On November 9, 2023 Ensysce Biosciences, Inc. ("Ensysce" or the "Company") (NASDAQ:ENSC), a clinical-stage company applying transformative chemistry to improve prescription drug safety, reported financial results for the third quarter of 2023 (Press release, Ensysce Biosciences, NOV 9, 2023, View Source [SID1234637401]).

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

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

                  Schedule Your 30 min Free Demo!

Dr. Lynn Kirkpatrick, Chief Executive Officer of Ensysce, commented, "We continue to execute steps toward meeting our near-term objective of entering Phase 3 evaluation of PF614 with a long-term goal of commercialization. Specifically, we’ve achieved multiple milestones in the third quarter which began with Institutional Review Board approval of the PF614-201 clinical protocol in August followed by the critically important completion of the Site Initiation Visit to commence our PF614-201 clinical trial, and additionally engagement of the strategic advisory group Alacrita Consulting to enhance our partnering and licensing opportunities. Behind the scenes we are continuing manufacturing activities of both PF614 and the combination product PF614-MPAR for overdose protection. The Company’s progress throughout 2023 has further supported our belief that PF614 and PF614-MPAR will become the ‘next generation’ of analgesics that are equally effective in treating severe pain with a safer outcome for pain management than currently marketed opioids.

Looking forward, we are on track for the important readout from the PF614-201 ‘time of onset’ study prior to year-end. The data will support the design of our Phase 3 protocols and add to the favorable results from the three clinical studies completed over the last year. All data generated to date have contributed to the regulatory submission for our End of Phase 2 discussions with the FDA scheduled for January 30, 2024, in advance of initiating Phase 3 studies in 2024. Lastly, we successfully completed a private placement funding round made possible by the support of our long-term investors signaling continued confidence in Ensysce’s PF614 pain management treatment and our mission to improve drug safety," concluded Dr. Kirkpatrick.

TAAPTM (Opioid Abuse Deterrent Program) Updates

Our lead product, PF614, is a Trypsin-Activated Abuse Protection (TAAPTM) extended-release oxycodone and a potential ‘next generation’ analgesic for severe pain. The Company’s TAAPTM technology is designed to control release, be highly resistant to tampering and reduce abuse through a unique chemical modification. PF614’s TAAPTM modification makes it inactive until it is swallowed, following which it is activated or ‘turned on’ to release oxycodone by the body’s owntrypsin, an enzyme in the small intestine. Ensysce completed three clinical trials over the last year and is working to complete enrollment for its latest clinical trial to evaluate the time of onset of pain relief with PF614. Ensysce believes it has a body of evidence showing that PF614 works as designed, is bioequivalent to OxyContin and has a good safety profile. Our most recent studies demonstrate that PF614 is less liked by recreational drug users when taken orally as compared to regular oxycodone, creating what we believe is a safer analgesic.

Most recently, on September 26, 2023, the Company completed the Site Initiation Visit for the PF614-201 study, ‘A Randomized, Double-Blind, Placebo-Controlled Crossover Study of PF614 on Analgesic Response in the Cold Pressor Test in Healthy Male Subjects’ to evaluate time of onset for PF614. The study is being conducted by Dr. George Atiee at Dr. Vince Clinical Research (DVCR) in Overland, KS. The Company expects results from this study in December 2023.

On September 14, 2023, the Company announced that two scale up manufactures had been completed for PF614 in 2023. The success of the scaled manufacturing work positions the Company to begin its strategy of commercial readiness consistent with timeline expectations as it enters the next phase of development, including planning its phase 3 studies.

MPAR (Opioid Abuse Deterrent and Overdose Protection Program) Updates

PF614-MPAR is a combination product to treat severe pain, designed with overdose protection. MPAR (Multi-Pill Abuse Resistance) reduces or ‘turns off’ the release of the opioid to prevent an overdose, providing an additional layer of protection to Ensysce’s TAAP medications. We believe that MPAR is the first technology that may reduce prescription drug overdoses stemming from oral abuse, which can save lives. The clinical data generated over the past year supported the approach that the MPAR combination technology reduces release and absorption of oxycodone from PF614, when consumed in overdose. A regulatory submission is planned to discuss our MPAR development program with the FDA.

