On August 9, 2023 Merus N.V. (Nasdaq: MRUS) ("Merus", the "Company," "we" and "our"), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics and Triclonics), reported the pricing of an underwritten public offering of 6,818,182 common shares, at a public offering price of $22.00 per share (the "Offer Shares") (Press release, Merus, AUG 9, 2023, View Source [SID1234634103]). Merus also granted the underwriters a 30-day option to purchase up to an additional 1,022,727 common shares (the "Option Shares" and together with the Offer Shares, the "Shares"). The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses and excluding the underwriters’ option to purchase the Option Shares, are expected to be approximately $150.0 million. All of the shares in the offering are to be sold by Merus.

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The offering is expected to close on or about August 14, 2023, subject to customary closing conditions.

Merus currently intends to use the net proceeds from the offering, together with its existing cash, cash equivalents and marketable securities, to advance the clinical development of its product candidates, for preclinical research and technology development, and for working capital and general corporate purposes.

Jefferies, BofA Securities, Guggenheim Securities and William Blair are acting as joint book-running managers for the offering. Van Lanschot Kempen is acting as lead manager for the offering.

The offering is being made pursuant to a shelf registration statement on Form S-3 that was filed with the Securities and Exchange Commission (SEC) on May 7, 2021 and was effective upon filing. The offering will be made only by means of a written prospectus and prospectus supplement that form a part of the registration statement, which, for the avoidance of doubt, will not constitute a "prospectus" for the purposes of (i) Regulation (EU) 2017/1129 (the "Prospectus Regulation") and has not been reviewed by any competent authority in any member state in the European Economic Area (the "EEA") and (ii) the Prospectus Regulation as it forms part of domestic law in the United Kingdom by virtue of the European Union (Withdrawal) Act 2018 (the "UK Prospectus Regulation") and has not been reviewed by the Financial Conduct Authority in the United Kingdom. A preliminary prospectus supplement to the prospectus describing the terms of the offering was filed with the SEC on August 9, 2023, and a final prospectus supplement will be filed with the SEC. Copies of the final prospectus supplement and the accompanying prospectus relating to the offering may be obtained, when available, from Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at (877) 821-7388, or by email at [email protected]; BofA Securities NC1-0220-02-25, Attention: Prospectus Department, 201 North Tryon Street, Charlotte, NC 28255‐0001, or by email at [email protected]; Guggenheim Securities, LLC, Attention: Equity Syndicate Department, 330 Madison Avenue, 8th Floor, New York, NY 10017, by telephone at (212) 518-9544, or by email at [email protected]; or William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, IL 60606, by telephone at (800) 621-0687, or by email at [email protected].

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

This press release is an advertisement and not a prospectus within the meaning of either the Prospectus Regulation or the UK Prospectus Regulation.

EEA:

In relation to each member state of the EEA (each, a "Relevant State"), no Shares have been offered or will be offered pursuant to the offering to the public in that Relevant State prior to the publication of a prospectus in relation to the Shares which has been approved by the competent authority in that Relevant State or, where appropriate, approved in another Relevant State and notified to the competent authority in that Relevant State, all in accordance with the Prospectus Regulation, except that Shares may be offered to the public in that Relevant State at any time:

to any legal entity which is a "qualified investor" as defined under Article 2 of the Prospectus Regulation;
to fewer than 150 natural or legal persons (other than qualified investors as defined under Article 2 of the Prospectus Regulation), subject to obtaining the prior consent of the representatives for any such offer; and
in any other circumstances falling within Article 1(4) of the Prospectus Regulation,
provided that no such offer of the Shares shall require us or any of the underwriters to publish a prospectus pursuant to Article 3 of the Prospectus Regulation or supplement a prospectus pursuant to Article 23 of the Prospectus Regulation. Each person who initially acquires any Shares or to whom any offer is made will be deemed to have represented, warranted, acknowledged and agreed to and with us and each of the underwriters that it is a "qualified investor" within the meaning of Article 2 of the Prospectus Regulation.

For the purposes of the above, the expression "offer to the public" in relation to the Shares in any Relevant State means the communication in any form and by any means of sufficient information on the terms of the offer and any Shares to be offered so as to enable an investor to decide to purchase or subscribe for any Shares, and the expression "Prospectus Regulation" means Regulation (EU) 2017/1129.

