | December 31, 2023 | |||||
| Assets | ||||||
| Cash, cash equivalents and marketable securities | $ |
139,065 |
$ |
279,938 |
||
| Other current assets |
|
13,862 |
|
8,256 |
||
| Total current assets |
|
152,927 |
|
288,194 |
||
| Other assets |
|
26,206 |
|
22,841 |
||
| Total assets | $ |
179,133 |
$ |
311,035 |
||
| Liabilities and Stockholders' Equity | ||||||
| Current liabilities | $ |
39,372 |
$ |
32,741 |
||
| Long-term liabilities |
|
60,565 |
|
53,076 |
||
| Total liabilities |
|
99,937 |
|
85,817 |
||
| Total stockholders' equity |
|
79,196 |
|
225,218 |
||
| Total liabilities and stockholders' equity | $ |
179,133 |
$ |
311,035 |
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20241112854138/en/
Scholar Rock:
Investors
Rushmie Nofsinger
Scholar Rock
rnofsinger@scholarrock.com
ir@scholarrock.com
857-259-5573
Media
Molly MacLeod
Scholar Rock
mmacleod@scholarrock.com
media@scholarrock.com
802-579-5995
Source: Scholar Rock
FAQ
What were the key results of SRRK's Phase 3 SAPPHIRE trial?
When will Scholar Rock (SRRK) submit regulatory applications for apitegromab?
What was Scholar Rock's (SRRK) financial position in Q3 2024?
When will SRRK release results from the Phase 2 EMBRAZE obesity trial?
