On May 11, 2022 Cyclacel Pharmaceuticals, Inc. (NASDAQ: CYCC, NASDAQ: CYCCP; "Cyclacel" or the "Company"), a biopharmaceutical company developing innovative medicines based on cancer cell biology, reported first quarter 2022 financial results and provided a business update (Press release, Cyclacel, MAY 11, 2022, View Source [SID1234614218]).

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"We are pleased to report another productive quarter for Cyclacel, which included continued expansion of our three, registration-directed, clinical trials and publication of research findings supporting our drug development strategy," said Spiro Rombotis, Chief Executive Officer of Cyclacel. "Oral fadraciclib, our CDK2/9 drug candidate, is proving to be well tolerated in 065-101, our Phase 1/2 solid tumor and lymphoma study, having reached dose level 5 in the dose escalation stage which provides for daily dosing over 4 out of 4 weeks. In our PLK1 program, we have dosed the first patients in the streamlined Phase 1/2 trial of CYC140 for the treatment of solid tumors and lymphomas. We have optimized the properties of CYC140 to fit its apoptosis-driven mechanism, including short half-life and differentiated structural and biological properties, compared to other PLK1 inhibitors in development. We therefore believe CYC140 has the potential to demonstrate activity across a wide range of solid tumors, as a single agent and in combinations."

"A growing body of preclinical research supports the clinical development plan of fadraciclib. In April, we announced publication of research from The University of Texas MD Anderson Cancer Center highlighting fadraciclib’s antileukemic activity in CLL. Fadraciclib treatment resulted in suppression of MCL1, a key target protein. In addition, synergy of fadraciclib in combination with venetoclax was observed against primary CLL cell lines, including those with 17p deletion. With funding estimated through mid-2023, we are continuing to execute on our clinical development plan. We look forward to presenting initial fadraciclib clinical data in solid tumors and lymphomas, in the coming weeks, determining the recommended Phase 2 dose and entering proof of concept stage in the second half of 2022."

Key Highlights

·Fadraciclib 065-101 Phase 1/2 study in advanced solid tumors: Phase 1 dose escalation has reached dose level 5 (100mg given twice a day for 5 days for 4 weeks in a 4-week cycle) with a favorable patient safety profile and appropriate pharmacokinetic data observed thus far. The study is enrolling at four sites with several additional sites planning to join the proof-of-concept stage of this registration-directed study in 2H 2022. The Phase 2 part includes seven histologically defined cohorts thought to be sensitive to the drug’s mechanism: breast, colorectal (including KRAS mutant), endometrial/uterine, hepatobiliary, ovarian cancers and lymphomas. The study also includes an eighth basket cohort which will enroll patients regardless of histology with biomarkers relevant to the drug’s mechanism, including MCL1, MYC and/or cyclin E amplified.

·Fadraciclib 065-102 Phase 1/2 study in patients with leukemias or myelodysplastic syndromes. This study is now enrolling at City of Hope and MD Anderson Cancer Center and is treating patients at dose level one. Once the recommended Phase 2 dose (RP2D) for single-agent, oral fadraciclib is determined, the study will enter into proof-of-concept, cohort stage, where fadraciclib will be administered, both as a single agent and in combinations, to patients in up to seven cohorts relevant to the drug’s mechanism of action and informed by the clinical activity of fadraciclib in previous studies. Single-agent cohorts will include patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) who have an inadequate response or have progressed on venetoclax combinations with hypomethylating agent (HMA) or low dose Ara C and relapsed/refractory AML or MDS patients with FLT3, KIT or MAPK pathways (including N and K RAS, BRAF, PTPN11, NF1). The trial will also include patients with CLL who have progressed after at least two lines of therapy including a BTK inhibitor and/or venetoclax.

