On August 10, 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 second quarter ended June 30, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, AUG 10, 2021, View Source [SID1234586232]).

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"Scholar Rock has had significant momentum in the first half of the year with further demonstration of apitegromab’s transformative potential to improve motor function for patients with SMA, as well as continued progression of dose escalation in the DRAGON trial evaluating SRK-181’s potential to overcome resistance to checkpoint inhibitors in solid tumors," said Nagesh Mahanthappa, Ph.D., Interim CEO of Scholar Rock. "I look forward to working closely with this exceptional team as we continue to advance our research and clinical programs to further elucidate the potential of our scientific platform and help make a difference in the lives of patients suffering from serious diseases."

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).

Positive Top-line Results and Additional Supportive Exploratory Analyses from the TOPAZ Trial were Presented at the Cure SMA Annual Conference. In June 2021, positive top-line data from the TOPAZ Phase 2 trial (NCT03921528) were presented by the lead principal investigator, Thomas Crawford, M.D. of Johns Hopkins Medicine. Treatment with apitegromab in conjunction with nusinersen in patients with Type 2 and 3 SMA led to meaningful motor function improvements of up to 20 points as measured by Hammersmith Functional Motor Scale Expanded (HFMSE). The majority (74%, 23/31) of non-ambulatory patients showed a clinical improvement, as defined by at least a 1-point increase in HFMSE. Further evidence that improvements in motor function may be attributed to apitegromab was supported by a post-hoc analysis that showed no correlation between change in HFMSE and duration of prior maintenance nusinersen therapy. In addition, 7/35 non-ambulatory patients also showed major functional achievements as measured by World Health Organization (WHO) Motor Development Milestones, with one patient achieving two and one patient achieving three new WHO motor milestones.
Phase 3 Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Anticipated to Initiate by Year-End 2021. Subject to feedback from regulatory agencies, Scholar Rock intends to conduct a randomized, double-blind, placebo-controlled Phase 3 trial to evaluate apitegromab as an add-on therapy to nusinersen or risdiplam in patients with non-ambulatory Type 2 and Type 3 SMA. In the TOPAZ Phase 2 trial, non-ambulatory patients experienced the largest increases in motor function (HFMSE scores) following treatment with apitegromab as add-on to chronic maintenance nusinersen. Patients with non-ambulatory Type 2 and Type 3 SMA are estimated to represent approximately two-thirds of the overall prevalent SMA patient population.
Apitegromab Granted Fast Track Designation by the FDA for the Treatment of Patients with Spinal Muscular Atrophy. In May 2021, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for apitegromab, offering Scholar Rock eligibility to submit a rolling Biologic License Application (BLA) for apitegromab if relevant criteria are met. Fast Track designation is intended to facilitate the development and expedite the review of drugs to treat serious conditions and get new drugs to patients earlier. In addition to Fast Track designation, apitegromab had previously received Orphan Drug and Rare Pediatric Disease designations from the FDA as well as PRIME and Orphan Medicine Product designations from the European Medicines Agency for the treatment of SMA, all recognizing the unmet medical needs of patients with SMA.
Additional Potential Indications Identified for Apitegromab. Scholar Rock has identified multiple diseases for which selectively inhibiting 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.
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.

Plan to Advance to Part B Dose Expansion Portion of the DRAGON Phase 1 Proof-of-Concept Trial Mid-Year. 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. Part A dose escalation, which is evaluating the safety and pharmacokinetics of SRK-181 as a single-agent and in combination with anti-PD-(L)1 therapy, continues to progress to identify the recommended dose for Part B of the trial. The Company is on-track to advance to Part B dose expansion, which 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. An update on dose escalation and initial clinical data from Part A of the DRAGON trial is anticipated by year-end.
Second Quarter 2021 Financial Results

For the quarter ended June 30, 2021, net loss was $30.7 million or $0.84 per share compared to a net loss of $19.3 million or $0.65 per share for the quarter ended June 30, 2020.

