On March 7, 2023 NanOlogy LLC, a clinical-stage oncology company, reported issuance of a US patent titled: Local Delivery of Antineoplastic Particles in Combination with Systemic Delivery of Immunotherapeutic Agents for the Treatment of Cancer (Press release, NanOlogy, MAR 7, 2023, View Source;utm_medium=rss&utm_campaign=nanology-granted-a-us-patent-for-its-investigational-drugs-in-combination-with-immune-checkpoint-inhibitors-in-the-treatment-of-cancer [SID1234628261]). The patent (11,583,499) is valid in the US until November 25, 2038, and has corresponding filings globally. It adds to an extensive global intellectual property portfolio of more than 250 issued or filed patents covering composition, formulation, use, and technology.

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The new patent covers a method of treating cancer by intratumoral administration of large surface area microparticle (LSAM) taxanes in combination with systemic administration of immune checkpoint inhibitors (ICIs).

ICIs are often used in combination with other systemic cancer therapies particularly in the treatment of solid tumors to prime the immune system to increase response. A growing body of preclinical and clinical data demonstrate local and systemic immunomodulation following local delivery of NanOlogy’s LSAM investigational drugs together with outside research pointing to the value of treating the primary tumor in neoadjuvant, locally advanced, and metastatic settings to improve clinical outcomes.

"The Cancer Research Institute estimates more than 5000 clinical trials are underway worldwide with immune checkpoint inhibitors; many are combination trials with other systemic cancer agents in solid tumors." said Gere DiZerega, MD, NanOlogy Chief Medical Officer. "The goal is to identify which combinations can increase the immune response thereby increasing the overall solid tumor response to treatment. Unfortunately, a key challenge is the stacking of severe toxicities that can occur when multiple systemic therapies are used in combination."

"With this patent added to our portfolio, NanOlogy is in a strong position to further investigate clinically whether the combination of its local LSAM investigational drugs with systemic ICIs increases response without adding toxicity as suggested in our preclinical and early clinical research", added Marc Iacobucci, a Managing Director for NanOlogy. "Our goal is to improve cancer treatment and potentially offer a strategy for product lifecycle extension for ICIs when used in combination with our drugs."

In all, NanOlogy clinical programs have advanced tumor directed LSAM investigational drugs in multiple solid tumors including pancreas, lung, bladder, peritoneal, ovarian, prostate, and dermal cancers. More than 170 patients have been treated to date across its clinical trials with signals of tumor and immune response and no confirmed drug-related serious adverse events.

On March 7, 2023 Alaunos Therapeutics, Inc. ("Alaunos" or the "Company") (Nasdaq: TCRT), a clinical-stage oncology-focused cell therapy company, reported financial results for the fourth quarter and full year ended December 31, 2022, and provided a corporate update (Press release, Alaunos Therapeutics, MAR 7, 2023, View Source [SID1234628257]).

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"Last year was transformative as we demonstrated leadership in the field of TCR-T cell therapies for solid tumors," said Kevin S. Boyle, Sr., Chief Executive Officer of Alaunos. "Our early clinical data shows exciting proof of concept. As a result, we have seen significant interest from clinicians and patients in our TCR-T Library Phase 1/2 trial. In 2023, we aim to build on this momentum by increasing patient enrollment to generate additional, meaningful clinical data. Through an expansive IND amendment, we have made several critical enhancements to accelerate enrollment, improve manufacturing and expand our TCR Library. We are confident that these amendments will allow us to achieve Phase 2 readiness in 2023."

Recent Developments and Upcoming Milestones

Actively enrolling patients in TCR-T Library Phase 1/2 trial; expect to report interim data on multiple patients in mid-2023: The Company continues to actively enroll patients in its TCR-T Library Phase 1/2 trial targeting KRAS, TP53 and EGFR hotspot mutations across six solid tumor indications. In the fourth quarter of 2022, the Company successfully dosed its third patient in the trial. The Company also made multiple enhancements to the trial IND to increase the patient screening match rate and treatment flexibility. With these enhancements, the Company expects to enroll multiple patients in the months ahead.

Translational assessments provide additional encouraging results and proof-of-concept for Sleeping Beauty TCR-T cell therapy: The Company is employing cutting-edge techniques to evaluate persistence, memory, exhaustion and function of infused TCR-T cells from blood and tumor specimens over time in patients treated on the TCR-T Library clinical trial. The Company has observed limited T-cell exhaustion markers such as PD-1 as well as multiple memory subsets of TCR-T cells in circulation, including T memory stem cells, which have been reported to have the greatest proliferative capacity and ability to regenerate more effector T cells. Functional TCR-T cells have been observed infiltrating solid tumors. These encouraging data corroborate clinical data, highlight the promise of TCR-T cell therapy targeting driver mutations and support next generation TCR-T cell efforts underway at the Company.