Announced publication confirming fadraciclib suppresses MCL1 and synergizes with venetoclax in chronic lymphocytic leukemia. Results from the study confirmed that fadraciclib inhibited CDK9 mediated transcription, reduced levels of the short-lived, anti-apoptotic protein MCL1, and induced apoptosis in primary CLL cells. The data highlighted the importance of continuous treatment to prevent recovery of MCL1 protein levels. Furthermore, fadraciclib was shown to combine synergistically with the BCL2 antagonist, venetoclax, and demonstrated even greater synergy when targeted against 17p deleted CLL cells which were not sensitive to either agent alone.

·CYC140 140-101 Phase 1/2 study in solid tumors and lymphomas. This registration-directed study opened in April at City of Hope and MD Anderson Cancer Center and is enrolling patients in the Phase 1 dose escalation stage. The study uses a streamlined design and will initially determine RP2D for single-agent oral CYC140. Following RP2D, the trial will immediately enter into proof-of-concept, cohort stage, using a Simon 2-stage design. In this stage CYC140 will be administered to patients in up to seven mechanistically relevant cohorts including patients with bladder, breast, colorectal (including KRAS mutant), hepatocellular and biliary tract, and lung cancers (both small cell and non-small cell), as well as lymphomas plus an eighth basket cohort which will enroll patients with biomarkers relevant to the drug’s mechanism.

More information on our clinical trials can be found here.

Financial Highlights

As of March 31, 2022, cash and cash equivalents totaled $29.6 million, compared to $36.6 million as of December 31, 2021. Subsequent to the end of the first quarter, the Company received $3.6 million of United Kingdom research & development tax credits and $1.3 million in royalty receipts providing pro forma March 31, 2022, cash and cash equivalents of $34.5 million. The Company estimates that its available cash will fund currently planned programs through June 2023.

Research and development (R&D) expenses were $5.0 million for the three months ended March 31, 2022, as compared to $2.6 million for the same period in 2021. R&D expenses relating to fadraciclib were $3.6 million for the three months ended March 31, 2022, as compared to $1.7 million for the same period in 2021 due to increase in clinical trial costs associated with ongoing clinical trials evaluating fadraciclib in Phase 1/2 studies along with an increase in non-clinical expenditures. R&D expenses related to CYC140 were $1.1 million for the three months ended March 31, 2022, as compared to $0.7 million for the same period in 2021 due to clinical trial costs associated with the opening of clinical sites for CYC140 Phase 1/2 studies.

General and administrative expenses for the three months ended March 31, 2022, were $1.6 million, compared to $1.7 million for the same period of the previous year due to a decrease in professional and recruitment costs.

Total other income, net, for the three months ended March 31, 2022, was $1.3 million, compared to $0.1 million for the same period of the previous year. The increase of $1.2 million for the three months ended March 31, 2022, is primarily related to royalty income received from Thermo Fisher Scientific Corporation.

United Kingdom research & development tax credits were $1.1 million for the three months ended March 31, 2022, as compared to $0.7 million for the same period in 2021 as a direct consequence of increased qualifying research and development expenditure. Tax credit receipts of $3.6 million in respect of the financial year ended December 31, 2021, were received in May 2022.

Net loss for the three months ended March 31, 2022, was $4.1 million, compared to $3.5 million for the same period in 2021.

For the live and archived webcast, please visit the Corporate Presentations page on the Cyclacel website at www.cyclacel.com. The webcast will be archived for 90 days and the audio replay for 7 days.

On May 11, 2022 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) ("Aeterna" or the "Company"), a specialty biopharmaceutical company developing and commercializing a diversified portfolio of pharmaceutical and diagnostic products, reported its financial and operating results for the first quarter ended March 31, 2022 (Press release, AEterna Zentaris, MAY 11, 2022, View Source;b=2533&ID=108323&m=rl&g=1592 [SID1234614236]). The Company also provided an update on its pre-clinical and clinical development programs.

