Scholar Rock Reports Second Quarter 2022 Financial Results and Highlights Business Progress

On August 8, 2022 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, 2022 (Press release, Scholar Rock, AUG 8, 2022, View Source [SID1234617816]).

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"We are pleased to have reported important progress with our two clinical programs during the second quarter. Enrollment continues to advance in our pivotal Phase 3 SAPPHIRE trial for apitegromab in SMA. We also presented new data from our Phase 2 TOPAZ trial highlighting sizeable and sustained improvement in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores at 24 months for non-ambulatory patients, reinforcing our belief in the unique potential of apitegromab as a muscle-targeted therapy to treat SMA, and we continue to evaluate these patients in their third year of treatment," said Nagesh Mahanthappa, Founding Chief Executive Officer & President of Scholar Rock. "In our SRK-181 program, enrollment is ongoing in the Phase 1 DRAGON trial Part B dose expansion. We look forward to providing updates on DRAGON as data become available. Further, our focused research continues to make excellent progress as we develop next-generation programs to build out our future pipeline."

Company Highlights and Upcoming Milestones

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

Positive 24-Month Phase 2 TOPAZ Extension Data Support Potential Benefit for Patients. Scholar Rock announced positive data at the Cure SMA Research & Clinical Care Meeting in June, demonstrating sizable and sustained motor function improvement at 24 months with apitegromab as measured by Hammersmith Functional Motor Scale-Expanded (HFMSE) scores for non-ambulatory patients with Types 2 and 3 SMA on nusinersen. Results also showed a substantial increase in Revised Upper Limb Module (RULM) scores, with no serious safety risks identified over 24 months of apitegromab treatment. Of the 55 patients who completed the 24-month extension period, 54 opted to continue into the 36-month extension period.
Enrollment Ongoing for Phase 3 SAPPHIRE Clinical Trial Evaluating Apitegromab in Non-Ambulatory Patients with Types 2 and 3 SMA. The randomized, double-blind, placebo-controlled Phase 3 clinical trial is evaluating apitegromab for patients on either nusinersen or risdiplam. Approximately 156 non-ambulatory patients aged 2-12 years old with Types 2 and 3 SMA are planned to be enrolled in the main efficacy population. Patients will be randomized 1:1:1 to receive for 12 months either apitegromab 20 mg/kg, apitegromab 10 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. SAPPHIRE is expected to be conducted across 55 sites in the U.S. and Europe. Scholar Rock presented on the trial design at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022) in July 2022, along with publication of the abstract in the peer-reviewed Journal of Neuromuscular Diseases.
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming primary resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Advancing Enrollment for Part B of the Phase 1 DRAGON Proof-of-Concept Clinical Trial for SRK-181. Part B of the Phase 1 DRAGON trial consists of multiple proof-of-concept cohorts focused on evaluating the ability of SRK-181 to overcome primary resistance to anti-PD-(L)1 therapy in patients with solid tumors. The biomarker strategy for DRAGON explores early signs of SRK-181 activity, including target engagement and pathway modulation. It includes measuring effects on both circulating and tumor immune contexture, such as CD8+ T cell infiltration and reductions in myeloid-derived suppressor cell (MDSC) populations, as well as analysis of TGFβ-related pathway signaling. Initial evidence of drug activity and safety data are anticipated in 2022.
Scholar Rock’s preclinical discovery pipeline includes a highly selective, context-dependent TGFβ1 antibody that inhibits proTGFβ1 activation selectively in the extracellular matrix via targeting the covalent complexes of proTGFβ1 with latent TGFβ binding proteins 1 and 3 ("LTBPs"— the targets are collectively referred to as "LTBP-TGFβ1").

Preclinical Data Supports Selective Targeting of Matrix-Associated TGFβ1 as an Attractive Approach for Anti-Fibrotic Therapy. Scholar Rock recently presented data on its targeted approach to LTBP-TGFβ1 that show reduction of TGFβ1 signaling and reduction of fibrosis in relevant preclinical in vivo models.
Second Quarter 2022 Financial Results

For the quarter ended June 30, 2022, net loss was $44.0 million or $1.06 per share compared to a net loss of $30.7 million or $0.84 per share for the quarter ended June 30, 2021.

