Category: Health

Release – Ocugen, Inc. Announces Positive Scientific Advice From The European Medicines Agency Related To The Approval Pathway For OCU400—Modifier Gene Therapy For Broad Retinitis Pigmentosa Indication

Posted on by [email protected]

Research News and Market Data on OCGN

April 10, 2024

MALVERN, Pa., April 10, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, and vaccines, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA).

EMA provided this opinion based on safety and tolerability of OCU400 demonstrated in the Phase 1/2 study. The Phase 3 liMeliGhT study will have a sample size of 150 participants primarily in the U.S.—one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic (representing multiple gene mutations associated with RP). In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.

The positive scientific advice from EMA is in alignment with U.S. FDA clearance of the IND amendment to initiate the Phase 3 liMeliGhT clinical trial of OCU400. OCU400 is the first gene therapy to enter Phase 3 with a broad RP indication. Previously, OCU400 received broad Orphan Drug Designation for RP and Leber congenital amaurosis in the EU.

“We are very grateful to EMA for their collaborative discussions and support in providing a gene-agnostic therapeutic option to RP patients with severe unmet medical need,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “This positive opinion is a critical step in providing our game-changing modifier gene therapies to patients globally.”

The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. With this milestone, OCU400 remains on track for 2026 BLA and MAA approval targets.

About OCU400
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 drive 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 RP
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100 genes.

There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene-replacement therapies are promising but are limited to treating just a single mutation. In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retina degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches targeting broader RP disease in a gene agnostic manner offer greater hope for patients.

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 X and LinkedIn.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, 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, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. 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
[email protected]

Release – Unicycive Therapeutics to Present At The Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference

Posted on by [email protected]

Research News and Market Data on UNCY

April 10, 2024 7:03am EDT

LOS ALTOS, Calif., April 10, 2024 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company or “Unicycive”), today announced that Shalabh Gupta, M.D., Chief Executive Officer will present a company update at the Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference on Thursday April 18, 2024 at 11:00 a.m. ET.

A link to the live and archived webcast may be accessed on the Unicycive website under the Investors section: Events and Presentations. An archive of the webcast will be available for three months.

About Unicycive Therapeutics

Unicycive Therapeutics is a biotechnology company developing novel treatments for kidney diseases. Unicycive’s lead drug candidate, oxylanthanum carbonate (OLC), is a novel investigational phosphate binding agent being developed for the treatment of hyperphosphatemia in chronic kidney disease patients on dialysis. UNI-494 is a patent-protected new chemical entity in clinical development for the treatment of conditions related to acute kidney injury. For more information, please visit Unicycive.com and follow us on LinkedIn and YouTube.

Investor Contact:

[email protected]
(650) 543-5470

SOURCE: Unicycive Therapeutics, Inc.

Released April 10, 2024

Release – Cadrenal Therapeutics Receives FDAOrphan Drug Designation For Tecarfarin For Prevention Of Thromboembolism And Thrombosis In Patients With LVADs, RVADs, Biventricular Assist Devices, And Total Artificial Hearts

Posted on by [email protected]

Research News and Market Data on CVKD

FDA designation provides potentially seven years of market exclusivity after approval and expanded partnering opportunities for tecarfarin

PONTE VEDRA, Fla., April 9, 2024 — Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage novel oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with rare cardiovascular conditions, announced today that the United States Food and Drug Administration (FDA) has granted tecarfarin Orphan Drug Designation (ODD) for the prevention of thromboembolism and thrombosis in patients with an implanted mechanical circulatory support device (left ventricular assist device (LVAD), right ventricular assist device (RVAD), collectively known as ventricular assist devices (VADs), biventricular assist device, and total artificial heart).



“This second orphan drug designation highlights the expanded need for tecarfarin where existing anticoagulation therapies are inadequate,” said Quang Pham, Founder, Chairman and Chief Executive Officer of Cadrenal Therapeutics. “We are dedicated to advancing tecarfarin through clinical development options as swiftly as possible.”

