On November 10, 2022 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 its financial results for the three and nine months ended September 30, 2022 and provided a business update (Press release, Caladrius Biosciences, NOV 10, 2022, View Source [SID1234623718]).

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"We are excited to report our first quarter as Lisata Therapeutics following the merger of Caladrius Biosciences and Cend Therapeutics," stated David J. Mazzo, Ph.D., Chief Executive Officer of Lisata. "The team has made tremendous progress over the past few months and now, as Lisata, we are building an enduring pharmaceutical company for the treatment of diseases with significant unmet medical needs. As such, our primary focus is the advancement of our clinical development pipeline of candidates targeting oncology and ischemic disease indications. LSTA1, our lead investigational product candidate from the CendR Platform, is the subject of multiple planned and ongoing clinical trials being conducted globally in a variety of solid tumor types and in combination with several chemotherapy and immunotherapy anti-cancer regimens. We believe that LSTA1 has the potential to become an integral part of a revised standard-of-care therapy for many difficult to treat cancers. Recent guidance from the U.S. Food and Drug Administration has given us further direction on what would be required for registration. We have discussed this guidance with our development partners and we are planning adjustments to protocols, including the modification and expansion of ongoing studies.
Dr. Mazzo continued, "Overall, we’re invigorated by the promise of our platform technologies and pipeline of product and partnering opportunities and look forward to providing updates on our progress in the coming months."
Development Portfolio Update
LSTA1 as a treatment for solid tumor cancers in combination with other anti-cancer agents
LSTA1, formerly known as CEND-1, is an investigational drug that actuates the CendR active transport mechanism while also having the potential to modify the tumor microenvironment ("TME") and make is less immunosuppressive. It is targeted to tumor vasculature by its affinity for alpha-v, beta-3 and beta-5 integrins that are selectively expressed in tumor vasculature, but not healthy tissue. LSTA1 is a specific cyclic internalizing RGD ("iRGD") peptide that, once bound to these integrins, is cleaved by proteases expressed in tumors to release a peptide fragment, called a CendR fragment, which binds to a second receptor, called neuropilin-1, to activate a novel uptake pathway that allows anticancer drugs to more selectively penetrate solid tumors. The ability of LSTA1 and iRGD peptides to modify the TME to enhance delivery and efficacy of co-administered drugs has been demonstrated in models of a range of solid tumors with the results from Lisata, collaborators and research groups around the world having been the subject of over 200 scientific publications. Lisata and its collaborators have also amassed significant non-clinical data demonstrating enhanced delivery of a range of emerging anti-cancer therapies, including immunotherapies and RNA-based therapeutics. Clinically, LSTA1 has demonstrated favorable safety, tolerability, and activity in clinical trials to enhance delivery of standard-of-care chemotherapy for pancreatic cancer. Lisata is exploring the potential of LSTA1 to enable a variety of treatment modalities to treat a range of solid tumors more effectively. Currently, LSTA1 is the subject of Phase 1b/2a and 2b clinical studies

