On November 13, 2023 Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, reported its recent accomplishments and financial results for the quarter ended September 30, 2023 (Press release, Soligenix, NOV 13, 2023, View Source [SID1234637563]).

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"We continue our collaborative discussions with the U.S. Food and Drug Administration (FDA) regarding the design of a second, confirmatory Phase 3 pivotal study evaluating HyBryte (synthetic hypericin sodium) in the treatment of cutaneous T-cell lymphoma (CTCL), where we successfully demonstrated statistically significant results in the first Phase 3 clinical trial," stated Christopher J. Schaber, PhD, President and Chief Executive Officer of Soligenix. "We continue to actively enroll patients in the Phase 2a trial of SGX302 (synthetic hypericin sodium) for the treatment of mild-to-moderate psoriasis after demonstration of a clear biological signal in all five of the initial patients, with the majority recording an improvement in their PASI (psoriasis area and severity index) score. Under our Public Health Solutions business segment, we continue to advance our heat stable vaccine platform technology, ThermoVax. Most recently, we successfully demonstrated two year stability of thermostabilized bivalent and trivalent filovirus vaccine candidates at temperatures of 40 degrees Celsius (104 degrees Fahrenheit) when formulated in a single vial, needing reconstitution only with sterile water immediately prior to use. This follows the previous successful demonstration of 100% protection of non-human primates against lethal Sudan ebolavirus and Marburg marburgvirus challenge with the bivalent vaccine."

Dr. Schaber continued, "With approximately $10.3 million in cash at September 30, 2023, not including our non-dilutive government funding, we are managing cash burn very carefully in order to achieve our near-term milestones. We continue to evaluate a number of strategic options, including but not limited to, partnership and merger and acquisition opportunities."

Financial Results – Quarter Ended September 30, 2023

Soligenix’s revenues for the quarters ended September 30, 2023 were $0.1 million as compared to $0.2 million for the quarter ended September 30, 2022. Revenues primarily relate to government contracts and grants awarded in support of SGX943, our therapeutic candidate for treatment of emerging and/or antibiotic-resistant infectious diseases; and CiVax, our vaccine candidate for the prevention of COVID-19.

Soligenix’s net loss was $1.7 million, or ($0.16) per share, for the quarter ended September 30, 2023, as compared to $3.3 million, or ($1.15) per share, for the quarter ended September 30, 2022. The decrease in net loss was primarily due to decreases in operating expenses and interest expense and an increase in other income.

Research and development expenses were $0.8 million as compared to $1.8 million for the quarters ended September 30, 2023 and 2022, respectively. The decrease was primarily due to the decrease in manufacturing and regulatory costs associated with the HyBryte new drug application filing.

General and administrative expenses were $1.0 million and $1.3 million for the quarters ended September 30, 2023 and 2022, respectively. This decrease in general and administrative expenses is primarily attributable to a reduction in legal and consulting expenses associated with the arbitration against Emergent BioSolutions, Inc. and certain of its subsidiaries.

As of September 30, 2023, the Company’s cash position was approximately $10.3 million.

On November 13, 2023 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that it recently concluded a Type C meeting with the U.S. Food and Drug Administration (FDA) regarding the Company’s Chemistry, Manufacturing, and Controls (CMC) sections in a potential biologics license application ("BLA") for SELLAS’ lead product candidate, galinpepimut-S (GPS) (Press release, Sellas Life Sciences, NOV 13, 2023, View Source [SID1234637562]). SELLAS submitted a CMC Briefing Package to the FDA which provided an up-to-date overview of the extensive work completed for the GPS CMC program and commercial manufacturing and regulatory plans. The FDA reviewed this package of data and accompanying questions to the agency and responded with favorable guidance.

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To date, the Company has successfully completed numerous clinical development and CMC objectives in advancing GPS monotherapy into its Phase 3 REGAL study in patients with acute myeloid leukemia ("AML") in the maintenance setting after achievement of second complete remission. CMC activities in support of the clinical development leading to this Type C meeting include:

Manufacturing lyophilized clinical GMP batches;
Qualifying processes;
Validating analytical methods; and
Monitoring the stability program.
The FDA also agreed on the Company’s proposed stability data generation plan for the commercial presentation of GPS. GPS is expected to be stored at 2-8 C (36 – 46 F) making it more accessible for end-users.