The FDA’s ODD program provides incentives to sponsor organizations for the development of innovative treatments for rare diseases that affect fewer than 200,000 people in the U.S. Since its adoption in 1983, the Orphan Drug Act has helped countless individuals living with these conditions gain access to life-enhancing and life-saving therapies. ODD also provides certain benefits to drug developers, including assistance in the drug development process, tax credits for certain clinical research, and a waiver of the New Drug Application user fee. The designation is made to promote safe and efficacious products for the treatment of rare conditions.

All patients with VADs require chronic anticoagulation to prevent the formation of thrombus (clot) which can cause the device to fail or can result in a clot breaking off (embolizing), resulting in a stroke or other vascular catastrophe.

The current market-leading direct oral anticoagulants (DOACs), such as Eliquis, are not indicated for patients with VADs due to a lack of evidence of benefit. Moreover, a recent study revealed that the level of anticoagulation achieved with warfarin, the only currently available Vitamin K Antagonist (VKA), is maintained in the target range only 56% of the time which has been shown to increase the risk of clotting and bleeding complications.

ABOUT CADRENAL THERAPEUTICS, INC.

Cadrenal Therapeutics is developing tecarfarin for unmet needs in anticoagulation therapy. Tecarfarin is a late-stage novel oral and reversible anticoagulant (blood thinner) to prevent heart attacks, strokes, and deaths due to blood clots in patients with rare cardiovascular conditions. Tecarfarin has orphan drug and fast-track designations from the FDA for the prevention of systemic thromboembolism (blood clots) of cardiac origin in patients with end-stage kidney disease (ESKD) and atrial fibrillation (AFib) and just received orphan drug designation for the prevention of thrombosis and thromboembolism in patients with ventricular assist devices (VADs). Cadrenal is also pursuing additional regulatory strategies for unmet needs in anticoagulation therapy for patients with thrombotic antiphospholipid syndrome (APS). Tecarfarin is specifically designed to leverage a different metabolism pathway than the oldest and most commonly prescribed Vitamin K Antagonist (warfarin). Tecarfarin has been evaluated in eleven (11) human clinical trials and more than 1,000 individuals. In Phase 1, Phase 2, and Phase 2/3 clinical trials, tecarfarin has generally been well-tolerated in both healthy adult subjects and patients with chronic kidney disease. For more information, please visit www.cadrenal.com.

Safe Harbor Statement

Any statements contained in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding tecarfarin addressing unmet needs in anticoagulation therapy and the ability to derive the anticipated and potential benefits from the recent Orphan Drug Designation for tecarfarin, including seven years of market exclusivity, and the expanded development and commercial partnering for tecarfarin as a result of the Orphan Drug Designation. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the Company’s ability to derive the anticipated benefits from the recent Orphan Drug Designation for tecarfarin and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s filings with the Securities and Exchange Commission, including periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.

For more information, please contact:

Cadrenal Therapeutics:
Matthew Szot, CFO
858-337-0766
[email protected]

Investors:
Lytham Partners, LLC
Robert Blum, Managing Partner
602-889-9700
[email protected]

View original content to download multimedia:https://www.prnewswire.com/news-releases/cadrenal-therapeutics-receives-fda-orphan-drug-designation-for-tecarfarin-for-prevention-of-thromboembolism-and-thrombosis-in-patients-with-lvads-rvads-biventricular-assist-devices-and-total-artificial-hearts-302111758.html

SOURCE Cadrenal Therapeutics, Inc.

Ocugen (OCGN) – Phase 3 IND Clearance Is Good News We’ve Been Waiting For

Posted on by [email protected]


Tuesday, April 09, 2024

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Phase 3 Trial Is Expected to Start Shortly. Ocugen announced that its amended IND for the OCU400 Phase 3 clinical trial has been cleared by the FDA. This allows the clinical trial to start enrolling patients, consistent with previous guidance for an April 2024 start. The company now has 3 clinical trials in progress in its ophthalmic disease programs.

Phase 3 Could Start Immediately. The trial is designed to compare patients treated with OCU400 to untreated controls. There are two arms, one enrolling patients with the RHO mutation and a “gene agnostic” arm enrolling patients who may have any of the mutations associated with RP. Each arm will randomize patients at 2:1, with 50 in each treatment group and 25 in each control group for a total enrollment of 150 patients. Patients will be treated with a single injection of OCU400.


