On May 13, 2021 Genetron Holdings Limited ("Genetron Health" or the "Company", NASDAQ:GTH), a leading precision oncology platform company in China that specializes in offering molecular profiling tests, early cancer screening products and companion diagnostics development, reported a strategic partnership with Siemens Healthineers at the China Medical Equipment Fair (Press release, Genetron Health Technologies, MAY 13, 2021, View Source [SID1234579960]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

This partnership aims to promote large scale application of Genetron’s S5 platform and lung cancer 8-gene IVD assay in Chinese hospitals, in order to provide non-small cell lung cancer (NSCLC) patients with efficient and accurate personalized diagnosis and treatment guidance. The companies also plan to work together to standardize cancer molecular testing in hospitals.

Representing Genetron Health at the signing ceremony in Shanghai were Co-founder and CEO Sizhen Wang, and Vice President Zhaohui Yin. Elisabeth Staudinger, President of Siemens Healthineers Asia Pacific, and Guo Yiming, Vice President of Siemens Healthineers Laboratory Diagnostic System, Greater China Region, also attended the event.

"Genetron Health focuses on the innovation, clinical transformation and commercialization of cancer genomics technologies. Both our lab developed test (LDT) and in vitro diagnostic (IVD) products’ business models have been progressing well," said Sizhen Wang, Co-founder and CEO of Genetron Health. "We are pleased to collaborate with Siemens Healthineers for our S5 instrument and lung 8 IVD assay, which helps diagnose and treat cancer patients. We also expect this partnership to help standardize cancer molecular testing in China, and reach more patients in need," Wang said.

"As a global leader in healthcare technology, Siemens Healthineers stays committed to addressing the needs of patients. To help Chinese cancer patients access quality care, we are not only introducing cutting-edge testing analyzers and reagents to China, but are also working with local innovators to offer personalized diagnostics. We are excited to forge a strategic partnership with Genetron Health, an industry leading precision oncology platform company. Meanwhile, we expect to push forward precision care in the field of lung cancer, therefore contributing to the vision of a Healthy China," said Guo Yiming, Vice President of Laboratory Diagnostics, Siemens Healthineers Greater China.

At present, lung cancer is one of the most serious diseases in China, placing the highest amongst morbidity and mortality rankings of all cancer types. In China, 787,000 cases of lung cancer are diagnosed every year, with about 631,000 deaths [1] caused annually as well. NSCLC is the most common type of lung cancer. In recent years, with the development of more advanced diagnostics and the emergence of targeted drugs, more NSCLC patients have been able to receive quicker and more accurate treatment. As the "first step" of targeted therapy for lung cancer, cancer molecular gene detection is the key to guided clinical treatment and drug applications. The U.S. National Comprehensive Cancer Network (NCCN) Guidelines for NSCLC also recommend that molecular testing of lung cancer-related genes be used to guide targeted therapies for NSCLC patients. Accurate lung cancer gene detection for NSCLC patients can help clinicians opt for more precise treatment.

Based on Genetron Health’s One-Step Seq technology (Chinese invention patent ZL 201710218529.4), the lung cancer 8-gene IVD assay can detect many mutations and fusions across 8 different genes at once, for targeted therapy selection in NSCLC. The assay can offer patients rapid and accurate molecular typing, medication guidance and drug resistance monitoring. Combined with Genetron S5, it offers advantages in detection efficiency, ease of use, economic cost, sample size, and can achieve a two-day turnaround time. It is suitable for independent clinical molecular testing in Chinese hospitals at all levels.

[1] Zheng R., Sun K., Zhang S., et al. Report of cancer epidemiology in China, 2015, Chinese Journal of Oncology, 2019, 41(1):19-28.

On May 13, 2021 Compass Therapeutics, Inc. ("Compass"; OTCQB: CMPX), a clinical-stage biotechnology company developing proprietary antibody therapeutics to treat cancer, and TRIGR Therapeutics, Inc. ("TRIGR"), a private biotechnology oncology company, reported that the companies have entered into a definitive merger agreement, under which Compass, through a subsidiary, will acquire TRIGR, a private cancer drug development company founded by George Uy, an industry veteran and a former commercial executive at Roche, in a stock-for-stock transaction (Press release, Compass Therapeutics, MAY 13, 2021, View Source [SID1234579959]). Under the terms of the agreement, Compass’ subsidiary will acquire all of the outstanding shares of TRIGR. In addition, Miranda Toledano, TRIGR’s Chief Financial Officer and Chief Operating Officer, will be joining the Compass Board of Directors. The transaction was unanimously approved by the Boards of Directors of both companies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

