On May 13, 2021 Chemomab Therapeutics Ltd. (Nasdaq: CMMB), a clinical-stage biotech company focused on the discovery and development of innovative therapeutics for fibrosis-related diseases with high unmet need, reported financial and operating results for the first quarter ended March 31, 2021 and provided a business update (Press release, Anchiano Therapeutics, MAY 13, 2021, View Source [SID1234579844]).

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"This quarter has been truly exciting for Chemomab as we accessed the public markets and began to trade on the Nasdaq exchange, successfully closed on a private offering of $45 million, announced positive data from our Phase Ib study in NAFLD, and initiated a Phase IIa study in PSC." said Dr. Adi Mor, CEO of Chemomab. "We also started treating patients in our Phase IIa liver fibrosis trial with our subcutaneous formulation of CM-101 and will look to build upon our substantial progress and positive momentum in the coming quarters with the initiation of our additional planned Phase II study of CM-101 in Systemic Sclerosis (SSc). CM-101 is a very promising therapy with the potential to treat multiple severe and life-threatening inflammatory and fibrotic diseases. With a strong financial position, and our unique development track record we believe we are well positioned to continue to advance our pipeline and execute our important milestones this year."

First Quarter and Recent Highlights

·Announced positive results from its Phase Ib SPARK study evaluating CM-101 in nonalcoholic fatty liver disease (NAFLD) patients. The SPARK study was a double-blind, placebo-controlled study designed to evaluate the safety, tolerability and pharmacokinetic (PK) profile of CM-101 in NAFLD patients with normal liver function. In this study repeated CM-101 administrations were found to be safe and well-tolerated for both tested doses when administered as intravenous (IV) infusion or subcutaneous (SC) injection. No safety signals or unexpected adverse events were observed for CM-101 in either the IV or SC formulation and all reported adverse events were mild or moderate in intensity. Exploratory analysis of multiple pharmacodynamic parameters, including measurement of collagen turnover and fibrotic biomarkers, demonstrated that CM-101 treatment resulted in reduction of fibrotic and fibrogenesis markers compared to no change or slight elevation in the placebo treated group. In addition, there was a reduction in liver stiffness measured by FibroScanTM in the CM-101 treated group.

·Enrolled the first patient in its Phase IIa SPRING clinical trial of CM-101 for the treatment of patients with primary sclerosing cholangitis (PSC). The SPRING study is a multi-center, randomized, double-blind, placebo-controlled, multiple dose study designed to assess the mechanism of action, safety, pharmacokinetics and pharmacodynamic effects, as well as the antifibrotic effect of IV CM-101 in PSC patients. The trial will enroll and randomize up to 45 patients and is anticipated to complete enrollment by early 2022 with data expected in 1H 2022.

·Enrolled the first patient in its Phase IIa SPLASH clinical trial of CM-101 for the treatment of patients with nonalcoholic steatohepatitis (NASH). The SPLASH study is a multi-center, randomized, double-blind, placebo-controlled, multiple dose study designed to assess the mechanism of action, safety, pharmacokinetics and pharmacodynamic effects, as well as the anti-fibrotic effects of SC CM-101 in NASH patients with fibrosis stage F2-F3. The trial will enroll 40 patients and is anticipated to complete enrollment by the end of 2021 with data expected in 1H 2022.

·Completed a merger with Anchiano Therapeutics Ltd, and began trading on the Nasdaq Capital Market exchange under the symbol "CMMB" on March 17, 2021.

·Completed the successful pricing of a private placement of $45 million into the combined company led by new and certain existing investors including Cormorant Asset Management, OrbiMed, Peter Thiel, Christian Angermayer’s Presight Capital and Apeiron Investment Group, as well as other healthcare-focused and institutional investors.

·Strengthened its Board of Directors with the appointment of four new directors: Dr. Alan Moses, Dr. Claude Nicaise, Mr. Joel Maryles, and Mr. Neil Cohen. Dr. Stephen Squinto remains as Chairman of Chemomab’s Board, with Dr. Adi Mor and Dr. Nissim Darvish continuing in their roles as Directors.