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"We are beginning to see tangible progress related to the development programs we in-licensed throughout 2021. In particular, we are very pleased to report data with our pre-clinical program for AIM biologicals, which have recently been accepted for presentation at two scientific conferences. We remain focused advancing our pipeline, executing on our strategic priorities and unlocking the full potential of our pipeline and value for all stakeholders," commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna.

Recent Highlights

Presented results from pre-clinical studies of Aeterna’s AIM Biologicals (Autoimmunity Modifying Biologicals) for the potential treatment of Parkinson’s Disease ("PD") at IMMUNOLOGY2022, the Annual Event of the American Association of Immunologists, held May 6-10, 2022.
Secured new European patent providing intellectual property protection of macimorelin in 27 countries within the European Union as well as additional European non-EU countries, such as the UK and Turkey, for macimorelin (Ghryvelin; Macrilen) for use to diagnose growth hormone deficiency (GHD) in adults.
Announced that results from pre-clinical studies of Aeterna’s AIM Biologicals for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD") were accepted for presentation at the 13th International Congress on Autoimmunity to be held June 10-13, 2022 in Athens, Greece.
Pre-Clinical and Clinical Programs Update:

Therapeutics Development Pipeline

AIM Biologicals: Targeted, highly specific autoimmunity modifying therapeutics for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD") and Parkinson’s disease (PD)

AIM Biologicals utilize a novel mechanism which is believed to demonstrate that peptide antigens presented on immunosuppressive MHC class I molecules can selectively and efficiently induce antigen-specific tolerance. Based on this mechanism, the targeted immunomodulating therapeutics are being designed as optimized soluble molecules with the goal that they may be adapted to selectively induce tolerance to various autoantigens. With AIM Biologicals, the Company aims to restore the tolerance against such proteins to treat autoimmune diseases.

Pre-clinical studies conducted by the University of Wuerzburg, Germany thus far indicate that tolerance induction appears to be achieved via selective elimination of antigen-specific immune effector cells and via induction of antigen-specific regulatory T cells from naïve T cells. AIM Biologicals thus have the potential to become highly specific and effective yet not personalized treatments of NMOSD. Data from the pre-clinical studies will be presented at the 13th International Congress on Autoimmunity to be held June 10-13, 2022 in Athens, Greece.

For the treatment of NMOSD, it is believed that the AIM Biologicals will present a specific antigen derived from the water channel protein aquaporin-4 (AQP4) loaded to soluble immunoregulatory HLA-G protein to selectively induce immunological tolerance in the central nervous system.

For the development of AIM Biologicals as potential PD therapeutics, Aeterna plans to utilize, among others, an innovative animal model on neurodegeneration by α-synuclein-specific T cells in AAV-A53T-α-synuclein Parkinson’s disease mice, which has recently been published by University of Wuerzburg researchers. Additionally, the Company recently presented pre-clinical data demonstrating that corresponding AIM Biologicals prevented mobility impairments and postmortem histopathological assessment confirmed the induction of favorable in-situ immune cell composition and the rescue of substantia nigra neurons. The pre-clinical data confirmed that the translation potential of the approach deserves further exploration.

The University of Wuerzburg continues to bolster its intellectual property (IP) protection and has filed new IP on AIM-Biologicals related to both NMOSD and PD.

Next Steps – NMOSD

Conduct in-vitro and in-vivo assessments to select an AIM Biologicals-based development candidate.
Manufacturing process development for a selected candidate.
Next Steps – Parkinson’s Disease

Design and produce antigen-specific AIM Biologics molecules for the potential treatment of Parkinson’s disease.
Conduct in-vitro and in-vivo assessments in relevant Parkinson’s disease models.
Delayed Clearance Parathyroid Hormone ("DC-PTH") Fusion Polypeptides: Potential treatment for primary hypoparathyroidism

In March 2021, Aeterna entered into an exclusive patent and know-how license agreement and research agreement with The University of Sheffield, United Kingdom, for the intellectual property relating to DC-PTH fusion polypeptides with delayed clearance for all human uses. In consultation with The University of Sheffield, Aeterna has selected AEZS-150 as the lead candidate in its DC-PTH program. AEZS-150 is being developed with the goal of providing a potential new treatment option for primary hypoparathyroidism in adults.