At the core of this transaction is a differentiated bispecific antibody targeting both DLL4 and VEGF-A, which inhibits both DLL4-mediated Notch signaling and VEGF signaling, which has been renamed CTX-009 (formerly designated TR009/ABL001/NOV1501). CTX-009 has completed a Phase 1 dose escalation study and is in a Phase 1b study in patients with solid tumors in S. Korea, where it has been observed to be well tolerated. In addition, there have been multiple confirmed partial responses (PRs) by RECIST criteria in several tumors in those studies, including PRs in colorectal cancer, cholangiocarcinoma, gastric cancer, and pancreatic cancer. The Phase 1b study in combination with chemotherapy is expected to be completed in the second half of this year. Compass plans to file an IND in the US in H2 2021 and initiate clinical studies in patients with cholangiocarcinoma, ovarian cancer and advanced colorectal cancer. Exclusive global rights to the program, except for S. Korea and China, are held by TRIGR through a license with ABL Bio Inc. (KOSDAQ: 298380). South Korean rights are held by Handok Inc. (KOSDAQ: 002390) and China rights were out-licensed to Elpiscience Biopharmaceuticals Co., Limited, under a license agreement executed on Jan 20th, 2021.

"The time now has come to maximize the therapeutic potential of this promising bispecific antibody, and I am confident that Compass has the management and development capabilities supporting the goal to advance CTX-009 to Phase 2 and Phase 3 studies in multiple indications" said George Uy, TRIGR Founder and Chief Executive Officer. "I am honored to join the Compass board and look forward to CTX-009’s success as part of Compass’ stellar immunotherapy pipeline" said Miranda Toledano, TRIGR’s Chief Operating Officer and Chief Financial Officer.

"CTX-009 presents an important addition to our pipeline of novel therapeutic antibodies and bispecific antibodies. It is uncommon to see multiple confirmed partial responses in a Phase 1 study in such an advanced patient population, and we are excited to begin to develop a robust Phase 2 program to evaluate the therapeutic potential of CTX-009 across all of the indications where this bispecific has shown substantial promise to-date" said Thomas Schuetz, MD, Ph.D., Co-Founder and Chief Executive Officer of Compass Therapeutics. "When we reviewed the data for CTX-009, we immediately saw the fit with our corporate strategy of advancing the best therapeutic candidates forward, regardless of origin" said Vered Bisker-Leib, Compass President and Chief Operating Officer. "We then moved quickly to conduct due diligence and reach agreement on a merger structure designed to deliver enhanced value for the shareholders of both companies".

Under the terms of the agreement, TRIGR will be acquired by Compass’ wholly owned subsidiary, Compass Acquisition Company, LLC, subject to certain customary conditions including the consent of the holders of a majority of TRIGR’s common shares. Consideration payable to TRIGR shareholders at closing totals an aggregate of 10,265,154 shares of Compass’ common stock, issued as unregistered shares in a private placement. In addition, TRIGR shareholders are eligible to receive up to $9 million, representing earnout payments that depend on certain events, including $5 million upon BLA approval of CTX-009 in the United States. Following the issuance of the share consideration, Compass has agreed to register such shares for resale by the recipients thereof. The transaction is expected to close in the second quarter of 2021.

About CTX-009

CTX-009 (formerly designated TR009/ABL001/NOV1501) is an anti-DLL4 X VEGF-A bispecific antibody. In November 2018, TRIGR licensed the exclusive, global rights to CTX-009, outside of South Korea, from ABL Bio, Inc. (KOSDAQ: 298380), a South Korea-based clinical-stage company focused on developing antibody therapeutics. A Phase 1 dose escalation study and a Phase 1b dose expansion monotherapy study have been completed and a Phase 1b combination study is ongoing in S. Korea. Data from CTX-009’s Phase 1 dose escalation and dose expansion monotherapy study demonstrates an approximately 20% Overall Response Rate (ORR) at the targeted therapeutic doses, with confirmed partial responses per RECIST criteria in heavily pre-treated colorectal and gastric cancer patients in whom multiple therapies have failed, including VEGF-targeted therapeutics, anti-PD-1/PD-L1 regimens and chemotherapies. Interim results from the ongoing Phase 1b combination study testing the tolerability and activity of CTX-009 in combination with irinotecan or paclitaxel, have also shown deep and sustained partial responses in difficult to treat intrahepatic cholangiocarcinoma (biliary tract cancer) patients in whom multiple lines of therapy have failed. In contrast to historical anti-DLL4 antibodies and other Notch targeted therapies, the administration of CTX-009 has not been associated with severe pulmonary hypertension. Full data from the ongoing Phase 1b studies is expected to be provided later in 2021.