Revenue was $4.6 million for the quarter ended June 30, 2021 compared to $3.9 million for the quarter ended June 30, 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 $25.6 million for the quarter ended June 30, 2021 compared to $17.0 million for the quarter ended June 30, 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 $9.3 million for the quarter ended June 30, 2021 compared to $6.4 million for the quarter ended June 30, 2020. The increase year-over-year was primarily attributed to higher personnel and facility-related costs and professional fees.

"We are executing towards key milestones and continue to work closely with regulatory authorities to finalize the design of the Phase 3 trial for apitegromab and to progress the DRAGON trial to Part B to evaluate SRK-181 across multiple tumor types," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We ended the second quarter with approximately $282 million in cash and cash equivalents, which will allow us to achieve meaningful milestones as we continue to execute against our plan."

On August 10, 2021 Olema Pharmaceuticals, Inc. ("Olema" or "Olema Oncology," Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted therapies for women’s cancers, reported second quarter financial results for the period ended June 30, 2021 (Press release, Olema Oncology, AUG 10, 2021, View Source [SID1234586231]).

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"We made important progress in the second quarter of 2021 as we advanced the clinical development of our lead candidate, OP-1250, an investigational complete estrogen receptor (ER) antagonist (CERAN), and strengthened our corporate foundation to ensure that we have the talent and resources in place to support our future success," said Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer of Olema Oncology. "We have seen robust enrollment in the ongoing Phase 1/2 clinical trial of OP-1250 and look forward to sharing interim dose-escalation data at a medical meeting in the fourth quarter of this year."

Corporate Highlights and Anticipated Milestones

Significant progress advancing the Phase 1/2 clinical trial of OP-1250 in patients with metastatic, ER+ / HER2- breast cancer. As of June 4, 2021, 28 patients have been enrolled across five dose-escalation cohorts. OP-1250 has demonstrated oral bioavailability and a dose-proportional pharmacokinetic profile consistent with predictions from Olema nonclinical models. A maximum tolerated dose has not been identified. The Company plans to present interim safety, tolerability, pharmacokinetic and initial efficacy data at a medical meeting in the fourth quarter of 2021, pending abstract acceptance.
Advance into monotherapy dose expansion in the second half of 2021.
Initiate a Phase 1b clinical trial of OP-1250 in combination with a CDK4/6 inhibitor in the first quarter of 2022.
Second Quarter 2021 Financial Highlights

Cash, cash equivalents and marketable securities as of June 30, 2021 were $318.1 million. Olema anticipates that this cash balance will be sufficient to fund operations through the end of 2023.
Research and development (R&D) expenses were $11.9 million for the quarter ended June 30, 2021, compared to $1.9 million for the same period of the prior year. The increase in R&D expenses was primarily due to increased expenditures to advance the Phase 1/2 clinical trial of OP-1250, the increase in nonclinical development activities, higher personnel-related expenses as headcount grew to support the advancement of the clinical and nonclinical programs, and higher non-cash stock-based compensation expenses.
General and administrative (G&A) expenses were $4.6 million for the quarter ended June 30, 2021, compared to $0.5 million for the same period of the prior year. The increase in G&A expenses was primarily due to higher personnel-related expenses associated with increases in the number of G&A personnel supporting the growth of the organization, public company-related expenses and other corporate costs, and non-cash share-based compensation expenses.
Net loss for the quarter ended June 30, 2021 was $16.4 million, compared to $2.5 million for the same period of the prior year.

On August 10, 2021 Day One Biopharmaceuticals (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for patients of all ages with genomically defined cancers, reported financial results for the second quarter of 2021 and highlighted recent corporate achievements (Press release, Day One, AUG 10, 2021, View Source [SID1234586230]).

