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Citius Pharmaceuticals, Inc. Reports Fiscal Full Year 2022 Financial Results and Provides Business Update

On December 22, 2022 Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products reported its business and financial results for the fiscal full year ended September 30, 2022 (Press release, Citius Pharmaceuticals, DEC 22, 2022, View Source [SID1234625555]).

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Fiscal Full Year 2022 Business Highlights and Subsequent Developments

Completed Pivotal Phase 3 trial of I/ONTAK (E7777) and submitted biologics license application (BLA) to the U.S. Food and Drug Administration (FDA);
FDA confirmed Prescription Drug User Fee Act (PDUFA) target action date of July 28, 2023;
Advanced Mino-Lok Phase 3 trial:
Expanded trial to additional sites in India for an anticipated total of 35 clinical trial locations globally,
Enrolled 169 patients to date, exceeding recruitment goal of 144 patients,
Observed 72 of 92 required catheter failure events, with 17 patients in active treatment or pending study completion data review (which may contribute additional failure events);
Initiated Phase 2b trial of Halo-Lido for the treatment of hemorrhoids in April 2022; patient enrollment ongoing with data readout expected 2H 2023;
Initiated clinical collaboration with the University of Pittsburgh to evaluate regulatory T-cell (T-reg) depletion with I/ONTAK (E7777) in combination with pembrolizumab in recurrent or metastatic solid cancer tumors in a Phase 1 investigator-initiated trial, with first patient enrolled in November 2022; and,
Approved for $3.6 million in non-dilutive capital through the New Jersey Economic Development Program to support ongoing research and development efforts.
Financial Highlights

Cash and cash equivalents of $41.7 million as of September 30, 2022;
R&D expenses were $17.7 million for the full year ended September 30, 2022, compared to $12.2 million for the full year ended September 30, 2021;
G&A expenses were $11.8 million for the full year ended September 30, 2022, compared to $9.8 million for the full year ended September 30, 2021;
Stock-based compensation expense was $3.9 million for the full year ended September 30, 2022, compared to $1.5 million for the full year ended September 30, 2021; and,
Net loss was $33.6 million, or ($0.23) per share for the full year ended September 30, 2022 compared to a net loss of $23.1 million, or ($0.23) per share for the full year ended September 30, 2021.
"In 2022, we focused on execution across our key development programs: I/ONTAK, Mino-Lok and Halo-Lido. These efforts, combined with a prudent use of funds, enabled us to meaningfully advance our pipeline. We believe we have sufficient runway through December 2023 to realize additional value-creating milestones, including a potential FDA approval and two anticipated trial completions in the coming calendar year," stated Leonard Mazur, Chairman and CEO of Citius.

"Our Phase 3 Mino-Lok trial is now significantly closer to completion. While we expected to achieve 92 catheter failure events with 144 patients by the end of 2022, the trial’s observed catheter failure event rate has proven to be lower than anticipated. Consequently, we must continue recruiting patients. By successfully re-engaging with our U.S. trial sites as they recovered from the impact of Covid, we were able to drive patient recruitment. We have now exceeded our targeted enrollment and have achieved 72 of the required catheter failure events, with additional patients under review. To augment our recruitment efforts and continue the positive momentum in enrollment, we expanded the Mino-Lok trial to include sites in India. Once all new trial sites are fully activated, we will have nearly doubled our clinical site footprint. With these additional sites helping to drive incremental enrollment, we anticipate that the 92-event threshold required to complete the trial is achievable in the coming months," added Mazur.

"During the year, we also completed a Phase 3 trial and submitted a BLA for I/ONTAK, an oncology asset we in-licensed just over a year ago. Upon further discussion with the FDA, the PDUFA target date has been set for July 28, 2023. We remain committed to establishing a robust commercial infrastructure to support I/ONTAK’s successful product launch, if approved. In the second half of 2022, we also extended our support for a Phase 1 investigator-initiated study of I/ONTAK in combination with pembrolizumab (Keytruda1) to treat patients with recurrent or metastatic solid tumors. This study has begun recruiting patients and is the second investigator-initiated trial to explore I/ONTAK’s potential as a combination therapy in much larger immuno-oncology markets. We continue to believe I/ONTAK’s value extends beyond a potential initial indication in persistent or recurrent cutaneous T-cell lymphoma. Earlier in the year, we announced our intention to spin off I/ONTAK. Given broader market conditions, we continue to evaluate opportunities to further unlock this asset’s value," continued Mazur.