On May 13, 2021 CytRx Corporation (OTCQB:CYTR) ("CytRx" or the "Company"), a specialized biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, reported on its results for the first quarter ended March 31, 2021 (Press release, CytRx, MAY 13, 2021, View Source [SID1234579957]). In addition, CytRx highlighted developments pertaining to its agreements with Orphazyme A/S (NASDAQ:ORPH) ("Orphazyme") and ImmunityBio, Inc. (NASDAQ:IBRX) ("ImmunityBio") as well as Centurion Biopharma. The Company’s 10-Q was filed on May 13, 2021.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, stated:

"CytRx continues to execute on its strategic plan to cut costs, maintain a stable capital position, and nimbly manage a portfolio of high-potential licensing agreements and strategic assets. We are very excited about the quarters ahead now that Orphazyme is preparing for prospective regulatory approvals for arimoclomol in the treatment of Niemann-Pick disease Type C and ImmunityBio is expanding its scope of clinical trials involving aldoxorubicin to treat advanced pancreatic cancer. We also continue to pursue viable partnership opportunities that can bring Centurion Biopharma’s platform into the clinical testing phase."

First Quarter Financial Overview

CytRx concluded the quarter ended March 31, 2021 with cash on hand of approximately $9.3 million, which management believes is sufficient to fund ongoing operations for the foreseeable future.
The Company recorded a net loss of $1.3 million for the quarter ended March 31, 2021, compared to a net loss of $1.2 million for the same period in 2020.
General and administrative expenses were $1.3 million for the quarter, compared with $1.2 million for the same period in 2020.
Based on a current projection of expenditures, the Company’s monthly cash burn rate is estimated to be approximately $430,000 per month. This number is largely unchanged from the close of the fourth quarter of fiscal year 2020.
Year-to-Date Highlights

Orphazyme Highlights

CytRx’s agreement with Orphazyme can deliver up to approximately $100 million in potential milestone payments and future single digit royalties paid on sales of arimoclomol.
CytRx is positioned to receive up to $10 million in potential milestone payments in 2021 based on possible U.S. and European approvals for arimoclomol to treat Niemann-Pick disease Type C ("NPC").
During the past quarter, multiple analysts issued notes that conveyed optimism regarding Orphazyme’s ability to obtain potential regulatory approvals for arimoclomol in the treatment of NPC.
Recently, Orphazyme announced the appointment of Christophe Bourdon as its new Chief Executive Officer, effective as of April 1, 2021.
As reported by Orphazyme, Mr. Bourdon has successfully launched a variety of products in demanding environments, making him an ideal individual to lead Orphazyme as it prepares for a potential commercial launch of arimoclomol.
He joins from Amgen, Inc., where he held the role of Senior Vice President, General Manager for the U.S. Oncology Business. He was leading commercialization planning and execution for several products.
Previously, Mr. Bourdon was Senior Vice President of Europe, Middle East, Africa and Canada at Alexion Pharmaceuticals Inc. as the company launched two breakthrough ultra-orphan drugs and negotiated payor access across the United Kingdom, Germany, France, Italy and Canada. He holds an MBA from IMD business school (Switzerland) and a BA from ISG (France).
Recently, Orphazyme also announced MIPLYFFA as the global brand name for arimoclomol and expanded its NPC Early Access Program in the U.S. and opened similar programs in France and Germany.
ImmunityBio Highlights

In January 2021, ImmunityBio and NantKwest announced that their ongoing Phase 2 clinical trials of a novel combination immunotherapy – which includes aldoxorubicin – for locally advanced or metastatic pancreatic cancer had produced early indications of increased survival rates for patients with no other approved treatment options.
Interim results of the three-cohort trials, known as QUILT 88, showed median survival rates of more than double that of the historic rate in patients with advanced metastatic pancreatic cancer (for which no other FDA approved treatment exists).
Recently, ImmunityBio and NantKwest announced the closing of their merger, with ImmunityBio now trading on the NASDAQ under ticker symbol IBRX.
Additional Corporate Highlights

In February 2021, CytRx announced that it is now a part of the LD Micro Index (the "Index").
The Index is designed to give the most accurate representation of the intraday activity of microcap stocks in North America.
In March 2021, CytRx participated in the H.C. Wainwright Global Life Sciences Conference and the Virtual 33rd Annual ROTH Conference.
Mr. Kriegsman’s presentation may be accessed on the News and Events page of our website.
With respect to Centurion Biopharma, Mr. Kriegsman and Lead Director Louis Ignarro, PhD have continued pursuing third-party financing and strategic partnership opportunities to advance clinical testing for the platform’s high-potential assets. Discussions with prospective partners are ongoing. There are no formal partnership updates to report at this time.
CytRx maintains federal and state net operating loss ("NOL") carryforwards of $327.6 million and $252.6 million, respectively, available to offset against future taxable income. Of this amount, $258.3 million of federal NOLs and $252.6 million of state NOL’s are unrestricted.