being conducted globally in various solid tumors, including metastatic pancreatic ductal adenocarcinoma, in combination with a variety of anti-cancer regimens. The combination of LSTA1 with corresponding standards-of-care in other solid tumor indications is planned for clinical study in the first half of 2023.
HONEDRA (LSTA12, aka CLBS12) 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 being reviewed by the PMDA, after which the PMDA is expected to provide important perspective to be considered in preparation for the formal consultation meetings which precede the Japanese new drug application. If successful in the pre-consultation process, Lisata expects formal clinical consultation to occur by mid-year 2023. Concomitantly, the Company is focusing its efforts to secure a Japanese partner to complete the remaining steps to produce registration in Japan.
XOWNA (LSTA16, aka CLBS16) for the treatment of coronary microvascular dysfunction ("CMD")
XOWNA is an experimental regenerative therapy for the treatment of CMD. It was the subject of a positive Phase 2a study (the "ESCaPE-CMD trial") reported in 2020 and is currently being evaluated in a U.S. Phase 2b study (the "FREEDOM Trial"). The FREEDOM Trial was originally designed as a 105-patient double-blind, randomized, placebo-controlled trial to further evaluate the efficacy and safety of intracoronary delivery of autologous CD34+ cells (XOWNA) in subjects with CMD and without obstructive coronary artery disease and was expected to complete enrollment in approximately 12 months. As previously disclosed, enrollment in the FREEDOM Trial initially proceeded as planned with the first patient treated in January 2021; however, the impact of the COVID-19 pandemic in the U.S., coupled with supply chain issues associated with the catheters used for diagnosis of CMD and/or administration of XOWNA, as well as with a contrast agent typically used in many catheter laboratories, have made and continue to make enrollment much slower than originally predicted and challenging to accelerate. As a result, the Company announced that enrollment in the FREEDOM Trial had been suspended and that it intended to conduct an interim analysis of the data from not less than the first 20 patients enrolled using the 6-month follow-up data to evaluate the efficacy and safety of XOWNA in subjects with CMD. Following the analysis of results of the FREEDOM Trial subjects completing 6-month follow-up along with Key Opinion Leaders’ input, the Company’s board of directors determined that execution of a redesigned FREEDOM-like trial would be the appropriate next step, but the cost of such a trial would be prohibitively expensive to undergo alone and without a strategic partner. Accordingly, the Company’s board of directors concluded that XOWNA development will only be continued if a strategic partner that can contribute the necessary capital for a redesigned trial is identified and secured.
LSTA201 (aka CLBS201) for the treatment of diabetic kidney disease ("DKD")
Progressive kidney failure is associated with attrition of the microcirculation of the kidney. Preclinical studies in kidney disease and injury models have demonstrated that protection or replenishment of the microcirculation results in improved kidney function. Based on these observations, 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. Patients selected for the study are in the pre-dialysis stage of kidney disease and exhibit rapidly progressing stage 3b disease. The protocol provides for a cohort of six patients overseen by an independent Data Safety Monitoring Board with the objective of determining the tolerance of intra-renal cell therapy injection in DKD patients as well as the ability of LSTA201 to regenerate kidney function. A key read-out of data will occur at the 6-month follow-up visit for all patients. The Company treated the first patient in April 2022 and completed treatment for all six subjects during the third quarter of 2022. Top-line data is anticipated from all subjects by the first quarter of 2023.
Third Quarter 2022 Financial Highlights
Research and development expenses were approximately $3.4 million for the three months ended September 30, 2022, compared to $4.1 million for the three months ended September 30, 2021, representing a decrease of $0.7 million or 18.1%. This was primarily due to a decrease in expenses associated with our XOWNA Phase 2b study (the FREEDOM Trial) as a result of the suspension in enrollment which commenced in the second quarter of 2022 and study close out activities in the third quarter of 2022, a decrease in expenses associated with HONEDRA in Japan related to study close out costs partially offset by the addition of Chemistry, Manufacturing, and Controls ("CMC") activities for LSTA1, and enrollment activities for Australasian Gastrointestinal Trials Group ("AGITG") ASCEND study. Research and development in both periods related to:
•expenses associated with our XOWNA Phase 2b study (the FREEDOM Trial);

•expenses associated with our registration-eligible study for HONEDRA in CLI in Japan as well as corresponding regulatory discussions and support expenses;
•expenses associated with the preparation of our filing of an Investigational New Drug Application, as well as study execution expenses for the clinical study of LSTA201 for treatment of DKD, a Phase 1b, open-label, proof-of-concept trial which includes six subjects in total;
•expenses associated with the addition of CMC activities for LSTA1, enrollment activities for the LSTA1 Phase 2b ASCEND study and preparatory activities associated with the design of a planned LSTA1 proof-of-concept basket trial in various solid tumors and in combination with the corresponding standards of care.
General and administrative expenses, which focus on general corporate related activities, were $3.9 million for the three months ended September 30, 2022, compared to $2.8 million for the three months ended September 30, 2021, representing an increase of 39%. This increase was primarily due to an increase in equity expense as a result of performance stock unit vesting, a one-time merger option assumption expense and departing board member restricted stock unit vesting in addition to an increase in expenses associated with our annual stockholder meeting and the merger.
Net losses were $37.4 million for the three months ended September 30, 2022, compared to $6.9 million for the three months ended September 30, 2021.
Balance Sheet Highlights
As of September 30, 2022, the Company had cash, cash equivalents and marketable securities of approximately $75.5 million.
Conference Call Information
Lisata will hold a live conference call today, November 10, 2022, at 4:30 p.m. Eastern time to discuss financial results, provide a business update and answer questions.
The Company is utilizing a new conference call service. Those wishing to participate must register for the conference call by way of the following link: https://register.vevent.com/register/BIb133855aa28547eda1ae5e1e1da0559c. Registered participants will receive an email containing conference call details for 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 (available at this link: View Source) and will be available for replay beginning two hours after the conclusion of the call for 12 months.

