On May 9, 2023 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a leader in the field of conditionally activated, localized biologics, reported first quarter 2023 financial results and provided a business update (Press release, CytomX Therapeutics, MAY 9, 2023, View Source [SID1234631237]).

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"As we entered 2023, CytomX continued the advancement of our diversified portfolio of innovative Probody therapeutic candidates for the treatment of cancer while ensuring disciplined resource allocation," said Sean McCarthy, D.Phil., chief executive officer and chairman of CytomX Therapeutics. "We remain intensely focused on execution towards key inflection points in our therapeutic pipeline including continued progress with CX-904 in Phase 1 and advancing our next-generation candidates, CX-2051 and CX-801, towards IND filings. Our scientific depth in conditional activation and biologics localization positions the company at the forefront of potential breakthroughs with potent biologic modalities such as ADCs, T-Cell engagers and cytokines. Additionally, our scientific leadership has attracted valued new relationships with Regeneron and Moderna, allowing us to maintain balance sheet strength. Moreover, with more than fifteen internal and partnered therapeutic programs, we are well positioned to deliver meaningful treatments to patients over time."

Dr. McCarthy continued, "I’m also thrilled to announce the promotion of Dr. Marcia Belvin to the position of chief scientific officer. Marcia’s skill and experience has played a central role in the translation of key learnings from our first wave of clinical programs into the next generation Probody therapeutic candidates that comprise our current pipeline. My colleagues and I look forward to Marcia’s continued success as we pursue our shared vision of building a long-term company that brings new and differentiated treatments to people with cancer."

First Quarter Business Highlights and Recent Developments

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Continued progress in Phase 1 dose escalation for CX-904, T-cell-engaging bispecific (TCB) targeted to EGFRxCD3 – CX-904 is a conditionally activated TCB designed to target the epidermal growth factor receptor (EGFR) on cancer cells and the CD3 receptor on T cells within the tumor microenvironment. CX-904 is partnered with Amgen and is being evaluated by CytomX in an ongoing Phase 1 study in patients with advanced solid tumors. The first patient was dosed in May 2022 and the dose escalation portion of the study continues to advance. The primary goal of dose escalation is to assess safety and reach dose levels and exposures by the end of 2023 at which enrollment into backfill cohorts in certain EGFR positive tumors can begin. In 2024, a key milestone will be the selection of the recommended Phase 2 dose and decision to potentially initiate expansion cohorts. This program is partnered with Amgen in a global co-development alliance.

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IND enabling activities on track for filings for CX-2051 (EpCAM-directed ADC) and CX-801 (Interferon alpha-2b) in the second half of 2023 – CytomX has selected previously validated anti-cancer targets, EpCAM and IFNa2b, respectively, that have been limited in their potential due to systemic toxicities, for its next generation molecules. The molecular design of CX-2051 and CX-801 has incorporated CytomX’s platform expertise and clinical learnings to optimize predicted therapeutic index in order to potentially broaden the clinical utility of these promising targets through tumor localized conditional activation.

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BMS advancement of BMS-986288 to Phase 2 – In February 2023, BMS published pipeline updates that included moving the Anti-CTLA-4 non-fucosylated Probody, BMS-986288, from Phase 1 to Phase 2. BMS prioritized the BMS-986288 Probody program over the other two molecules in its CTLA-4 pipeline – the Probody, BMS-986249, and the antibody, BMS-986218. Clinical evaluation of BMS-986288 is ongoing.

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CD71-Targeting strategies under evaluation – In March 2023, AbbVie notified CytomX that it would not advance the CD71-targeting, conditionally activated ADC, CX-2029, into additional clinical studies and provided notice of termination of the 2016 CD71 License and Collaboration Agreement. CytomX is assessing acquisition of full rights to CX-2029 whilst also evaluating potential next generation CD71 targeting strategies.

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Clinical candidate milestone achievement in Astellas TCB collaboration – In January 2023, Astellas nominated a collaboration clinical candidate, the first Probody TCB molecule to progress in the alliance, triggering a $5 million dollar milestone payment to CytomX. CytomX and Astellas are collaborating on additional conditionally activated TCB programs, and CytomX is eligible to receive future preclinical, clinical, and commercial milestones. CytomX retains a cost share and co-commercialization option on a select number of targets.