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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Release – Ocugen, Inc. Announces U.S. FDA Clearance of IND Amendment to Initiate OCU400 Phase 3 Clinical Trial — First Gene Therapy to Enter Phase 3 with a Broad Retinitis Pigmentosa Indication

Posted on by cpatches

Research News and Market Data on OCGN

MALVERN, Pa., April 08, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, and vaccines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) amendment to initiate a Phase 3 clinical trial of OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).

“The initiation of the OCU400 Phase 3 clinical trial is a significant milestone for patients with RP and a pivotal event for Ocugen as a company,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “OCU400 is the first gene therapy program to enter Phase 3 with a broad RP indication. Until now, there has been only one marketed product to treat one of the 100 gene mutations associated with RP. Now there is real hope for all RP patients who haven’t had a treatment option.”

The Phase 3 study will have a sample size of 150 participants—one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene agnostic. In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.

In the Phase 1/2 OCU400 clinical trial, a Multi-Luminance Mobility Testing (MLMT) scale was the primary functional endpoint. For the Phase 3 OCU400 clinical trial, an updated mobility course will be used—Luminance Dependent Navigation Assessment (LDNA)— that includes a wider range of light intensity (0.04-500 Lux) and Lux Levels (0-9) with a uniform correlation between Lux level and Lux intensity.

“A sensitive mobility course, LDNA, was developed by Ocugen in collaboration with FDA for the Phase 3 clinical trial to allow enrollment of patients with early to advanced stages of disease,” said Dr. Arun Upadhyay, Chief Scientific Officer at Ocugen. “We are extremely encouraged that with this Phase 3 study design more than 50% of intent to treat RHO patients would meet the responder criteria, which is demonstrating 2 or greater Lux level of improvement after one year of treatment based on Phase 1/2 study results.”

Currently there are approximately 110,000 patients in the United States with RP and 1.6 million patients globally. Of these patients, more than 10% have the RHO genetic mutation.

“We believe that the gene-agnostic clinical trial design provides an appropriate therapeutic option to include patients who have greater potential of benefiting from treatment,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “We are looking forward to working with our selected trial sites and leading retinal surgeons to deliver this novel modifier gene therapy to potentially address unmet medical needs.”

Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA. With the initiation of the Phase 3 clinical trial, OCU400 remains on track for the 2026 BLA approval target.

About OCU400
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 drive 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 RP
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100 genes.

There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene-replacement therapies are promising but are limited to treating just a single mutation. In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retina degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches targeting broader RP disease in a gene agnostic manner offer greater hope for patients.

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 X and LinkedIn. 

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, 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, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. 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
[email protected] 

Release – Maia Biotechnology To Present At Two Investor Conferences In April 2024

Posted on by [email protected]

Research News and Market Data on MAIA

April 05, 2024 8:01am EDTDownload as PDF

CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that Chairman and Chief Executive Officer Vlad Vitoc, M.D. will present at two investor conferences during the month of April: the 14th Annual LD Micro Invitational taking place April 8-9, 2024, in New York City; and the Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference taking place April 17-18, 2024.

LD Micro:

Presentation:04:30PM ET on April 09, 2024*
Format:In-person and virtual
Location:Sofitel New York, New York City
Conference registration:Visit the conference website
Livestream registration:Visit Sequire Events
1×1 meetings:Upon request by registered investors

Noble Capital Markets:

Presentation:11:00AM ET on April 17, 2024*
Format:Virtual
Conference registration:Visit the conference website
1×1 meetings:Upon request by registered investors

*Please note that the presentation date and time are subject to change. Participants should refer to the final program agendas for up-to-date information.

MAIA’s lead candidate is THIO, a small molecule telomere-targeting anticancer agent that acts by producing direct telomeric DNA damage and inducing cancer-specific immune responses. THIO’s efficacy in non-small cell lung cancer (NSCLC) is being evaluated in THIO-101, a Phase 2 go-to-market clinical trial nearing completion, which is expected to be the first completed clinical study of a telomere-targeting agent in the field of cancer drug discovery and treatment. MAIA plans to pursue the FDA’s accelerated approval program for THIO.