Andrew Elnatan, Vice President of Regulatory Affairs, CMC & Quality at SELLAS, stated, "We are pleased with the positive outcome of the Type C meeting and FDA’s guidance on the advancement of the CMC plans as part of the GPS program development. The responses to questions regarding our proposed potency assay and manufacturing processes validation are aligned with our expectations and will help guide our plans towards a potential future successful BLA assuming positive data from the REGAL study."

"We look forward to completing our enrollment in the Phase 3 REGAL trial, outside of China, this month and working closely with the FDA as we continue to advance our GPS development program. GPS, through its unique mechanism of action and novel technology, has the potential to become the first immunotherapy, cancer vaccine to reach the market for AML. There are currently no FDA-approved drugs to address the high unmet medical need that exists in treating patients in the maintenance setting in second complete remission," said Angelos Stergiou, MD, ScD hc, President and Chief Executive Officer at SELLAS. "Our off-the-shelf therapy, GPS, was precisely and deliberately designed to target Wilms Tumor 1 and the multivalent approach of GPS targets 25 carefully selected WT1 epitopes, allowing it to be maximally immunogenic by eliciting both CD4 and CD8 immune responses. GPS has the potential to offer clinical benefit to this population of leukemia patients and the positive CMC regulatory feedback is a critical step in its development."

On November 13, 2023 Selecta Biosciences, Inc. (NASDAQ: SELB) (the Company) reported that it has merged with Cartesian Therapeutics, Inc., a clinical-stage biotechnology company pioneering RNA cell therapies for autoimmune diseases (Press release, Selecta Biosciences, NOV 13, 2023, View Source [SID1234637561]). In connection with the merger, Selecta announced a $60.25 million private financing led by Timothy A. Springer, Ph.D., member of the Selecta Board of Directors.

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With the cash from both companies at closing and the proceeds of the concurrent private financing, the combined company is expected to have over $110 million on hand to support the development of the Cartesian pipeline through the Phase 3 study of lead product candidate, Descartes-08, a potential first-in-class RNA-engineered chimeric antigen receptor T-cell therapy (rCAR-T) for the treatment of MG, as well as the advancement of additional RNA cell therapy programs.

Concurrent with the merger, the combined company has been renamed Cartesian Therapeutics, Inc. The Company’s Nasdaq ticker symbol will change to "RNAC" effective prior to the opening of trading tomorrow, November 14, 2023.

"We are thrilled to announce our merger with Cartesian, a true pioneer in the RNA cell therapy space," said Carsten Brunn, Ph.D., who will continue to serve as President and Chief Executive Officer of the combined company. "With several potential value-driving milestones expected in the near-term, including data from the ongoing Phase 2b study of Descartes-08 in MG expected in mid-2024, we are confident that this merger represents a significant opportunity for Selecta stockholders. Cartesian’s mission aligns seamlessly with Selecta’s commitment to advancing innovative therapies for the treatment of autoimmune diseases, and we look forward to working toward maximizing the potential of this robust pipeline and technology."

"RNA cell therapy has the potential to overcome the challenges of using conventional, costly DNA-engineered cell therapies to treat autoimmune diseases, including their toxicity and the need for preconditioning chemotherapy," said Murat Kalayoglu, M.D., Ph.D., Co-Founder and former Chief Executive Officer of Cartesian. "With a shared vision of bringing meaningful therapeutic options to patients with autoimmune diseases, we are confident that our novel approach can thrive under Carsten’s leadership."

Cartesian’s Portfolio and Proprietary RNA Armory Platform

Cartesian’s internally manufactured portfolio includes RNA cell therapies that are purposefully designed to be administered conveniently in an outpatient setting. Cartesian’s RNA-engineering approach has the potential to expand the reach of cell therapy to autoimmunity with potentially safer, potent, and less expensive therapies versus DNA analogs.

Cartesian’s proprietary technology platform, RNA Armory, is designed to enable precision control and optimization of engineered cells for diverse cell therapies leveraging multiple modalities, including autologous, allogeneic, and in vivo transfection. In addition, Cartesian’s wholly owned, state-of-the-art GMP manufacturing and internal research and development capabilities potentiates the optimization of processes in a rapid and iterative manner.