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Marcia Belvin, Ph.D. promoted to Chief Scientific Officer – In March 2023, Marcia Belvin, Ph.D. was promoted to the position of chief scientific officer. Dr Belvin has served as the company’s senior vice president, head of research since April 2020 and joined the company as head of oncology research in 2018. Prior to joining CytomX, Dr. Belvin held roles of increasing responsibility at Genentech, where for over 13 years, she led multiple preclinical pipeline teams and oversaw programs in cancer signaling, cancer metabolism, and cancer immunology. Dr. Belvin began her career at Exelixis, where she managed teams responsible for preclinical pipeline discovery within the oncology and inflammation portfolios.

Priorities for 2023

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CX-904 (EGFRxCD3): Continue patient enrollment and dose escalation in ongoing Phase 1 study and initiate backfill cohorts by the end of 2023
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File 2 New INDs (wholly-owned): CX-801 (IFNa2b) and CX-2051 (EpCAM) projected in the second half of 2023
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Next-Generation CTLA-4 Program: Continued clinical progress for BMS-986288
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CX-2029 (CD71): Determine next steps for CD71 program, including CX-2029
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Collaborations: Continuation of drug discovery activities within R&D alliances including those with our newest collaborators, Regeneron and Moderna

First Quarter 2023 Financial Results

Cash, cash equivalents and investments totaled $204.5 million as of March 31, 2023, compared to $193.7 million as of December 31, 2022. Operating cash received in the first quarter included a $35.0 million upfront payment received as a result of the execution of the Moderna collaboration agreement in the fourth quarter of 2022 and a $5.0 million milestone earned under the Astellas collaboration.

Total revenue was $23.5 million for the three months ended March 31, 2023, compared to $9.0 million for the corresponding period in 2022. The increase in revenue was driven primarily by a higher percentage of completion versus the corresponding period in 2022 for projects under the company’s projects with Bristol Myers Squibb, the milestone earned under the agreement with Astellas, and revenue recognition of the remaining deferred revenue upon termination of the AbbVie CD71 Agreement.

Research and development expenses decreased by $9.4 million during the three months ended March 31, 2023 to $21.2 million, compared to $30.6 million for the corresponding period in 2022. The decrease in research and development expenses for the three months ended March 31, 2023 compared to the corresponding period of 2022 was primarily due to a decrease in personnel related expenses, as well as winding down of laboratory contract services and clinical study activities related to the CX-2009 and CX-2029 programs, partially offset by an increase in laboratory contract services related to IND enabling activities.

General and administrative expenses decreased by $2.6 million during the three months ending March 31, 2023 to $8.0 million, compared to $10.5 million for the corresponding period in 2022. General and administrative expenses decreased primarily due to a decrease in personnel related expenses due to the workforce reduction in 2022 and patent related legal expenses.

On May 9, 2023 Neurix SA reported that CYTEA|BIO SAS has selected our BlastomaBrain platform to perform its pre-clinical validation in anticipation of its IND filing with the FDA (Press release, CYTEA BIO, MAY 9, 2023, View Source [SID1234631236]). CYTEA is developing a novel immunotherapy approach to address an urgent unmet medical need for glioblastoma patients. Glioblastoma models developed by Neurix using patient-derived glioblastoma samples offer the opportunity to test personalised therapies for the disease.

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"We are very proud of the fact that our platform has been selected for such an exciting new therapy that offers to transform the lives of patients, and provide an alternative to animal testing for an FDA IND dossier" said Neurix’s CEO Nikolay Zhukovsky.

CYTEA|BIO’s CEO, Alan Cookson said: CYTEA|BIO relishes this opportunity to employ the BlastomaBrainTM platform to evaluate the safety and efficacy of our PinTM immunotherapy treatment for GBM. Pre-clinical platforms that offer more predictive outcomes to animal studies are an important innovation for the pharmaceutical industry.

On May 9, 2023 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported a business update and provides financial results for the first quarter ended March 31, 2023 (Press release, Cogent Biosciences, MAY 9, 2023, View Source [SID1234631235]).