On November 10, 2022 Fresh Tracks Therapeutics, Inc. (the "Company" or "Fresh Tracks") (Nasdaq: FRTX), a clinical-stage pharmaceutical company striving to transform patient lives by developing innovative and differentiated prescription therapeutics for the treatment of autoimmune, inflammatory, and other debilitating diseases, reported financial results for the third quarter ended September 30, 2022 and provided a corporate update (Press release, Vical, NOV 10, 2022, View Source [SID1234623717]).

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"This year has been a transformational period for our company, as we executed a fundamental shift in strategy via the acquisition and development of a pipeline of new chemical entities targeting novel mechanisms of action for the treatment of a broad array of autoimmune and inflammatory diseases. To better align with our new strategic vision and plans, we recently rebranded the Company to Fresh Tracks Therapeutics," commented Robert Brown, Chief Executive Officer of Fresh Tracks. "This rebrand highlights our commitment to develop targeted, science-driven therapies, such as FRTX-02, our potential first-in-class oral DYRK1A inhibitor that is currently being evaluated in a Phase 1 clinical trial. We are pleased with the progress in this first-in-human study and remain on track to report topline results by early 2023."
Research and Development Highlights
FRTX-02: a potential first-in-class DYRK1A inhibitor for the treatment of autoimmune and inflammatory diseases
•Continue to progress the Phase 1 clinical trial of FRTX-02, which is a randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of FRTX-02 capsules in both healthy subjects and patients with atopic dermatitis ("AD"). Part 1A of the study is a single ascending dose ("SAD") assessment, which enrolled a total of 56 healthy volunteers at one study center across seven cohorts, each of which included six subjects receiving a single dose of FRTX-02 and two subjects receiving a placebo. Part 1B of the study is a multiple ascending dose ("MAD") assessment of FRTX-02 or placebo in healthy adult subjects. In the MAD assessment, up to 33 healthy volunteers are being enrolled across three cohorts, each of which includes nine subjects who receive FRTX-02 and two subjects who receive a placebo once daily for 14 days. After completing Part 1, the Company intends to initiate Part 2 of the study, which will compare FRTX-02 to placebo in patients with moderate-to-severe AD over 28 days of treatment and will include a preliminary assessment of efficacy. Additional information on this clinical trial can be found on www.clinicaltrials.gov under identifier NCT05382819.
•Completed all SAD cohorts and initiated MAD part of the study in September 2022.
•On track to report topline results from the SAD and MAD parts of the Phase 1 study by early 2023.
•Progressing FRTX-02 nonclinical development program in parallel with the ongoing Phase 1 study.
FRTX-10: a covalent Stimulator of Interferon Genes (STING) inhibitor candidate for the potential treatment of autoimmune, inflammatory, and rare genetic diseases
•Preclinical development activities for FRTX-10 are underway.