Expanded TCR library with two new TCRs, doubling potential addressable market: In the fourth quarter of 2022, the Company submitted an IND amendment to the U.S. Food and Drug Administration (FDA) adding two new TCRs targeting frequent mutations and HLAs to its library, doubling the addressable market of its TCR-T program. This illustrates the Company’s two-pronged strategy to expand the library by adding more HLAs to the existing mutations with a TCR targeting KRAS-G12V and HLA-DRB1*07:01 and adding new mutations with a TCR targeting TP53-R273C and HLA-DPB1*04:02. In over 700 patients screened at MD Anderson Cancer Center with gastrointestinal or lung tumors, the Company has improved its match rate from 5% to 10%, including roughly one in five patients matching two TCRs in the current library providing a potential opportunity for multiplexed TCR-T cell therapy in the future.

Executing against multi-pronged strategy to optimize manufacturing process and improve treatment flexibility: The Company doubled its manufacturing capacity in 2022, allowing for production of two products simultaneously. In the fourth quarter of 2022 IND amendment, the manufacturing process was enhanced to move from fresh to cryopreserved cell product. The use of cryopreserved cell products has reduced manufacturing process time from 30 days to 26 days, a 13% decrease. This transition also allows for greater flexibility for patient scheduling and treatment and supports the Company’s expected increase in patient accruals in 2023. The Company will continue to execute its multi-pronged strategy to further optimize and reduce manufacturing time.

Expanding application of the hunTR TCR discovery platform to add additional TCRs to the TCR Library in 2023: In November 2022, the Company presented a poster at the SITC (Free SITC Whitepaper) annual meeting, highlighting its proprietary hunTR (human neoantigen T-cell Receptor) discovery platform. The poster highlighted the ability of hunTR to identify neoantigen-reactive TCRs, including shared mutations that could potentially be added to the Company’s TCR Library. The Company is working to expand the application of hunTR to screen for shared KRAS, TP53 and EGFR mutations to rapidly advance new TCR library candidates from the lab through to clinical translation. Alaunos expects to expand its TCR library to 15 TCRs by the end of 2023.

Completed $15.0 million follow-on public offering of common stock to extend cash runway: In November 2022, Alaunos raised $15.0 million in gross proceeds from a follow-on offering of 24.2 million shares of its common stock in a challenging financing environment for biotechs. Alaunos intends to use the net proceeds from the offering to fund the continued development of the product candidates in its pipeline, and for working capital, capital expenditures and general corporate purposes.

Fourth Quarter Ended December 31, 2022 Financial Results

Research and Development Expenses: Research and development expenses were $5.6 million for the fourth quarter of 2022, compared to $8.2 million for the fourth quarter of 2021, a decrease of approximately 32%. The decrease was primarily due to lower program-related costs of $1.3 million, a $0.9 million decrease in employee-related expenses due to reduced headcount and a $0.4 million decrease in consulting expenses.

General and Administrative Expenses: General and administrative expenses were $2.9 million for the fourth quarter of 2022, compared to $2.1 million for the fourth quarter of 2021, an increase of approximately 40%. The increase was primarily due to higher professional services fees of $0.8 million.

Net Loss: Net loss was $9.2 million, or $(0.04) per share, for the fourth quarter of 2022, compared to a net loss of $11.8 million, or $(0.05) per share, for the same period in 2021.

Cash, Cash Equivalents and Restricted Cash: As of December 31, 2022, Alaunos had approximately $53.0 million in cash balances, which includes restricted cash of approximately $13.9 million. Based on current operating plans, the Company expects its operating outflows, excluding debt service costs, for 2023 to be between approximately $35 million and $40 million. The Company expects to have sufficient cash resources to fund research and development programs and operations into the fourth quarter of 2023.

Full Year 2022 Financial Results

Collaboration Revenue: Collaboration revenue was $2.9 million for the full year ended December 31, 2022, compared to $0.4 million for the full year ended December 31, 2021. The increase in collaboration revenue was primarily due to the achievement of sales-based milestones of darinaparsin in Japan pursuant to a license and collaboration agreement with Solasia Pharma K.K., which was largely offset by a one-time corresponding $2.5 million Research and Development expense.

Research and Development Expenses: Research and development expenses were $25.0 million for the full year ended December 31, 2022, compared to $49.6 million for the full year ended December 31, 2021. The decrease in research and development expenses was primarily due to lower program-related costs of $9.7 million, a $15.5 million decrease in employee-related expenses due to reduced headcount, a $1.4 million decrease in consulting expenses and a $0.5 million decrease in lease expense. These decreases were partially offset by a one-time $2.5 million expense to MD Anderson under the terms of our License Agreement, which was associated with the achievement of sales-based milestones of darinaparsin in Japan.