"In addition to advancing our Phase 3 trials, we initiated a Phase 2b trial for Halo-Lido, our prescription strength topical formulation for hemorrhoids. The trial began enrolling patients with symptomatic Grade II or III hemorrhoids in the second quarter of 2022. Recent recruitment has accelerated and we expect complete trial data available in the second half of 2023," added Mazur.

"As financial stewards, we continuously evaluate the optimal capital structure for the company. We believe our anticipated catalysts, along with a healthy cash position, provide us with several strategic and financial options with which to continue advancing our pipeline. This may include the previously announced potential spinoff of I/ONTAK into a standalone oncology company, pending market conditions, and other standalone financing alternatives available to us. We are encouraged by the multiple value-driving catalysts anticipated in calendar 2023, including a potential drug approval and two trial completions, and look forward to extending our positive momentum in the months ahead," concluded Mazur.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA

FULL YEAR 2022 FINANCIAL RESULTS:

Liquidity

As of September 30, 2022, the Company had $41.7 million in cash and cash equivalents.

As of September 30, 2022, the Company had 146,211,130 common shares outstanding.

The Company estimates that its available cash resources will be sufficient to fund its operations through December 2023.

Research and Development (R&D) Expenses

R&D expenses were $17.7 million for the full year ended September 30, 2022, compared to $12.2 million for the full year ended September 30, 2021. The increase of $5.5 million is primarily associated with the completion of the I/ONTAK (E7777) Phase 3 trial and the preparation and submission of the related Biologics License Application to the FDA, incremental Mino-Lok Phase 3 trial costs related to the addition of a global clinical research organization, Biorasi, and the opening of international sites in India, as well as costs associated with the initiation of the Halo-Lido Phase 2 study. The increase was offset primarily by a one-time $5 million license fee paid to Novellus in the year ended September 30, 2021, which did not recur.

We expect that research and development expenses will continue to increase in fiscal 2023 as we continue to focus on the anticipated commercialization of E7777, our Phase 3 trial for Mino-Lok, our Phase 2b trial for Halo-Lido, and accelerate our research and development efforts related to Mino-Wrap and ARDS.

General and Administrative (G&A) Expenses

G&A expenses were $11.8 million for the full year ended September 30, 2022, compared to $9.8 million for the full year ended September 30, 2021. The increase was primarily due to additional compensation costs for new employees and investor relations expenses. General and administrative expenses consist primarily of compensation costs, consulting fees for our financing activities and corporate development services, and investor relations expenses.

Stock-based Compensation Expense

For the full year ended September 30, 2022, stock-based compensation expense was $3.9 million as compared to $1.5 million for the prior year. The increase reflects expenses related to new grants made by Citius and the NoveCite stock option plan. In fiscal year 2022, we granted options to our new employees and additional options to other employees, directors, and consultants. Stock-based compensation expense includes options granted to directors, employees, and consultants.

At September 30, 2022, unrecognized total compensation cost related to unvested options for Citius common stock of $5.3 million is expected to be recognized over a weighted average period of 1.9 years and unrecognized total compensation cost related to unvested options for NoveCite common stock of $0.2 million is expected to be recognized over a weighted average period of 1.5 years

Net loss

Net loss was $33.6 million, or ($0.23) per share for the year ended September 30, 2022, compared to a net loss of $23.1 million, or ($0.23) per share for the year ended September 30, 2021. The increase in net loss is primarily due to the $5.4 million increase in our research and development expenses, a $1.9 million increase in general and administrative expenses, and a $2.4 million increase in stock-based compensation expense.

Targovax announces that the TG01 study in multiple myeloma at Oslo University Hospital has received regulatory approvals to proceed

On December 22, 2022 Targovax ASA (OSE: TRVX), a clinical-stage immuno-oncology company developing immune activators to target solid tumors, reported that the planned clinical trial with mutant RAS cancer vaccine TG01 in multiple myeloma has received regulatory approval to proceed from NOMA and REK (Press release, Targovax, DEC 22, 2022, View Source [SID1234625554]).

The trial is a collaboration between OUS and Targovax and will test TG01 vaccination as a monotherapy in 20 KRAS or NRAS mutated multiple myeloma patients who continue to have measurable disease after completion of standard of care treatment. The aim is to assess whether anti-RAS T-cell priming induced by TG01 can enhance the clinical response.

The trial will be sponsored by OUS and led by Dr. Fredrik Schjesvold, an international leader in the field and founder of the Oslo Myeloma Center, the largest Myeloma center in the Nordics. Targovax will provide TG01 drug supply, scientific support and a financial contribution through grants awarded from Innovation Norway and the Norwegian Research Council.

