Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. ANJESO is the first and only 24-hour, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO, Baudax Bio has a pipeline of other innovative pharmaceutical assets including two novel neuromuscular blocking agents (NMBs) and a proprietary chemical reversal agent specific to these NMBs. For more information, please visit www.baudaxbio.com.
Gregory Aurand, Senior Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.
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All patients met Good or Excellent intubating conditions. In the 2nd interim pre-planned analyis released Monday, March 27th, 41 patients from 4 cohorts (at least 10 from each) met Good or Excellent intubating conditions, with 40 patients intubated at 60 seconds and 1 patient at 90 seconds. Treatments were generally well tolerated, and while there were no serious events, one patient experienced a treatment-emergent adverse event that could possibly be related to treatment. Based on the analysis, none of the four cohorts (3 different dose groups of BX1000, and a standard dose group of rocuronium) are expected to be dropped nor is the full 80-patient trial enrollment number expected to be adjusted.
Results similar to the 1st interim analysis. In late January, Baudax Bio released the 1st interim analysis that showed the 20 patients (5 patients from each of the four cohorts) met the criteria for Good or Excellent intubating conditions with no adverse events. As a reminder, the primary endpoints assess the time frame needed to reach intubation conditions (time frame is within 2 minutes of administration) and also assess, using a standardized scale (Poor, Good, Excellent), the proportion of patients meeting Good or Excellent conditions. Secondary endpoints assess the safety and tolerability profile of BX1000.
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U.S. Food & Drug Administration (FDA) approves enrolling pediatric patients in the ongoing OCU400 Phase 1/2 trial who have: 1) RP associated with NR2E3 and RHO mutations and 2) LCA associated with CEP290 gene mutations
Ocugen has completed enrollment of adult RP patients with NR2E3 and RHO mutations in the Phase 1/2 trial and expanded enrollment in LCA patients with CEP290 mutations
MALVERN, Pa., March 27, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the FDA approved enrolling pediatric patients in the ongoing OCU400 Phase 1/2 trial.
“This approval moves us one step closer in our efforts to bring OCU400, a novel gene-agnostic modifier gene therapy, to market as a potential life-changing treatment for children afflicted with inherited retinal diseases, such as RP and LCA,” noted Arun Upadhyay, PhD, Ocugen’s Chief Scientific Officer. “This approval further demonstrates the consistent, positive, and timely progress we are making with the Phase 1/2 trial in adult patients. Since a significant number of individuals in the pediatric age group are diagnosed with RP and LCA, it is very important for us to cover this age group in our clinical trials.”
Enrollment of adult RP patients in the Phase 1/2 trial is complete—per protocol—and enrollment continues among patients with LCA. The Company plans to initiate the Phase 3 trial near the end of 2023.
Unlike single-gene replacement therapies, which only target one genetic mutation, Ocugen believes that its modifier gene therapy platform, through its use of Nuclear Hormone Receptors (NHRs), represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and potentially address complex diseases that are caused by imbalances in multiple gene-networks. While single-gene replacement therapies have shown tremendous promise in rare retinal diseases, they are highly specific and cannot improve a multitude of disease-causing genetic defects. For example, RP and LCA are associated with mutations in more than 100 and in more than 25 genes, respectively. Ocugen is the only company with a gene-agnostic modifier platform that aims to alter this single-gene therapy paradigm through the introduction of a functional gene to modify the expression of multiple genes and gene-networks. We believe that patient prevalence in the United States alone would provide significant long-term value, with RP and LCA affecting 110,000 and 15,000 people, respectively.
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its diverse functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.
About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.
Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact: Tiffany Hamilton Head of Communications IR@ocugen.com
BOTHELL, Wash., March 22, 2023 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) announces that James Martin, CFO and co-CEO, will present a company overview at the Virtual Investor Summit Conference being held on Wednesday, March 29, 2023 at 10:30 a.m. Eastern time.