Recent news from MAIA’s THIO-101 trial includes:

About MAIA Biotechnology, Inc.

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.

Forward Looking Statements

MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240405374193/en/

Investor Relations Contact
+1 (872) 270-3518
[email protected]

Source: MAIA Biotechnology, Inc.

Released April 5, 2024

Release – Ocugen Announces Positive Data And Safety Monitoring Board Review And Initiation Of Enrollment In Medium Dose For OCU410—A Modifier Gene Therapy—In Phase 1/2 Armada Study For Geographic Atrophy

Posted on by [email protected]

Research News and Market Data on OCGN

April 5, 2024

  • Established Low Dose as Safe and Tolerable Dose in Current OCU410 Clinical Trial
  • DSMB Approval to Proceed with Medium Dose Cohort Dosing

Malvern, Pa., April 05, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Data and Safety Monitoring Board (DSMB) for the Phase 1/2 ArMaDa clinical trial for OCU410 recently convened and approved to proceed dosing with the medium dose of OCU410 in the dose-escalation phase of the study.

Three subjects with geographic atrophy (GA) were dosed in the Phase 1/2 clinical trial to date. An additional three subjects will be dosed with the medium dose (Cohort 2) and three patients with the high dose (Cohort 3) of OCU410 in the dose-escalation phase.

“The DSMB has recommended moving forward to medium dose for dosing subjects with GA,” said Dr. Peter Chang, MD, FACS, DSMB Chair for the OCU410 clinical trial. “No serious adverse events (SAEs) related to OCU410 have been reported to date. I believe that this marks a critical next step towards determining the optimal dosing regimen and an important milestone for the clinical development of OCU410.”

“The positive DSMB review for the first modifier gene therapy for GA significantly builds on the favorable safety and tolerability profile exhibited by OCU410,” said Huma Qamar, M.D., MPH, Chief Medical Officer of Ocugen. “We are very enthusiastic about the potential of OCU410 as a one-time treatment for life with a single sub-retinal injection.”

Currently approved products to treat GA have significant limitations, as they require multiple injections per year and only target one pathway contributing to GA. OCU410 regulates multiple pathways involved with the disease including: lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement).

The ArMaDa clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group.

Ocugen is committed to finding solutions for inherited retinal diseases as well as blindness diseases affecting millions. GA is an advanced form of dry age-related macular degeneration (dAMD) and affects approximately 1 million people in the United States.

About dAMD
dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula. The macula is the part of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population.

About OCU410
OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies 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 X 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
[email protected] 

Johnson & Johnson Flexes Its M&A Muscle with $12.5 Billion Shockwave Medical Buy

Posted on by [email protected]

Healthcare giant Johnson & Johnson announced on Friday that it is acquiring Shockwave Medical for a whopping $12.5 billion in cash, in a move that further bolsters its cardiovascular device portfolio. The deal allows J&J to add Shockwave’s innovative intravascular lithotripsy (IVL) system to its offerings.

IVL is a minimally invasive technique that uses sonic pressure waves to crack calcified plaque in arteries prior to inserting stents – similar in concept to how shockwaves are used to break up kidney stones. This novel approach helps improve outcomes for certain challenging arterial calcification cases that traditional treatment can struggle with.

Under the terms of the agreement, J&J will pay $335 per share for Shockwave, representing a 17% premium over the company’s stock price in late March when acquisition rumors first surfaced. The total enterprise value of the transaction is approximately $13.1 billion when including the cash on Shockwave’s balance sheet.

The acquisition comes hot on the heels of J&J’s $16.6 billion purchase of heart pump maker Abiomed last year, as the company doubles down on expanding its cardiovascular capabilities. Analysts see significant opportunity in this space, with RBC estimating the total addressable market for IVL and similar calcified plaque treatments at around $10 billion annually.