Next-Generation Kinase Inhibitors: a cutting-edge platform with the potential to produce treatments for autoimmune, inflammatory, and other debilitating diseases
•The Company is planning to identify and characterize both brain penetrant and non-brain penetrant new chemical entities from this next-generation kinase inhibitor platform that specifically inhibit LRRK2, TTK, and CLK kinases.
•A number of these drug candidates have the potential to penetrate the blood-brain barrier, presenting an opportunity to address neuroinflammatory conditions of high unmet need, such as Down Syndrome, Alzheimer’s Disease, and Parkinson’s Disease, while other peripherally acting novel LRRK2, TTK, and CLK kinase inhibitors could be developed in additional therapeutic areas within autoimmunity, inflammation, and oncology.
Recent Corporate Highlights
Scientific Advisory Board
In September 2022, Fresh Tracks announced the formation of a Scientific Advisory Board ("SAB") consisting of five renowned scientists and clinicians with deep experience across immunology and inflammation, including Drs. Kate Fitzgerald, Bernard Khor, Pui Lee, Peter Nigrovic, and Bridget Wagner. The SAB members are offering their expertise and guidance to the Company as it develops its novel pipeline of differentiated therapies for the treatment of autoimmune and inflammatory diseases, including FRTX-02, FRTX-10, and next-generation kinase inhibitor platform.
Sofpironium Bromide
In the second quarter of 2022, Fresh Tracks entered into an asset purchase agreement ("APA") with Botanix SB Inc. ("Botanix"). Under the terms of the APA, Botanix acquired and assumed control of rights, title, and interests to assets primarily related to the proprietary compound sofpironium bromide in exchange for an upfront fee, near-term regulatory milestone payments, and sales-based milestone payments, as well as tiered earnout payments on net sales of sofpironium bromide gel. In connection with the sale of sofpironium bromide, Fresh Tracks and Botanix entered into a transition services agreement ("TSA") whereby Fresh Tracks is providing consulting services to Botanix as an independent contractor in support of and through filing and potential approval of a U.S. new drug application ("NDA") for sofpironium bromide gel, 15%.
In September 2022, Botanix submitted an NDA for sofpironium bromide gel, 15% to the U.S. Food and Drug Administration ("FDA"). If the FDA accepts the NDA for filing, Fresh Tracks will receive a milestone payment of $2.0 million from Botanix. On November 10, 2022, Fresh Tracks paid its former licensor of sofpironium bromide $1.0 million in cash in lieu of issuing $1.0 million in shares of its common stock as originally provided for in the license agreement.
Third Quarter 2022 Financial Results
The Company reported cash and cash equivalents of $11.3 million as of September 30, 2022, compared to $26.9 million as of December 31, 2021. The Company expects its cash and cash equivalents as of September 30, 2022, combined with $6.0 million from expected near-term payments under the APA with Botanix, will be sufficient to fund its operations for at least the next 12 months.
Revenue was $0.5 million for the third quarter of 2022, compared to $0.1 million for the third quarter of 2021. Revenue for the three months ended September 30, 2022 consisted of contract revenue recognized under the APA and TSA with Botanix, while revenue for the three months ended September 30, 2021 was driven by royalty revenue earned on a percentage of net sales of ECCLOCK (sofpironium bromide gel, 5%) in Japan under the Kaken agreement. Contract revenue for the three months ended September 30, 2022 consisted of fees for consulting services the Company provided to Botanix under the TSA of $0.4 million and sublicense income under the APA of $0.1 million.
Research and development expenses were $3.6 million for the third quarter of 2022, compared to $10.2 million for the third quarter of 2021, which decrease was driven primarily by lower clinical expenses related to sofpironium bromide, upfront license expense incurred in the third quarter of 2021 under the license agreement with Voronoi Inc., and lower regulatory and compliance fees, partially offset by increased clinical costs for FRTX-02. Throughout 2021, the Company was executing its U.S. Phase 3 pivotal clinical program for sofpironium bromide gel, 15%, which concluded in the fourth quarter of 2021. During the second quarter of 2022, the Company initiated its Phase 1 clinical trial for FRTX-02 and began incurring research and development expenses related to the clinical trial.

General and administrative expenses were $3.0 million for the third quarter of 2022, compared to $3.3 million for the third quarter of 2021. The decrease was primarily related to lower expenses incurred in the third quarter of 2022 associated with compensation and other administrative fees.
The Company’s net loss was $6.0 million for the third quarter of 2022 compared to $13.3 million for the third quarter of 2021.
Conference Call and Webcast Information
Fresh Tracks’ management will host a conference call today at 4:30 p.m. EDT to discuss the financial results and recent corporate developments. The dial-in number for the conference call is 1-877-407-3982 for domestic participants and 1-201-493-6780 for international participants, with Conference ID #: 13732536. A live webcast of the conference call can be accessed at (View Source;tp_key=49e1233eb2) or through the Investors section of Fresh Tracks’ website at View Source A replay will be available on this website shortly after conclusion of the event for approximately 90 days.

On November 10, 2022 GENFIT (Nasdaq and Euronext: GNFT), a late-stage biopharmaceutical company dedicated to improving the lives of patients with severe chronic liver diseases, reported its cash position as of September 30, 2022 and revenue for the first nine months of 2022 (Press release, Genfit, NOV 10, 2022, View Source [SID1234623716]).

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Cash Position

As of September 30, 2022, the Company’s cash and cash equivalents amounted to €163.6 million compared with €91.5 million a year earlier.

As of June 30, 2022, cash and cash equivalents totaled €209.1 million.