On September 7, 2023, the Company hosted a symposium at PAINWeek 2023 to formally present the recent clinical data for PF614 and PF614-MPAR to a global community of leading pain medical professionals. Ensysce’s objectives included conveying an understanding of the current landscape for severe pain treatment and drug use and abuse in America. This overview dovetailed with the Company’s thesis of the unmet medical and societal need for the safety and effectiveness of PF614 and PF614-MPAR, the Company’s two leading next-generation agents which are designed to reduce opioid abuse and prevent overdose deaths.

The results from our clinical trials are evidence that Ensysce’s PF614 and PF614-MPAR analgesics reflect an inflection point in the management of severe pain as a safe and equally effective alternative to prescription opioids that have become less accessible due to the direct correlation to abuse and overdose.

Third Quarter 2023 Financial Results

Cash – Cash and cash equivalents were $1.5 million as of September 30, 2023, as compared to $3.8 million as of June 30, 2023. Subsequent to quarter end, on October 23, 2023, the Company entered into a securities purchase agreement with investors in the form of senior secured convertible notes with a dedicated syndicate of investors for an aggregate investment of $1.7 million to occur in two closings. The initial closing occurred on October 25, 2023, providing $0.6 million prior to fees and offering expenses. The second closing is anticipated prior to year-end.
Federal Grants – Funding under federal grants totaled to $0.4 million for the third quarter of 2023 compared to $0.3 million in the comparable year ago quarter. The increase is due to the timing of research activities eligible for funding, particularly relative to the MPAR program.
Research & Development Expenses – R&D expenses decreased to $1.9 million for the third quarter of 2023 compared to $4.8 million for the same period in 2022. The decrease was primarily the result of changes in timing of external research and development costs related to clinical and pre-clinical programs for PF614 and PF614-MPAR.
General & Administrative Expenses – G&A expenses decreased to $1.2 million for the third quarter of 2023 compared to $1.7 million for the same period of 2022. The decrease was primarily a result of reduced stock-based compensation, employee bonus accrual and consulting fees.
Other Income (Expense) – Total other income (expense), net was income of $16,508 for the third quarter of 2023 compared to expense of $3.7 million for the same period of 2022. The change in other income is primarily due to non-cash fair value adjustments for convertible notes and warrants.
Net Loss – Net loss attributable to common stockholders for the third quarter of 2023 was $2.7 million compared to $9.9 million for the third quarter of 2022. As a clinical stage biotech company, our continued research and development efforts toward regulatory approvals for our product candidates are expected to result in losses for the foreseeable future.

On November 9, 2023 Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, reported financial results for the third quarter ended September 30, 2023, and provided clinical development and operational highlights (Press release, Aura Biosciences, NOV 9, 2023, View Source [SID1234637400]).

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

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

                  Schedule Your 30 min Free Demo!

"In the third quarter we made meaningful progress across our portfolio," said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. "We are encouraged by the agreement with the FDA under the SPA, as it reinforces the plan for our CoMpass trial and further supports our goal of having the first approved vision-preserving therapy for patients with early-stage choroidal melanoma. The Phase 2 data presented at AAO, with 90% of patients at twelve months follow-up, show results that are highly consistent with, and strongly support, the assumptions for the design of the CoMpass Phase 3 trial."

Dr. de los Pinos added, "We are pleased with the preliminary data from our ongoing Phase 1 trial in bladder cancer, where the first patient who received a single dose of bel-sar with light activation demonstrated a clinical complete response evidenced by absence of cancer cells on histopathology, and we look forward to additional data from the program in mid-2024. With a strong balance sheet from our recent financing, we are well-positioned to execute our pipeline to meaningful clinical milestones."

Recent Pipeline Developments

•
The CoMpass trial is designed as a superiority trial comparing bel-sar versus sham. The trial is a global Phase 3, randomized, multi-center, masked study, and it is intended to enroll approximately 100 patients randomized 2:1:2 to receive high dose regimen of bel-sar, low dose regimen of bel-sar with suprachoroidal (SC) administration, or a sham control.

•
The Company received written agreement from the FDA under an SPA for the overall design of the CoMpass trial. The primary endpoint is time to tumor progression and the first key secondary endpoint is a composite time to event analysis that will compare the tumor control and visual acuity of the bel-sar high dose regimen to sham when the last patient completes their 15 months of follow up.
•
The Company is planning to dose the first patient in Q4 2023.

Positive updated Phase 2 data evaluating SC administration of bel-sar for the first-line treatment of adult patients with early-stage CM was presented at AAO 2023.