Revenue was $0 for the quarter ended June 30, 2022, compared to $4.6 million for the quarter ended June 30, 2021.
Research and development expense was $32.1 million for the quarter ended June 30, 2022, compared to $25.6 million for the quarter ended June 30, 2021. The increase was primarily attributable to increased clinical costs associated with apitegromab and higher personnel costs, including severance expenses associated with the recent restructuring.
General and administrative expense was $11.1 million for the quarter ended June 30, 2022, compared to $9.3 million for the quarter ended June 30, 2021. The increase was primarily due to higher personnel costs, including severance expenses associated with the recent restructuring.
As of June 30, 2022, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $371 million, which is expected to fund the Company’s anticipated operating and capital expenditure requirements into 2025.
"We were thrilled to announce the completion of a $205 million registered direct offering during the quarter. This financing included several high-quality, long-term oriented, fundamentals-based biotechnology investors who saw the promise of our platform and our programs. Importantly, this capital puts us in a strong financial position to fully fund the Phase 3 SAPPHIRE trial, continue advancing Part B of the Phase 1 DRAGON trial for SRK-181, while investing in selected early-stage programs that exemplify the power of our scientific platform," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock.

Initial Analysis of Tumor Profiling From a Prospective Study by Foundation Medicine, Flatiron Health and Genentech Demonstrates Value of Tissue and Liquid Comprehensive Genomic Profiling in Patients with Advanced Non-Small Cell Lung Cancer

On August 8, 2022 Foundation Medicine, Inc., Flatiron Health, and Genentech, members of the Roche Group, in partnership with a network of community oncology practices, reported new research in the Journal of Thoracic Oncology Clinical and Research Reports on tumor profiling results from the ongoing, multicenter Prospective Clinicogenomic (PCG) Study (NCT04180176) (Press release, Foundation Medicine, AUG 8, 2022, View Source [SID1234617815]). In this investigation, researchers compared the ability of FoundationOneCDx and FoundationOneLiquid CDx comprehensive genomic profiling (CGP) to limited tissue testing in detecting actionable, guideline-recommended biomarkers for targeted treatment planning in patients with advanced non-squamous non-small cell lung cancer (aNSCLC).

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Despite continued adoption of CGP, most patients with advanced cancer still receive limited biomarker testing. The tumor profiling data from a diverse group of 515 aNSCLC patients in the PCG study demonstrates the superiority of both tissue- and blood-based CGP over more limited tissue testing in detecting actionable biomarkers. Additionally, the findings reinforce the value of blood-based CGP as a compelling and convenient complement to tissue-based CGP, especially when an adequate tissue sample is not immediately available.

Nine genes recommended for testing by clinical guidelines were included in the analysis: EGFR, KRAS, ALK, ROS1, RET, BRAF, MET exon 14, NTRK, as well as emerging biomarkers ERBB2 mutations and MET amplification. At study enrollment, patients with non-squamous aNSCLC received Foundation Medicine’s liquid biopsy CGP testing, and a subset also received Foundation Medicine tissue testing. Results demonstrate that tissue-based FoundationOneCDx and blood-based FoundationOneLiquid CDx both provided superior biomarker detection over more limited tissue testing of up to 5 biomarkers. In particular, Foundation Medicine’s liquid biopsy test detected guideline-recommended biomarkers in 48 additional patients who received single biomarker or hotspot testing.

"Precision oncology is transforming the way patients with cancer are treated, and there has been an explosion of targeted therapy options in lung cancer specifically," said Foundation Medicine’s Chief Medical Officer, Dr. Mia Levy. "With the amount of diverse diagnostic tools now available to physicians, a key clinical question is how to piece these diagnostics together to optimally identify precision treatment options for patients with advanced cancer. Results from this research reinforce the unique benefits, and complementary value of both tissue- and blood-based CGP tests, depending on the patient’s tissue availability, health status and tumor type."

Results from this research reinforce that, in certain patient cases where an adequate tissue sample is immediately available, tissue CGP offers the most reliable detection of guideline-recommended biomarkers. In cases where tissue is not immediately available, blood-based CGP can be used for expeditious CGP testing, though reflex tissue testing may be beneficial if blood-based CGP does not detect an actionable alteration to inform therapy selection. An important reason for this distinction is that liquid biopsy sensitivity is dependent upon the amount of tumor shed into the blood stream, called circulating tumor DNA (ctDNA). When ctDNA shed for a patient is low, a blood-based test may not be able to detect the ctDNA in the blood and tissue testing could yield additional insights.

