– Optimistic 24-month knowledge from ongoing Section 2 TOPAZ trial extension helps potential advantage of apitegromab for sufferers with Varieties 2 and three Spinal Muscular Atrophy (SMA)

– Actively enrolling in pivotal Section three SAPPHIRE scientific trial for apitegromab

– Discovery pipeline developments made for LTBP-TGFβ1 goal to deal with a variety of fibrotic illnesses

– Considerably strengthened steadiness sheet with the completion of $205 million registered direct providing

CAMBRIDGE, Mass., August 08, 2022–(BUSINESS WIRE)–Scholar Rock (NASDAQ: SRRK), a Section three clinical-stage biopharmaceutical firm targeted on the therapy of great illnesses wherein protein development elements play a elementary function, at this time reported monetary outcomes and company updates for the second quarter ended June 30, 2022.

“We’re happy to have reported vital progress with our two scientific applications through the second quarter. Enrollment continues to advance in our pivotal Section three SAPPHIRE trial for apitegromab in SMA. We additionally offered new knowledge from our Section 2 TOPAZ trial highlighting sizeable and sustained enchancment in Hammersmith Purposeful Motor Scale-Expanded (HFMSE) scores at 24 months for non-ambulatory sufferers, reinforcing our perception within the distinctive potential of apitegromab as a muscle-targeted remedy to deal with SMA, and we proceed to guage these sufferers of their third 12 months of therapy,” mentioned Nagesh Mahanthappa, Founding Chief Government Officer & President of Scholar Rock. “In our SRK-181 program, enrollment is ongoing within the Section 1 DRAGON trial Half B dose growth. We look ahead to offering updates on DRAGON as knowledge develop into accessible. Additional, our targeted analysis continues to make wonderful progress as we develop next-generation applications to construct out our future pipeline.”

Firm Highlights and Upcoming Milestones

Apitegromab is a selective inhibitor of myostatin activation being developed as the primary muscle-targeted remedy with the potential to deal with spinal muscular atrophy (SMA).

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• Optimistic 24-Month Section 2 TOPAZ Extension Knowledge Help Potential Profit for Sufferers. Scholar Rock introduced constructive knowledge on the Remedy SMA Analysis & Medical Care Assembly in June, demonstrating sizable and sustained motor perform enchancment at 24 months with apitegromab as measured by Hammersmith Purposeful Motor Scale-Expanded (HFMSE) scores for non-ambulatory sufferers with Varieties 2 and three SMA on nusinersen. Outcomes additionally confirmed a considerable enhance in Revised Higher Limb Module (RULM) scores, with no critical security dangers recognized over 24 months of apitegromab therapy. Of the 55 sufferers who accomplished the 24-month extension interval, 54 opted to proceed into the 36-month extension interval.

• Enrollment Ongoing for Section three SAPPHIRE Medical Trial Evaluating Apitegromab in Non-Ambulatory Sufferers with Varieties 2 and three SMA. The randomized, double-blind, placebo-controlled Section three scientific trial is evaluating apitegromab for sufferers on both nusinersen or risdiplam. Roughly 156 non-ambulatory sufferers aged 2-12 years outdated with Varieties 2 and three SMA are deliberate to be enrolled in the primary efficacy inhabitants. Sufferers shall be randomized 1:1:1 to obtain for 12 months both apitegromab 20 mg/kg, apitegromab 10 mg/kg, or placebo by intravenous (IV) infusion each Four weeks. SAPPHIRE is predicted to be carried out throughout 55 websites within the U.S. and Europe. Scholar Rock offered on the trial design on the 17th Worldwide Congress on Neuromuscular Ailments (ICNMD 2022) in July 2022, together with publication of the summary within the peer-reviewed Journal of Neuromuscular Ailments.

SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the purpose of overcoming main resistance to and growing the variety of sufferers who could profit from checkpoint inhibitor remedy.