“This is a highly eventful time a Cocrystal with significant advancements and upcoming milestones with our antiviral influenza A, COVID-19 and norovirus programs,” said Mr. Martin. “We look forward to sharing our progress with the many investors tuning into this event.”
Registration for the live and archived virtual presentation is available here and on the IR Calendar of the Cocrystal website.
About Cocrystal Pharma, Inc.
Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.
FLORHAM PARK, N.J., March 16, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, today announced that the Company will release financial results for the fourth quarter of 2022 on Tuesday, March 28, 2023, before the market opens. Following the release, management will host a conference call to review the financial results and provide a business update.
Tuesday, March 28, 2023, 8:00 AM EDT Domestic: 877-407-3088 International: 201-389-0927 Conference ID: 13736455 Webcast: PDS Biotech Earnings Webcast
After the live webcast, the event will be archived on PDS Biotech’s website for six months.
About PDS Biotechnology PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune®, Versamune® plus PDS0301, and Infectimune™ T cell-activating platforms. We believe our targeted immunotherapies have the potential to overcome the limitations of current immunotherapy approaches through the activation of the right type, quantity and potency of T cells. To date, our lead clinical candidate, PDS0101, has demonstrated the ability to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV16-associated cancers. Our Infectimune™ based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.
Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-15001 which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-29002 for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-8013, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-18504, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-35005 (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL6, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022. Finally, TNX-13007 is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.
Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
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Tonix Reported FY2022 and Gave Clinical Milestone Updates. Tonix reported a loss of $34.1 million or ($0.56) per share for 4Q22 and $116.9 million or $(3.27) per share for FY2022. The company reaffirmed its guidance for a 2Q23 announcement of interim analysis for TNX102 SL in the Phase 3 RESILIANT trial testing in fibromyalgia. The full analysis is expected in 4Q23, one of several clinical milestones expected in 2023. Tonix had $120.2 million in cash on December 31.
Interim Analysis Expected For Phase 3 in Fibromyalgia. The RESILIANT Phase 3 trial is a double-blind, placebo-controlled study for fibromyalgia. It reached the 50% patient milestone in December 2022, with the interim analysis results expected in 2Q23. If successful, the RESILIANT trial could lead to an NDA filing for product approval.
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MALVERN, Pa., March 06, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced the appointment of Quan A. Vu to Chief Financial Officer & Chief Business Officer.
“Quan has quickly become a tremendous asset for Ocugen and his comprehensive healthcare and financial expertise is providing valuable insight toward reaching our corporate goals,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “With Quan now overseeing both finance and business development, there will be a fully integrated approach to financial strategy—pursuing new partnerships and engaging with the investment community.”
Mr. Vu joined Ocugen on February 1, 2023, and has been working closely across multiple, key corporate functions—understanding short- and long-term business and financial needs of the Company along with the potential of its differentiated scientific platforms. With his additional appointment as Chief Financial Officer, and aside from his management and oversight of the finance and accounting functions, Mr. Vu will partner closely with Dr. Musunuri to enhance the Company’s forward-looking, value-additive financial initiatives by leveraging the Company’s anticipated clinical milestone achievements to bolster fundamental shareholder composition, expand interactions with the investor community, and ensure reputable capital markets and equity research support.
“I am excited about this new role and the ability to work alongside the leadership team to advance Ocugen at such a pivotal time,” said Mr. Vu. “We will continue to work collaboratively to position the Company for future success.”
Before joining Ocugen, Mr. Vu was most recently Chief Operating Officer/Chief Business Officer for 180 Life Sciences. He began his career in healthcare investment banking at both Morgan Stanley and Goldman Sachs, and then held leadership roles of increasing responsibility at Opiant Pharmaceuticals, Impax Laboratories, Anthem, and Amgen. Mr. Vu obtained his BA in Economics from UCLA, graduating summa cum laude with College Honors and Economics Departmental Honors. He is also a Certified Treasury Professional (inactive).
About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs.
Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact:
Tiffany Hamilton Head of Communications IR@ocugen.com
National Cancer Institute (NCI)-led research of PDS0301, a novel investigational tumor-targeting IL-12 fusion protein, shows dose-dependent, immune responses and association with improved clinical outcomes
FLORHAM PARK, N.J., March 01, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, announced that clinical research conducted by the NCI, one of the Institutes of the National Institutes of Health, was published in the peer-reviewed journal, International Immunopharmacology. The clinical study assessed immune changes in relation to the dose level and dosing schedule of PDS0301 (NHS-IL12), a novel investigational fusion protein consisting of a tumor-targeting antibody conjugated to Interleukin 12 (IL-12). The study also evaluated the correlation of several treatment-related immunological changes with clinical responses.
As described in the paper, titled, “Immune correlates with response in patients with metastatic solid tumors treated with a tumor-targeting immunocytokine NHS-IL12,” the researchers at the NCI evaluated a subset of 23 patients with advanced cancers who participated in a Phase 1 clinical trial of PDS0301. Patients were dosed every 2 weeks with PDS0301 at one of two levels – 12.0 mcg/kg and 16.8 mcg/kg – or every 4 weeks at 16.8 mcg/kg to identify dosing amounts and regimens of PDS0301 that correlate with higher levels of immune activation, including quantities of CD8 T cells, immune suppressive T regulatory cells, natural killer cells (NK) and natural killer T cells (NKT).
Patients receiving the higher dose of PDS0301 generated “a more robust immune activation compared to a lower dose,” including a greater expansion of NK, NKT, and CD8 T cells. Additionally, patients treated with the higher dose at two-week intervals had a greater response than patients receiving treatment every four weeks in the study. Importantly, greater increases were seen at the higher dose level in the serum pro-inflammatory cytokines IFNγ and TNFα, and soluble PD-1 (sPD-1). Studies found that increases in sPD-1 post-therapy have been associated with improved survival in various cancers and may indicate re-activation of CD8 T cells.
The ability to limit exposure of IL-12 in the circulating blood, and to increase its presence within the tumors constitutes a significant advancement in the development of cytokine-based immunotherapy. “The research published by the NCI demonstrates the potential of PDS0301 as a tumor-targeting IL-12 and its ability to stimulate immune activation and increase the frequency of CD8 T cells and certain NK cell subsets to potentially overcome the immunosuppressive tumor microenvironment,” stated Dr. Lauren V. Wood, Chief Medical Officer of PDS Biotech. “Importantly, this study appears to demonstrate the safety and tolerability of biologically active doses of PDS0301 and reports increases in specific immune cells that were associated with PDS0301 administration and improved clinical outcomes.”
The publication also summarizes clinical results from a study combining PDS0301, with a checkpoint inhibitor and PDS0101, PDS Biotech’s HPV-targeted immunotherapy that has been shown to promote induction of multifunctional, tumor-infiltrating killer T-cells. In this study in advanced HPV-positive anal, cervical, head and neck, vaginal, and vulvar cancers patients with checkpoint inhibitor refractory disease, 63% of patients who received the 16.8 mcg/kg dose had an objective response.
About PDS0301
PDS0301 is a novel investigational tumor-targeting Interleukin 12 (IL-12) that enhances the proliferation, potency and longevity of T cells in the tumor microenvironment. Together with Versamune® based immunotherapies PDS0301 works synergistically to promote a targeted T cell attack against cancers. PDS0301 is given by a simple subcutaneous injection. Clinical data suggest the addition of PDS0301 to Versamune® based immunotherapies may demonstrate significant disease control by shrinking tumors and/or prolonging survival in recurrent/metastatic cancers with poor survival prognosis.
About PDS Biotechnology
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune®, PDS0301, and Infectimune™ T cell-activating platforms. We believe our targeted Versamune® and PDS0301 based candidates have the potential to overcome the limitations of current immunotherapy approaches through the activation of the right type, quantity and potency of T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the ability to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV16-associated cancers in multiple Phase 2 clinical trials. Our Infectimune™ based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.