The Company has selected a contract manufacturing organization for the development of its manufacturing for AEZS-150.

Next Steps

Work with The University of Sheffield to conduct in depth characterization of development candidate (in-vitro and in-vivo).
Ongoing development of manufacturing process.
Formalize pre-clinical development of AEZS-150 in preparation for a potential IND filing for conducting the first in-human clinical study.
Macimorelin Therapeutic: Ghrelin agonist in development for the treatment of ALS (Lou Gehrig’s disease)

In January 2021, the Company entered into a material transfer agreement with the University of Queensland, Australia, to provide macimorelin for the conduct of pre-clinical and subsequent clinical studies evaluating macimorelin as a potential therapeutic for the treatment of ALS (Lou Gehrig’s disease). The University of Queensland researchers have filed for supportive grants and aim to conduct pre-clinical studies in multiple pre-clinical models to demonstrate the therapeutic potential of macimorelin to slow disease progression and disease-specific pathology.

Macimorelin, a potent ghrelin agonist, is an orally active small molecule that stimulates the secretion of growth hormone from the pituitary gland. Acting via this mechanism, which was established during the development as a diagnostic test for growth hormone deficiency, it is believed that macimorelin may slow the progression of certain neurodegenerative diseases like ALS.

Apart from already available pre-clinical and clinical data on macimorelin for the development as a diagnostic, Aeterna may utilize the established supply chain to support this development. Alternative formulations are currently also under development, as a further option in addition to the existing oral solution already approved for the diagnostic use in adult growth hormone deficiency (AGHD).

Next Steps

Continue investigating macimorelin efficacy in an ALS specific SOD1 mouse model.
Assess alternative formulations.
Formalize pre-clinical development plan.
Diagnostics Development Pipeline

Macimorelin Diagnostic: Ghrelin agonist in development for diagnostic use in childhood-onset growth hormone deficiency ("CGHD")

Aeterna is currently conducting its pivotal Phase 3 safety and efficacy study AEZS-130-P02 (the "DETECT-trial") evaluating macimorelin for the diagnosis of CGHD.

Children and adolescents from two to less than 18 years of age with suspected growth hormone deficiency are to be included. The study is expected to include approximately 100 subjects in Europe and North America, with at least 40 subjects in pre-pubertal and 40 subjects in pubertal status. Macimorelin growth hormone stimulation test ("GHST") will be performed twice for repeatability data and two standard GHSTs will be used as controls: arginine (i.v.) and clonidine (p.o.).

On April 22, 2021, the U.S. FDA Investigational New Drug Application associated with this clinical trial became active.

The first clinical sites in the U.S. and in Europe are open for patient recruitment. In Europe, national clinical trial approval procedures and site initiation activities are ongoing. Site activation and patient enrollment continues to be impacted by the ongoing COVID-19 pandemic. The Company is actively monitoring delays to mitigate potential impact of COVID-19 on estimated trial completion dates. Additionally, clinical trial sites originally planned in the Ukraine and Russia are being halted due to the conflict in Ukraine intensifying following the Russian invasion. As a result, further delays with enrollment are expected as the DETECT-trial planned to recruit at least 25% (25 subjects) within those countries. Due to these circumstances and the resulting feasibility data from the Company’s CRO on potential options, Aeterna believes recruitment for the DETECT-trial may now continue until later into 2023.

The Company continues to advance its ongoing business development discussions to secure commercialization partners for macimorelin in additional markets. In addition to its previously established agreements, Aeterna recently entered into a license agreement with NK Meditech Ltd., for the development and commercialization of macimorelin in the Republic of Korea, and a distribution agreement with Er-Kim Pharmaceuticals Bulgaria EOOD for the commercialization of macimorelin in Turkey and some Balkan countries.