The decrease in cash and cash equivalents between June 30, 2022, and September 30, 2022, takes into account notably the initial consideration of CHF40.0 million for the acquisition of Versantis AG, which GENFIT paid at the closing of this acquisition on September 29, 2022. It also includes the consolidation of the cash and cash equivalents of Versantis for the first time, which amounted to €5.1 million.

Revenue

Revenue2 for the first nine months of 2022 amounted to €14,129 thousand compared to €20 thousand for the same period in 2021.

The increase in revenue is mainly attributable to the partial recognition of the €40.0 million deferred income, which was accounted for in accordance with IFRS 15 following the conclusion of the strategic licensing and collaboration agreement with Ipsen on December 17, 2021. The revenue recognized out of this deferred income amounted to €13,050 thousand for the first nine months of 2022.

On November 10, 2022 Bellicum Pharmaceuticals, Inc. (Nasdaq: BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported financial results for the third quarter 2022 and provided an operational update (Press release, Bellicum Pharmaceuticals, NOV 10, 2022, View Source [SID1234623715]).

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"We are encouraged by the progress we are making in our patient enrollment efforts for the BPX-601 and BPX-603 trials despite a national fludarabine shortage, and are on track to present data updates for both programs in the first half of 2023," said Rick Fair, President and Chief Executive Officer. "We are also excited about new data from the Baylor College of Medicine supporting the potential benefit of CaspaCIDe in resolving the adverse events associated with cell therapies, and remain committed to expanding CaspaCIDe use through collaborations with other developers of cell therapies."

Program Highlights and Current Updates

BPX-601 GoCAR-T

Enrollment in the Phase 1/2 dose escalation clinical trial in patients with previously treated metastatic castration-resistant prostate cancer (mCRPC) is ongoing. The company expects to provide interim data on BPX-601 in the first quarter of 2023.
BPX-603 GoCAR-T

Enrollment is ongoing in the Phase 1/2 clinical trial for BPX-603 in patients with solid tumors that express human epidermal growth factor 2 (HER2), including breast, endometrial, ovarian, gastric, and colorectal cancers. The company expects to announce interim data from this trial in the first half of 2023.
Fludarabine Shortage

In May 2022, the FDA announced a shortage of fludarabine in the U.S. that is affecting the company’s clinical trial sites. Fludarabine is a chemotherapeutic agent used as part of a lymphodepletion regimen administered to patients prior to receiving CAR-T cells and is required as part of the protocols in the BPX-601 and BPX-603 Phase 1/2 trials. Some of the BPX-601 and BPX-603 trial sites have been unable to procure fludarabine, or have had restrictions placed on fludarabine use, which has affected enrollment and patient dosing at these trial sites. The company has begun to procure a fludarabine supply for patients at these sites and expects to receive fludarabine and resume dosing of patients at these sites in the fourth quarter of 2022.
CaspaCIDe (Inducible Caspase-9)

At the 54th annual meeting of the International Society of Paediatric Oncology (SIOP Congress September 28 to October 1, 2022), David Steffin, M.D., and colleagues from the Center for Cell and Gene Therapy at the Baylor College of Medicine reported initial findings from a study of GPC3 CAR-T cells co-expressing IL-15 and inducible caspase-9 in children with liver cancer. Infusion of these CAR-T cells into a 20-year-old hepatocellular carcinoma (HCC) patient led to robust lymphocyte expansion associated with grade 4 cytokine release syndrome not controlled by IL-6, IL-1 or tumor necrosis factor-alpha inhibition. The patient’s symptoms were rapidly relieved by administration of rimiducid to trigger inducible caspase-9. Anti-tumor activity in the primary liver lesion and lung metastases four weeks after CAR-T cell infusion was reported.
Financial Results for the Third Quarter 2022

Revenues: Bellicum reported revenue of $1.0 million for the three and nine months ended September 30, 2022, compared to $5.0 million and $5.7 million for the same periods in 2021. Revenue in 2022 was from licensing, and in 2021 from licensing and from sale of rimiducid for use in clinical trials.