For Shockwave, being acquired by the deep-pocketed J&J provides the resources to ramp up commercialization of its breakthrough IVL system, which generated $730 million in sales last year. Meanwhile, the deal aligns with J&J’s strategic efforts to augment its medical device segment amid increasing competitive pressures in its pharmaceutical arm.

The Shockwave acquisition exemplifies a broader trend of large healthcare conglomerates snapping up promising smaller companies and technologies to drive future growth. With organic drug pipelines drying up and patent expirations looming, “big pharma” players are turning to M&A to inject innovation into their product portfolios.

Just last week, pharma giant AbbieVie announced multi-million dollar buyouts of smaller biotech firm Landos. Earlier this year, AstraZeneca shelled out $2.4 billion for oncology innovator Fusion Pharmaceuticals.

For investors interested in identifying the next potential M&A targets in healthcare’s hot growth areas, one upcoming event to mark on the calendar is the Noble Capital Markets Emerging Growth Virtual Healthcare Conference on April 17-18. This two-day virtual investor conference will feature presentations from emerging public and private healthcare companies spanning biotech, medical devices, healthcare IT and services. You can register at no cost for this event here.

The Noble virtual conference provides an ideal opportunity for institutional investors, financial advisors and independent investors alike to gain insights into cutting-edge healthcare innovations that could be tomorrow’s M&A prizes for industry titans like J&J. Presenting companies will span an array of therapeutic areas including oncology, neurology, xenotransplantation and more.

As the Shockwave deal demonstrates, big pharma isn’t shying away from spending big to stay ahead of the healthcare innovation curve. For investors, uncovering the next game-changing therapies and technologies could uncover lucrative future buyout candidates.

Release – Zyversa Therapeutics Highlights Published Data Demonstrating NLRP3 Inflammasome Inhibition Has Potential To Decrease Atherosclerotic Lesions In Patients With Diabetes

Posted on by [email protected]

Research News and Market Data on ZVSA

Apr 4, 2024

PDF Version

  • Atherosclerosis (AS) and its sequelae are the most common cause of death in diabetic patients and one of the reasons why diabetes has entered the top 10 causes of death worldwide.
  • The published data show that inhibiting the NLRP3 inflammasome pathway significantly reduces atherosclerotic lesions and improves hyperglycemic-induced plaque instability.
  • ZyVersa is developing IC 100, a monoclonal antibody targeting inflammasome ASC and ASC specks from multiple types of inflammasomes, including NLRP3, to block initiation and perpetuation of damaging inflammation that promotes atherosclerosis and its progression, among numerous other inflammatory diseases.

WESTON, Fla., April 04, 2024 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of inflammatory and renal diseases, highlights data from a peer-reviewed article published in Biochemical and Biophysical Research Communications. This article demonstrates that NLRP3 inhibition results in improved glucose tolerance and markedly smaller and more stable atherosclerotic lesions in a diabetic mouse model.

In the paper titled, “High glucose levels accelerate atherosclerosis via NLRP3-IL/ MAPK/ NF-κB-related inflammation pathways,” the authors evaluated serum and coronary artery tissues from patients with coronary artery disease (CAD), with and without diabetes and they conducted a study in diabetic mouse models. Key findings include:

  • Patients with comorbid CAD and diabetes had higher serum levels and expression of NLRP3 in their coronary arteries, and increased serum levels of IL-1β and IL-6 than those with CAD only.
  • Diabetic mouse models showed a significantly higher atherosclerotic plaque/vessel area ratio than non-diabetic mice, which was markedly reduced with NLRP3 inhibition and the resulting reduction in levels of proinflammatory cytokines and inflammation.

The authors concluded, “Our research offers new understanding of the pathological mechanisms of diabetes-accelerated AS and provide a novel and promising target for treating diabetes-accelerated AS.” To review the publication, Click Here.

“We are excited about the data published in Biochemical and Biophysical Research Communications demonstrating that inhibiting inflammasome NLPR3 pathways has potential to attenuate the development and progression of AS in patients with diabetes, a leading cause of morbidity and mortality,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO, and President. “We look forward to seeing our preclinical data with Inflammasome ASC Inhibitor IC 100 in an animal model of atherosclerosis in the first half of this year. We believe that by inhibiting multiple types of inflammasomes and disrupting the structure and function of their associated ASC specks to attenuate initiation and perpetuation of inflammation, that IC 100 has promise to effectively control AS development and progression.” To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.