Cartesian’s wholly owned pipeline includes:

Descartes-08 is designed to be an autologous anti-BCMA rCAR-T. Descartes-08 is currently in clinical development for autoimmune diseases, including MG, a chronic autoimmune disorder that causes disabling muscle weakness and fatigue. Compared to conventional DNA-based CAR T-cell therapies, rCAR-T is designed to not require preconditioning chemotherapy, to have predictable and controllable pharmacokinetics, and to avoid the risk of genomic integration. Descartes-08 has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of MG.

Cartesian previously reported positive data from its Phase 2a study of 14 patients with MG who received six weekly infusions of Descartes-08 in the outpatient setting without preconditioning chemotherapy. In the study, the results of which were published in The Lancet Neurology, Descartes-08 was observed to be safe and well-tolerated and to induce deep and durable responses. Enrollment is ongoing in a Phase 2b randomized, double-blind, placebo-controlled trial (NCT04146051) in patients with MG, and topline results are expected in mid-2024.

Beyond MG, initiation of a Phase 2 study of Descartes-08 in patients with systemic lupus erythematosus, a chronic autoimmune disease that causes systemic inflammation which affects multiple organ systems, is expected in the first half of 2024. In addition, initiation of a Phase 2 ocular autoimmune basket study and a Phase 2 vasculitic autoimmune basket study is planned for mid-2024 and the second half of 2024, respectively.
Descartes-15 is designed to be a next-generation, autologous anti-BCMA rCAR-T. In preclinical studies, Descartes-15 was observed to be significantly more potent than Descartes-08. Cartesian plans to leverage its clinical observations to date from its Descartes-08 clinical program to inform the clinical strategy for Descartes-15 for the treatment of autoantibody-associated autoimmune diseases (AAAD).

Descartes-33 is designed to be an off-the-shelf (allogeneic) mesenchymal stem cell therapy (rMSC) for the treatment of AAAD. In preclinical studies, Descartes-33 was observed to induce potent degradation of disease-enabling neutrophil extracellular traps.

Management and Organization

The combined company will be led by Selecta’s Chief Executive Officer, Carsten Brunn, Ph.D., and current Chief Financial Officer, Blaine Davis, as well as several members of the legacy Cartesian team, including Metin Kurtoglu, M.D., Ph.D., as Chief Operating Officer, Milos Miljkovic, M.D., as Chief Medical Officer, Chris Jewell, Ph.D., as Chief Scientific Officer, and Emily English, Ph.D., as Vice President of Quality. Matthew Bartholomae, J.D., Selecta’s General Counsel, will continue to serve in this role.

The combined company’s Board of Directors will be led by current Selecta Chairman Carrie S. Cox and will include, among others, current Selecta board member Timothy Springer, Ph.D., as well as Cartesian Co-Founders Murat Kalayoglu, M.D., Ph.D., and Michael Singer, M.D., Ph.D. All members of the Selecta Board of Directors prior to the merger will continue to serve on the Board of Directors following the closing of the transaction.

Merger Terms

The merger was structured as a stock-for-stock transaction pursuant to which all of Cartesian’s outstanding equity interests were exchanged based on a fixed exchange ratio for consideration as a combination of approximately 6.7 million shares of Selecta common stock and approximately 0.38 million shares of Selecta Series A Non-Voting Convertible Preferred Stock ("Series A Preferred Stock") (or approximately 385 million shares on an as-converted-to-common basis). Concurrently with the acquisition of Cartesian, Selecta entered into a definitive agreement for a PIPE investment to raise $60.25 million in which the investors will be issued approximately 0.15 million shares of Series A Preferred Stock (or approximately 149.3 million shares on an as-converted-to-common basis) at a price of $403.46851 per share. Subject to approval of the Company’s stockholders, each share of Series A Preferred Stock will automatically convert into 1,000 shares of common stock, subject to certain beneficial ownership limitations. On a pro forma basis, based upon the number of shares of Selecta common stock and Series A Preferred Stock issued in the acquisition and prior to the private financing, stockholders of Selecta immediately prior to the acquisition will own approximately 26.9% of the Company on an as-converted basis immediately after giving effect to this transaction. The acquisition was approved by the Board of Directors of Selecta and the Board of Directors and stockholders of Cartesian. The closings of the transactions are not subject to the approval of Selecta stockholders. On an as-converted basis, assuming the approval of the Company’s stockholders of the conversion of the Series A Preferred Stock into common stock, and after accounting for these transactions, the total number of shares of Selecta common stock will be approximately 696.2 million.