Forward Looking Statements
This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune® and Infectimune™ based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune® and Infectimune™ based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; the success of the Company’s license agreements, including the potential for the clinical and nonclinical data available under the Company’s exclusive license agreement with Merck KGaA to aid in the development of the Versamune® platform; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product, Covaxin, is a killed-virus vaccine for COVID-19 in-licensed from Bharat Biotech (India). The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.
Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Financial Results Were Within Expectations. Ocugen reported 4Q22 and FY2022 Net Loss of $21.9 million or $(0.10) per share, and $81.4 million or $(0.38) per share for FY2022. Cash on hand as of December 31 was $90.9 million or $0.41 per share, which management projects will fund operations through 1Q24. On its quarterly conference call, management discussed recent milestones and development programs in gene therapy, cellular therapy, and vaccines.
Ophthalmology Programs Are Advancing. Ocugen filed an IND for OCU200 to begin Phase 1 clinical trial in diabetic macular edema (DME), meeting the expected timeframe. Trial enrollment is expected to begin in 2Q23. A second IND filing is expected during 2Q23 for OCU410 in dry AMD and OCU410ST in Stargardt disease. The Phase 1/2 dose testing OCU400 in retinitis pigmentosa (RP) escalation phase previously completed enrollment for the RP cohort, with enrollment continuing in the Leber congenital amaurosis (LCA) patient cohort. Phase 3 is expected to start by year-end 2023.
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Continued progress for programs targeting eye diseases with the submission of an IND application for OCU200
Expanded portfolio now includes inhaled vaccines for COVID-19, seasonal flu, and a combination COVID-19+seasonal flu vaccine
MALVERN, Pa., Feb. 28, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today reported fourth quarter and full year 2022 financial results along with a general business update.
“We continue to grow and advance as a diversified biotechnology organization as reflected in our accomplishments of 2022,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “Our pipeline has been expanded to appropriately address the current challenges and gaps in the fight against COVID-19, application of OCU410 to address Stargardt (a rare eye disease), and our novel approach to address dry age-related macular degeneration (dAMD)—a disease affecting vision in over 266 million people worldwide.
“Following FDA concurrence in the fourth quarter of 2022 on a confirmatory Phase 3 clinical trial design for NeoCart®, we are developing internal capabilities to move our regenerative medicine asset, NeoCart®, into the clinic next year.”
“The FDA has granted expanded orphan drug designations to OCU400 for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA),” said Dr. Musunuri. “These broad, gene-agnostic designations are encouraging at this stage in the development of OCU400.”
“During our first decade, we have built a strong foundation for addressing the diseases and conditions we aim to treat. We delivered on our promise to file an OCU200 IND in the first quarter of 2023 and look forward to delivering on important milestones in 2023, especially regarding preliminary efficacy data for gene therapy product OCU400, as we progress toward realizing our long-term vision to address unmet medical needs through courageous innovation,” Dr. Musunuri concluded.
Business Updates
Ophthalmic Gene Therapies
OCU400 – Established the high dose as the maximum tolerable dose, completed retinitis pigmentosa patient enrollment, and continuing to enroll patients with LCA to receive the high dose. Ocugen intends to initiate a Phase 3 clinical trial near the end of 2023.
OCU410 and OCU410ST – Executing IND-enabling studies and intend to submit IND applications in the second quarter of 2023 to initiate Phase 1/2 clinical trials in dry AMD (geographic atrophy) and Stargardt disease.
Ophthalmic Biologic Product
OCU200 – Submitted an IND application on February 27, 2023 to initiate a Phase 1 clinical trial targeting diabetic macular edema.
Regenerative Cell Therapies
NeoCart® – Received concurrence from the FDA on the confirmatory Phase 3 clinical trial design. Ocugen intends to initiate the Phase 3 clinical trial in the first half of 2024. Ocugen is renovating its facility to accommodate cGMP manufacturing of NeoCart® for clinical trials and beyond.