On May 13, 2021 Systems Oncology, a biotechnology company, reported that Dr. Spyro Mousses, CEO and Co-Founder of Systems Oncology, will participate in a fireside chat at the 2021 RBC Capital Markets Global Healthcare Conference on Thursday, May 20th at 12:40pm EST (Press release, Systems Oncology, MAY 13, 2021, View Source [SID1234579956]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Management will host one-on-one investor meetings at the conference. To request a meeting with management, please contact your registered RBC representative or Andrew Matricaria, CFO of Systems Oncology, at [email protected].

On May 13, 2021 -Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results for the first quarter ended March 31, 2021 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAY 13, 2021, View Source [SID1234579955]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Company Updates and Upcoming Milestones

Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

TOPAZ 12-Month Data Show Transformative Potential in SMA of Apitegromab as Add-on to SMN Upregulator Therapy. In April 2021, positive top-line data were announced from the TOPAZ Phase 2 trial (NCT03921528) in patients with Type 2 and Type 3 SMA. Both younger and older non-ambulatory patients treated with apitegromab and background SMN upregulator therapy over the 12-month period attained meaningful motor function improvements with 74% (23/31) of patients demonstrating a clinical improvement of ≥1-point increase in Hammersmith Functional Motor Scale Expanded (HFMSE).

In the younger non-ambulatory TOPAZ cohort (ages 2-6 years), treatment with apitegromab led to a mean increase from baseline in HFMSE of +6.2 points across both evaluated doses; a mean increase of +7.1 points for the 20 mg/kg dose and +5.3 points with the 2 mg/kg dose.

Increase in HFMSE from baseline across both apitegromab doses:

− 82% (14/17) of patients attained a >1-point increase
− 59% (10/17) of patients attained a >3-point increase
− 59% (10/17) of patients attained a >5-point increase
− 35% (6/17) of patients attained a >10-point increase

In the older non-ambulatory TOPAZ cohort (age 8-19 years), treatment with apitegromab 20 mg/kg led to a mean increase from baseline in HFMSE of +0.6 points by the primary intent-to-treat analysis and +1.2 points by the prespecified per protocol analysis.

Increase in HFMSE from baseline for this patient population:

− 64% (9/14) of patients attained a >1-point increase
− 29% (4/14) of patients attained a >3-point increase
Patients enrolled in TOPAZ were receiving chronic maintenance doses of nusinersen; both the younger and older non-ambulatory cohorts had received more than five mean maintenance doses of nusinersen (approximately two years of treatment) at baseline. Clinical data from the CHERISH and SHINE studies of nusinersen offer background insights into this patient population.1 These studies observed that nusinersen-treated patients primarily experience stabilization or only slight increases in HFMSE scores beyond the initial 15-month treatment period.2 In addition, even in the initial 15-month treatment period, older patients (age 5-12) on average experience declines in HFMSE and rarely attain a >3-point increase in HFMSE in a 12-month timeframe.3

The five most frequently reported treatment-emergent adverse events included headache, pyrexia, upper respiratory tract infection, cough, and nasopharyngitis​. All 57 patients who completed the 12-month treatment period elected to opt into the extension period.

