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Scholar Rock Reports Full Year 2022 Financial Results and Highlights Business Progress

On March 7, 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 full year ended December 31, 2022 (Press release, Scholar Rock, MAR 7, 2023, View Source [SID1234628255]).

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"2023 is off to a great start as we continue to build upon our momentum from last year. Importantly, we are on track to complete the enrollment of our pivotal SAPPHIRE trial of apitegromab and are looking forward to providing 36-month extension data from the Phase 2 TOPAZ trial of apitegromab in the coming months. Additionally, we look forward to sharing biomarker and clinical updates throughout the year from the SRK-181 Phase 1 DRAGON trial in patients with relapsed and/or refractory cancers resistant to checkpoint inhibitor therapy," said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock.

Company Highlights and Upcoming Milestones

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

Continue to progress and complete enrollment of Phase 3 SAPPHIRE clinical trial. The last patient is expected to be enrolled in SAPPHIRE in 2023, with the top-line data readout expected in 2024. If successful, and if approved, the company expects to initiate a commercial product launch in 2025. SAPPHIRE is a randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating apitegromab in patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan designations to apitegromab for the treatment of SMA.
36-month extension data from Phase 2 TOPAZ trial to be presented in mid-2023. As of December 31, 2022, approximately 90 percent of patients who entered the trial’s long-term extension study (51/57) remained on study. At the Cure SMA Research & Clinical Care Meeting in June 2022, the company presented 24-month extension data from the TOPAZ trial which demonstrated sustained gains in Hammersmith Functional Motor Scale Expanded (HFMSE), increased Revised Upper Limb Module (RULM) scores, and positive trends in quality-of-life data for nonambulatory patients with Types 2 and 3 SMA receiving an SMN therapy.
SRK-181 is an investigational selective inhibitor of latent TGFβ-1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.

Continue to progress Phase 1 DRAGON trial. The company expects to provide biomarker and clinical updates from the DRAGON proof-of-concept trial throughout 2023. In November 2022, Scholar Rock presented data from DRAGON at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) Annual Meeting that showed SRK-181 continued to be generally well tolerated with early indications of efficacy (as of the data cut-off date of August 29, 2022).
Encore data from DRAGON will be presented at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress in Paris, France on March 7 at 11:05-12:30 p.m. EST. The presentation is titled "Safety and efficacy results of SRK-181 (a latent TGFβ1 inhibitor) from a phase 1 trial (DRAGON trial)."
Corporate

Appointed Tracey M. Sacco as Chief Commercial Officer. In February 2023, Ms. Sacco joined Scholar Rock to build and lead all commercial functions, including sales, marketing, market access, and commercial operations. She will help the company prepare for the potential commercial launch of apitegromab, if approved. Ms. Sacco brings extensive biopharmaceutical industry experience, including commercial strategy and global launch experience of rare disease products.
Full Year 2022 Financial Results

For the full year ended December 31, 2022, net loss was $134.5 million or $2.26 per share compared to a net loss of $131.8 million or $3.59 per share for the full year ended December 31, 2021.

Revenue was $33.2 million for the year ended December 31, 2022, compared to $18.8 million for the year ended December 31, 2021. Revenue recognized during both periods was associated with the Gilead collaboration which ended in 2022.
Research and development expense was $124.4 million for the year ended December 31, 2022, compared to $108.5 million for the year ended December 31, 2021. The increase was primarily attributable to costs associated with running the company’s Phase 3 SAPPHIRE pivotal trial for apitegromab in SMA, including clinical trials costs as well as costs associated with employee compensation and benefits.
General and administrative expense was $43.1 million for the year ended December 31, 2022, compared to $40.3 million for the year ended December 31, 2021. The increase was due to an increase in employee compensation and benefits, primarily associated with equity-based compensation and professional fees.
As of December 31, 2022, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $315 million, which is expected to fund the Company’s anticipated operating and capital expenditure requirements into 2025.
"As Scholar Rock works to develop transformative therapies for patients with our highly differentiated platform, we are entering 2023 with an experienced team, a strong balance sheet, and a clear line of sight to important anticipated milestones, all of which are expected to help advance the next phase of growth for the company," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock.

Panbela Announces First Patient Enrolled in South Korea for ASPIRE Trial Studying Ivospemin (SBP-101)

On March 7, 2023 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, reported its first patient enrolled in South Korea for its ASPIRE global clinical trial in the first-line treatment of metastatic pancreatic cancer (Press release, Panbela Therapeutics, MAR 7, 2023, View Source [SID1234628252]). ASPIRE is a global randomized, double-blind placebo-controlled clinical trial to evaluate ivospemin in combination with gemcitabine and nab-Paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma. Detailed information on the trial can be located at View Source

"We’re really excited to continue the Country and site initiation for the ASPIRE trial as we aim to have the full complement of sites on board by the middle of this year," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela. "Having South Korean enrollment underway is highly encouraging as we continue to ramp up the trial. We’re targeting the interim analysis in early 2024."

With approximately 95 sites planned throughout the United States, Europe, Australia, and South Korea, Panbela is continuing to focus on site initiation and enrollment to ultimately deliver a more effective treatment for pancreatic cancer, a deadly disease with few treatment options.