General and Administrative Expenses: General and administrative expenses were $13.1 million for the full year ended December 31, 2022, compared to $27.6 million for the full year ended December 31, 2021. The decrease in general and administrative expenses was primarily due to lower employee-related expenses of $12.3 million as a result of reduced headcount, a $1.9 million decrease in professional services fees and a $0.2 million decrease in lease expense.

Net loss: Net loss was $37.7 million, or $(0.17) per share for the full year ended December 31, 2022, compared to a net loss of $78.8 million, or $(0.37) per share for the full year ended December 31, 2021.

Conference Call and Webcast

Alaunos will host a conference call and webcast today, March 7, 2023, at 8:30 a.m. ET. Participants may access the live webcast using the link here or by visiting the "Investors" section of the Alaunos website at www.alaunos.com. To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. The event will be archived on the Company’s website for approximately 30 days after the call.

On March 7, 2023 Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, reported fourth quarter and full year 2022 financial results and provided business updates (Press release, Seres Therapeutics, MAR 7, 2023, View Source [SID1234628256]).

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"We are eagerly looking forward to the upcoming potential FDA approval of SER-109, an investigational first-in-class oral microbiome therapeutic for recurrent C. difficile infection (rCDI). Pending FDA approval, we anticipate a commercial launch in the weeks following a favorable decision. With nearly 156,000 cases in the U.S. this year, rCDI places an extraordinary burden on patients and the healthcare system. If SER-109 is approved, we look forward to offering a novel oral therapeutic with a compelling safety and clinical profile, and capturing what we expect to be a sizable commercial opportunity," said Eric Shaff, President and Chief Executive Officer at Seres.

"We have also made meaningful progress advancing additional microbiome therapeutic candidates. Enrollment is ongoing in Cohort 2 of our Phase 1b study of SER-155, designed to prevent infections and/or GvHD in medically compromised individuals and we plan to report safety and pharmacological data from study Cohort 1 in May of this year," added Mr. Shaff.

Fourth Quarter and Recent Program and Corporate Updates

SER-109 Phase 3 program in recurrent C. difficile infection: SER-109, an investigational oral, live microbiome therapeutic, achieved its primary endpoint of superiority to placebo in reducing recurrence in patients with rCDI in the ECOSPOR III study. These results, initially published in the New England Journal of Medicine (NEJM), showed that 88% of SER-109 patients were free of recurrence compared to 60% on placebo at eight weeks. SER-109 was observed to be well tolerated with no drug-related serious adverse events in the Phase 3 study.

A SER-109 Biologics License Application has been accepted for Priority Review by the FDA based on its Breakthrough Therapy designation and a PDUFA target action date has been set for April 26, 2023.

In February 2023, Seres announced the publication of Phase 3 ECOSPOR IV trial results in JAMA Network Open and results were also shared at the IDWeek and American College of Gastroenterology (ACG) 2022 Annual Meetings. The SER-109 Phase 3 ECOSPOR IV trial enrolled 263 participants with a history of rCDI, including individuals that have experienced only a single recurrence of CDI. While comorbidities were prevalent among study participants, a well-tolerated safety profile was observed in ECOSPOR IV, consistent with the safety-profile observed in ECOSPOR III, with no treatment-related adverse events leading to withdrawal from the study. At the 8- and 24-week primary endpoints, 91.3% and 86.3% of patients remained free of recurrence, respectively, supporting positive data from the SER-109 placebo-controlled ECOSPOR III study. Similar results were observed in all subgroups, including those with a single recurrence of CDI. A separate publication from JAMA Network Open, based on secondary data analysis from the ECOSPOR III Phase 3 study, suggests that SER-109 administration may be associated with a rapid and steady improvement in Health-Related Quality Of Life (HRQOL), an important patient-reported outcome, compared with placebo through 8 weeks.

In October 2022, Seres announced the publication of additional ECOSPOR III results in the Journal of the American Medical Association (JAMA), highlighting that the clinical benefits of SER-109 in preventing recurrent CDI were apparent as early as two weeks post-treatment and sustained for at least 24 weeks.

On December 8, 2022, Seres held an investor event highlighting the anticipated SER-109 commercial opportunity and launch plans. In preparation for the potential FDA approval of SER-109, Seres and collaborator Nestlé Health Science have been executing pre-commercialization activities including appropriate market education and data dissemination to the medical community. In addition, activities are ongoing to engage payers in accordance with FDA guidance on pre-approval information exchange. At approval the existing Nestlé 150-person gastroenterology sales force will be deployed to educate this important specialty about SER-109. In order to complement Nestle’s current gastroenterology sales force, Nestlé has also hired a 20-person hospital selling team to profile institutions with the highest rCDI patient volume pre-launch.