R&D Expenses: Research and development expenses were $6.9 million and $16.4 million for the three and nine months ended September 30, 2022, respectively, compared to $6.3 million and $19.5 million for the three and nine months ended September 30, 2021, respectively. The increase for the quarter was primarily due to increased R&D headcount and associated personnel costs. At the end of the third quarter of 2022, Bellicum had 11 full time equivalent R&D employees, compared to 5 at the end of the third quarter of 2021. The decrease for the three quarters in 2022 compared to 2021, was due to continued reduction of expenses related to rivo-cel activities and patient enrollment delays in ongoing clinical trials.

G&A Expenses: General and administrative expenses were $1.3 million and $4.2 million for the three and nine months ended September 30, 2022, respectively, compared to $1.7 million and $5.5 million for the three and nine months ended September 30, 2021, respectively. The decrease in G&A expenses for the third quarter 2022 was primarily due to reduced personnel costs, share-based compensation expenses and depreciation expenses.

Loss from Operations: Bellicum reported a loss from operations of $7.2 million and $19.6 million for the three and nine months ended September 30, 2022, respectively, compared to a loss from operations of $3.0 and $19.8 million for the three and nine months ended September 30, 2021, respectively.

Net Income/Loss: Bellicum reported a net loss of $7.2 million for the three months ended September 30, 2022, and a net loss of $18.8 million for the nine months ended September 30, 2022, compared to a net income of $1.2 million and a net loss of $12.2 million for the three and nine months ended September 30, 2021, respectively.

Shares Outstanding: As of November 4, 2022, Bellicum had 8,613,527 shares of common stock and 452,000 shares of preferred stock outstanding. Each share of preferred stock is convertible into 10 shares of common stock.

Cash Position: Bellicum reported cash and cash equivalents totaling $28.8 million as of September 30, 2022, compared to cash, cash equivalents and restricted cash totaling $47.7 million as of December 31, 2021.

On November 10, 2022 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, reported its financial results for the quarter ended September 30, 2022 and provided a pipeline update (Press release, Moleculin, NOV 10, 2022, View Source [SID1234623714]). As previously announced, the Company will host its inaugural quarterly conference call and live audio webcast, today, November 10, 2022, at 5:00 PM ET (details below).

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"We have made fundamental progress across our pipeline this past quarter. Of note, we have three active Phase 1/2 clinical trials for Annamycin and patients treated thus far continue to demonstrate no evidence of cardiotoxicity. Additionally, we have gained valuable insight in all of our ongoing development programs and are well-positioned to successfully execute operationally through the remainder of the year. Looking ahead, as we continue delivering on our initiatives, I believe the fourth quarter is poised to be an exciting conclusion of a transformational year and foundational for what is to come," commented Walter Klemp, Chairman and Chief Executive Officer of Moleculin.

Recent Highlights

Opened enrollment in the Phase 1/2 Study of Annamycin in combination with cytarabine for the treatment of Acute Myeloid Leukemia ("AML") in Europe.
Initiated and began dosing in the Phase 2 portion of the U.S. Phase 1b/2 clinical trial evaluating Annamycin for the treatment of soft tissue sarcoma lung metastases ("STS lung mets").
An investigator-funded, second Phase 1b/2 clinical trial of Annamycin in STS lung mets in Europe was commenced and began dosing.
Continued expanding the safety profile of Annamycin with a third report from an independent cardiology expert assessing recent subjects. This assessment again confirmed that there was no evidence of cardiotoxicity associated with Annamycin. This brings the total to 42 subjects where no evidence of cardiotoxicity has been identified by an independent expert cardiologist.
Enrolled what is expected to be the final subject in the Phase 1 portion of a clinical trial of WP1066 being conducted in collaboration with Emory University for the treatment of pediatric brain tumors, including medulloblastoma and diffuse interstitial pontine glioma ("DIPG").
Concluded Phase 1a first-in-human clinical trial of WP1122 for the treatment of COVID-19 in the United Kingdom establishing a safe and tolerable dose in healthy volunteers.
Announced the selection of WP1096 (a compound in the WP1122 portfolio) as novel potential antiviral, by the National Institute of Allergy and Infectious Diseases ("NIAID"), part of the National Institutes of Health ("NIH"), for NIH-funded animal studies.
Received U.S. Food and Drug Administration ("FDA") Orphan Drug Designation of WP1122 for the treatment of Glioblastoma Multiforme ("GBM").
Pipeline Update

Next Generation Anthracycline – Annamycin

Annamycin is the Company’s next-generation anthracycline that has been designed to be non-cardiotoxic and has been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin (a commonly prescribed anthracycline), as well as demonstrating the ability to avoid the multidrug resistance mechanisms that typically limit the efficacy of doxorubicin and other currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory AML and STS lung metastases and the Company believes it may have the potential to treat additional indications.