Dr. Ingunn Munch Lindvig, VP & Head of Regulatory Affairs, Targovax ASA, said: "We are very pleased that the planned collaborative studies for the TG mutant RAS program continue to obtain the necessary regulatory approvals to proceed in both the EU and USA. The multiple myeloma trial plan is now approved both by the Norwegian regulatory agency NOMA and the ethics committee REK under the new European system, and Oslo University Hospital can proceed with identifying patients for inclusion into the trial."

AIkido Pharma Announces Name and Stocker Ticker Symbol Change to Dominari Holdings Inc. (Nasdaq: DOMH)

On December 22, 2022 AIkido Pharma Inc. (NASDAQ: AIKI) (the "Company") reported that it has changed its name from AIkido Pharma Inc. to Dominari Holdings Inc. and its ticker symbol from "AIKI" to "DOMH (Press release, AIkido Pharma, DEC 22, 2022, View Source [SID1234625553])." The Company expects that its shares of common stock will begin trading on The Nasdaq Stock Market under the new name and new stock ticker symbol "DOMH" on or about December 22, 2022.

The name change was approved at a meeting of the board of directors held on December 5, 2022.

Anthony Hayes, CEO of Dominari Holdings Inc., commented, "Rebranding of the Company reflects the board’s long-range strategic goal to diversify away from the healthcare sector and into the financial services sector. We are actively recruiting new team members and building out operations space for our newly created financial services subsidiary, Dominari Financial Inc., which is acquiring the financial services business of a registered broker-dealer. We have deployed approximately $3.2 million of capital in support of the establishment and operations of this new subsidiary, including the broker-dealer acquisition, and expect to continue to make additional contributions as required from time to time."

PureTech Provides End of Year Report on Key Progress

On December 22, 2022 PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to changing the treatment paradigm for devastating diseases, reported on the key progress made across its Wholly Owned Programs[1] and Founded Entities[2] in 2022 (Press release, PureTech Health, DEC 22, 2022, View Source [SID1234625550]).

Daphne Zohar, Founder and Chief Executive Officer of PureTech, commented: "2022 has been a particularly successful and productive year in both crystallizing value and shaping the future of PureTech. Our primary focus is to progress our Wholly Owned Pipeline to commercialization and deliver new classes of medicines for patients with serious, debilitating conditions. We are advancing toward this goal by deepening our commitment to programs with compelling clinical data that are moving into late-stage studies and that we believe have the potential for broad impact.

"Over the next 12 months, we anticipate multiple important catalysts that will further guide how we prioritize our pipeline, informing our decisions regarding which programs we will drive to commercial launches ourselves and which programs could be most successfully advanced through other avenues such as a partnership, sale, or spinout into another entity."

PureTech’s Wholly Owned Programs are centered on developing breakthrough medicines for patients with underserved and serious diseases. The Company’s therapeutic candidates are being advanced for the potential treatment of devastating conditions including idiopathic pulmonary fibrosis (IPF), metastatic solid tumors, leukemia and certain neurological and neuropsychological indications.

Additionally, PureTech’s Founded Entities are advancing 20 therapeutics and therapeutic candidates, of which two (Plenity for weight management[3] and EndeavorRx for treating inattention in ADHD ages 8-12[4]) have received both U.S. FDA clearance and European marketing authorization and a third (KarXT for schizophrenia) will soon be filed for FDA approval, as of the most recent update by the company. Key highlights include the following:

Key Wholly Owned Program Updates

LYT-100 (deupirfenidone) is in development for the potential treatment of conditions involving inflammation and fibrosis, including IPF, for which current standards of care are associated with significant tolerability issues, resulting in approximately three out of four patients in the U.S. foregoing treatment with these otherwise efficacious medicines.[5]LYT-100 is a deuterated form of one of these treatments, pirfenidone, which has proven efficacy and has been shown to improve survival in these patients by approximately three years, but its side effects cause patients to discontinue or dose reduce, thereby limiting its effectiveness.[6]

o Announced results from a randomized, double-blind crossover study in healthy older adults demonstrating that approximately 50% fewer subjects treated with PureTech’s LYT-100 experienced gastrointestinal-related adverse events compared to subjects treated with pirfenidone.

Initiated a global, randomized double blind placebo-controlled trial of LYT-100 in patients with IPF. This trial is expected to serve as the first of two registration-enabling trials, and topline results are expected by the end of 2023.

Initiated a preclinical study of LYT-100 for the prevention and treatment of radiation induced fibrosis, an indication for which the United States government stockpiles medical countermeasures. This program is subject to the Animal Rule, which allows for the approval of drugs based on validated animal models when human efficacy studies are not feasible.