•
The results, with 90% of patients at twelve months of follow-up who received three cycles of therapy in Cohorts 5 and 6, and who match the criteria for the global Phase 3 trial, showed a tumor control rate of 80% (8/10) and the visual acuity preservation rate was 90% (9/10), with the majority of patients being at high-risk for vision loss with tumors close to the fovea or optic disk. For the 80% of patients that responded, data showed a statistically significant reduction in tumor growth rate (-0.382 mm/yr, p = <0.0001) compared to each patient’s documented growth rate at study entry. The overall tolerability profile of bel-sar was favorable, with no dose-limiting toxicities, treatment-related SAEs or significant AEs reported as of August 3, 2023. There was no posterior inflammation and only mild anterior inflammation (Grade 1) in ~18% of the patients which was self-limited or resolved with a short course of topical steroids. Treatment-related AEs were predominantly mild and resolved without sequelae.
•
Beyond early-stage CM, the Company continues to build its ocular oncology franchise. The Company’s goal is to initiate clinical development in choroidal metastasis, an indication with a high unmet medical need and no approved therapies, as the second ocular oncology indication. The Company is on track to initiate the Phase 2 trial in 2024, with initial data by year-end 2024.
•
The Phase 1 trial of bel-sar for the treatment of muscle invasive bladder cancer (MIBC) and non-muscle invasive bladder cancer (NMIBC) is currently ongoing, and the Company expects to report data in mid-2024. This represents an area of high unmet need with approximately 80,000 patients diagnosed in the United States every year. The Company received Fast Track Designation from the Oncology Division of the FDA for NMIBC in June 2022.
•
The FDA has allowed an amendment to the protocol of the ongoing Phase 1 trial allowing the inclusion of adult patients with MIBC, in addition to NMIBC.
•
The ongoing Phase 1 multi-center, open-label clinical trial is expected to enroll approximately 19 adult patients. The trial is designed to assess the safety and tolerability of bel-sar as a single agent. The trial will provide histopathological evaluation after the local treatment to assess bel-sar’s biological activity. The trial has completed enrollment of the cohort that received bel-sar injection without light activation. Protocol mandated safety review found no safety issues and the study has proceeded to the bel-sar injection plus light activation cohorts. Preliminary data from the first patient in the light activated cohort of the trial, utilizing a single dose of bel-sar with light activation, demonstrated a clinical complete response demonstrated by absence of cancer cells on histopathology with evidence of extensive necrosis and immune activation.
•
The Company expects to provide additional data in mid-2024.

Recent Corporate Events

•
Raised Gross Proceeds of $99.0 million in an underwritten public offering. In November 2023, the Company announced the pricing of an underwritten public offering of 11,000,000 shares of its common stock at a price to the public of $9.00 per share. The offering closed on November 9, 2023. In addition, Aura has granted the underwriters a 30-day option to purchase up to 1,650,000 additional shares of common stock at the public offering price, less the underwriting discount.
•
Strengthened the clinical leadership team with the following key appointments:
•
J. Jill Hopkins, M.D., appointed as Chief Medical Officer and President of Research and Development. Dr. Hopkins previously served as Senior Vice President, Global Head of Ophthalmology and Exploratory Development at Novartis, and Chief Executive Officer of Gyroscope Therapeutics, a Novartis Company. Dr. Hopkins brings over 30 years of cross-sector experience in ophthalmology, spanning clinical care, academia, education, industry, advocacy and innovation.
•
Mark Plavsic, Ph.D., appointed as Chief Technology Officer. Dr. Plavsic previously served as Chief Technology Officer at Fate Therapeutics, a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline stem celled-derived cellular immunotherapies to patients with cancer and autoimmune disorders. Dr. Plavsic brings 30 years of global biopharmaceutical experience including end-to-end technical operations in the United States, Europe, and Australasia and successful translation and scale-up of complex biologics from preclinical development through commercial launch and distribution.