Vaccines Portfolio
OCU500 Series – Developing a novel mucosal vaccine platform which includes OCU500, a bivalent COVID-19 inhaled vaccine; OCU510, a seasonal quadrivalent flu inhaled vaccine; and OCU520 a combination quadrivalent seasonal flu and bivalent COVID-19 inhaled vaccine.
COVAXIN™ (BBV152) – Completed enrollment in Phase 2/3 immuno-bridging and broadening clinical trial in fourth quarter 2022.
Financial Results
Fourth quarter — Research and development expenses for the three months ended December 31, 2022, were $17.2 million compared to $7.1 million for the three months ended December 31, 2021. General and administrative expenses for the three months ended December 31, 2022, were $6.9 million compared to $7.5 million for the three months ended December 31, 2021. Ocugen reported a $0.10 net loss per common share for the three months ended December 31, 2022, compared to a $0.07 net loss per common share for the three months ended December 31, 2021.
Full year — Research and development expenses for the year ended December 31, 2022, were $49.8 million compared to $35.1 million for the year ended December 31, 2021. General and administrative expenses for the year ended December 31, 2022, were $35.1 million compared to $22.9 million for the year ended December 31, 2021. Ocugen reported a $0.38 net loss per common share for the year ended December 31, 2022, compared to a $0.30 net loss per common share for the year ended December 31, 2021.
Ocugen’s cash, cash equivalents, restricted cash, and investments totaled $90.9 million as of December 31, 2022, compared to $95.1 million as of December 31, 2021. The Company estimates that its current cash, cash equivalents, and investments will enable it to fund its operations into the first quarter of 2024. The Company had 221.6 million shares of common stock outstanding as of December 31, 2022.
Conference Call and Webcast Details
Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights. Ocugen’s senior management team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.
Attendees are invited to participate on the call or webcast using the following details:
Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers Conference ID: 8912239 Webcast: Available on Ocugen’s investor site
A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.
About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs.
Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact: Tiffany Hamilton Head of Communications IR@ocugen.com
MALVERN, Pa., Feb. 27, 2023 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that it has submitted an Investigational New Drug application (IND) with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1 clinical trial of OCU200, a fusion protein with a distinct mechanism of action (MOA), for the treatment of diabetic macular edema (DME). This regulatory milestone fulfills the Company’s commitment to file the IND for OCU200 within the first quarter of 2023.
“Today’s achievement is an important step towards fulfilling our mission to bring novel therapeutics to address limitations of the current standard of care or unmet medical needs in hard-to-treat blindness diseases,” said Dr. Arun Upadhyay, Chief Scientific Officer at Ocugen. “We are encouraged by the potential for OCU200 to provide a new treatment option for the significant percentage of people living with DME, including non-responders to the current standard of care.”
The planned Phase 1 clinical study will assess the unilateral intravitreal administration of OCU200 alone or in combination with an approved anti-VEGF therapy in participants with DME. This is a multicenter, open-label, dose-ranging study with 3 cohorts in the dose-escalation portion of the study and 1 cohort in the combination therapy portion of the study.
DME is one of the most common vision-threatening diseases occurring in people with diabetes and includes blurriness in vision and progressive vision loss as the disease progresses. Approximately 745,000 people in the United States are affected with DME, and this number is expected to further increase as the number of people with diabetes increases.
The Company intends to pursue additional indications for OCU200 to potentially treat diabetic retinopathy and wet age-related macular degeneration, which combined affect nearly 9.0 million Americans.
About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.
About OCU200 OCU200 is a novel fusion protein consisting of human transferrin linked to human tumstatin. It exerts anti-proliferative, anti-inflammatory, and anti-oxidative effects by selective targeting to the retinal and choroidal tissues. OCU200 potentially showcases better bioavailability and tissue penetrance than tumstatin alone due to transferrin and provides distinct MOA binding through αVβ3 integrin pathways that can potentially reduce the number of injections for patients.