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"Cogent continued to make important progress in the first quarter," said Andrew Robbins, the Company’s President and Chief Executive Officer. "With three active clinical programs, 2023 will be a year rich in data and milestones that build upon our scientific achievements. We look forward to sharing lead-in data of bezuclastinib plus sunitinib from our PEAK trial in GIST patients at ASCO (Free ASCO Whitepaper). We are pleased with the progress in our SUMMIT trial in NonAdvSM patients and remain on track to report initial clinical data in the second half of 2023. Additionally, we are planning a robust clinical update from approximately 30 patients with AdvSM from our APEX Part 1 study, also in the second half of the year. With these catalysts, a preclinical pipeline with best-in-class potential and a cash runway into 2025, we believe we are well positioned to build on our momentum to bring important therapies to patients fighting rare, genetically driven diseases."

Recent Business Highlights

Initiated Part 2 of the ongoing Phase 2 APEX trial evaluating bezuclastinib in Advanced Systemic Mastocytosis (AdvSM) following completion of enrollment in Part 1.
Part 2 will enroll approximately 65 patients treated at a once-daily 150 mg optimized dose and if successful, is designed to support regulatory submission. Enrollment is expected to be complete by the end of 2024.
Presented preclinical data from the company’s ErbB2 and FGFR2 research programs at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting.
Preclinical data described a novel EGFR-sparing, brain-penetrant ErbB2 inhibitor with potency across key oncogenic ErbB2 mutations.
In vivo characterization showed a novel, selective, reversible FGFR2 inhibitor that has potency against molecular brake and gatekeeper mutations, with potential advantages over covalent approaches.
Received approvals from European regulatory authorities to initiate the Phase 2 SUMMIT trial in patients with nonadvanced systemic mastocytosis (NonAdvSM) and rapidly activating clinical trial sites across major countries in the European Union.
FDA granted Orphan Drug Designation for bezuclastinib for the treatment of mastocytosis, including systemic mastocytosis (SM) with an estimated 30,000 SM cases diagnosed annually in the United States.
Upcoming Milestones

Present a poster highlighting updated safety and tolerability data from Part 1 of PEAK, the Company’s ongoing Phase 3 trial evaluating bezuclastinib in combination with sunitinib in patients with Gastrointestinal Stromal Tumors (GIST), at the 2023 ASCO (Free ASCO Whitepaper) Annual Meeting taking place June 2-6, 2023, in Chicago, IL.
Title: Safety, Pharmacokinetics (PK), and Clinical Activity of Bezuclastinib + Sunitinib in Previously-Treated Gastrointestinal Stromal Tumor (GIST): Results from Part 1 of the Phase 3 Peak Study
Presenter: Dr. William Tap
Abstract number: 11537
Date and time: 6/3/2023, 1:15-4:15pm CT
Session: Sarcoma
Data will include measures of safety and tolerability, along with clinical activity, including duration of therapy and objective response rate. Cogent will host an investor webcast to discuss these results. Details will be provided closer to the event.
Present initial clinical data from SUMMIT, a randomized, double-blind, placebo-controlled, global, multicenter, Phase 2 clinical trial of bezuclastinib in patients with NonAdvSM in the second half of 2023. Data will include safety/tolerability, pharmacokinetics and measures of clinical activity.
Present clinical data from approximately 30 patients in Part 1 of APEX in patients with AdvSM at a scientific meeting in the second half of 2023.
First Quarter 2023 Financial Results

Cash and Cash Equivalents: As of March 31, 2023, cash, cash equivalents and marketable securities were $220.3 million as compared to $259.3 million as of December 31, 2022. Total cash spent was $39.0 million, including a one-time payment of $5.1 million related to performance-based bonus compensation. Based on its current plans, the company expects its existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into 2025.

R&D Expenses: Research and development expenses were $36.0 million for the first quarter of 2023 compared to $25.5 million for the first quarter of 2022. The increase was primarily due to costs associated with the on-going APEX, SUMMIT and PEAK clinical trials and costs related to development of the research pipeline. R&D expenses include non-cash stock compensation expense of $3.0 million for the first quarter of 2023 as compared to $1.9 million for the first quarter of 2022.

G&A Expenses: General and administrative expenses were $7.2 million for the first quarter of 2023 compared to $5.9 million for the first quarter of 2022. The increase was primarily due to the growth of the organization. G&A expenses include non-cash stock compensation expense of $2.9 million for the first quarter of 2023 as compared to $2.3 million for the first quarter of 2022.

Net Loss: Net loss was $38.6 million for the first quarter of 2023 compared to a net loss of $30.6 million for the first quarter of 2022.