About Inflammasome ASC Inhibitor IC 100

IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP2, NLRP3, NLRC4, AIM2, and Pyrin. Intracellularly, IC 100 binds to ASC monomers, inhibiting inflammasome formation, thereby blocking activation of IL-1β early in the inflammatory cascade. IC 100 also binds to ASC in ASC Specks, both intracellularly and extracellularly, further blocking activation of IL-1β and the perpetuation of the inflammatory response that is pathogenic in inflammatory diseases. Because active cytokines amplify adaptive immunity through various mechanisms, IC 100, by attenuating cytokine activation, also attenuates the adaptive immune response.

About ZyVersa Therapeutics, Inc.

ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs for patients with renal and inflammatory diseases who have significant unmet medical needs. The Company is currently advancing a therapeutic development pipeline with multiple programs built around its two proprietary technologies – Cholesterol Efflux Mediator™ VAR 200 for treatment of kidney diseases, and Inflammasome ASC Inhibitor IC 100, targeting damaging inflammation associated with numerous CNS and other inflammatory diseases. For more information, please visit www.zyversa.com.

Cautionary Statement Regarding Forward-Looking Statements

Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.

New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.

This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.

Corporate, Media, and IR Contact:
Karen Cashmere
Chief Commercial Officer
[email protected]
786-251-9641

Onconova Therapeutics (ONTX) – Transaction Forms A New Company In Virology and Oncology

Posted on by [email protected]


Wednesday, April 03, 2024

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation. Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in a combination trial with estrogen blockade in advanced endometrial cancer. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also evaluating opportunities for combination studies with narazaciclib in additional indications. Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies. These studies include a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Onconova Merges With Trawsfynydd Therapeutics To Form Traws Pharma, Inc. Onconova announced a business combination with Trawsfynydd Therapeutics, a private company developing products for influenza and COVID-19. The new company will be called Traws Pharma Inc, and will add antivirals to narazaciclib and rigosertib. Upon completion, it will raise $14 million in a private placement to bring its cash balance to about $28 million.

Combination Brings New Drugs With Several Near Term Milestones. Trawsfynydd brings antiviral drugs for influenza and COVID-19. TRX100 (viroksavir) inhibits the cap-dependent endonuclease required by the influenza virus for replication. Preclinical studies have shown efficacy against wild-type virus and strains that have resistance to current drugs. It has completed Phase 1, with data showing safety, tolerability, and pharmacokinetics that have potential dosing advantages. A dose-extension study will evaluate two increased doses. Following dose selection, Phase 2 is expected to begin in 2H24.


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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Ocugen (OCGN) – Progress In Clinical Trials Overshadows FY2023 Filing Delay

Posted on by [email protected]


Wednesday, April 03, 2024

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

10-K Filing Has Been Delayed. Ocugen announced that it has not filed its 10-K for FY2023 and will be restating FY2022 and first three quarters of FY2022. The restatement is due to errors in non-cash cost recognition related to collaborative agreements. We do not believe these restatements affect operating cash flow for these periods or the current cash balance. Cash on December 31, 2023, was reported at $39.5 million, which we estimate is sufficient to fund operations through 4Q24.

OCU400 Phase 3 Trial Is Expected To Begin Shortly. Ocugen reported that it is awaiting final clearance from the FDA to begin its Phase 3 trial testing OCU400 in retinitis pigmentosa (RP). Although timing of FDA actions can be unpredictable, patient treatment is expected to begin during April 2024. The trial will test OCU400 in RP with an enrollment of 300 patients, divided into an arm with the RHO mutation and a “mutation agnostic” arm with any RP-associated mutation. Each will randomize patients 2:1, with 50 treated patients and 25 control patients in each arm.