Third Quarter 2023 Financial Results

•
As of September 30, 2023, the Company had cash and cash equivalents and marketable securities totaling $149.1 million. This excludes the net proceeds from the underwritten public offering received in November 2023. The Company believes its current cash and cash equivalents, marketable securities, and proceeds from the underwritten public offering are sufficient to fund its operations into the second half of 2026.
•
Research and development expenses increased to $15.4 million for the three months ended September 30, 2023 from $11.3 million for the three months ended September 30, 2022, primarily due to ongoing clinical costs associated with the progression of the Company’s Phase 2 study and CRO costs associated with the start of the Company’s Global Phase 3 trial, and manufacturing and development costs for bel-sar.
•
General and administrative expenses increased to $5.1 million for the three months ended September 30, 2023 from $4.8 million for the three months ended September 30, 2022. General and administrative expenses include $1.2 million and $1.1 million of stock-based compensation for the three months ended September 30, 2023 and 2022, respectively. The increase was primarily driven by personnel expenses, as well as increases in general corporate expenses related to growth of the Company.
•
Net loss for the three months ended September 30, 2023 was $18.5 million compared to $15.9 million for the three months ended September 30, 2022.

On November 9, 2023 Monopar Therapeutics Inc. (Monopar or the Company) (Nasdaq: MNPR), a clinical­-stage biopharmaceutical company focused on developing innovative treatments for cancer patients, reported third quarter 2023 financial results and summarized recent developments (Press release, Monopar Therapeutics, NOV 9, 2023, View Source [SID1234637382]).

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

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

                  Schedule Your 30 min Free Demo!

Recent Developments

MNPR­-101 for Radiopharmaceutical Use – First-in-Human Study to Start Potentially As Early As December of This Year

Monopar continues to advance its MNPR-101 radiopharmaceutical program towards a first-in-human study in patients with advanced solid tumors. MNPR‐101 is a highly selective antibody against uPAR, a promising target overexpressed in multiple solid tumors, being developed as a precision radiopharmaceutical for both imaging and treatment of cancer. Much of the work with MNPR-101 is being done in collaboration with NorthStar Medical Radioisotopes LLC.

•Preclinical data with MNPR-101 labeled with zirconium-89 (imaging radioisotope) as well as actinium-225 (a powerful alpha-emitting therapeutic radioisotope) so far have shown selective, high, and durable tumor uptake across multiple aggressive cancers including pancreatic, colorectal, and triple negative breast cancers. In addition, a strong, dose-dependent anti-tumor effect has been seen in in vivo pancreatic and triple-negative breast cancer models with favorable biodistribution profiles.

Camsirubicin – Phase 1b Dose­-Escalation Trial, Treating Fifth Dose­-Level Cohort (650 mg/m2)

•The Phase 1b open-label, dose-escalating clinical trial of camsirubicin in patients with advanced soft tissue sarcoma (ASTS) is in the fifth dose-level cohort (650 mg/m2), which is nearly 2.5x the highest dose evaluated in any prior camsirubicin clinical trial (265 mg/m2). We have dosed to date two patients in the fifth dose cohort, and both experienced tumor size reductions, one of approximately 18% and the other of approximately 20%.

•At the 2023 Connective Tissue Oncology Society (CTOS) Annual Meeting, Monopar presented Phase 1b clinical trial results to-date. So far, 9 out of the 14 enrolled ASTS patients have had stable disease (SD, as defined by RECIST 1.1 criteria) after camsirubicin treatment, including all patients in the fourth and fifth dose cohorts. No dose-limiting toxicity, as defined in the protocol, has been observed to-date. A medically complex patient in the fifth dose cohort has an ongoing left ventricular ejection fraction ("LVEF") decrease and is being assessed for potential anthracycline (camsirubicin) induced cardiotoxicity. This patient has a BMI of 42.5, one kidney, hypertension, a long-standing heart murmur, and a maternal history of heart failure.

MNPR­-202 – Encouraging Preclinical Results Support Further Research

•MNPR-202 is a camsirubicin analog that retains the same potentially non-cardiotoxic backbone as camsirubicin but is modified at other positions which may enable it to work against cancers that are resistant to doxorubicin, one of the most commonly-used cancer drugs worldwide.

•In collaboration with Dr. Anand Jeyasekharan at National University of Singapore, preclinical studies are showing that MNPR-202 has a similar cytotoxic potency to doxorubicin but that it works in a distinct way as compared to doxorubicin.

•In a preclinical study using a human iPSC cardiomyocyte model to evaluate heart toxicity, cells treated with MNPR-202 maintained a greater contractile amplitude and a more consistent and regular beat rate relative to doxorubicin-treated cells, indicating that MNPR-202 has a broader therapeutic window than doxorubicin with respect to cardiotoxicity.