In connection with the transactions, a transferrable contingent value right (a "CVR") will be distributed to Selecta stockholders and holders of Selecta’s warrants issued in 2022 (the "2022 Warrants") of record as of the close of business on December 4, 2023, but will not be distributed to holders of shares of common stock or Series A Preferred Stock issued to Cartesian or the PIPE investors in the transaction. Additionally, holders of Selecta’s warrants other than the 2022 Warrants will be entitled to receive CVRs when and if such warrants are exercised. Holders of the CVR will be entitled to receive certain cash payments from proceeds received by the Company, if any, from its legacy assets, including SEL-212, following the closing of the transaction.

Leerink Partners is serving as exclusive financial advisor and private placement agent to Selecta. Covington & Burling LLP is serving as legal counsel to Selecta. Foley Hoag LLP is serving as legal counsel to Cartesian.

Conference Call and Webcast

Selecta and Cartesian will host a conference call today, Monday, November 13, 2023, at 9:00 am ET to discuss the merger. To access the conference call, please dial (844) 845-4170 (local) or (412) 717-9621 (international) at least 10 minutes prior to the start time and ask to be joined into the Selecta Biosciences call. The live audio webcast, along with accompanying slides, can be accessed on the Events & Presentations section of Selecta’s website at View Source A replay of the webcast will be available for a limited time following the event on Selecta’s website.

On November 13, 2023 Roivant (Nasdaq: ROIV) reported its financial results for the second quarter ended September 30, 2023, and provided an update on the business (Press release, Roivant Sciences, NOV 13, 2023, View Source [SID1234637560]).

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"In the last few weeks, we announced a historic deal with Roche for the sale of Telavant for $7.25 billion. While we intend to be very thoughtful about capital deployment, we expect that the resulting cash will be sufficient to fund our programs through profitability, in addition to enabling other opportunities and investments," said Matt Gline, CEO of Roivant. "This was also another significant quarter for our clinical programs with a data readout from IMVT-1402’s Phase 1 SAD study and 300 mg MAD cohort. The data represent what we believe is the best-case scenario for our FcRn franchise and truly broadens the horizon for what is possible in the landscape of autoimmune therapies and for patients suffering from autoimmune diseases. We are excited about the recent progress and look forward to announcing additional clinical results for 1402 and brepocitinib in the final quarter of the calendar year. 2023 has continued to deliver on being an incredibly catalyst-rich year, and certainly Roivant’s biggest year yet."

Recent Developments

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Telavant: In October 2023, Roivant entered into a definitive agreement with Roche for the sale of Telavant. Roche will gain the rights to develop, manufacture and commercialize RVT-3101 in the US and Japan for the treatment of inflammatory bowel disease and potentially other diseases. Under the terms of the agreement, Roche will pay a purchase price of $7.1 billion upfront and a milestone payment of $150 million payable upon the initiation of a Phase 3 trial in ulcerative colitis. Roivant owns 75% of the issued and outstanding shares of common stock and preferred stock of Telavant and Pfizer owns the remaining 25%, in each case on an as-converted basis. Roivant’s net proceeds from the transaction are expected to be approximately $5.2 billion plus $110 million from the milestone payment. Regulatory filings in connection with the transaction have been submitted and the closing of the transaction remains on track for the fourth quarter of 2023 or the first quarter of 2024.

•
Immunovant: In September 2023, Immunovant announced initial data from the Phase 1 clinical trial evaluating the safety, tolerability, and pharmacodynamic profiles of IMVT-1402 in healthy adults showed that subcutaneously administered doses of IMVT-1402 produced dose-dependent reductions in Immunoglobulin G, with no statistically significant dose-related decrease in serum albumin or increase in LDL cholesterol, strengthening IMVT-1402 as a potential best-in-class neonatal fragment crystallizable receptor (FcRn) inhibitor. In October 2023, Immunovant announced the closing of an underwritten public offering and concurrent private placement offering of common stock yielding approximately $467 million in net proceeds to Immunovant, after deducting underwriting commissions and estimated offering expenses. Roivant owns approximately 55.2% of Immunovant as of November 3, 2023.