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Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Release – Ocugen Provides Business Update With Certain Financials For The Year Ending 2023

Posted on by [email protected]

Research News and Market Data on OCGN

April 2, 2024

CONFERENCE CALL AND WEBCAST TODAY AT 8:30 A.M. ET

  • Received alignment with FDA for broad retinitis pigmentosa (RP) indication in Phase 3 clinical trial of OCU400—first gene therapy program to receive a broad indication for RP. OCU400 Phase 3 clinical trial expected to commence in April 2024.
  • Regenerative Medicine Advanced Therapy (RMAT) designation granted by FDA to OCU400
  • Completed Cohort 1 dosing for OCU410 and OCU410ST gene therapy clinical studies for geographic atrophy (GA) and Stargardt disease, respectively

MALVERN, Pa., April 02, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today provided a general business update with certain financials for the year ending 2023.

“In 2023, our diligent efforts laid the foundation for continued advancement towards our clinical and operational goals with a focus on our game-changing modifier gene therapy platform,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “2024 is already proving to be a pivotal year with considerable developments in our modifier gene therapy programs for blindness diseases, and an increased understanding of the value of these assets among our stakeholders. Promising initial OCU400 Phase 1/2 study results for RP patients have been recognized by the FDA through the RMAT designation, potentially accelerating our path to deliver this critical therapy. With the FDA’s support, we feel confident that this trial will continue to demonstrate the benefits of our gene-agnostic mechanism utilizing a potential one-time treatment for life via a single sub-retinal injection.”

Additionally, the Company’s OCU410 and OCU410ST modifier gene therapy programs are currently enrolling patients with GA secondary to dry AMD (dAMD) and Stargardt disease, respectively. In February 2024, Ocugen completed dosing patients in the first cohort of its Phase 1/2 OCU410ST trial for Stargardt disease, and in March, dosing was completed for the first cohort of the OCU410 trial for GA.

The current treatment landscape for both GA and Stargardt disease is extremely limited. The estimated 1 million patient GA market in the U.S. saw some momentum with recent drug approvals. However, these treatment options have significant limitations, as they require multiple injections per year (impacting compliance) and only target one pathway contributing to GA. OCU410 -regulates multiple pathways involved with the disease including: lipid metabolism, regulation of inflammation, oxidative stress, and membrane attack complex (complement); and has the potential to provide a one-time treatment for life. Presently, there is no approved treatment for people living with Stargardt disease – an orphan blindness disease that affects approximately 40,000 people in the U.S. alone.

“As we pursue our pioneering efforts to change the paradigm for gene therapy, our dedication is unwavering to patients living with a constant fear of losing their sight,” said Dr. Musunuri. “With millions of people affected by these conditions, our mission is clear: to deliver treatments that cannot only stop disease progression but potentially help to preserve or improve sight and allow patients to maintain independence.”

Ocugen’s team has strategically allocated resources to drive the ongoing progress of its gene therapy trials and continues to pursue government funding to support its vaccines programs. During the fourth quarter of 2023, the Company announced its mucosal vaccine candidate, OCU500, was chosen for the multi-billion-dollar NIAID Project NextGen initiative. As a result, OCU500 is slated to enter clinical trials mid-2024. In the planned Phase 1 clinical trial, OCU500 will be tested via two different mucosal routes: inhalation into the lungs and as a nasal spray. All administration of the clinical trial is being led by NIAID.

NeoCart®, the Company’s 3-D regenerative cell therapy platform for cartilage repair, remains on track to begin a Phase 3 trial by the latter half of 2024 subject to availability of funding. Ocugen completed renovations on a world-class cGMP facility last year to produce NeoCart®, which has since received its full final clearance and occupancy certificate. Simultaneously, the Company is evaluating opportunities for NeoCart to maximize value for shareholders and patients.