Results for the Third Quarter Ended September 30, 2023 Compared to the Third Quarter Ended September 30, 2022

Cash and Net Loss

Cash, cash equivalents and short-term investments as of September 30, 2023 were $8.5 million. Monopar expects that its current funds will be sufficient for Monopar to advance the Company’s MNPR-101 radiopharmaceutical program into its first-in-human clinical trial, obtain topline results from its ongoing open-label Phase 1b camsirubicin clinical trial by mid-2024 (but this may not be the case if camsirubicin reaches even higher dose levels than anticipated and topline results are deferred as dosing continues beyond mid-2024), and continue preclinical studies of MNPR-202. The Company estimates its cash, cash equivalents and short-term investments will fund the Company’s planned operations at least through November 2024. Monopar will require additional funding to advance its clinical and preclinical programs beyond that and anticipates seeking to raise additional capital within the next 12 months to fund its future operations.

Net loss for the third quarter of 2023 was $2.0 million or $0.14 per share compared to net loss of $2.4 million or $0.19 per share for the third quarter of 2022.

Research and Development (R&D) Expenses

R&D expenses for the three months ended September 30, 2023 were $1,317,000, compared to $1,732,000 for the three months ended September 30, 2022. This represents a decrease of $415,000 primarily attributed to a decrease of $673,000 in Validive clinical trial-related and clinical material manufacturing-related expenses offset by (1) an increase of $161,000 in non-clinical studies related to the MNPR-101 radiopharmaceutical program, (2) an increase of $78,000 in camsirubicin clinical trial expenses including patient dosing and manufacturing-related expenses, and (3) a net increase of $19,000 due to other R&D expenses.

General and Administrative (G&A) Expenses

G&A expenses for the three months ended September 30, 2023 were $749,000, compared to $675,000 for the three months ended September 30, 2022. This represents an increase of $74,000 primarily attributed to an increase in G&A salaries and benefits.

On November 9, 2023 MEI Pharma, Inc. (Nasdaq: MEIP), a clinical-stage pharmaceutical company evaluating novel drug candidates to address know resistance mechanisms to standard-of-care cancer therapies, reported results for the quarter ended September 30, 2023 and highlighted recent corporate events (Press release, MEI Pharma, NOV 9, 2023, View Source [SID1234637381]).

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

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

                  Schedule Your 30 min Free Demo!

"Our ongoing clinical studies evaluating the combination of voruciclib, our CDK9 inhibitor, with Venclexta in relapsed/refractory AML patients and ME-344, our mitochondrial inhibitor, combined with Avastin in metastatic colorectal cancer patients, continue to have strong investigator support and cohort enrollment remains on track in each program," said David M. Urso, president and chief executive officer of MEI Pharma. "We expect to report data from the dose escalation portion of the Phase 1 clinical trial evaluating voruciclib in combination with venetoclax in early calendar 2024, and data from the first cohort of patients in Phase 1b clinical trial evaluating ME-344 in the first half of 2024."

First Quarter Fiscal Year 2024 and Recent Highlights

•
In August 2023, MEI announced the dosing of the first patient in a Phase 1b study evaluating ME-344 in combination with bevacizumab (AVASTIN) in patients with previously treated metastatic colorectal cancer. ME-344 is a novel mitochondrial inhibitor targeting energy production through the OXPHOS pathway, which is important for supporting tumor cell survival and proliferation for many forms of cancer, including colorectal cancer. Bevacizumab, a vascular endothelial growth factor (VEGF) inhibitor, and other antiangiogenics, inhibit energy production through glycolysis and, thereby, increase tumor reliance on mitochondrial energy production, providing an opportunity to evaluate a combination with ME-344 to inhibit energy production in tumor cells and induce an antitumor effect. The Company anticipates announcing safety and efficacy data from the first cohort of 20 patients in the first half of 2024.

•
In November 2023, MEI announced that an abstract highlighting clinical data from the monotherapy dose escalation stage of the ongoing Phase 1 study evaluating voruciclib, a selective oral cyclin-dependent kinase 9 (CDK9) inhibitor, alone and in combination with venetoclax (Venclexta), a B-cell lymphoma 2 ("BCL2") inhibitor, in patients with acute myeloid leukemia (AML) or B-cell malignancies, will be presented during a poster session at the upcoming 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition to be held December 9 – 12, 2023.