Upcoming Milestones:

Chemomab is advancing in parallel three Phase 2 clinical trials with CM-101 in three distinct fibrotic indications; Systemic Sclerosis (SSc) is planned to be initiated by the end of 2021, and clinical readouts from the ongoing clinical trials in PSC and NASH are expected during 2022.

First Quarter 2021 Financial Highlights

·Cash and cash equivalents as of March 31, 2021 were $58.2 million which includes $45.5 million of gross proceeds from the private placement completed on March 22, 2021.

·Research and Development expenses for the three months ended March 31, 2021 were $1.2 million, compared to $1.6 million for the three months ended March 31, 2020. The decrease of $0.4 million was primarily related to a decrease in expenses to sub-contractors.

·General and administrative expenses were $0.5 million for the three months ended March 31, 2021, compared to $0.1 million for the three months ended March 31, 2020. The increase of $0.4 million was primarily related to merger related expenses.

·Net loss for the three months ended March 31, 2021 and 2020 was $1.7 million.

On May 12, 2021 Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage biopharmaceutical company that is genetically engineering red blood cells to create an entirely new class of cellular medicines called Red Cell Therapeutics, reported the publication of preclinical data in the peer-reviewed journal Nature Communications, for its lead artificial antigen-presenting (aAPC) cell program, RTX-321, for the potential treatment of human papillomavirus (HPV) 16-positive cancers (Press release, Rubius Therapeutics, MAY 12, 2021, View Source [SID1234584705]). RTX-321 is an allogeneic, off-the-shelf Red Cell Therapeutic product candidate that is engineered as an aAPC with a dual mechanism of action: to boost HPV 16-specific CD8+ T cell responses and promote broad stimulation of both innate and adaptive immune responses. Rubius Therapeutics is currently enrolling patients with persistent, recurrent, or metastatic, unresectable, HPV 16-positive cancers, including cervical cancer, head and neck squamous cell carcinoma (HNSCC) and anal cancer, in a Phase 1 clinical trial.

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The paper entitled "Engineered red blood cells as an off-the-shelf allogeneic anti-tumor therapeutic" highlights preclinical findings demonstrating that the surrogate model of RTX-321 induced a target antigen-specific immune response, epitope spreading, memory formation as well as broad immune stimulation. This suggests that in patients, an effective immune response could be generated against multiple HPV antigens, and potentially enable the patient’s own immune system to remember a cancer’s identity, which could lead to long-term protection from tumor recurrence. The paper is available here: View Source

"These preclinical findings support the potential of RTX-321 as an effective antigen-specific therapy for advanced HPV 16-positive cancers, where few treatment options exist in the metastatic setting for this group of patients. Our publication describes an elegant mechanism of action for RTX-321, combining antigen-specific responses with the addition of broad immune stimulation," said Laurence Turka, M.D., chief scientific officer of Rubius Therapeutics. "We are very excited about the potential of this investigational therapy and plan to share initial clinical results from our Phase 1 clinical trial in the first quarter of 2022."

For additional background on the paper, see accompanying article on Rubius Therapeutics’ RED PLATFORM from the authors in Nature’s Behind the Paper Channel, here.

About HPV 16-Positive Cancers
Human papillomavirus (HPV) 16 is associated with approximately 70 percent of cervical cancers, approximately 40 percent of head and neck squamous cell carcinoma (HNSCC) arising in the oropharynx, approximately 25-40 percent of HNSCC arising in other locations and approximately 80-85 percent of anal cancers. A critical need remains for better treatment options for advanced HPV 16-associated cancers. The prognosis remains poor for patients with metastatic disease with few treatment options beyond the first-line setting.

About the RTX-321 Clinical Trial
Rubius Therapeutics is enrolling patients in a Phase 1 open-label, multicenter, monotherapy dose escalation, first-in-human study of RTX-321 for the treatment of patients that are HLA-A*02:01-positive with persistent, recurrent, or metastatic, unresectable, HPV 16-positive cancers, including unresectable cervical cancer (squamous, adeno, or adenosquamous histology), head and neck squamous cell carcinoma (including of the nasal and oral cavities, larynx, hypopharynx, nasopharynx, and oropharynx) and squamous cell cancer of the anal canal that is not amenable to curative therapy. The purpose of the trial is to determine the safety and tolerability, recommended Phase 2 dose and pharmacology, and antitumor activity of RTX-321. For more information about the Phase 1 clinical trial of RTX-321, please visit clinicaltrials.gov (NCT04672980).