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"Day One made significant progress across multiple clinical and corporate initiatives during the second quarter of 2021, including dosing the first patients in our ongoing FIREFLY-1 pivotal study of DAY101 in pediatric low-grade glioma," said Jeremy Bender, Ph.D., chief executive officer of Day One. "The success of our recent IPO reflects a strong commitment from our investors who, like all of us at Day One, recognize the therapeutic potential of DAY101. Entering the second half of 2021, we remain well positioned to advance our pipeline through key data readouts with the goal of fulfilling our mission of developing novel medicines to improve the lives of patients of all ages living with cancer." Program Highlights The Company announced first patients dosed in the FIREFLY-1 pivotal clinical trial of DAY101 in pediatric low-grade glioma (pLGG). FIREFLY-1 is being conducted in collaboration with the Pacific Pediatric Neuro-Oncology Consortium (PNOC) and is designed to support the regulatory approval of DAY101. Initial data from FIREFLY-1 is expected in the first half of 2022. Day One has initiated a Phase 2 monotherapy trial of DAY101 in adult patients with recurrent, progressive, or refractory solid tumors harboring MAPK pathway aberrations. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to DAY101 for the treatment of low-grade gliomas harboring an activating RAF alteration that disproportionately affects children. If a New Drug Application in the United States for DAY101 is approved, Day One may be eligible to receive a Priority Review Voucher (PRV) from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application or may be sold or transferred. The European Commission granted DAY101 Orphan Designation for the treatment of glioma based upon a positive opinion from the European Medicines Agency Committee for Orphan Medicinal Products. Corporate Highlights The Company announced the successful closing of its upsized initial public offering, raising gross proceeds of $184.0 million, bringing total cash, cash equivalents and marketable securities to $310.0 million at the end of June 30, 2021. The company expects its current cash position to fund operations into the second half of 2023 and through key clinical milestones. Day One appointed Saira Ramasastry to its Board of Directors. Ms. Ramasastry currently serves as the Managing Partner of Life Sciences Advisory, LLC, and brings more than 20 years of experience to the Board as a life sciences-focused strategic consultant and investment banker. Second Quarter 2021 Financial Highlights Cash Position: Cash and cash equivalents and short-term investments totaled $310.0 million at June 30, 2021. Based on Day One’s current operating plan, management believes it has sufficient capital resources to fund anticipated operations into the second half of 2023. R&D Expenses: Research and development expenses were $9.9 million for the second quarter 2021 and $1.4 million for the second quarter 2020. The increase was primarily due to additional employee compensation costs, clinical trial expenses, CMC activity and a milestone payment for DAY101. G&A Expenses: General and administrative expenses were $5.5 million for the second quarter 2021 and $0.9 million for the second quarter 2020. The increase was primarily due to additional employee compensation costs, legal, and professional expenses associated with being a public company. Net Loss: Net loss totaled $15.5 million and $2.4 million for the second quarter 2021 and 2020, respectively, with non-cash stock compensation expense of $2.5 million and $0.1 million for the second quarter of 2021 and 2020, respectively. Upcoming Events 12th Annual Wedbush PacGrow Healthcare Conference: Day One’s chief executive officer Jeremy Bender will be a participant on the Panel, "Bullseye – Targeted Oncology Part 2". The panel discussion will take place on Wednesday, August 11th at 10:20 am ET. Day One will also be available for one-on-one investor meetings during the conference. About DAY101 DAY101 is an investigational, oral, brain-penetrant, highly-selective type II pan-RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway. Studies have shown DAY101 has high brain distribution and exposure in comparison to other MAPK pathway inhibitors, thus potentially benefiting patients with primary brain tumors or brain metastases of solid tumors. DAY101 is a type II RAF inhibitor found to selectively inhibit both monomeric and dimeric RAF kinase, which may broaden its potential clinical application to treat an array of RAF-altered tumors. DAY101 has been studied in over 250 patients, and as a monotherapy demonstrated good tolerability and encouraging anti-tumor activity in pediatric and adult populations with specific MAPK pathway-alterations. In November 2020, Day One announced preliminary results from PNOC014, an ongoing Phase 1 Pacific Pediatric Neuro-Oncology Consortium (PNOC) network study with DAY101 sponsored by the Dana-Farber Cancer Institute. Preliminary results demonstrated that of the eight relapsed pLGG patients in the study with RAF fusions, two patients achieved a complete response by Response Assessment for Neuro-Oncology (RANO), three had a partial response, two achieved prolonged stable disease, and one experienced progressive disease. DAY101 also demonstrated a tolerable safety profile with the most common side effects being skin rash and hair color changes. DAY101 has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with pLGG harboring an activating RAF alteration who require systemic therapy and who have either progressed following prior treatment or who have no satisfactory alternative treatment options. The FDA has also granted Rare Pediatric Disease Designation to DAY101 for the treatment of low-grade gliomas harboring an activating RAF alteration that disproportionately affects children. In addition, DAY101 has received Orphan Drug designation from the FDA for the treatment of malignant glioma and orphan designation from the European Commission for the treatment of glioma. Day One is conducting a pivotal Phase 2 trial (FIREFLY-1) of DAY101 in pediatric, adolescent and young adult patients with pLGG. Day One also plans to study DAY101 alone or in combination with other agents that target key signaling nodes in the MAPK pathway, such as the Company’s MEK inhibitor pimasertib, in patient populations where various RAS and RAF alterations are believed to play an important role in driving disease.