OCU200 can potentially be used for the treatment of diabetic macular edema, diabetic retinopathy, and wet age-related macular degeneration. These diseases, combined, account for approximately 10 million cases in the U.S.
Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact: Tiffany Hamilton Head of Communications IR@ocugen.com
Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. ANJESO is the first and only 24-hour, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO, Baudax Bio has a pipeline of other innovative pharmaceutical assets including two novel neuromuscular blocking agents (NMBs) and a proprietary chemical reversal agent specific to these NMBs. For more information, please visit www.baudaxbio.com.
Gregory Aurand, Senior Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.
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Baudax Bio reported 4Q 2022. While we had removed 4Q ANJESO revenues with the announced ANJESO discontinuation at the end of December, the Company reported 4Q revenues of $0.310 million in the quarter. Net loss per share for 4Q 2022 was $12.33 per share as compared to our outlook of $9.76 per share loss as the Company took additional write-offs related to ANJESO.
Baudax Bio reported full year 2022. For the year 2022, Baudax Bio generated revenues of $1.27 million, up from $1.08 million in the 2021 year. The Company took a write-down for impairment of intangibles of $19.7 million. The full year impairment write-down for property and equipment totaled $4.2 million. The reported full year 2022 net loss was $58.8 million or a loss of $177.30 per share compared to our full year 2022 expected loss of $55.3 million or $189.44 per share.
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Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
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Webinar. BIO (Biotechnology Innovation Organization, an industry group) sponsored a webinar titled, “Long COVID: What Will It Take To Accelerate Therapeutic Progress?” Speakers included officials from government, academia, and drug development companies to discuss the scientific basis of the condition, its public health implications, and its potential treatments. Presenting companies included Axcella Health (AXLA) and Tonix Pharmaceuticals (TNXP).
Long COVID Symptoms and Prevalence. The program began with how Long COVID became recognized as a condition, its incidence, and its patient population. Scientific presentations showed the biological effects of the SARS-CoV-2 virus on cells in the body, including the connection between Long COVID and other chronic conditions such as CFS/ME (chronic fatigue syndrome/myalgic encephalomyelitis). Symptoms and their duration can vary widely, including inflammation, fatigue, organ dysfunction, and reactivation of latent viral reservoirs. The presenters agreed that the range of symptoms and their variability will require numerous drugs.
Axcella Health Presented AXA1125. Chief Scientific Officer at Axcella Health, Dr. Margaret Koziel, presented the scientific basis for the use of AXA1125 in Long COVID, including its effects on inflammation, mitochondrial dysfunction, and fatigue. Data from the Phase 2a trial was presented, showing improvements in function, and biomarkers that correlate with reductions in inflammation and mitochondrial dysfunction. The design of the Phase 2b/3 trial was also presented.
Tonix Pharmaceuticals Presented Data From TNX-102 SL. Tonix is currently enrolling patients in its Phase 2 PREVAIL trial testing TNX-102 SL, sublingual cyclobenzaprine, in Long COVID. This trial is based on common symptoms with fibromyalgia and chronic fatigue syndrome, such as pain, fatigue, and insomnia. Projected enrollment is about 470 patients with a primary endpoint of change in daily pain score from baseline over the 14-week treatment period.
Conclusion. The BIO webinar speakers agreed that the high variability and large population would require several drugs to adequately treat patients. For Axcella, we believe the presentation could raise awareness of AXA1125 and its “Phase 2b/3 Ready” status, allowing it obtain trial funding through partnerships, government funding, or from investors. Tonix was able to present how TNX-102 SL overlaps with other pain syndromes and how it could treat symptoms, as well as to highlight its clinical milestones in the coming year.