About RTX-321
RTX-321 is an allogeneic, off-the-shelf aAPC therapy product candidate that is engineered to induce a tumor-specific immune response by expanding antigen-specific T cells. RTX-321 expresses hundreds of thousands of copies of an HPV peptide antigen bound to major histocompatibility complex (MHC) class I proteins, the costimulatory molecule 4-1BBL and the cytokine IL-12 on the cell surface to mimic human T cell-APC interactions.

On May 12, 2021 Panbela Therapeutics, Inc. (Nasdaq: PBLA), a clinical stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with cancer reported a business update and reports financial results for the quarter ended March 31, 2021 (Press release, Panbela Therapeutics, MAY 12, 2021, View Source [SID1234583759]). Management is hosting an earnings call today at 4:30 p.m. ET.

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The first quarter 2021 was marked by meaningful clinical progress.

Q1 and Recent Highlights

Abstract accepted with poster presentation at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting June 4-8, 2021.
Partial clinical hold lifted from the company’s Phase 1 first-line study of SBP-101.
Research agreement entered into with Johns Hopkins University School of Medicine; Preclinical studies underway.
As previously announced, in April the U.S. Food and Drug Administration (FDA) lifted the partial clinical hold on the company’s Phase 1 first-line study of SBP-101 when used in combination with standard of care agents gemcitabine and nab-paclitaxel for treatment of patients with metastatic pancreatic ductal adenocarcinoma. The company has agreed to include in the design of future studies the exclusion of patients with a history of retinopathy or at risk of retinal detachment and scheduled periodic ophthalmologic monitoring for all patients, and in future dose-finding studies screening for retinal toxicity will be included.

"Year to date, we have focused on advancing SBP-101 in its first indication and exploring the broader potential of polyamine metabolic inhibition," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer. "With the hold lifted, we are excited to move the pancreatic cancer program forward. Additionally, the research initiated with Johns Hopkins will help to inform development outside of pancreatic cancer as well as potentially in combination with a checkpoint inhibitor."

Based on interim data from our Phase I trial, SBP-101 demonstrated a 62% objective response rate in combination with gemcitabine & abraxane (G&A); more than double the historical standard of care for metastatic pancreatic cancer with G&A.

We believe SBP-101 has the potential to expand into other cancers with known elevated levels of polyamine metabolism.

Upcoming Milestones

Public release of additional data from phase 1 trial – ASCO (Free ASCO Whitepaper) Annual Meeting June 4-8, 2021
Initiation of randomized phase 2 study mid-year
Public release of preclinical data across tumors outside of pancreatic cancer 2H’21
First Quarter ended March 31, 2021 Financial Results

General and administrative expenses were $1.1 million in the first quarter of 2021, compared to $0.5 million in the first quarter of 2020. The change in the quarter is primarily associated with increased headcount and other increased costs associated with maintaining our common stock on the Nasdaq Capital Market.

Research and development expenses were $1.1 million in the first quarter of 2021, compared to $0.6 million in the first quarter of 2020. The change in quarter is due primarily to higher manufacturing costs in preparation for future clinical trials.

Net loss was $2.3 million, or $0.23 per diluted share, compared to a net loss of $1.8 million, or $0.27 per diluted share, in the first quarter of 2020.

Total cash was $8.1 million as of March 31, 2021. Total current assets were $8.8 million and current liabilities were $1.3 million as of the same date. The company had no debt as of March 31, 2021.

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About SBP-101

SBP-101 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. The molecule has shown signals of tumor growth inhibition in clinical studies of US and Australian metastatic pancreatic cancer patients, suggesting potential complementary activity with an existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, SBP-101 has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Recently observed serious visual adverse events are being 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 current Panbela sponsored clinical trial provides support for continued evaluation of SBP-101 in a randomized clinical trial. For more information, please visit View Source .

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

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

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