Completed a Phase 2a trial of LYT-100 in patients with breast cancer-related, upper limb secondary lymphedema, which met the primary endpoint of safety and tolerability, adding to the growing body of data demonstrating a favorable tolerability profile for LYT-100. Secondary endpoints assessed exploratory biomarkers related to lymphedema, and the data did not provide support for a clear development path for this indication. Given the Company’s current priorities in IPF, where the regulatory path is well-defined and there is a wealth of strong efficacy data with pirfenidone, the Company has deprioritized lymphedema as an indication.

PureTech is also exploring the potential evaluation of LYT-100 in other inflammatory and fibrotic conditions, such as progressive fibrosing interstitial lung diseases, myocardial and other organ system fibrosis, based on the strength of existing clinical data around the use of pirfenidone in these indications.

LYT-200 (anti-galectin-9 mAb) is in development for the potential treatment of metastatic solid tumors that have poor survival rates as well as hematological malignancies, such as acute myeloid leukemia (AML), where more than 50% of patients either don’t respond to initial treatment or experience relapse after responding to initial treatment.[8]

Completed the bi-monthly and weekly monotherapy dose escalation portion of the Phase 1 program assessing the safety and tolerability of escalating doses of LYT-200 as a potential treatment for metastatic solid tumors. No dose-limiting toxicities were reported, and the full results will be presented in an upcoming scientific forum. Evaluation of LYT-200 in combination with tislelizumab is expected to begin in the first quarter of 2023.

Shared new preclinical data supporting the clinical potential of LYT-200 as a therapeutic agent for the treatment of leukemia in a scientific poster at the American Society of Hematology (ASH) (Free ASH Whitepaper) 64th Annual Meeting. The data presented support the role of galectin-9 in multiple types of leukemia and the ability of anti-galectin-9 antibodies to provide effective anti-tumor activity in these cancers. Based on these and other compelling preclinical data generated with LYT-200 in blood cancers, PureTech initiated a clinical trial to evaluate LYT-200 as a single agent for the treatment of AML, with results expected in 2023.

Given the expansion of the clinical evaluation of LYT-200, the Company has deprioritized preclinical development of LYT-210.

LYT-300 (oral allopregnanolone) is in development for the potential treatment of neurological and neuropsychological conditions where there is a need for more effective treatments that work quickly, have more favorable tolerability and can be administered conveniently.

o Completed the multi-part Phase 1 trial of LYT-300, which demonstrated oral bioavailability in healthy adults, achieving blood levels of allopregnanolone at or above those associated with therapeutic effect and ninefold greater than orally administered allopregnanolone, based on third-party published data.[9] The data also demonstrated target engagement with GABAA receptors, which are known to regulate mood and other neurological conditions. LYT-300 was generally well-tolerated with no treatment-related severe or serious adverse events observed. A Phase 1b/2a trial is expected to begin in the first half of 2023.

LYT-310 (oral cannabidiol [CBD]) is in development to expand the therapeutic application of CBD across a range of neurological disorders.

o Announced nomination of a new therapeutic candidate, LYT-310, which is expected to enter the clinic in Q4 of 2023. As with LYT-300, PureTech scientists applied the Glyph Platform to enable advantages such as oral capsule dosing and the potential for improved safety and tolerability. These differentiated features of LYT-310 could expand CBD use across a broad range of patient populations (such as adolescents and adults) and indications, including rare and common forms of epilepsy as well as other central nervous system disorders. LYT-310 has the potential to be a readily scalable and consistent product that can be produced in a cost-effective manner.

LYT-503/IMB-150 (partnered non-opioid pain program) is being advanced through a collaboration for the potential treatment of interstitial cystitis/bladder pain syndrome, a chronic bladder condition that consists of discomfort or pain in the bladder or surrounding pelvic region that is not adequately controlled for many patients.

o First patient, first visit was achieved in the Phase 1 clinical trial evaluating LYT-503/IMB -150 as a potential non-opioid treatment for female patients with Interstitial Cystitis/Bladder Pain Syndrome.

LYT-510 (oral inflammation-targeting formulation of tacrolimus) is in development for the potential treatment of chronic pouchitis and inflammatory bowel disease (IBD), a condition for which current treatments require multiple injections over time and are associated with several limitations, including dose-limiting adverse events, loss of efficacy over time, a lack of efficacy entirely and the potential for opportunistic infections or malignancies.

LYT-510 is the lead candidate generated from the Alivio Platform, with two others, LYT-503/IMB-150 and LYT-500, also developed using PureTech’s proprietary inflammation-targeting technology. PureTech will prioritize the development of LYT-510 ahead of LYT-500.