"This initial analysis of the PCG Study data provides meaningful insights to help oncologists select the appropriate molecular diagnostic platform for their patients with lung cancer," said Dr. Neal Meropol, Vice President, Research Oncology and Scientific and Clinical Lead, Clinical Research, Flatiron Health. "This research reinforces how advances in precision oncology are benefitting patients with advanced non-small cell lung cancer."

The PCG Study, funded and sponsored by Genentech, pilots the use of a technology-enabled prospective data collection platform to facilitate, streamline and simplify the execution of clinical trials for patients living with advanced lung cancer, and secondarily aims to better understand how genomic changes in a patient’s tumor may predict response or impact resistance to treatment. A complete report of the PCG Study will be forthcoming to more fully describe the genomics of the pre-treatment and on-treatment comprehensive genomic profiling and its relationship to patient outcomes.

Pyramid Biosciences Names Nushmia Khokhar, MD, as Chief Medical Officer

On August 8, 2022 Pyramid Biosciences, Inc., a Boston-based, clinical-stage biotechnology company focused on developing new and highly differentiated precision therapies for cancer and other serious diseases, reported the expansion of its senior leadership team (Press release, Pyramid Biosciences, AUG 8, 2022, View Source [SID1234617814]). Nushmia Khokhar, MD has joined the company as Chief Medical Officer, where she will oversee the advancement and expansion of the Company’s clinical pipeline.

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"Dr. Khokhar has a proven track record of successfully bringing novel oncology compounds through clinical development to global approval," said CEO of Pyramid Biosciences, Brian Lestini, MD, PhD. "We are looking forward to Dr. Khokhar’s leadership of our clinical programs during this important growth stage of our company."

Prior to joining Pyramid Biosciences, Dr. Khokhar served as Chief Medical Officer at Umoja Biopharma, a multi-platform, immuno-oncology company based in Seattle, WA. In this capacity, Dr. Khokhar oversaw Umoja’s clinical development, operations, and regulatory functions. Prior to joining Umoja, Dr. Khokhar was the Senior Vice President & Head of Clinical Development at Autolus Therapeutics, where she played a critical role in advancing the company’s autologous CAR T-cell products and clinical programs. Prior to that, Dr. Khokhar held numerous roles at Janssen Oncology, a subsidiary of Johnson & Johnson (NYSE: JNJ) where she led several successful clinical trials, directed the Phase 3 registration trial of trabectedin (YONDELIS) in soft tissue sarcomas, and served as the Global Clinical Leader for the breakthrough therapy daratumumab (DARZALEX) for hematologic cancers.

"I look forward to working with the outstanding team at Pyramid Biosciences, progressing the Company’s promising therapies through clinical development and building a robust pipeline," said Dr. Khokhar.

"We are extremely pleased to have Nushmia join our senior leadership team as we continue to grow our clinical programs and develop new medicines for underserved patients," said Jordan Leef, Pyramid Biosciences’ Co-Founder & COO.

Dr. Khokhar is a graduate of Aga Khan University and completed her post-doctoral fellowship at Memorial Sloan-Kettering Cancer Center, where she focused on pharmacology and molecular therapeutics. Following her post-doctoral fellowship, Dr. Khokhar completed a residency in internal medicine at Washington Hospital Center and a fellowship in Hematology/Oncology at the Northwestern University Hospital. Dr. Khokhar is board certified in medical oncology.

Gamida Cell Announces the Date of Its Second Quarter 2022 Financial Results and Webcast

On August 8, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, reported that the company will host a conference call and live audio webcast on Monday, August 15, 2022, at 8:00 a.m. ET to review its second quarter 2022 financial results and provide an update on the company (Press release, Gamida Cell, AUG 8, 2022, View Source [SID1234617813]).

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To access the conference call, please register here and be advised to do so at least 10 minutes prior to joining the call. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.

Review of SURPASS-ET Trial Evaluating Ropeginterferon Alfa-2b-njft for Essential Thrombocythemia (ET) Published in Future Oncology

On August 8, 2022 PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TPEx:6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, reported that the clinical rationale and protocol for the SURPASS-ET trial evaluating ropeginterferon alfa-2b-njft as a second-line treatment option for adults with high-risk essential thrombocythemia (ET) has been published in the journal, Future Oncology (Press release, PharmaEssentia, AUG 8, 2022, View Source [SID1234617812]).