• Advancing Enrollment for Half B of the Section 1 DRAGON Proof-of-Idea Medical Trial for SRK-181. Half B of the Section 1 DRAGON trial consists of a number of proof-of-concept cohorts targeted on evaluating the flexibility of SRK-181 to beat main resistance to anti-PD-(L)1 remedy in sufferers with stable tumors. The biomarker technique for DRAGON explores early indicators of SRK-181 exercise, together with goal engagement and pathway modulation. It consists of measuring results on each circulating and tumor immune contexture, similar to CD8+ T cell infiltration and reductions in myeloid-derived suppressor cell (MDSC) populations, in addition to evaluation of TGFβ-related pathway signaling. Preliminary proof of drug exercise and security knowledge are anticipated in 2022.

Scholar Rock’s preclinical discovery pipeline features a extremely selective, context-dependent TGFβ1 antibody that inhibits proTGFβ1 activation selectively within the extracellular matrix by way of concentrating on the covalent complexes of proTGFβ1 with latent TGFβ binding proteins 1 and three (“LTBPs”— the targets are collectively known as “LTBP-TGFβ1”).

• Preclinical Knowledge Helps Selective Focusing on of Matrix-Related TGFβ1 as an Engaging Strategy for Anti-Fibrotic Remedy. Scholar Rock not too long ago offered knowledge on its focused method to LTBP-TGFβ1 that present discount of TGFβ1 signaling and discount of fibrosis in related preclinical in vivo fashions.

Second Quarter 2022 Monetary Outcomes

For the quarter ended June 30, 2022, internet loss was $44.Zero million or $1.06 per share in comparison with a internet lack of $30.7 million or $0.84 per share for the quarter ended June 30, 2021.

• Income was $Zero for the quarter ended June 30, 2022, in comparison with $4.6 million for the quarter ended June 30, 2021.

• Analysis and improvement expense was $32.1 million for the quarter ended June 30, 2022, in comparison with $25.6 million for the quarter ended June 30, 2021. The rise was primarily attributable to elevated scientific prices related to apitegromab and better personnel prices, together with severance bills related to the current restructuring.

• Basic and administrative expense was $11.1 million for the quarter ended June 30, 2022, in comparison with $9.three million for the quarter ended June 30, 2021. The rise was primarily as a result of increased personnel prices, together with severance bills related to the current restructuring.

• As of June 30, 2022, Scholar Rock had money, money equivalents, and marketable securities of roughly $371 million, which is predicted to fund the Firm’s anticipated working and capital expenditure necessities into 2025.

“We have been thrilled to announce the completion of a $205 million registered direct providing through the quarter. This financing included a number of high-quality, long-term oriented, fundamentals-based biotechnology buyers who noticed the promise of our platform and our applications. Importantly, this capital places us in a powerful monetary place to completely fund the Section three SAPPHIRE trial, proceed advancing Half B of the Section 1 DRAGON trial for SRK-181, whereas investing in chosen early-stage applications that exemplify the facility of our scientific platform,” mentioned Ted Myles, Chief Working Officer and Chief Monetary Officer of Scholar Rock.

About Scholar Rock

Scholar Rock is a clinical-stage biopharmaceutical firm targeted on the invention and improvement of modern medicines for the therapy of great illnesses wherein signaling by protein development elements performs a elementary function. Scholar Rock is making a pipeline of novel product candidates with the potential to rework the lives of sufferers affected by a variety of great illnesses, together with neuromuscular issues, most cancers, and fibrosis. Scholar Rock’s method to concentrating on the molecular mechanisms of development issue activation enabled it to develop a proprietary platform for the invention and improvement of monoclonal antibodies that regionally and selectively goal these signaling proteins on the mobile degree. By growing product candidates that act within the illness microenvironment, the Firm intends to keep away from the historic challenges related to inhibiting development elements for therapeutic impact. Scholar Rock believes its deal with biologically validated development elements could facilitate a extra environment friendly improvement path. For extra info, please go to www.ScholarRock.com or observe Scholar Rock on Twitter (@ScholarRock) and LinkedIn (https://www.linkedin.com/firm/scholar-rock/). Buyers and others ought to be aware that we talk with our buyers and the general public utilizing our firm web site www.scholarrock.com, together with, however not restricted to, firm disclosures, investor displays and FAQs, Securities and Alternate Fee filings, press releases, public convention name transcripts and webcast transcripts, in addition to on Twitter and LinkedIn. The data that we put up on our web site or on Twitter or LinkedIn could possibly be deemed to be materials info. Because of this, we encourage buyers, the media and others to assessment the knowledge that we put up there frequently. The contents of our web site or social media shall not be deemed integrated by reference in any submitting beneath the Securities Act of 1933, as amended.