On August 10, 2021 Equillium, Inc. (Nasdaq: EQ), a clinical-stage biotechnology company developing itolizumab to treat severe autoimmune and inflammatory disorders with high unmet medical need, reported financial results for the second quarter 2021, and provided an update on its clinical programs (Press release, Equillium, AUG 10, 2021, View Source [SID1234586229]).

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"The second quarter of the year was highlighted by positive data from our Phase 1b EQUATE study in acute graft-versus-host disease," said Bruce Steel, chief executive officer at Equillium. "These data were critical for achieving a positive outcome from our End-of-Phase 1 meeting with the FDA and accelerating our plans to immediately advance to a single, pivotal Phase 3 clinical study. This strategy, if successful, may position itolizumab to become the first approved therapy to treat patients with acute graft-versus-host disease in the first-line setting."

Corporate & Clinical Highlights Since Beginning of Q2 2021:

Announced positive topline results from the EQUATE study in first-line treatment of acute graft-versus-host disease presented at the 2021 Virtual Congress of the European Hematology Association (EHA) (Free EHA Whitepaper), and plans to initiate a Phase 3 pivotal study following an End-of-Phase 1 meeting with the FDA
Itolizumab continues to demonstrate favorable safety and efficacy profile
Rapid and durable complete responses resulted in clinically meaningful reduction in corticosteroid use
Data support clinical advancement of itolizumab in first-line treatment of aGVHD
Presented multiple posters at the 104th annual meeting of the American Association of Immunologists; research highlighted:
Itolizumab’s novel mechanism of action and its effect on modulating T cell responses through inhibition of the CD6-ALCAM pathway
Development of a pharmacodynamic biomarker assay to monitor target engagement and fate of CD6 on T cells in patients treated with itolizumab
Upcoming Catalysts:

EQUALISE Phase 1b study: interim data from Type B patients (lupus nephritis) expected 2H 2021
EQUIP Phase 1b study: topline data in uncontrolled asthma expected 2H 2021
Initiate pivotal study in first-line aGVHD expected Q4 2021
Second Quarter 2021 Financial Results

Research and development (R&D) expenses for the second quarter of 2021 were $6.0 million, compared with $3.9 million for the same period in 2020. The increase was driven by greater employee compensation and benefit expenses primarily related to increased headcount, an increase in clinical development expenses primarily related to the EQUATE and EQUALISE studies, as well as greater consulting, overhead, and non-clinical research expenses.

General and administrative (G&A) expenses for the second quarter of 2021 were $2.9 million, compared with $2.7 million for the same period in 2020. The increase was driven by greater employee compensation and benefits primarily related to increased headcount, partially offset by a decrease in non-cash stock-based compensation mainly due to retention option grants issued to certain officers and directors in the second quarter of 2020.

Net loss for the second quarter of 2021 was $9.2 million, or $(0.31) per basic and diluted share, compared with a net loss of $6.5 million, or $(0.37) per basic and diluted share for the same period in 2020. The increase in net loss was largely attributable to greater operating expenses.

Cash used in operations for the second quarter of 2021 was $7.0 million compared to $7.9 million in the first quarter of 2021.

Cash, cash equivalents and short-term investments totaled $97.6 million as of June 30, 2021, compared to $104.1 million as of March 31, 2021. Equillium believes that its cash and investments will be sufficient to fund its currently planned operations into 2023.