Phase 3 Registrational Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Anticipated to Initiate by Year-End 2021. TOPAZ Phase 2 top-line efficacy and safety results support the continued evaluation of apitegromab in a Phase 3 trial. Subject to feedback from regulatory agencies, Scholar Rock intends to conduct a Phase 3 trial to evaluate apitegromab with background SMN upregulator therapy in non-ambulatory Type 2 and Type 3 SMA, the patient population where apitegromab demonstrated the largest increases in motor function (HFMSE scores) in TOPAZ. Patients with non-ambulatory Type 2 and Type 3 SMA represent approximately two-thirds of the overall prevalent SMA patient population.
Additional Data and Analyses from the TOPAZ Trial to be Presented at Upcoming Medical Congresses. Scholar Rock is conducting additional data analyses, including exploratory analyses using patient-level data, evaluating additional outcome measures, and reviewing additional safety data from the TOPAZ Phase 2 trial. The Company plans to present the TOPAZ top-line results as well as findings from these analyses at upcoming medical congresses.
Apitegromab Granted Priority Medicines (PRIME) Designation by the European Medicines Agency (EMA). In March 2021, the EMA granted PRIME designation to apitegromab, recognizing its potential to address unmet medical needs of patients with SMA. With the PRIME designation, Scholar Rock has greater access and enhanced dialogue with the regulatory agency to optimize development plans and evaluations.
Two U.S. Patents Issued Adding Further Protection for Inhibitors of Myostatin Activation and Related Methods, Including Apitegromab. In March 2021, the United States Patent Office (USPTO) issued U.S. Patent No. 10,946,036 with an expiry of June 2037, protecting both add-on and combination therapy with a myostatin inhibitor and a neuronal corrector therapy (such as SMN upregulator therapy) for the treatment of SMA. In April 2021, the USPTO issued U.S. Patent No. 10,981,981 with an expiry of May 2034, broadly covering methods for making inhibitors of myostatin (GDF8) activation based on Scholar Rock’s proprietary platform approach of targeting the precursor forms of growth factors.
Additional Potential Indications Identified for Apitegromab. Scholar Rock has identified multiple diseases for which the selective inhibition of the activation of myostatin may offer therapeutic benefit, including additional patient populations in SMA (such as Type 1 SMA and ambulatory Type 3 SMA) and potential indications outside of SMA, such as Becker Muscular Dystrophy (BMD).
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Published Preclinical Development Data for SRK-181 in International Journal of Toxicology. In March 2021, Scholar Rock announced the publication of "Nonclinical Development of SRK-181: An Anti-latent TGFβ1 Monoclonal Antibody for the Treatment of Locally Advanced or Metastatic Solid Tumors," in the peer-reviewed International Journal of Toxicology. This publication provided a comprehensive preclinical assessment of the pharmacology, pharmacokinetics, and safety of SRK-181, which provided support for the dose selection strategy for the ongoing DRAGON Phase 1 trial.
Initial Clinical Response and Safety Data from Part A of the DRAGON Phase 1 Trial Anticipated by Year-End 2021. SRK-181 is being evaluated in the two-part DRAGON trial (NCT04291079) in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Dose escalation continues to progress in Part A of the trial, which evaluates the safety and pharmacokinetics of SRK-181, and the Company expects to advance to the Part B dose expansion portion of the trial in mid-2021. Part B will consist of multiple cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Each cohort will enroll up to 40 patients who have demonstrated primary resistance to anti-PD-(L)1 therapy and will be treated with SRK-181 in combination with an approved anti-PD-(L)1 therapy.
"2021 is off to a great start as we are delighted with the positive 12-month top-line data from our TOPAZ study, which shows the transformative potential of apitegromab in SMA," said Tony Kingsley, President and CEO of Scholar Rock. "Based on these exciting results and the potential to bring apitegromab to the SMA community, we will be working with urgency on behalf of patients to initiate our Phase 3 registrational trial in patients with non-ambulatory Type 2 and 3 SMA by the end of the year."

First Quarter 2021 Financial Results

For the quarter ended March 31, 2021, net loss was $27.7 million or $0.76 per share compared to a net loss of $17.1 million or $0.58 per share for the quarter ended March 31, 2020.

Revenue was $4.7 million for the quarter ended March 31, 2021 compared to $5.0 million for the quarter ended March 31, 2020 and was related to the Gilead fibrosis-focused collaboration (the "Gilead Collaboration Agreement") that was executed in December 2018.
Research and development expense was $22.5 million for the quarter ended March 31, 2021 compared to $16.9 million for the quarter ended March 31, 2020. The increase year-over-year primarily reflects manufacturing costs associated with apitegromab and higher personnel and facility-related costs, partially offset by lower manufacturing costs associated with SRK-181.
General and administrative expense was $9.4 million for the quarter ended March 31, 2021 compared to $5.8 million for the quarter ended March 31, 2020. The increase year-over-year was primarily attributed to higher personnel and facility-related costs.
"We are focused on advancing our portfolio of clinical and preclinical programs with several near-term milestones, including the initiation of a Phase 3 trial evaluating apitegromab in SMA, completion of dose escalation in Part A and initiation of the Part B dose expansion portion of the DRAGON Phase 1 trial of SRK-181 in patients with solid tumors," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We are well positioned to continue to execute on our plans as we ended the first quarter with approximately $315 million in cash, which can fund operations into 2023."

1This information from third-party studies is provided for background purposes only and is not intended to convey or imply a comparison to the TOPAZ clinical trial results.
2 Source: "Longer-term treatment with nusinersen: results in later-onset spinal muscular atrophy from the SHINE study" P.257, World Muscle Society Congress 2020
3 Source: Mercuri E, et.al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378:625-635.