Vaccine Development Pipeline

Bacterial Vaccine Platform: Orally active, live-attenuated bacterial vaccine platform with potential application against viruses and bacteria, such as coronavirus types, including COVID-19 (SARS-CoV-2) and Chlamydia

In February 2021, Aeterna entered into an exclusive option agreement with the University of Wuerzburg to evaluate a pre-clinical, potential COVID-19 vaccine developed at the University of Wuerzburg. In March 2021, the Company exercised its option and entered into a license agreement where the Company was granted an exclusive, world-wide, license to certain patent applications and know-how owned by the University of Wuerzburg to research and develop, manufacture, and sell a potential COVID-19 vaccine. The Company’s vaccine platform is currently undergoing pre-clinical studies for the prevention of coronavirus diseases, including COVID-19 (SARS-CoV-2) with the planned start of clinical development targeted for H1 2023.

In September 2021, the Company exercised its option under the agreement with the University of Wuerzburg on a then undisclosed field, now known to be Chlamydia. Chlamydia trachomatis is a sexually transmitted bacterium infecting over 130 million subjects annually. Asymptomatic disease can spread to the reproductive tract eventually inducing infertility, miscarriage, or ectopic pregnancy, which is a life-threatening condition. Ocular infections can lead to inclusion conjunctivitis or trachoma, which is the primary source of visual impairment or infectious blindness. Additionally, Prof. Dr. Thomas Rudel of the University of Wuerzburg was engaged by the Company in September 2021 as a scientific consultant to support development of the salmonella-based vaccine platform for the coronavirus and Chlamydia vaccines.

Recently, the Company expanded its research agreement with the University of Wuerzburg to conduct supplementary research activities and pre-clinical development studies on the potential vaccines, the results of which are covered within the scope of the license agreements. Under the expanded research program, the University of Wuerzburg will validate and utilize innovative human 3D intestinal tissue models to study the infection biology of Salmonella strains towards clinical development.

Next Steps – Coronavirus Vaccine

Evaluate administration route, dose and immunization scheme.
Initiate in-vivo immunology experiments with antigen variant candidates in relevant mice models.
Conduct virus challenge experiments in immunized transgenic animals.
Start manufacturing process assessment / development.
Conduct pre-clinical safety and toxicology assessment.
Next Steps – Chlamydia Vaccine

Design and prepare candidate vaccine strains.
Evaluate administration route, dose and immunization scheme.
Conduct In-vivo immunology experiments with candidate strains in relevant mouse models.
Summary of First Quarter 2022 Financial Results

All amounts in this press release are in U.S. dollars unless otherwise noted.

Results of operations for the three-month period ended March 31, 2022

For the three-month period ended March 31, 2022, we reported a consolidated net loss of ($2.6 million), or ($0.02) net loss per common share (basic), as compared with a consolidated net loss of ($1.5) million, or ($0.02) net income per common share (basic) for the three-month period ended March 31, 2021. The $1.1 million increase in net loss is primarily due to an increase of $1.3 million in total operating costs, $0.2 million decline in total revenues and offset by favorable foreign currency exchange rates of $0.4 million

Revenues

Our total revenue for the three-month period ended March 31, 2022 was $1.5 million as compared with $1.7 million for the same period in 2021, representing a decline of $0.2 million. The 2022 revenue was comprised of $0.43 million in licensing revenue (2021 – $0.52 million), $1.0 million in development revenue (2021 – $1.1), $0.04 million in supply chain revenue (2021 – $0.04 million), $0.02 million in royalty income (2021 – $0.01 million). and $0.06 in product sales (2021 – $nil)
Operating expenses

Our total operating expense for the three-month period ended March 31, 2022 was $4.3 million as compared with $3.0 million for the same period in 2021, representing an increase of $1.3 million. This increase arose primarily from a $0.9 increase research and development, $0.3 million increase in general and administrative expenses and an increase of $0.1 million in selling expenses.
Net finance (costs) income

Our net finance (costs) for the three-month period ended March 31, 2022 was $0.2 million as compared with net finance cost of $(0.3) million for the same period in 2021, representing an increase in net finance income of $0.5 million.
The Company had $63.6 million cash and cash equivalents at March 31, 2022 (December 31, 2021 – 65.3 million).