STS Lung Mets

The Company recently initiated its Phase 2 portion of the U.S. Phase 1b/2 clinical trial evaluating Annamycin for the treatment of STS lung mets ("MB-107") and has begun enrollment and dosing.

In the Phase 1b portion of the study, 15 subjects were enrolled and treated per the protocol in four cohorts to determine the maximum tolerable dose and/or the RP2D. Each cohort had three subjects, except for the fourth cohort, which (per the protocol) was expanded to six subjects after a dose-limiting toxicity (DLT) occurred in a single subject. The Company concluded the Phase 1b portion after the fourth cohort of 390 mg/m2 was documented to be safe. Based on findings from the Phase 1b position of the trial, 360 mg/m2 demonstrated it may be tolerated by subjects initially, however continued treatment was deemed at risk to be delayed or interrupted due to adverse events (primarily myelosuppression, which is anticipated with high doses of anthracycline therapy), hence lowering the dose from 360 mg/m2 to 330 mg/m2 was contemplated in advance of beginning the Phase 2 expansion pending results from the first three subjects in the expansion phase. Adverse Events, primarily myelosuppression, in the first three subjects at 360 mg/m2 led the Company to lower the RP2D to 330 mg/m2 in October 2022, which the Company believes will enable continued treatment of subjects with fewer interruptions.

The Company expects to treat at least 25 subjects in this Phase 2 portion of the clinical trial. For more information about the Phase 1b/2 study evaluating Annamycin for the treatment of STS lung mets, please visit clinicaltrials.gov and reference identifier NCT04887298.

Additionally, the investigator-funded, second Phase 1/2b clinical trial of Annamycin for the treatment of STS lung mets was recently initiated. The grant-funded clinical trial is being led by Prof. Piotr Rutkowski, MD, PhD, Head of Department of Soft Tissue/Bone Sarcoma and Melanoma at the Maria Sklodowska-Curie National Research Institute of Oncology in Warsaw, Poland. The trial will use a dosing regimen of once weekly for three weeks in a 28-day cycle rather than once every 21 days as in the US trial. Two subjects have been dosed to date.

AML

Patient enrollment and screening has been initiated in the Company’s Phase 1/2 clinical trial in Poland evaluating Annamycin in combination with Cytarabine (also known as "Ara-C" and for which the combination of Annamycin and Ara-C is referred to as AnnAraC) for the treatment of subjects with AML who are refractory to or relapsed after induction therapy ("MB-106"). Ongoing efforts to open additional clinical sites in Poland and other European countries for the MB-106 clinical trial are underway.

Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of STS lung metastases, in addition to Orphan Drug and Fast Track Designation for the treatment of relapsed or refractory AML.

Upcoming Milestones Expectations

Q4 2022: Commence dosing and open additional clinical sites in Poland and other European countries in the Phase 1/2 study of Annamycin in combination with Ara-C in Acute Myeloid Leukemia.
Q1 2023: Report Phase 2 interim data from ongoing Phase 1b/2 study of Annamycin for the treatment of STS lung mets in the US and Europe.
Flagship Immune/Transcription Modulator – WP1066

WP1066 is designed to stimulate the immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (Tregs) while also inhibiting key oncogenic transcription factors, including p-STAT3 (phosphorylated signal transducer and activator of transcription 3), c-Myc (a cellular signal transducer named after a homologous avian virus called Myelocytomatosis) and HIF-1α (hypoxia inducible factor 1α). These transcription factors are widely sought targets that are believed to contribute to an increase in cell survival and proliferation, and the angiogenesis (coopting vasculature for blood supply), invasion, metastasis and inflammation associated with tumors. They may also play a role in the inability of immune checkpoint inhibitors to affect more resistant tumors.

Moleculin is in ongoing discussions with multiple academic institutions in separate programs evaluating WP1066 for the treatment of brain tumors. The Company expects the investigator-sponsored clinical trials or programs for the treatment of adult and pediatric brain tumor to commence in the first half of 2023.