•
Dermavant: For the second quarter ended September 30, 2023, Roivant reported VTAMA net product revenue of $18.4M, representing a 28% gross-to-net yield for the quarter. As of November 2023, over 250,000 VTAMA prescriptions have been written by approximately 12,800 unique prescribers for psoriasis, based on IQVIA data. Coverage has been expanded to 137 million US commercial lives and includes coverage by all three of the top pharmacy benefit managers.

In October 2023, Dermavant reported that in adult patients, VTAMA showed positive results from a Phase 4 open-label trial for the treatment of intertriginous plaque psoriasis – 82.8% achieved an intertriginous Physician Global Assessment (iPGA) Score of 0 (clear) or 1 (almost clear) and ≥2-grade improvement from baseline at Week 12, demonstrating compelling efficacy. Additionally, Dermavant reported in adults and children down to two years of age with atopic dermatitis, VTAMA showed rapid and significant onset of pruritus (itch) relief as early as 24 hours after initial application.

•
Roivant: In September 2023, Roivant raised approximately $200 million in a follow-on offering. Roivant reported cash, cash equivalents and restricted cash of approximately $1.4 billion at September 30, 2023. Giving effect to Immunovant’s October 2023 follow-on offering and expected cash proceeds from the pending sale of Telavant (including one-time milestone), Roivant’s cash, cash equivalents and restricted cash would have been approximately $7.0 billion. The acquisition of Telavant is subject to customary closing conditions and is expected to close in the fourth quarter of 2023 or the first quarter of 2024.

Major Upcoming Milestones

•
Immunovant expects additional IMVT-1402 data from the 600 mg multiple-ascending dose cohort in November 2023. Additionally, for batoclimab: Top-line data from the Phase 3 clinical trial in MG are expected in the second half of calendar year 2024. For the Phase 3 program in TED, top-line data are expected in the first half of calendar year 2025. Immunovant also expects to have initial results from period 1 of the Phase 2B clinical trial in CIDP in the first half of calendar year 2024, and initial Phase 2 proof-of-concept data in Graves’ disease by the end of calendar year 2023.

•
Priovant plans to announce topline results from the potentially registrational trial evaluating brepocitinib for the treatment of patients with systemic lupus erythematosus (SLE) in the fourth quarter of calendar year 2023. Priovant also expects to announce topline results from the Phase 2 POC study in non-infectious uveitis (NIU) in the first quarter of calendar year 2024 and topline results from the Phase 3 trial in dermatomyositis (DM) in calendar year 2025.

•
Dermavant plans to submit its sNDA for VTAMA in atopic dermatitis to the FDA in the first quarter of calendar year 2024.

•
Hemavant plans to announce data from the ongoing open-label Phase 1/2 trial evaluating RVT-2001 for the treatment of transfusion-dependent anemia in lower-risk myelodysplastic syndromes (MDS) patients in the first quarter of calendar year 2024.

•
Kinevant plans to report topline data from the ongoing Phase 2 trial of namilumab for the treatment of sarcoidosis in the second half of calendar year 2024.

Second Quarter Ended September 30, 2023 Financial Summary

Cash Position

As of September 30, 2023, the Company had consolidated cash, cash equivalents and restricted cash of $1.4 billion.

Research and Development Expenses

Research and development expenses were $132.0 million for each of the three months ended September 30, 2023, and 2022. Changes in the components of research and development expenses included a decrease in personnel-related expenses of $5.4 million and increases in share-based compensation expense of $1.5 million and program-specific costs of $1.2 million.

Within program-specific costs, the primary drivers of change during the three months ended September 30, 2023 as compared to the three months ended September 30, 2022 were an additional expense of $18.6 million related to RVT-3101, which was acquired in November 2022, and a decrease in expenses related to other development and discovery programs of $18.2 million, which in part resulted from the deconsolidation of Proteovant in August 2023 along with the reprioritization of certain programs and drug discovery efforts.