Consolidated Financial Statements and Management’s Discussion and Analysis

For reference, the Management’s Discussion and Analysis of Financial Condition and Results of Operations for the fourth quarter and full year 2021, as well as the Company’s unaudited consolidated interim financial statements as of December 31, 2021, will be available on the Company’s website (www.zentaris.com) in the Investors section or at the Company’s profile at www.sedar.com and www.sec.gov.

On May 11, 2022 Pulse Biosciences, Inc. (Nasdaq: PLSE), a novel bioelectric medicine company commercializing the CellFX System powered by Nano-Pulse Stimulation (NPS) technology, reported financial results for the first quarter of 2022 (Press release, Pulse Biosciences, MAY 11, 2022, View Source [SID1234614219]).

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Company Updates

Increased the overall CellFX System commercial session utilization four week moving average1 during Q1 with decreasing trend in early Q2 due to transition in commercial strategy.
Appointed new commercial leadership and began initiation of the CellFX System utilization program in May with nine commercial clinics to establish commercial integration best practices. Established program goal for each clinic of 40 commercial sessions per month. Average monthly utilization of the nine participating clinics during Q1 was 14 sessions per month.
Generated first quarter 2022 revenue of $444 thousand.
Completed one commercial sale of a CellFX System in the first quarter of 2022.
Transitioned 10 Controlled Launch Program participants to commercial use in the first quarter totaling 39 commercial conversions at the end of the first quarter. There are 20 clinics remaining in the Controlled Launch program after a total of 11 clinics have opted out as of the end of Q1.
Met with FDA regarding the Additional Information (AI) letter response to the sebaceous hyperplasia 510(k). Provided additional analysis of the clinical data following the meeting, at FDA’s request, and anticipate further communication prior to any formal response to the AI letter.
1 Utilization is measured as commercial sessions defined as individual patient treatments, regardless of the number of lesions treated, performed using CellFX Systems that have been purchased or converted to commercial use from the controlled launch program.

"In the first quarter of 2022 we took steps to refocus our CellFX dermatology efforts by bringing in new commercial leadership. We have prioritized increasing CellFX System utilization at a subset of our commercial clinics, with the goal of developing commercial integration best practices that will drive utilization across all clinics. While these best practices are being established there will be a reduced focus on capital sales," said Darrin Uecker, President and CEO of Pulse Biosciences. "We also continue to prioritize indication expansion for the CellFX System and are actively working with FDA on this process."

First Quarter 2022 Results

Revenue for the three months ended March 31, 2022 was $444 thousand. System revenue for the three months ended March 31, 2022 was $367 thousand. Cycle units revenue for the three months ended March 31, 2022 was $77 thousand resulting from the purchase of cycle units to be used with commercial systems. Total revenues of $331 thousand were recognized on a non-cash basis resulting from the Controlled Launch Participants opting to acquire CellFX Systems during the quarter.

Total GAAP cost and expenses representing cost of revenues, research and development, sales and marketing and general and administrative expenses for the three months ended March 31, 2022 were $17.7 million, compared to $18.5 million for the prior year period. Non-GAAP cost and expenses for the three months ended March 31, 2022 were $14.7 million, compared to $11.3 million for the same period in the prior year. The year-over-year increase in non-GAAP cost and expenses was primarily driven by the expansion of commercial and operational infrastructure, including increased headcount, to support commercialization activities. The first quarter of 2022 also included a discrete restructuring charge of $733 thousand, of which $706 thousand remains in accrued expenses as of March 31, 2022.