Additionally, WP1066 is currently being evaluated in collaboration with Emory University for the treatment of pediatric brain tumors, including medulloblastoma and DIPG. In October 2022, the Emory trial enrolled the last subject in the last cohort at 8 mg/kg. Discussions are underway to explore WP1066 in combination with radiation on similar tumors in a Phase 2 clinical trial in the near future.

In April 2022, the Company received IND clearance from the FDA to conduct a Phase 1 study of WP1066 for the treatment of recurrent malignant glioma. Since that time, the Company has been evaluating strategic partnerships and collaborations to utilize this IND. WP1066 has received Orphan Drug Designation for the treatment of brain tumors, as well as Rare Pediatric Disease designation for three other pediatric indications. Additionally, WP1066 + radiation is being evaluated, pre-clinically, in the treatment of Glioblastoma Multiforme (GBM).

Upcoming Milestones Expectations

H1 2023: Commencement of investigator-sponsored clinical trials or programs for the treatment of adult and pediatric brain tumors.
Metabolism/Glycosylation Inhibitor – WP1122 Portfolio

WP1122 was developed as a 2-DG prodrug to provide a more favorable pharmacological profile and was found to have greater potency than 2-DG alone in preclinical models where tumor cells require higher glycolytic activity than normal cells. WP1122 has also been shown to have a greater antiviral effect than 2-DG against SARS-CoV-2 in MRC-5 cells in culture. The improved pharmacokinetic and pharmacodynamic (PK/PD) profile of WP1122 compared to 2-DG was noted in mice following oral dosing at equimolar (i.e., equivalent levels of 2-DG) doses. The WP1122 Portfolio includes numerous analogs, including WP1096, which has demonstrated the potential for broad antiviral capabilities in a wide range of in vitro models including multiple arenaviruses, Zika virus, and HIV.

Glioblastoma Multiforme

In September of 2022, Moleculin was granted Orphan Drug Designation of WP1122 for the treatment of GBM from the FDA. Additionally, based on preclinical data indicating the potential for WP1122 as a treatment for GBM, Moleculin received FDA clearance of its Investigational New Drug application to initiate a Phase 1 open label, single arm, dose escalation study of the safety, pharmacokinetics and efficacy of oral WP1122 in adult patients with GBM. The Company is currently evaluating opportunities for collaboration in clinical development.

COVID-19

In October 2022, the Company concluded its Phase 1a, first-in-human, randomized, double-blind, placebo-controlled, overlapping SAD and MAD clinical trial investigating the effects of WP1122 administered as an oral solution in healthy human volunteers. A safe and tolerable dose was established for WP1122 in this trial, which now provides a starting point for a range of potential Phase 2 clinical trials. Approximately 80 subjects were enrolled in the trial.

Upcoming Milestones Expectations

Identify investigators interested in initiating a Phase 1 open label, single arm, dose escalation study of the safety, pharmacokinetics and efficacy of oral WP1122 in adult patients with GBM.
Report preliminary findings of NIH-funded animal testing of WP1096 in the Tacaribe Arenavirus by the end of the first quarter 2023.
Summary of Financial Results for the Third Quarter 2022

Research and development (R&D) expense was $14.8 million and $11.2 million for the nine months ended September 30, 2022 and 2021, respectively. The increase of $3.6 million is mainly related to increased clinical trial activity as described above, a license termination fee, and costs related to manufacturing of additional drug product.

General and administrative expense was $8.7 million and $6.4 million for the nine months ended September 30, 2022 and 2021, respectively. The increase of $2.3 million is mainly related to an increase in regulatory and legal services, consulting and advisory fees.

For the nine months ended September 30, 2022 and 2021, the Company incurred net losses of $22.3 million and $13.1 million, respectively, and had net cash flows used in operating activities of $20.4 million and $14.7 million, respectively.

The Company ended the quarter with $50.4 million of cash. The Company believes that this cash is sufficient to meet its projected operating requirements, which include a forecasted increase over its current R&D rate of expenditures, beyond mid-2024.

Conference Call and Webcast

Moleculin management will host its inaugural quarterly conference call and live audio webcast for investors, analysts, and other interested parties today, Thursday, November 10, 2022, at 5:00 PM ET.

Interested participants and investors may access the conference call by dialing (877) 407-0832 (domestic) or (201) 689-8433 (international) and referencing the Moleculin Biotech Conference Call. The live webcast will be accessible on the Events page of the Investors section of the Moleculin website, moleculin.com, and will be archived for 90 days.