United Kingdom:

No Shares have been offered or will be offered pursuant to this offering to the public in the United Kingdom prior to the publication of a prospectus in relation to the Shares which has been approved by the Financial Conduct Authority in the United Kingdom, except that the Shares may be offered to the public in the United Kingdom at any time:

a) to any legal entity which is a qualified investor as defined under Article 2 of the UK Prospectus Regulation;

b) to fewer than 150 natural or legal persons (other than qualified investors as defined under Article 2 of the UK Prospectus Regulation), subject to obtaining the prior consent of the representatives for any such offer; or

c) in any other circumstances falling within Section 86 of the Financial Services and Markets Act 2000 (the "FSMA")

provided that no such offer of the Shares shall require us or any of the underwriters to publish a prospectus pursuant to Section 85 of the FSMA or Article 3 of the UK Prospectus Regulation or supplement a prospectus pursuant to Article 23 of the UK Prospectus Regulation. Each person in the United Kingdom who initially acquires any Shares or to whom any offer is made will be deemed to have represented, warranted, acknowledged and agreed to and with us and each of the underwriters that it is a "qualified investor" within the meaning of the UK Prospectus Regulation.

For the purposes of this provision, the expression an "offer to the public" in relation to the Shares in the United Kingdom means the communication in any form and by any means of sufficient information on the terms of the offer and any Shares to be offered so as to enable an investor to decide to purchase or subscribe for any Shares.

In addition, in the United Kingdom, the transaction to which this press release relates will only be available to, and will be engaged in only with persons who are "qualified investors" (as defined in the UK Prospectus Regulation) (i) who have professional experience in matters relating to investments falling within Article 19(5) of the FSMA (Financial Promotion) Order 2005, as amended (the Order), and/or (ii) who are high net worth entities (or persons to whom it may otherwise be lawfully communicated) falling within Article 49(2)(a) to (d) of the Order (all such persons together being referred to as "relevant persons"). In the United Kingdom, the securities referred to herein are only available to, and any invitation, offer or agreement to subscribe, purchase or otherwise acquire such securities will be engaged in only with relevant persons. Any person in the United Kingdom who is not a relevant person should not act or rely on this communication or any of its contents.

On August 9, 2023 aTyr Pharma, Inc. (Nasdaq: LIFE) ("aTyr" or the "Company"), a biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, reported second quarter 2023 results and provided a corporate update (Press release, aTyr Pharma, AUG 9, 2023, View Source [SID1234634102]).

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"Throughout the second quarter we have continued to progress and invest in our clinical development program for our lead therapeutic candidate, efzofitimod, in interstitial lung disease (ILD)," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "Our global pivotal Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, the most prevalent form of ILD, continues to enroll and our Phase 2 EFZO-CONNECT study in patients with systemic sclerosis (SSc, or scleroderma)-associated ILD (SSc-ILD), is expected to enroll the first patient in the third quarter."

Second Quarter 2023 and Subsequent Period Highlights

Continued enrollment in the global pivotal Phase 3 EFZO-FIT study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a randomized, double-blind, placebo-controlled, 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously monthly for a total of 12 doses. The study intends to enroll up to 264 subjects with pulmonary sarcoidosis. The study is open for enrollment at nearly all of the centers intended in the U.S., Europe and Japan and is expected to expand to include centers in Brazil.
Progressed plans to initiate the Phase 2 EFZO-CONNECT study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with SSc-ILD. This proof-of-concept study will be a randomized, double-blind, placebo-controlled, 28-week study consisting of three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo dosed intravenously monthly for a total of 6 doses. The study is expected to enroll 25 patients at multiple centers in the U.S. The primary objective of the study will be to evaluate the efficacy of multiple doses of intravenous efzofitimod on pulmonary, cutaneous and systemic manifestations in patients with SSc-ILD. The study is expected to initiate in the third quarter of 2023.
Received European Commission orphan drug designation for efzofitimod for the treatment of SSc based on the opinion of the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. The EMA grants orphan status to products intended for the treatment, prevention or diagnosis of a disease with a prevalence no more than five in 10,000 people in the EU that is life-threatening or chronically debilitating for which either no satisfactory method of diagnosis, prevention, or treatment exists, or if such a method exists, the medicine is of significant benefit to those affected by such condition. EMA orphan drug designation provides certain benefits, including the potential for up to 10 years of marketing exclusivity following regulatory approval in the EU, reduction in regulatory fees and a centralized EU approval process.
Announced two posters for efzofitimod accepted for presentation at the upcoming European Respiratory Society (ERS) International Congress 2023. The conference is scheduled to take place September 9 – 13, 2023, in Milan, Italy. The Company will present new data from a pooled, post hoc analysis from the Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis that further supports efficacy measures in these patients. Additionally, new mechanistic data supports the rationale for efzofitimod as a potential treatment for patients with SSc-ILD.
Poster PA419 – Efzofitimod: A Novel Therapeutic Candidate for SSc-ILD on Sunday, September 10, 2023, from 8:00 a.m. to 9:30 a.m. CEST.
Poster PA1744 – Therapeutic Doses of Efzofitimod Significantly Improve Multiple Pulmonary Sarcoidosis Efficacy Measures on Sunday, September 10, 2023, from 4:00 p.m. to 5:30 p.m. CEST.
Second Quarter 2023 Financial Highlights and Cash Position