GAAP net loss for the three months ended March 31, 2022 was ($17.3) million compared to ($18.6) million for the three months ended March 31, 2021. Non-GAAP net loss for the three months ended March 31, 2022 was ($14.2) million compared to ($11.4) million for the three months ended March 31, 2021.

Cash, cash equivalents and investments totaled $12.7 million as of March 31, 2022 compared to $59.9 million as of March 31, 2021 and $28.6 million as of December 31, 2021. Cash used in the first quarter of 2022 totaled $15.9 million compared to $10.7 million used in the same period in the prior year and $13.4 million used in the fourth quarter of 2021.

Reconciliations of GAAP to non-GAAP cost and expenses and net loss have been provided in the tables following the financial statements in this press release. An explanation of these measures is also included below under the heading "Non-GAAP Financial Measures."

Webcast and Conference Call Information

Pulse Biosciences’ management will host a conference call today, May 11, 2022, beginning at 1:30pm PT. Investors interested in listening to the conference call may do so by dialing 1-877-704-4453 for domestic callers or 1-201-389-0920 for international callers. A live and recorded webcast of the event will be available at View Source

On May 11, 2022 Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) ("Innate" or the "Company") reported that members of its senior management team are scheduled to participate in the following upcoming investor conferences (Press release, Innate Pharma, MAY 11, 2022, View Source [SID1234614237]):

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H.C. Wainwright Annual Global Life Sciences Conference
Event Date: May 23-25, 2022
Citi’s European Healthcare Conference
Event Date: June 16, 2022

On May 11, 2022 TRACON Pharmaceuticals, Inc. (Nasdaq:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted cancer therapeutics and utilizing a cost efficient, CRO-independent product development platform to partner with companies to develop and commercialize innovative products in the U.S., reported financial results for the first quarter ended March 31, 2022 (Press release, Tracon Pharmaceuticals, MAY 11, 2022, View Source [SID1234614259]). The Company will host a conference call and webcast today at 4:30 PM Eastern Time / 1:30 PM Pacific Time.

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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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"We are pleased with the pace of enrollment under the amended ENVASARC protocol and remain on track to deliver interim efficacy data in the second half of this year," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Additionally, with the arbitration hearing now completed we look forward to the outcome of the binding arbitration for the TJ4309 and Bispecific Antibody agreements during the second half of this year. We also continue preparations to initiate dosing of the Phase 1/2 clinical trial of envafolimab with our potential best-in-class CTLA-4 antibody, YH001, as well as doxorubicin chemotherapy in the second half of 2022."

Recent Corporate Highlights

In March, we announced the amended ENVASARC protocol using a higher dose of envafolimab was approved by the FDA.

In April, we announced the amended ENVASARC protocol was approved by internal review boards or ethic committees at each of the 30 clinical sites in the U.S. and U.K. and that all sites were open for enrolment, with more than 10 patients enrolled.
Expected Key Upcoming Milestones

Two interim safety reviews and the interim efficacy data review by the ENVASARC Independent Data Monitoring Committee (IDMC) in the second half of 2022.

Initiate dosing of a Phase 1/2 clinical trial of envafolimab with our potential best in class CTLA-4 antibody YH001 as well as with doxorubicin chemotherapy in the second half of 2022.

Report the International Chamber of Commerce (ICC) Arbitration Panel’s binding decisions on the legal disputes involving the TJ4309 and bispecific antibody agreements with I-Mab this year.

Complete the TJ4309 Phase 1 trial permitting I-Mab the opportunity to terminate the license for $9M this year.