On May 9, 2023 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address inflammatory, cancer and liver diseases, reported that it will initiate an open-label Phase 2 exploratory trial to assess the efficacy and safety of Namodenoson in the treatment of patients with pancreatic cancer who have received at least one previous systemic therapy (Press release, Can-Fite BioPharma, MAY 9, 2023, View Source [SID1234631234]).

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Patients will receive Namodenoson at a dose of 25 mg orally, twice daily. Efficacy endpoints will include objective response, progression-free survival, duration of response, disease control (defined as an objective response or stable disease), and overall survival. Safety will be assessed as well. The study will be conducted by Dr. Salomon Stemmer, an oncology key opinion leader and Professor at the Institute of Oncology, Rabin Medical Center, Israel.

Namodenoson is currently under a pivotal Phase 3 study for the treatment of advanced liver cancer and has completely cleared cancer in an advanced liver cancer patient who remains cancer-free 6 years after starting treatment.

"This relatively small exploratory trial is designed as an open-label study, enabling us to assess the potential efficacy of Namodenoson in pancreatic cancer. We’ve seen Namodenoson’s potent anti-cancer effects in treating people with liver cancer and believe that based on recent pre-clinical results, our drug may be equally effective in pancreatic cancer, an indication in need of more effective treatments," stated Can-Fite CEO Dr. Pnina Fishman.

The highest incidence rates for pancreatic cancer are in Asia, Europe, and North America. According to the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), in 2020, an estimated 496,000 people were diagnosed with pancreatic cancer globally and an estimated 466,000 died from the disease. The 5-year survival rate for people with pancreatic cancer in the U.S. is 11%. Acumen Research estimates the global pancreatic cancer therapeutics market was valued at approximately $3.6 billion in 2021 and is projected to grow to approximately $6.6 billion by 2030.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson was evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

On May 9, 2023 Lisata Therapeutics, Inc. (Nasdaq: LSTA) ("Lisata" or the "Company"), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, reported a business update and provides financial results for the three months ended March 31, 2023 (Press release, Lisata Therapeutics, MAY 9, 2023, View Source [SID1234631233]).

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"During the first quarter of 2023, our team continued its focus on the advancement of multiple ongoing and planned clinical studies evaluating LSTA1, our lead investigational product," stated David J. Mazzo, Ph.D., Chief Executive Officer of Lisata. "We expect to report progress on several of these activities over the coming months and quarters. Just recently, we, along with our research partner, WARPNINE, announced the treatment of the first patient in the iLSTA Trial in Australia evaluating LSTA1 in combination with standard-of-care chemotherapy and immunotherapy as a first-line treatment in locally advanced non-resectable pancreatic ductal adenocarcinoma. We are hopeful that this and our other trials will continue to show the potential of LSTA1 in combination with corresponding standards-of-care as well as with emerging treatment modalities, such as immunotherapies, as an effective treatment against various solid tumors.

Dr. Mazzo continued, "Our overarching goal is to report meaningful clinical data to benefit patients and to support our development pipeline in the most expeditious manner possible. Positive data should also bring value to shareholders and encourage additional partnering opportunities."

Development Portfolio Update

LSTA1 as a treatment for solid tumor cancers in combination with other anti-cancer agents

LSTA1 is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to penetrate solid tumors more effectively. LSTA1 actuates this active transport system in a tumor-specific manner, resulting in systemically co-administered anti-cancer drugs more efficiently penetrating and accumulating in the tumor, while normal tissues are not expected to be affected. LSTA1 also has the potential to modify the tumor microenvironment, with the objective of making tumors more susceptible to immunotherapies. We and our collaborators have amassed significant non-clinical data demonstrating enhanced delivery of a range of existing and emerging anti-cancer therapies, including chemotherapeutics, immunotherapies, and RNA-based therapeutics. To date, LSTA1 has also demonstrated favorable safety, tolerability and activity in completed and ongoing clinical trials designed to test its ability to enhance delivery of standard-of-care chemotherapy for pancreatic cancer. Currently, LSTA1 is the subject of Phase 1b/2a and 2b clinical studies being conducted globally in metastatic pancreatic ductal adenocarcinoma in combination with each of the two standards-of-care for this disease. The combination of LSTA1 with corresponding standards-of-care in other solid tumor indications is planned for clinical study commencing before the end of the second quarter of 2023.