Cash & Investment Position: Cash, restricted cash, cash equivalents and investments as of June 30, 2023, were $112.0 million. Based on the Company’s current operational plans and existing cash, the Company maintains its prior guidance and believes its cash runway will extend into 2026.
R&D Expenses: Research and development expenses were $9.8 million for the second quarter of 2023, which consisted primarily of clinical trial costs for the Phase 3 EFZO-FIT study, manufacturing costs for the efzofitimod program and research and development costs for the efzofitimod and discovery programs.
G&A Expenses: General and administrative expenses were $3.7 million for the second quarter of 2023.
About Efzofitimod

aTyr is developing efzofitimod as a potential therapeutic for patients with fibrotic lung disease. Efzofitimod, a fusion protein comprised of the immunomodulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody, is a selective modulator of neuropilin-2 that downregulates innate immune responses in inflammatory disease states. aTyr’s lead indication for efzofitimod is pulmonary sarcoidosis, a major form of interstitial lung disease. Clinical proof-of-concept for efzofitimod was recently established in a Phase 1b/2a multiple-ascending dose, placebo-controlled study of efzofitimod in patients with pulmonary sarcoidosis, which demonstrated safety and a consistent dose response and trends of benefit of efzofitimod compared to placebo on key efficacy endpoints, including steroid reduction, lung function, clinical symptoms and inflammatory biomarkers. aTyr is currently conducting EFZO-FIT, a Phase 3 study of efzofitimod in pulmonary sarcoidosis patients.

On August 9, 2023 Mirati Therapeutics, Inc. (NASDAQ: MRTX), a commercial stage biotechnology company, reported Cancer Discovery published preclinical and initial clinical data from a first-in-human Phase 1/2 clinical trial of MRTX1719, a PRMT5 / methylthioadenosine (MTA)-cooperative inhibitor evaluated in methylthioadenosine phosphorylase (MTAP) deleted cancers (Press release, Mirati, AUG 9, 2023, View Source [SID1234634100]). These results provide preclinical and early clinical proof-of-concept for this differentiated approach and demonstrate that MRTX1719 may represent a promising targeted therapy for the ~10% of cancer patients with this biomarker. The publication can be found here.

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In preclinical studies, MRTX1719 was demonstrated to be a potent, selective inhibitor of the PRMT5/MTA complex. This compound exploits the elevated MTA levels present in MTAP-deleted cancers and turns this altered metabolic state into a therapeutic vulnerability to selectively kill MTAP deleted cancer cells while sparing normal cells. Preclinical results showed marked anti-tumor activity of MRTX1719, including regression, across lung, pancreatic, mesothelioma, and other solid tumor models.

As of June 13, 2023, there were 18 patients with solid tumors harboring MTAP deletions in the Phase 1/2 trial of MRTX1719 that were evaluable for clinical response at dose levels of 100mg QD or greater. Initial dose level started at 50mg administered once-daily orally over 21-day cycles followed by 100% dose escalations for subsequent dose levels. Six individual case narratives were selected for inclusion in the Cancer Discovery publication:

There were six confirmed objective responses observed in the Phase 1 study including one patient who achieved a response which subsequently confirmed post-data cutoff
MRTX1719 achieved apparent complete PRMT5 inhibition in MTAP deleted tumor cells at a dose of 200 mg QD as evidenced by pretreatment and on-treatment tumor biopsies of the PRMT5 biochemical biomarker symmetric dimethyl arginine (SDMA)
It was notable that four patients were on therapy for multiple cycles before experiencing an initial objective response, with their tumors continuing to decrease in size over the course of treatment.
These findings suggest that tumor response may continue to deepen over time indicating the importance of evaluating tumor response kinetics and response rates across a broader patient population with longer follow up.
MRTX1719 was well-tolerated with no dose limiting toxicities observed at dose levels up to 400mg QD. None of the patients treated with MRTX1719 experienced dose-limiting adverse events associated with first generation PRMT5 inhibitors such as thrombocytopenia, anemia or neutropenia.
"Many cancers with a prevalence of MTAP deletion have few treatment options so it is encouraging to see these initial responses in the clinic. These data, including the favorable safety profile, demonstrate the development of a possible therapeutic approach for a significant population of patients with MTAP-deleted cancers in need of new treatment options," said Jordi Rodon Ahnert, MD, PhD, The University of Texas MD Anderson Cancer Center. "It will be important for next-generation sequencing tests to expand their panel of targets to include for markers of PRMT5 inhibition."

"The targeting strategy of MRTX1719 is differentiated from first generation PRMT5 inhibitors and has so far shown to be well tolerated in this patient population with dire unmet need," said James Christensen, Ph.D., chief scientific officer, Mirati Therapeutics, Inc. "We are pleased that Cancer Discovery recognized the importance and potential impact of this early data from MRTX1719, and we look forward to pursuing continued development of MRTX1719 for the benefit of patients living with cancer."

About MRTX1719 (MTA-cooperative PRMT5 inhibitor)

MRTX1719, discovered at Mirati, is an orally bioavailable, methylthioadenosine (MTA)-cooperative PRMT5 inhibitor that exhibits synthetic lethality in MTAP-deleted cancers. MTAP deletion leads to MTA accumulation in cancer cells and MRTX1719 is designed to selectively bind the PRMT5-MTA complex ultimately inhibiting PRMT5 function in MTAP-deleted cancer cells while sparing healthy non-tumor cells.1MTAP deletions occur in around 10% of all cancers2, including a high percentage of pancreatic cancer, non-small cell lung cancer, and mesothelioma which are associated with a poor prognosis, representing a significant unmet medical need. The PRMT5-MTA complex creates a novel drug target for the treatment of MTAP-deleted cancers. Targeting this complex involves a new and potentially improved therapeutic approach. MRTX1719 is being evaluated as monotherapy and in combination with other therapeutic options to treat patients with advanced, unresectable or metastatic solid tumor malignancy with homozygous deletion of the MTAP gene.

For more information visit Mirati.com/science.

On August 9, 2023 Zentalis Pharmaceuticals, Inc. (Nasdaq: ZNTL), a clinical-stage biopharmaceutical company discovering and developing clinically differentiated small molecule therapeutics targeting fundamental biological pathways of cancers, reported financial results for the quarter ended June 30, 2023, and highlighted recent corporate accomplishments (Press release, Zentalis Pharmaceuticals, AUG 9, 2023, View Source [SID1234634092]).

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"We have strong azenosertib data that creates the potential to build a meaningful franchise around this exciting product candidate with the possibility of near-, mid- and long-term opportunities across multiple tumor types," said Kimberly Blackwell, M.D., Chief Executive Officer of Zentalis. "As a monotherapy, azenosertib achieved an objective response rate of 36.8% in ovarian cancer and uterine serous carcinoma patients who received intermittent dosing. Azenosertib plus chemotherapy combinations also demonstrated positive results in women facing heavily pretreated ovarian cancer. Taking these results together, we believe there is the potential for azenosertib to transform the standard of care for patients with ovarian and uterine cancers, and ultimately other solid tumors. We are well positioned to advance this program with a strong cash position resulting from our recent offering that extended our cash runway and will sustain our corporate and clinical strategy for the next several years."

WEE1 Inhibitor (Azenosertib) Program Highlights

•Identified monotherapy recommended Phase 2 dose (RP2D) and reported positive safety and efficacy data. Based on encouraging Phase 1 dose optimization clinical data, the Company identified 400 mg daily (QD) on a 5 days on 2 days off (5:2) administration schedule as the monotherapy RP2D. Intermittent dosing more than doubled steady state drug exposure to achieve an objective response rate (ORR) of 36.8% in ovarian cancer and uterine serous carcinoma (USC) patients, while maintaining safety and improving tolerability in comparison to continuous dosing. For a more detailed summary of the Phase 1 monotherapy dose optimization data, click here. To listen to a replay of the call, click here.