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ET is a myeloproliferative neoplasm (MPNs), a group of rare blood cancers caused by genetic mutations that trigger the overproduction of blood cells; ET is characterized by the overproduction of platelets. Without proper treatment, these disorders carry a higher risk of thrombosis or other cardiovascular complications, or may progress toward myelofibrosis or secondary acute myeloid leukemia (sAML). Yet many patients who receive conventional approved treatments for ET experience resistance or intolerance, or the efficacy wanes over time, so new therapeutic options are needed to help address these limitations and improve treatment outcomes.

Ropeginterferon alfa-2b-njft is a unique monopegylated, long-acting interferon that was recently approved to treat adults with polycythemia vera (PV), another type of MPN. Given the well-established safety and efficacy profile demonstrated in prior studies, the treatment may represent a useful alternative to approved options for ET.

"Physicians treating people with MPNs have lacked effective, durable therapeutics that are designed specifically for these cancers. To help improve the long-term outlook for these patients, we need to focus not only on normalizing symptoms and improving quality of life, but also targeting driver mutations to more completely control the disease," said Srdan Verstovsek, M.D., Ph.D., Director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms, Department of Leukemia at the University of Texas MD Anderson Cancer Center. "This trial will provide critical data on how use of a monopegylated interferon for the first time in this disease state could reduce the risk of progression for these patients over time."

The global phase 3, randomized, open-label, multicenter SURPASS-ET (NCT04285086) trial is evaluating the safety, efficacy, tolerability, and pharmacokinetics of ropeginterferon alfa-2b-njft compared to anagrelide as second-line therapy in high-risk ET. Approximately 160 patients are being enrolled from 61 study sites across the United States, Canada and multiple regions across Asia, and will be randomized to receive treatment with ropeginterferon alfa-2b-njft (via subcutaneous injection every two weeks starting at 250 mcg, with a target optimal dose of 500 mcg by week four and onwards) or a daily anagrelide capsule (0.5 mg).

The primary efficacy endpoint is durable patient response, as defined by modified ELN response criteria (peripheral blood count remission; improvement or non-progression in disease-related signs; improvement or non-progression based on the MPN-SAF TSS; absence of hemorrhagic or thrombotic events; and durability at months 9 and 12). Other endpoints being evaluated include quality of life and change in allelic burden (the proportion of mutated cells in the blood). Data from the trial are expected by 2024.

"With nearly a decade of ongoing research with interferons in MPNs, we have strong evidence supporting the use of ropeginterferon alfa-2b-njft to treat individuals with ET," said Raymond Urbanski, M.D., Ph.D., U.S. Head of Clinical Development and Medical Affairs, PharmaEssentia. "The series of clinical trials currently underway are providing an increasingly clear picture of the profile of this treatment to potentially improve care standards and patient outcomes across the MPN category."

About Ropeginterferon alfa-2b

Ropeginteferon alfa-2b-njft is an innovative monopegylated, long-acting interferon. With its unique pegylation technology, the product has a long duration of activity in the body.

Ropeginteferon alfa-2b-njft (marketed as BESREMi) has orphan drug designation for treatment of polycythemia vera (PV) in adults in the United States. The product was approved by the European Medicines Agency (EMA) in 2019, in the United States in 2021, and has recently received approval in Taiwan and South Korea. The product was invented by PharmaEssentia and is manufactured in the company’s Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018. PharmaEssentia retains full global intellectual property rights for the product in all indications.

BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infectious disorders.

About Essential Thrombocythemia

Essential thrombocythemia (ET) is a myeloproliferative neoplasm (MPN) characterized by an overproduction of platelets in the blood that results from a genetic mutation; data indicates a JAK2 gene mutation is present in approximately half of diagnosed patients. ET is estimated to affect up to 57 per 100,000 people in the U.S. The disease is most commonly diagnosed through routine blood work, and is most common in people over the age of 50, with women 1.5 more times more likely to be diagnosed than men. As a chronic, progressive disease, ET requires regular monitoring and appropriate treatment. Over time, the disease may progress into more deadly conditions such as myelofibrosis or acute leukemia.1,2