PureTech’s technology platforms, which include the Glyph, Alivio and Orasome- platforms, contribute to the Company’s advancement by generating new wholly-owned candidates (Glyph: LYT-300 and LYT-310) and enabling strategic partnerships (Alivio: LYT-503/IMB-150). As part of its pipeline prioritization, the Company may opportunistically enter into additional strategic partnerships or spinout programs into new entities around one or several of its platforms or related candidates, especially as resources are increasingly dedicated to late-stage clinical development.

Key Founded Entity Updates:

Karuna (Nasdaq: KRTX)

Announced positive topline Phase 3 data evaluating the efficacy, safety and tolerability of KarXT in adults with schizophrenia, meeting its primary endpoint and key secondary endpoints. Karuna plans to submit a New Drug Application (NDA) for KarXT in schizophrenia with the U.S. FDA in mid-2023, as of the most recent update by the company.

Announced the initiation of its Phase 3 ADEPT program, which is evaluating KarXT for the treatment of psychosis related to Alzheimer’s Disease (AD.)

Gelesis (NYSE: GLS)

Generated $36.8M in revenue and obtained 185,000 customers since the launch of Plenity.3

Announced that based on the extensive safety data and differentiation due to affordability, broad label and safety profile, it is preparing an application to the FDA to broaden the classification of Plenity in the U.S. from prescription-only to over the counter. Gelesis plans to submit the application to the FDA during the first quarter of 2023 and could receive market clearance by the third quarter of 2023.

Vor (Nasdaq: VOR)

o Announced initial clinical data from VBP101, Vor’s Phase 1/2a multicenter, open-label, first-in-human study of tremtelectogene empogeditemcel or "trem-cel" (formerly VOR33) in patients with AML. The data observed from the first treated patient support the potential of a trem-cel transplant to be successfully manufactured, to engraft normally and to maintain blood counts following treatment with the CD33-targeted therapy Mylotarg. The clinical trial continues to enroll patients and additional data are expected in 2023.

Akili (Nasdaq: AKLI)

Deployed first wave of its EndeavorRx4 go-to-market sales force in 14 priority territories across the U.S. with a focus on Integrated Behavioral Health Centers and pediatric providers.

Announced that Highmark, the fourth largest Blue Cross Blue Shield organization, included EndeavorRx in its medical policy for the reimbursement of prescription digital therapeutics.

Announced that its partner, Shionogi, started a pivotal Phase 3 randomized, controlled study of SDT-001 in children with attention-deficit hyperactivity disorder (ADHD).

Vedanta

Opened a new facility designed to manufacture clinical and commercial supply for its therapeutic portfolio, including for the planned Phase 3 study (expected to begin in the first half of 2023) and potential commercial launch of lead candidate, VE303, in Clostridioides difficile infection. Vedanta believes it was the first company to manufacture CGMP-grade defined bacterial consortia in powdered form, which enables stable, consistent oral formulations.

Board Composition & Committee Changes:

As noted in PureTech’s 2022 Half-Year Report, Dame Marjorie Scardino, Senior Independent Director, chair of the Nomination Committee and member of the Audit Committee, will retire as of the close of business on December 31, 2022. Raju Kucherlapati, Ph.D., will assume the role of PureTech’s Senior Independent Director as well as the chair of its Nomination Committee, effective as of Dame Scardino’s retirement.

Christopher Viehbacher, Chair of PureTech’s Board, was recently appointed President, Chief Executive Officer and a member of the Board of Biogen Inc. (Nasdaq: BIIB). Given the time commitment required by this new role, Mr. Viehbacher will not stand for re-election at the Company’s 2023 Annual General Meeting. The Nomination Committee will initiate a process to identify a new Chair.

Mr. Viehbacher noted: "It has been truly rewarding to work with such a talented group of people who share a genuine passion for helping patients. Together, we have developed a pharmaceutical pioneer delivering innovative medicines with a proven track record that is truly differentiated across the industry. The foundation for these successes is supported by a strong cash position and unique business model to support continued growth. I’m proud of these collective accomplishments and I look forward to continuing to interact with my colleagues at PureTech in this next exciting phase for the company."

Ms. Zohar commented: "For nearly a decade, Chris has brought a balance of enthusiasm and skepticism to the PureTech Board that is emblematic of our approach. His commercial expertise, deal-making ethos and patient-centered focus have helped to shape our mission and laid the foundation for our future growth. I am grateful that Chris will remain a trusted advisor, and we wish him much success as he takes the helm at Biogen."

Processa Pharmaceuticals Investor Presentation dated December 2022.

On December 22, 2022 Processa Pharmaceuticals presented its investor presentation (Presentation, Processa Pharmaceuticals, DEC 22, 2022, View Source [SID1234625549]).