•Presented positive azenosertib + chemotherapy combination data at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. The Company presented positive data from its Phase 1b chemotherapy combination trial in platinum-resistant ovarian cancer at the ASCO (Free ASCO Whitepaper) Annual Meeting in Chicago on June 5, 2023. Azenosertib was well tolerated in combination with multiple types of chemotherapy and demonstrated strong anti-tumor activity, with noteworthy improvements in ORRs and median progression free survival (mPFS) in all patients, especially those with Cyclin E1+ tumors, a subgroup recognized to have a poor prognosis and relatively poor outcomes following chemotherapy. Azenosertib combined with paclitaxel resulted in an ORR of 50.0% and mPFS of 7.4 months. An ORR of 35.7% and mPFS of 10.4 months was observed in azenosertib combined with carboplatin. Zentalis is preparing for a Phase 3 trial of azenosertib using intermittent dosing in combination with chemotherapy in Cyclin E1+ platinum-sensitive ovarian cancer. To review the Phase 1b results in more detail, click here.
•Biomarker enrichment strategies. Zentalis is exploring biomarker enrichment strategies for azenosertib targeting tumors of high genomic instability, such as Cyclin E1+ tumors and homologous recombination deficient tumors. In April 2023, the Company presented preclinical data supporting the rationale for the Cyclin E1 enrichment strategy at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2023.

Corporate Highlights

•In August 2023, the Company announced the appointment of Kimberly Freeman as Chief Strategy Officer. In this role, she will support and implement Zentalis’ long-term portfolio strategy. Ms. Freeman joins Zentalis with over 25 years of clinical development and strategic leadership experience, including deep expertise in oncology, particularly in gynecologic malignancies and the DNA damage response (DDR) pathway. Ms. Freeman brings an outstanding track record of building and executing drug development and franchise strategies that will be invaluable to the Company as it seeks to capitalize on the significant opportunity with azenosertib across a broad array of tumor types.
•In June 2023, the Company closed an underwritten public offering of 11,032,656 shares of its common stock at a public offering price of $22.66 per share. The total gross proceeds were approximately $250.0 million.
•In May 2023, the Company appointed Diana Hausman, M.D., to its Board of Directors. Dr. Hausman is an oncologist with extensive experience in all aspects of drug development, including development and implementation of clinical strategy. Dr. Hausman is currently serving as Chief Medical Officer of Link Immunotherapeutics. Dr. Hausman previously served as Chief Medical Officer of Lengo Therapeutics, Zymeworks and Oncothyreon. Prior to Oncothyreon, Dr.

Hausman held positions of increasing responsibility at ZymoGenetics, Berlex Laboratories and Immunex. Dr. Hausman currently serves on the Board of Directors of Immuneering.
•In May 2023, the Company announced that it had appointed Funda Meric-Bernstam, M.D., a widely recognized Phase 1 trial expert and experimental therapeutics researcher in oncology, to its Scientific Advisory Board. Dr. Meric-Bernstam is the Chair of the Department of Investigational Cancer Therapeutics — the Phase 1 Program at The University of Texas MD Anderson Cancer Center. Her clinical research is focused on novel therapeutics, novel combination therapies and biomarkers to predict and monitor drug response.
Second Quarter 2023 Financial Results

•Cash and Marketable Securities Position: As of June 30, 2023, Zentalis had cash, cash equivalents and marketable securities of $553.0 million. The Company believes that its existing cash, cash equivalents and marketable securities as of June 30, 2023 will be sufficient to fund its operating expenses and capital expenditure requirements into 2026.
•Research and Development Expenses: Research and development (R&D) expenses for the three months ended June 30, 2023 were $42.7 million, compared to $43.8 million for the three months ended June 30, 2022. The decrease of $1.1 million was primarily due to $1.7 million in decreased collaboration costs, a $1.1 million decrease related to clinical trials and R&D supplies, and a $0.6 million reduction in personnel and related expense. These decreases were partially offset by a $1.5 million increase in Zentera Therapeutics cost sharing and a $0.8 million increase in consulting and other expense.
•General and Administrative Expenses: General and administrative expenses for the three months ended June 30, 2023 were $15.7 million, compared to $19.6 million during the three months ended June 30, 2022. This decrease of $3.9 million was primarily attributable to a $4.2 million decrease in non-cash, stock-based compensation expense and a $0.8 million decrease related to other personnel expenses. These decreases were partially offset by an increase of $0.8 million in allocated overhead expenditures and a $0.3 million increase in outside services and other costs.
•Zentera-Related Expenses: On June 15, 2023, the Company announced that it had regained worldwide development and commercialization rights to azenosertib, ZN-d5 and ZN-c5 as a result of the termination of its collaboration with Zentera Therapeutics in certain Asian countries, including China. In connection with the Zentera termination, the Company incurred one-time expenses totaling $45.6 million.