About Panbela’s Pipeline

The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of catalysts with programs ranging from pre-clinical to registration studies.

Ivospemin (SBP-101)

Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nabpaclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapyrelated adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI profile observed in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial.

Flynpovi

Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increase polyamine export and catabolism. In a Phase 3 clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase 3 trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), Flynpovi showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety profile for Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP.

CPP-1X

CPP-1X (eflornithine) is being developed as a single agent tablet or high dose power sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase 1 or Phase 2 investigator-initiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity.

Nuvectis Pharma, Inc. Reports Fiscal Year 2022 Financial Results and Business Highlights

On March 7, 2023 Nuvectis Pharma, Inc (NASDAQ: NVCT) ("Nuvectis" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of innovative precision medicines for the treatment of serious conditions of unmet medical need in oncology, reported its financial results for the fiscal year 2022 and provided an update on recent business progress (Press release, Nuvectis Pharma, MAR 7, 2023, View Source [SID1234628251]).

Ron Bentsur, Chairman and Chief Executive Officer of Nuvectis commented: "2022 was a very busy year for Nuvectis with significant progress made on both development programs. For NXP800, we continued to advance the Phase 1a dose escalation study in patients with various advanced solid tumors, and in December we announced that the Food and Drug Administration ("FDA") granted Fast Track Designation status to the NXP800 development program in platinum resistant, ARID1a-mutated ovarian carcinoma. Moreover, we recently announced that the European Network of Gynecological Oncology Trial Groups ("ENGOT") and the GOG Foundation, Inc. ("GOG-F"), the world’s premier gynecology oncology clinical trials consortia, will lead the upcoming Phase 1b clinical trial in ARID1a-mutated ovarian carcinoma, a disease comprised mostly of clear cell and endometrioid histologies. Lastly, the discovery and optimization work performed at the Institute of Cancer Research ("ICR") was showcased in the prestigious New Drugs on the Horizon session of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR"), and additional in-vivo preclinical data was generated that provides further potential development opportunities for NXP800."

Mr. Bentsur continued: "For NXP900, we are very pleased with the progress made toward submission of an IND and commencement of the Phase 1 clinical trial, expected in 1H2023. NXP900’s unique mechanism of action translated into substantial single-agent activity in several in-vivo xenograft models. Additionally, publications in the scientific literature outlined opportunities to potentially reverse resistance to osimertinib (Tagrisso) in non-small cell lung cancer and enzalutamide (Xtandi) in metastatic, castration resistant prostate cancer, in combination with these agents, highlighting the importance of NXP900 key targets, YES1 and SRC kinases, in these disease settings

Mr. Bentsur concluded, "On the corporate front, we completed the initial public offering in February 2022 and a private placement in July 2022, providing us with sufficient working capital to continue the momentum for both programs into the second half of 2024. As we continue to make significant progress with NXP800 and NXP900, our exciting precision medicine drug candidates, we believe that 2023 is shaping up to be a potentially transformational year for Nuvectis

Full Year 2022 Financial Results

Cash, cash equivalents, and short-term investments were $20.0 million as of December 31, 2022, compared to $5.7 million as of December 31, 2021. The increase of $14.3 million was primarily a result of the Company’s initial public offering in February 2022 and the private placement in July 2022.

The Company’s net loss was $19.1 million for the year ended December 31, 2022, compared to $12.9 million for the year ended December 31, 2021, an increase of $6.2 million. Net loss for the 2022 fiscal year included $1.7 million in non-cash expenses and $6.1 million in one-time expenses, primarily related to milestone, fee payments and one-time development costs in connection with NXP800 and NXP900.

Research and development expenses were $13.2 million for the year ended December 31, 2022, compared to $9.5 million for the year ended December 31, 2021, an increase of $3.7 million. The increase in research and development expenses is primarily attributed to one-time expenses related to milestone and fee payments in connection with the license agreements, and an increase in preclinical and clinical development costs. Research and development expenses for the 2022 fiscal year included $0.9 million in non-cash expenses.

General and administrative expenses were $ 6.0 million for the year ended December 31, 2022, compared to $3.3 million for the year ended December 31, 2021, an increase of $2.7 million. The increase in general and administrative expenses is primarily attributed to costs associated with operating as a public company following our 2022 initial public offering. General and administrative expenses for the 2022 fiscal year also included $0.8 million in non-cash expenses.

Monopar to Present at the 35th Annual Roth Conference

On March 7, 2023 Monopar Therapeutics Inc. (Nasdaq: MNPR), a clinical-stage biopharmaceutical company focused on developing proprietary therapeutics designed to extend life or improve the quality of life for cancer patients, reported that Chandler Robinson, MD, Monopar’s Chief Executive Officer, will participate in a fireside chat at the 35th Annual Roth Conference (Press release, Monopar Therapeutics, MAR 7, 2023, View Source [SID1234628250]).

Presentation Details:

Date: March 13, 2023
Time: 12:30-1:00 pm Pacific Time
Location: The Ritz Carlton, Laguna Niguel in Dana Point, California

View Source

Corporate Presentation

On March 7, 2023 Milestone Pharmaceuticals presenting its corporate presentation (Presentation, Milestone Pharmaceuticals, MAR 7, 2023, View Source [SID1234628249]).