Expected Drug Candidate Pipeline Developments

Voruciclib – Oral CDK9 inhibitor in Phase 1 Study

img183464774_0.jpg

•
Report clinical data from the dose escalation portion of the ongoing Phase 1 clinical trial evaluating voruciclib plus Venclexta (venetoclax) in patients with AML early in calendar 2024.

ME-344 – Mitochondrial inhibitor in Phase 1b Study

•
Report clinical data from Cohort 1 of the Phase 1b clinical trial evaluating ME-344 plus Avastin (bevacizumab) in patients with relapsed colorectal cancer in the first half of calendar-year 2024.

First Quarter Fiscal Year 2024 Financial Results

•
As of September 30, 2023, MEI had $82.2 million in cash, cash equivalents, and short-term investments with no outstanding debt.

•
For the quarter ended September 30, 2023, cash used in operations was $18.5 million, compared to $14.8 million during the quarter ended September 30, 2022. The increase in cash used in operations was primarily due to changes in working capital associated with the wind down of zandelisib activities with Kyowa Kirin and professional services primarily related to advisory and legal fees associated with various stockholder-related activities, including stockholder-initiated consent solicitations.

•
Research and development expenses were $3.5 million for the quarter ended September 30, 2023, compared to $19.5 million for the quarter ended September 30, 2022. The decrease was primarily related to a reduction in zandelisib costs as we continued the wind down of development activities announced in December 2022, as well as reduced personnel and related costs from our fiscal year 2023 reduction in headcount.

•
General and administrative expenses decreased by $1.0 million to $6.5 million for the quarter ended September 30, 2023, compared to $7.5 million for the quarter ended September 30, 2022. The net decrease was primarily related to reduced personnel and related costs from our fiscal year 2023 reduction in headcount, partially offset by higher external professional services and legal expenses.

•
MEI recognized revenue of $65.3 million for the quarter ended September 30, 2023, compared to $8.7 million for the quarter ended September 30, 2022. The increase in revenue is due to the recognition of deferred revenue associated primarily with the termination of the Kyowa Kirin Commercialization Agreement in July 2023. As of September 30, 2023, all deferred revenue associated with that agreement has been recognized.

img183464774_0.jpg

•
Net income was $56.4 million, or $8.46 per share, for the quarter ended September 30, 2023, compared to net loss of $16.6 million, or $2.49 per share for the quarter ended September 30, 2022. The Company had 6,662,857 shares of common stock outstanding as of September 30, 2023.

The Company believes its cash balance is sufficient to fund operations for at least the next 12 months, and through the reporting of clinical data readouts from the ongoing and planned voruciclib and ME-344 Phase 1 and Phase 1b clinical programs, respectively.

On November 9, 2023 MaaT Pharma, a clinical-stage biotech company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to improving survival outcomes for patients with cancer, reported a business update and reported its cash position as of September 30, 2023 (Press release, MaaT Pharma, NOV 9, 2023, View Source [SID1234637380]).

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

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

                  Schedule Your 30 min Free Demo!

"The positive DSMB review of MaaT013 in its Phase 3 trial and the commencement of the MaaT033 Phase 2b trial underscores our leadership position in oncology-focused microbiome therapeutics. It also demonstrates our ability to execute our clinical plan and prepare for commercialization through expanded production capacities that were completed in Q3. We are proud to achieve these milestones and we will continue to work on generating value for our shareholders," stated Siân Crouzet, CFO of MaaT Pharma. "Our progress and achievements put us in good stead as we look towards the months ahead and the completion of patient recruitment in the Phase 3 clinical study in 2024."

Pipeline highlights

MET-N

MaaT013

In hemato-oncology:
In July 2023, the Company announced that clinical data on MaaT013 as a treatment for aGvHD was published in eClinicalMedicine, one of the Lancet Discovery Science suite of journals.
As a post period event, in October 2023, the Company announced that the DSMB unanimously recommended that the open-label, single arm pivotal Phase 3 clinical trial evaluating MaaT013 in acute Graft-versus-Host Disease (aGvHD), named ARES, can continue without modification. The Overall Response Rate (ORR) was superior to pre-defined protocol assumptions. Therefore, the DSMB concluded that the benefit/risk ratio with "high efficacy and low toxicity" was favorable in this patient population.
As a post period event, in November 2023, the Company announced that extended results from its Early Access Program of MaaT013 in 111 patients (additional 30 patients included in the Program compared to last year) with aGvHD have been selected for poster presentations at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
In immuno-oncology:
The Phase 2a PICASSO trial, evaluating MaaT013 in combination with immune checkpoint inhibitors in metastatic melanoma, is on schedule, and results should be available in late 2024 or early 2025.
MaaT033