About Azenosertib

Azenosertib is a potentially first-in-class and best-in-class small molecule WEE1 inhibitor in development for the treatment of cancer. Inhibition of WEE1, a DNA damage response kinase, drives cancer cells into mitosis without being able to repair damaged DNA, resulting in cell death. Currently, there are no FDA-approved WEE1 inhibitors, and azenosertib has been designed for superior selectivity and pharmacokinetic properties. Azenosertib is being developed in therapeutic areas of high unmet need and is being evaluated as a monotherapy, in combination with chemotherapy, and in combination with molecularly targeted agents.

On August 9, 2023 Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results and corporate updates for the second quarter ended June 30, 2023 (Press release, Scholar Rock, AUG 9, 2023, View Source [SID1234634091]).

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"We continue to advance our apitegromab program, notably with the recent presentation of 36-month data from the Phase 2 TOPAZ trial which supports apitegromab’s therapeutic potential to meaningfully improve motor function for patients with SMA," said Jay Backstrom, M.D., M.P.H., President and Chief Executive Officer of Scholar Rock. "The continued success of TOPAZ increases our conviction in SAPPHIRE and we look forward to completing enrollment this quarter and sharing a topline data readout in 2024."

Recent 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.

Presented 36-month extension data from Phase 2 TOPAZ trial at Cure SMA Research & Clinical Care Meeting in June. The company shared new data evaluating outcomes after 36 months of treatment with apitegromab, which showed substantial and sustained improvement in motor function, as well as improvements in patient-reported outcome measures in patients with nonambulatory Types 2 and 3 SMA receiving survival motor neuron (SMN) therapy. The company also hosted a virtual investor day event in July featuring key opinion leaders to discuss the current SMA treatment landscape and apitegromab’s potential to advance the standard of care.
Continued progress towards completion of enrollment for Phase 3 SAPPHIRE clinical trial. The randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating the safety and efficacy of apitegromab in patients with nonambulatory Types 2 and 3 SMA receiving either nusinersen or risdiplam is actively enrolling SMA patients across sites in the U.S. and Europe. Enrollment completion is expected in the third quarter of 2023, with top-line data expected in 2024. If successful and if approved, the company expects to initiate a commercial product launch in 2025.
Initiated the ONYX trial, the long-term extension study for patients from both the TOPAZ and SAPPHIRE studies. As of July 2023, more than 90 percent of patients from TOPAZ receiving apitegromab and a SMN therapy remained in the extension study.
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 in patients with advanced cancer.

Advancing Phase 1 DRAGON proof-of-concept trial. DRAGON is evaluating SRK-181 in patients with locally advanced or metastatic solid tumors that exhibit resistance to anti-PD-(L)1 antibodies. The Company will provide biomarker and clinical updates from Part B of the DRAGON trial in the second half of 2023.
Second Quarter 2023 Financial Results

For the quarter ended June 30, 2023, net loss was $37.9 million compared to a net loss of $44.0 million for the quarter ended June 30, 2022.

The Company did not record any revenue for either the quarter ended June 30, 2023 or June 30, 2022.
Research and development expense was $26.9 million for the quarter ended June 30, 2023, compared to $32.1 million for the quarter ended June 30, 2022. The decrease was primarily attributable to a decrease in external research and development costs, including costs for the apitegromab clinical trials and associated clinical trial material manufacturing costs. In addition, there was a decrease in employee compensation and benefits costs, resulting from the restructuring in May 2022.
General and administrative expense was $12.2 million for the quarter ended June 30, 2023, compared to $11.1 million for the quarter ended June 30, 2022.
As of June 30, 2023, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $249 million, which is expected to fund the company’s anticipated operating and capital expenditure requirements into 2025.
"We are relentlessly focused on execution, and we are encouraged by the continued strong data from TOPAZ and momentum we’re seeing in the enrollment of SAPPHIRE. Our overall execution, along with our spend for the first six months of 2023, is aligned with our strategic plan," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. "We ended the quarter with $249 million in cash, which we expect to provide runway through a number of anticipated important milestones."