Initiate dosing of a randomized Phase 2 trial of TRC102 in locally advanced non-small cell lung cancer sponsored and funded by the National Cancer Institute this year.
First Quarter 2022 Financial Results

Cash, cash equivalents and short-term investments were $16.6 million at March 31, 2022, compared to $24.1 million at December 31, 2021. The Company expects that its current cash and cash equivalents will fund operations into 2023.

Research and development expenses for the first quarter of 2022 were $3.0 million, compared to $2.3 million for the first quarter of 2021.

General and administrative expenses for the first quarter of 2022 were $6.5 million, compared to $2.7 million for the first quarter of 2021. The increase was primarily attributable to legal expenses incurred due to the arbitration hearing held in February 2022 with I-Mab related to the TJ4309 and bispecific antibody agreements, and the Company expects general and administrative expenses to decrease significantly for the remainder of the year.

Net loss for the first quarter of 2022 was $9.5 million, compared to $5.1 million for the first quarter of 2021.
Conference Call Details

A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Envafolimab

Envafolimab (KN035), a single-domain antibody against PD-L1 invented by Alphamab Oncology, is the first approved subcutaneously injected PD-(L)1 inhibitor. Envafolimab was approved by the Chinese NMPA in November 2021 in adult patients with MSI-H/dMMR advanced solid tumors who failed systemic treatment and have no satisfactory alternative treatment options. In December 2019, Alphamab Oncology, 3D Medicines and TRACON entered into a collaboration whereby TRACON has the right to develop and commercialize envafolimab in soft tissue sarcoma in North America. Envafolimab is currently being studied in the pivotal ENVASARC Phase 2 trial in the United States sponsored by TRACON and a Phase 3 pivotal trial in combination with gemcitabine and oxaliplatin in advanced biliary tract cancer patients in China sponsored by TRACON’s corporate partners, Alphamab Oncology and 3D Medicines.

About ENVASARC (NCT04480502)

The ENVASARC pivotal trial is a multicenter, open label, randomized, non-comparative, parallel cohort study at 30 top cancer centers in the United States and the United Kingdom that began dosing in December 2020. TRACON expects the trial to enroll more than 160 patients with UPS or MFS who have progressed following one or two lines of prior treatment and have not received an immune checkpoint inhibitor, with 80 patients enrolled into a cohort of treatment with single agent envafolimab at 600 mg every three weeks and 80 patients enrolled into a cohort of treatment with envafolimab at 600 mg every three weeks with Yervoy. The primary endpoint is objective response rate by central review with duration of response a key secondary endpoint.

About YH001

YH001 is an IgG1 antibody against CTLA-4 that has shown enhanced antibody dependent cellular cytotoxicity (ADCC) and complement dependent cytotoxicity (CDC) in vitro. In preclinical studies YH001 demonstrated superior T cell activation and superior tumor growth inhibition activity compared to ipilimumab. YH001 also demonstrated superior activity compared to ipilimumab in human transgenic mouse tumor models when combined with a PD-(L)1 antibody. In these models, single agent YH001 depleted regulatory T cells and increased CD8+ T cells in tumor tissue. YH001 is being dosed as a single agent in a Phase 1 trial in China (NCT04699929) and in combination with the PD-1 antibody toripalimab in a Phase 1 trial in Australia (NCT04357756).

About TRC102

TRC102 (methoxyamine) is a novel, small molecule inhibitor of the DNA base excision repair pathway, which is a pathway that causes resistance to alkylating and antimetabolite chemotherapeutics. TRC102 is currently being studied in multiple Phase 1 and Phase 2 clinical trials sponsored by the National Cancer Institute through a Cooperative Research and Development Agreement (CRADA) and has orphan drug designation from the FDA in malignant glioma, including glioblastoma.

About TJ004309

TJ004309 is a novel, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine, which is highly immunosuppressive. TJ004309 is currently being studied in an ongoing Phase 1 trial to assess safety and preliminary efficacy as a single agent and when combined with the PD-L1 checkpoint inhibitor Tecentriq in patients with advanced solid tumors.