Scholar Rock® is a registered trademark of Scholar Rock, Inc.

Ahead-Wanting Statements

This press launch incorporates “forward-looking statements” throughout the which means of the Personal Securities Litigation Reform Act of 1995, together with, however not restricted to, statements concerning Scholar Rock’s future expectations, plans and prospects, together with with out limitation, Scholar Rock’s expectations concerning its development, technique, progress and timing of its scientific trials for apitegromab, SRK-181, and different product candidates and indication choice and improvement timing, its money runway, the flexibility of any product candidate to carry out in people in a way according to earlier nonclinical, preclinical or scientific trial knowledge, and the potential of its product candidates and proprietary platform. Using phrases similar to “could,” “may,” “might,” “will,” “ought to,” “anticipate,” “plan,” “anticipate,” “imagine,” “estimate,” “venture,” “intend,” “future,” “potential,” or “proceed,” and different related expressions are meant to establish such forward-looking statements. All such forward-looking statements are based mostly on administration’s present expectations of future occasions and are topic to a lot of dangers and uncertainties that would trigger precise outcomes to vary materially and adversely from these set forth in or implied by such forward-looking statements. These dangers and uncertainties embrace, with out limitation, that preclinical and scientific knowledge, together with the outcomes from the Section 2 scientific trial of apitegromab, or Half A of the Section 1 scientific trial of SRK-181, should not predictive of, could also be inconsistent with, or extra favorable than, knowledge generated from future scientific trials of the identical product candidate, together with, with out limitation, the Section three scientific trial of apitegromab in SMA or Half B of the Section 1 scientific trial of SRK-181, Scholar Rock’s potential to offer the monetary assist, assets and experience essential to establish and develop product candidates on the anticipated timeline, the info generated from Scholar Rock’s nonclinical and preclinical research and scientific trials, info offered or choices made by regulatory authorities, competitors from third events which are growing merchandise for related makes use of, Scholar Rock’s potential to acquire, keep and defend its mental property, Scholar Rock’s dependence on third events for improvement and manufacture of product candidates together with, with out limitation, to produce any scientific trials, Scholar Rock’s potential to handle bills and to acquire further funding when wanted to assist its enterprise actions and set up and keep strategic enterprise alliances and new enterprise initiatives, and the impacts of public well being pandemics similar to COVID-19 on enterprise operations and expectations, in addition to these dangers extra absolutely mentioned within the part entitled “Threat Components” in Scholar Rock’s Quarterly Report on Kind 10-Q for the quarter ended June 30, 2022, in addition to discussions of potential dangers, uncertainties, and different vital elements in Scholar Rock’s subsequent filings with the Securities and Alternate Fee. Any forward-looking statements characterize Scholar Rock’s views solely as of at this time and shouldn’t be relied upon as representing its views as of any subsequent date. All info on this press launch is as of the date of the discharge, and Scholar Rock undertakes no responsibility to replace this info until required by regulation.

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Contacts

Scholar Rock:

Buyers
Rushmie Nofsinger
Scholar Rock
rnofsinger@scholarrock.com
ir@scholarrock.com
857-259-5573

Media
Ariane Lovell
Finn Companions
ariane.lovell@finnpartners.com
media@scholarrock.com
917-565-2204