Non-GAAP R&D expenses were $121.9 million for the three months ended September 30, 2023, compared to $123.3 million for the three months ended September 30, 2022.

Selling, General and Administrative Expenses

Selling, general and administrative expenses increased by $6.7 million to $164.4 million for the three months ended September 30, 2023, compared to $157.7 million for the three months ended September 30, 2022, primarily due to an increase in selling, general and administrative expenses of $21.8 million at Dermavant as a result of the progression of the commercial launch of VTAMA, partially offset by a decrease of $14.2 million of share-based compensation expense.

Non-GAAP SG&A expenses were $122.1 million for the three months ended September 30, 2023, compared to $101.5 million for the three months ended September 30, 2022. The majority of non-GAAP SG&A expenses were related to Dermavant’s SG&A and ongoing VTAMA commercial launch activities.

Net Loss

Net loss was $331.1 million for the three months ended September 30, 2023, compared to $315.9 million for the three months ended September 30, 2022. On a per common share basis, net loss was $0.40 for the three months ended September 30, 2023, and $0.42 for the three months ended September 30, 2022. Non-GAAP net loss was $225.4 million for the three months ended September 30, 2023, compared to $226.8 million for the three months ended September 30, 2022.

On November 13, 2023 Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of retinal and refractive eye disorders, reported financial results for the third quarter ended September 30, 2023 and provided a corporate update (Press release, Ocuphire Pharma, NOV 13, 2023, View Source [SID1234637559]).

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"We achieved important regulatory and clinical milestones in recent months, particularly in advancing oral APX3330 towards a registrational Phase 3 program in diabetic retinopathy," said Chief Executive Officer George Magrath, M.D., M.B.A., M.S., "We now have agreement with the FDA on the Phase 3 primary endpoint of 3-step worsening (measuring delay or prevention of progression) on a binocular Diabetic Retinopathy Severity Scale, and plan to finalize the protocol and statistical analysis plan with the FDA through a Special Protocol Assessment submission. If approved, APX3330 has the potential to be the first non-invasive, early treatment to delay or prevent vision-threatening complications in millions of patients with non-proliferative DR. We are very pleased to have recently received FDA approval of RYZUMVI (phentolamine ophthalmic solution) 0.75% eye drops for the reversal of pharmacologically induced mydriasis and look forward to our partner Viatris’ commercial launch in the first half of 2024. We are also excited to share that the VEGA-2 Phase 3 trial in presbyopia met its primary endpoint, and Viatris is expected to continue the Phase 3 development in the first half of 2024."

Key Anticipated Future Milestones

•
APX3330: Special Protocol Assessment ("SPA") submission and agreement with U.S. Food and Drug Administration ("FDA")

Recent Business Highlights

Clinical and Regulatory Updates

•
In October 2023, Ocuphire had a successful End-of-Phase 2 ("EOP2") meeting with the FDA for oral APX3330 in Diabetic Retinopathy and agreed on the Phase 3 primary endpoint of 3-step worsening (measuring delay or prevention of progression) on a binocular diabetic retinopathy severity scale. The Company plans to submit a SPA to the FDA to agree on the protocol and statistical analysis plan of the first Phase 3 trial.

•
In September 2023, Ocuphire and Viatris announced FDA approval of RYZUMVI (phentolamine ophthalmic solution) 0.75% for the treatment of pharmacologically induced mydriasis produced by adrenergic agonists (e.g., phenylephrine) or parasympatholytic agents (e.g., tropicamide). Ocuphire received a $10 million milestone payment from Viatris upon the approval. RYZUMVI is expected to be commercially available in the U.S. in the first half of 2024.

•
The VEGA-2 Phase 3 study evaluating phentolamine ophthalmic solution 0.75% in presbyopia achieved its primary endpoint. Viatris is expected to continue Phase 3 development for this indication in the first half of 2024.

•
The Company has submitted a SPA to the FDA for night vision disturbances or dim light vision and Viatris is expected to continue Phase 3 development of phentolamine ophthalmic solution 0.75% for this indication in the first half of 2024.