About Itolizumab

Itolizumab is a clinical-stage, first-in-class anti-CD6 monoclonal antibody that selectively targets the CD6-ALCAM pathway. This pathway plays a central role in modulating the activity and trafficking of T cells that drive a number of immuno-inflammatory diseases. Equillium acquired rights to itolizumab through an exclusive partnership with Biocon Limited.

On August 10, 2021 CohBar, Inc. (NASDAQ: CWBR), a clinical stage biotechnology company developing mitochondria based therapeutics to treat chronic diseases and extend healthy lifespan, reported its financial results for the second quarter ended June 30, 2021 (Press release, CohBar, AUG 10, 2021, View Source [SID1234586228]).

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"Our positive CB4211 topline data announcement today marks an important milestone in our path towards demonstrating the full potential of our novel therapeutic platform sourced from the mitochondrial genome," stated Dr. Joseph Sarret, Chief Executive Officer. "In parallel with our ongoing analysis of the promising data from the CB4211 study, we are diligently working to enable CB5138-3 to enter the clinic next year for the treatment of idiopathic pulmonary fibrosis. This is an exciting time for the company as we look forward to continued progress in the development of product candidates designed to address serious unmet medical needs."

Second Quarter 2021 and Recent Highlights

Announced Positive Topline Data from the Phase 1a/1b Study of CB4211 Being Developed for the Treatment of Nonalcoholic Steatohepatitis (NASH) and Obesity: Today, the company announced topline results from the multi-center, randomized, double-blind, placebo-controlled Phase 1a/1b clinical study of CB4211, under development for NASH and obesity. The study met its primary endpoints as CB4211 was well-tolerated and appeared safe with no serious adverse events. The evaluation of the exploratory endpoints showed robust and statistically significant improvements in key biomarkers of liver damage, ALT and AST, and in glucose levels in the CB4211 group compared to placebo. There was a trend towards lower body weight in the CB4211 group after four weeks of treatment.
Appointed Joseph J. Sarret, M.D., J.D. as Chief Executive Officer and Director: In April, the company announced the appointment of Joseph J. Sarret, M.D., J.D. as Chief Executive Officer and Director. Dr. Sarret is a seasoned executive with a track record of success in biotechnology.
Second Quarter 2021 Financial Highlights

Cash and Investments: CohBar had cash and investments of $13.8 million as of June 30, 2021, compared to $21 million as of December 31, 2020. The cash burn for the quarter ended June 30, 2021, was approximately $4.6 million.
R&D Expenses: Research and development expenses were $2.6 million for the three months ended June 30, 2021, compared to $1.5 million in the prior year quarter. The increase in research and development expenses was primarily due to the investment in the company’s research programs focused on the continued development of its peptides, and an increase in clinical trial costs due to the timing of those expenses, partially offset by a decrease in stock based compensations costs.
G&A Expenses: General and administrative expenses were $2.6 million for the three months ended June 30, 2021, compared to $1.4 million in the prior year quarter. The increase in general and administrative expenses was primarily due to higher compensation and stock-based compensation costs and increased D&O insurance premiums.
Net Loss: For the three months ended June 30, 2021, net loss, which included $1.0 million of non-cash expenses, was $5.2 million, or $0.08 per basic and diluted share. For the three months ended June 30, 2020, net loss, which included $1.8 million of non-cash expenses, was $4.1 million, or $0.09 per basic and diluted share.
Second Quarter Investor Call:

Please visit View Source and enter password CWBR, or
Go to www.cohbar.com and click on CohBar Q2 2021 Investor Presentation at the top of homepage.
For individuals participating in the Investor Call and Slide Presentation, please call into the conference audio and log into Zoom approximately 10 minutes prior to its start. Please note, no audio will be available through Zoom.

An audio replay of the call will be available beginning at 8:00 p.m. Eastern Time on August 10, 2021, through 11:59 p.m. Eastern Time on August 31, 2021. To access the recording please dial (844) 512-2921 in the U.S. and Canada, or (412) 317-6671 internationally, and reference Conference ID# 10159293. The audio recording along with the slide presentation will also be available at www.cohbar.com during the same period.