HONEDRA (LSTA12) for the treatment of critical limb ischemia ("CLI")

•HONEDRA is the Company’s SAKIGAKE-designated product candidate for the treatment of CLI and Buerger’s disease in Japan, which is now in the pre-consultation phase of the registration process

with the Pharmaceuticals and Medical Devices Agency ("PMDA") in Japan. Data from the follow-up of all patients completed in the registration-eligible clinical trial in Japan have been compiled and are the subject of discussions with the PMDA as part of the Japanese regulatory pre-consultation process and in preparation for the formal clinical consultation meetings which precede a Japanese new drug application. To date, the PMDA has provided advice on how to prepare for the formal consultation meeting. Concomitantly, the Company has reinforced its efforts to secure a partner to complete the remaining steps of development/registration and potential commercialization in Japan through the engagement of an advisory firm specializing in Japanese partnerships.

LSTA201 for the treatment of diabetic kidney disease ("DKD")

The Company initiated a Phase 1b, open-label, proof-of-concept trial evaluating LSTA201, a CD34+ regenerative cell therapy investigational product, for intra-renal artery administration in patients with DKD. Preclinical studies in kidney disease and injury models have demonstrated that protecting or replenishing the microcirculation of the kidney may result in improved kidney function. A key criterion for continued development of LSTA201 was determined, a priori, to be the ability of LSTA201 to regenerate kidney function as indicated by increased Glomerular Filtration Rate. The Company treated the first patient in the LSTA201 proof-of-concept study in April 2022 and completed treatment for all six subjects during the third quarter of 2022. Top line results, which were reported on February 6, 2023, showed that LSTA201 was safe and well-tolerated by patients with no serious adverse events related to the therapy. However, the study did not demonstrate a consistent improvement in kidney function among patients. Nevertheless, the Company, based on the encouragement of the study’s principal investigator/key opinion leader, believes there may still be potential for use of CD34+ cell therapy for the treatment of DKD. However, it is expected that further development of LSTA201 would require significantly larger studies and capital investment. Thus, LSTA201 development will only be continued if a strategic partner that can contribute the necessary capital for future development is identified.

First Quarter 2023 Financial Highlights

Research and development expenses were approximately $3.2 million for the three months ended March 31, 2023, compared to $3.3 million for the three months ended March 31, 2022, representing a decrease of $0.1 million or 3.2%. This was primarily due to expenses associated with our XOWNA Phase 2b study (the FREEDOM Trial) in the prior year, partially offset by study start up activities in the current year associated with the planned LSTA1 Phase 2 proof-of-concept basket trial in various solid tumors in combination with the corresponding standards of care, enrollment activities for the LSTA1 Phase 2b ASCEND study and chemistry, manufacturing and control activities for LSTA1.

General and administrative expenses were approximately $3.7 million for the three months ended March 31, 2023, compared to $3.3 million for the three months ended March 31, 2022, representing an increase of $0.3 million or 9.8%. This was primarily due to the addition of one employee acquired through the Company’s merger with Cend Therapeutics, Inc., an increase in external legal fees and an increase in accounting and tax-related fees.
Overall, net losses were $6.2 million for the three months ended March 31, 2023, compared to $4.2 million for the three months ended March 31, 2022.

Balance Sheet Highlights

As of March 31, 2023, the Company had cash, cash equivalents and marketable securities of approximately $61.1 million. These figures do not include the recently announced $2.2 million in non-dilutive funding received as an approved participant of the Technology Business Tax Certificate Transfer Program (the "Program") sponsored by the New Jersey Economic Development Authority, which will be recorded in the second quarter of 2023. The Program enables qualifying New Jersey-based biotechnology or technology companies to sell a percentage of their New Jersey net operating losses and research and development tax credits to unrelated qualifying corporations. The Company is confident that its projected capital will fund its operations into the first quarter of 2026 encompassing anticipated data milestones from all of its ongoing and planned clinical trials.

Conference Call Information

Lisata will hold a live conference call today, May 9, 2023, at 4:30 p.m. Eastern time to discuss financial results, provide a business update and answer questions.
Those wishing to participate must register for the conference call by way of the following link: https://register.vevent.com/register/BI0ea7c01f84154b159cddca509bd575cd. Registered participants will receive an email containing conference call details with dial-in options. To avoid delays, we encourage participants to dial into the conference call fifteen minutes ahead of the scheduled start time.
A live webcast of the call will also be accessible under the Investors & News section of Lisata’s website and will be available for replay beginning two hours after the conclusion of the call for 12 months.