Corporate

On August 10, 2023, Ocuphire entered into a common share purchase agreement with Lincoln Park Capital Fund, LLC ("LPC"). Subject to the terms and conditions of the purchase agreement, Ocuphire has the right to sell, and LPC is obligated to purchase, up to $50 million of Ocuphire’s common shares over a 30-month period at prices per share as computed under the purchase agreement. Ocuphire, in its sole discretion, controls the timing and amount of all sales of common shares within a pre-specified range. There are no warrants or other share classes associated with the purchase agreement. Proceeds from share sales are expected to fund the future development of APX3330 and to be used for general corporate purposes.

Presentations, Publications, and Conferences

Beginning in the third quarter of 2023, several presentations at medical meetings featured previously announced data from the ZETA-1 Phase 2 trial of APX3330 in diabetic retinopathy. Highlights include:

•
A presentation at the Eyecelerator American Academy of Ophthalmology 2023 Retina Showcase by Jay Pepose, M.D. Ph.D., Chief Medical Advisor, highlighting the agreement of Phase 3 registration endpoint at the EOP2 FDA meeting.

•
A paper presentation delivered by Veeral Sheth, M.D., at the 23rd Euretina Congress in October 2023.

•
An oral presentation delivered by Anat Lowenstein, M.D., at the 56th Annual Retina Society Scientific Meeting in October 2023.

•
An oral presentation delivered by Priya Vakharia, M.D., at the Women in Ophthalmology Summer Symposium in August 2023. The abstract was rated as one of the top three scoring abstracts out of nearly 600 submissions and received the Joanne Angle Abstract of Distinction Award.

Third Quarter ended September 30, 2023, Financial Highlights

As of September 30, 2023, Ocuphire had cash and cash equivalents of approximately $42.4 million. Based on current projections, management believes the present cash on hand will be sufficient to fund operations into 2025.

License and collaborations revenue was $11.9 million and $17.4 million for the three and nine months ended September 30, 2023, respectively. There was no license and collaborations revenue during the three and nine months ended September 30, 2022. Revenue during the three and nine months ended September 30, 2023, was derived from the achievement of a $10.0 million milestone attributed to the FDA’s approval of RYZUMVI for reversal of mydriasis and from the reimbursement of research and development services under the License Agreement with Viatris in the amount of $1.9 and $7.4 million, respectively.

General and administrative expenses for the three and nine months ended September 30, 2023, were $2.1 million and $8.7 million, respectively, compared to $1.7 million and $5.2 million, respectively, for the three and nine months ended September 30, 2022. The increases from the comparable periods in 2022 were attributable to professional services and personnel related and other costs. General and administrative expenses included stock-based compensation expenses.

Research and development expenses for the three and nine months ended September 30, 2023, were $3.5 million and $13.8 million, respectively, compared to $2.8 million and $10.8 million, respectively, for the three and nine months ended September 30, 2022. The increases from the comparable periods in 2022 were primarily attributable to increased drug manufacturing, toxicology services and payroll and consulting related costs. Research and development expenses also included stock-based compensation expenses.

Income (loss) from operations for the three and nine months ended September 30, 2023, was $6.4 million and ($5.1) million, respectively, compared ($4.5) million and ($16.0) million, respectively, for the three and nine months ended September 30, 2022.

Net income (loss) for the three and nine months ended September 30, 2023, was $5.6 million and ($5.2) million, respectively, compared to ($4.5) million and ($16.1) million, respectively, for the three and nine months ended September 30, 2022. Basic net income (loss) per share for the three and nine months ended September 30, 2023, was $0.26 and ($0.25) per share, respectively, compared to ($0.22) and ($0.82) per share, respectively, for the three and nine months ended September 30, 2022.

For further details on Ocuphire’s financial results, refer to the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, to be filed with the Securities and Exchange Commission.

Conference Call and Webcast Details:

Date: December 5th, 2023
Time: 10:00 AM ET
Dial-in information: 1-877-407-4018 (US); 1-201-689-8471 (International); Call me
Passcode: 13742669
Webcast link

Participants can use Guest dial-in #s above and be answered by an operator OR click the Call me link for instant telephone access to the event. Call me link will be made active 15 minutes prior to scheduled start time.