The Company has SER-109 drug supply ready in anticipation of product approval and continues to make progress expanding commercial-scale production of SER-109 to prepare for anticipated future market demand. An ongoing agreement with Bacthera, a global leader in biopharmaceutical product manufacturing, is designed to increase longer-term SER-109 product supply and adds to existing manufacturing capabilities.

SER-155 Phase 1b clinical study: SER-155 is an investigational oral, rationally designed, cultivated microbiome therapeutic designed to reduce the incidence of gastrointestinal (GI) infections, bloodstream infections, and GvHD in patients receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT). The SER-155 Phase 1b study includes two cohorts with Cohort 1 designed to assess safety and drug pharmacology including the engraftment of drug bacteria in the gastrointestinal tract.

In Cohort 1, 13 subjects received SER-155 (i.e., safety population). The study’s Data and Safety Monitoring Board (DSMB) reviewed available Cohort 1 clinical data and cleared advancement to Cohort 2. The Company expects to report preliminary SER-155 Cohort 1 safety and pharmacology data in May 2023.

Study Cohort 2 incorporates a randomized, double-blinded placebo-controlled design to further evaluate safety and engraftment, as well as clinical outcomes, and will enroll approximately 60 subjects administered either SER-155 or placebo at a 1:1 ratio. The trial will assess the impact of SER-155 administration on infections and/or graft versus host disease (GvHD) in adult subjects who are undergoing allo-HSCT. The study is being conducted at leading medical institutions including Memorial Sloan Kettering Cancer Center, University of Chicago Medical Center, Harvard Medical School—Massachusetts General Hospital Cancer Center, and Mayo Clinic (Scottsdale, Arizona).

SER-155 is a consortium of bacterial species selected using Seres’ reverse translation discovery and development platform technologies. The design incorporates microbiome biomarker data from human clinical data and nonclinical human cell-based assays and in vivo disease models. The SER-155 composition aims to decrease the colonization and abundance of bacterial pathogens that can harbor antibiotic resistance and to enhance epithelial barrier integrity in the GI tract to both reduce the likelihood of pathogen translocation and decrease the incidence of bloodstream infections. Further, SER-155 is designed to modulate host immune responses to decrease GvHD.

Infection Protection research: The Company continues to conduct research to bring forward new microbiome therapeutics as a novel approach for Infection Protection for medically compromised individuals, including those with cancer neutropenia, cirrhosis, or solid organ transplant. Preclinical studies are evaluating the potential to reduce the abundance of targeted pathogens to decrease the potential for pathogen transmission, strengthen epithelial barriers to further reduce translocation and the frequency of bloodstream infections, and to modulate immune responses to tackle medical complications such as graft versus host disease GvHD. The Company plans to announce an additional Infection Protection clinical development program in 2023.

Ulcerative Colitis (UC) research: The Company previously reported clinical, microbiome and metabolomic data from the SER-287 Phase 2b study and the first cohort of its SER-301 Phase 1b study. Available data for these investigational microbiome therapeutics suggest that there may be an opportunity to utilize biomarker-based patient selection and stratification for future studies. Research activities remain ongoing to inform potential further development activities.

Financial Results

Seres reported a net loss of $250.2 million for the full year of 2022, as compared to a net loss of $65.6 million for the prior year. Seres reported a net loss of $68.8 million for the fourth quarter of 2022, as compared to a net loss of $50.0 million for the same period in 2021.

Research and development expenses for the fourth quarter of 2022 were $46.2 million, compared with $36.8 million for the same period in 2021. The research and development expenses were primarily related to Seres’ late-stage SER-109 clinical development program and manufacturing costs, as well as personnel expenses.

General and administrative expenses for the fourth quarter of 2022 were $22.4 million, compared with $20.5 million for the same period in 2021. General and administrative expenses were primarily related to personnel expenses, professional fees, including SER-109 commercial readiness and pre-launch expenses, and facility costs.

As of December 31, 2022, Seres had approximately $181.3 million in cash, cash equivalents and marketable securities as compared with $291.2 million at the end of 2021. Pending FDA approval of SER-109, the Company anticipates receiving a $125 milestone payment from Nestlé Health Science.

Conference Call Information

Seres’ management will host a conference call today, March 7, 2023, at 8:30 a.m. ET. To access the conference call, please dial 800-715-9871 (domestic) or 646-307-1963 (international) and reference Conference ID 4218669. To join the live webcast, please visit the "Investors and News" section of the Seres website at www.serestherapeutics.com.

A webcast replay will be available on the Seres website beginning approximately two hours after the event and will be archived for at least 21 days.

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.

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

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"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.