Summary. BIO (Biotechnology Innovation Organization, and industry group) sponsored a webinar titled, “Long COVID: What Will It Take To Accelerate Therapeutic Progress?” Speakers included officials from government, academia, and drug development companies to discuss the scientific basis of the condition, its public health implications, and its potential treatments. Presenting companies included Axcella Health (AXLS) and Tonix Pharmaceuticals (TNXP).
The program began with the history of Long COVID, how it became recognized as a condition, and its prevalence. Scientific presentations discussed the SARS-CoV-2 viral infection and its effects on different organs, as well as the mechanisms of infection that lead to fatigue, cerebral effects (brain fog), and chronic pain. The speakers pointed out that the range and severity of symptoms can vary widely, making it likely that numerous drugs will be needed.
The overlap between Long COVID and other chronic conditions, such as CFS/ME (chronic fatigue syndrome/myalgic encephalomyelitis), was also discussed. The common features in these conditions were cited in the “National Research Action Plan on Long COVID”, published by the Department of Health and Human Services in August 2022.
Axcella Health Spoke About AXA1125. Chief Scientific Officer at Axcella Health, Dr. Margaret Koziel, presented the scientific basis for the use of AXA1125 in Long COVID, including its effect on inflammation, mitochondrial dysfunction, epithelial dysfunction, and fatigue. Data from the Phase 2a trial was presented, showing improvements in physical and mental function. These data included biomarkers that correlate with improvements in inflammation and mitochondrial function. The design of the Phase 2b/3 trial was also presented.
Tonix Pharmaceuticals Presented Data From TNX-102 SL. Tonix is currently enrolling patients in its Phase 2 PREVAIL trial testing TNX-102 SL, sublingual cyclobenzaprine, in Long COVID. This trial is based on common symptoms with fibromyalgia and chronic fatigue syndrome, such as pain, fatigue, and insomnia. Projected enrollment is about 470 patients with a primary endpoint of change in daily pain score from baseline over the 14-week treatment period.
TNX-102 SL showed positive results in its Phase 3 RELIEF study in fibromyalgia, although the confirmatory Phase 3 RALLY missed its primary endpoint. After analyzing the RALLY trial, the Phase 3 RESILIENT trial began. An interim analysis from the RESILIENT trial is expected in 2Q23.
Conclusion. The BIO webinar gave different perspectives on Long COVID and discussed different approaches to treatment. For Axcella, we believe the presentation could raise awareness of AXA1125 data and its “Phase 2b/3” ready status, allowing it obtain trial funding through partnerships, government funding, or from investors. For Tonix, presentations on the common features of Long COVID and chronic pain syndromes support the use of Tonix’s TNX-102 SL, and show how its Phase 2 PREVAIL clinical trial design could become an effective treatment.
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Senior Equity Analyst focusing on Basic Materials & Mining. 20 years of experience in equity research. BA in Business Administration from Westminster College. MBA with a Finance concentration from the University of Missouri. MA in International Affairs from Washington University in St. Louis. Named WSJ ‘Best on the Street’ Analyst and Forbes/StarMine’s “Best Brokerage Analyst.” FINRA licenses 7, 24, 63, 87
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MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
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THIO Is A First-In-Class Cancer Therapeutic With A Dual Mechanism Of Action. MAIA Biotechnology is in Phase 2 development with THIO, a drug directed at the telomeres of cancer cell chromosomes. These are structures at the distal tips of the chromosomes that protect the coding DNA regions and have functions needed for DNA replication. THIO is a modified nucleoside analogue that targets the chromosome’s telomeres, leading to cell death, and stimulates the immune system to attack remaining cancer cells.
THIO Is Phase 2 With A Checkpoint Inhibitor. The direct killing and stimulation of an immune response increases the number of immune killing cells in the tumor that can attack remaining cancer cells. Preclinical studies testing THIO in combination with checkpoint inhibitors showed complete killing and long-term durability of its effects. The current Phase 2 clinical trial program is testing THIO in combination with Libtayo, a PD-1 inhibitor from Regeneron, in non-small cell lung cancer (NSCLC).
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