In hemato-oncology:
In September 2023, the Company announced that the European Medicines Agency (EMA) had granted MaaT033 an orphan drug designation. MaaT033 aims to improve overall survival in patients undergoing hematopoietic stem cell transplantation (HSCT) and the EMA had recognized the significant benefit that MaaT033 could therefore bring to this patient population. The status offers key benefits including market exclusivity, clinical protocol assistance, waivers or reductions in regulatory fees.
As a post period event, in November 2023, the Company announced that the first patient has been treated as part of its Phase 2b trial (PHOEBUS) investigating the efficacy of MaaT033 in improving overall survival at 12 months for patients with blood cancer receiving allo-HSCT. The international, multi-center, randomized, double-blind, placebo-control study (NCT05762211), will be conducted in up to 56 clinical investigation sites and is expected to enroll 387 patients. It is, to date, the largest randomized controlled trial assessing a microbiome therapy in oncology.
As a post period event, in November 2023, the Company announced that the design of its Phase 2b study evaluating MaaT033 has been selected for a poster presentation at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
In neurodegenerative diseases:
In September 2023, the Company announced that the first patient was dosed in the IASO Phase 1b pilot study (NCT05889572) in ALS (also known as Lou Gehrig’s disease in the U.S. and Charcot’s disease in French-speaking countries).
MET-C

MaaT034

In immuno-oncology:
As a post period event, in November 2023, the Company had two presentations at the 38th Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting including in vitro results for its new Artificial Intelligence (AI)-generated lead product, MaaT034, designed to improve responses to immunotherapy for patients with solid tumors. MaaT034 is the first member of the MET-C platform. Data presented at SITC (Free SITC Whitepaper) 2023 shows that MaaT034 replicates, at large industrial scale, the richness and diversity of healthy native-based microbiome ecosystems, restores the integrity of a damaged gut barrier, activates AhR pathway involved in gut homeostasis, and stimulates both myeloid and lymphoid immune cells and improves immune cell response to immune checkpoint inhibitor (ICI) therapy. The first clinical batches are expected to be produced in 2024 and the first-in-human testing is planned for 2025.
Corporate update

In July 2023, MaaT Pharma joined the Microbiome Therapeutics Innovation Group (MTIG).
In September 2023, the Company and Skyepharma announced completion of the cGMP manufacturing facility and the transfer of MaaT Pharma’s Production and Development teams to the new site.
Cash position1

As of September 30, 2023, total cash and cash equivalents were EUR 31.7 million, as compared to EUR 35.1 million as of June 30, 2023, and EUR 35.2 million as of December 31, 2022. The net decrease in cash of EUR 3.4 million during the third quarter 2023 reflects continued investment in R&D activities across the pipeline, offset in part by partial reimbursement of the 2022 R&D tax credit of EUR 0.5 million. The Company believes it has sufficient cash to cover needs of the development programs into the second quarter of 2024.
Revenues in Q3 2023

MaaT Pharma reported revenues from its compassionate access program of EUR 0.4 million for the quarter ended September 30, 2023 comparable with the third quarter of 2022. Total revenues for the first three quarters of 2023 amount to EUR 1.8 million compared with EUR 0.9 million for the first three quarters of 2022[1]. This trend is a direct reflection of the continued demand from the medical community for MaaT Pharma’s drug candidate MaaT013.

Upcoming investor and medical conference participation

November 14-17, 2023 – London, UK Investor Meetings
November 14-16, 2023 – Boston, MA – USA, Microbiome Connect
November 15-17, 2023 – Lille, France, 22nd Société Francophone de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC) Congress – Booth #12
December 9-12, 2023 – San Diego, CA – USA, 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting – Posters presentation
January 8-12, 2024 – San Francisco, CA – USA, Investor Meetings
[1] Unaudited data

[2] The Company would like to correct a clerical error that was present in its Q2 results press release of July 27, 2023. The revenue from H1 2022 was EUR 0.5 million, and not EUR 0.9 million as reported in the press release. The EUR 0.9 million figure was the difference in revenue between H1 2023 and H1 2022 (EUR 1.4 million vs. EUR 0.5 million).