Modifier Gene Therapies

  • OCU400 – Received alignment with FDA for broad RP indication in the Phase 3 clinical trial of OCU400—the first gene therapy program to receive a broad indication for RP. The modified Phase 3 trial design will include 150 adult and pediatric RP patients with RHO and other gene mutations associated with RP. In December, the FDA granted RMAT designation to OCU400 for the treatment of RP. RP affects more than 100,000 people in the U.S. and 1.6 million globally.
  • OCU410 – Currently in Phase 1/2 stage of clinical development with active patient enrollment. The first patient was dosed in the Phase 1/2 trials to assess the safety and efficacy of OCU410 for GA secondary to dAMD in December 2023. Dosing is complete for Cohort 1 (low dose).
  • OCU410ST – Currently in Phase 1/2 stage of clinical development with active patient enrollment. The first patient was dosed in the Phase 1/2 trials to assess the safety and efficacy of OCU410ST for Stargardt disease in November 2023. Dosing is complete for Cohort 1 (low dose). The Data and Safety Monitoring Board for the OCU410ST clinical trial determined that the safety and tolerability profile for OCU410ST is favorable and approved to proceed dosing with the medium dose of OCU410ST in the dose-escalation phase of the study.

Financial Results

  • The Company intends to restate its consolidated financial statements as of and for the year ended December 31, 2022, in connection with the filing of its 2023 Form 10-K. Similarly, the Company will include restated unaudited financial information for each of the first three quarters of 2023 and 2022 in its 2023 Form 10-K (each such annual and quarterly period to be restated, a “Restated Period”).
  • The identified errors in each of the Restated Periods relate to the Company’s non-cash accounting for the estimated costs in one of its collaboration arrangements. However, the Company does not expect the errors to result in any impact on its cash position, cash runway, or financial projections.
  • Ocugen’s cash, cash equivalents, and investments totaled $39.5 million as of December 31, 2023, compared to $90.9 million as of December 31, 2022. The Company estimates that its current cash, cash equivalents, and investments will enable it to fund its operations into the fourth quarter of 2024. The Company had 256.6 million shares of common stock outstanding as of December 31, 2023.

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: 4947142
Webcast: Available on the events section of the Ocugen 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 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 X 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 include, but are not limited to, statements regarding the Company’s clinical development activities and related anticipated timelines; strategy, business plans and objectives for its clinical stage programs; plans and timelines for the preclinical and clinical development of its product candidates, including the therapeutic potential, clinical benefits and safety thereof; expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials; the ability to initiate new clinical programs; Ocugen’s financial condition and the expected impact of the restatement of certain financials. 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, as well as discussions of potential risks, uncertainties, and other important factors in Ocugen’s subsequent filings with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. 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
[email protected]

Release – Traws Pharma Announces New Employee Inducement Grants

Posted on by [email protected]

Research News and Market Data on ONTX

Apr 02, 2024

NEWTOWN, Pa., April 02, 2024 (GLOBE NEWSWIRE) — Traws Pharma, Inc. (Nasdaq: TRAW, “Traws”, the “Company”) today announced it has granted equity awards to new employees who joined Traws in connection with the recent acquisition of Trawsfynydd Therapeutics, Inc. by Onconova Therapeutics, Inc. (the “Merger”).

On April 1, 2024, Traws granted restricted stock equity awards to Werner Cautreels, Iain Dukes, Nikolay Savchuk, C. David Pauza and Robert Redfield, as an inducement for each of them to accept employment, which equity awards relate to, 200,000, 67,500, 67,500, 97,500 and 97,500 shares of Traws common stock. One-quarter (1/4) of the shares subject to each restricted stock equity award will vest on each of the first four anniversaries of the grant date, conditioned upon each employee’s continued employment on the vesting date(s).

The inducement grants were approved by Traws’ Compensation Committee of the Board of Directors, as required by NASDAQ Rule 5635(c)(4), and were granted as a material inducement to employment in accordance with NASDAQ Rule 5635(c)(4).

About Traws Pharma, Inc.

Traws is developing next-generation, best-in-class antivirals for influenza, COVID and other respiratory infections and narazaciclib in oncology. Traws was formed from the business combination of Onconova Therapeutics, Inc. and Trawsfynydd Therapeutics, Inc. and is headquartered in Newtown, PA and will trade on NASDAQ as “TRAW” starting on April 3, 2024.

Forward-Looking Statements

This communication contains “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including but not limited to, statements regarding the timing of the change in the Nasdaq symbol; the development plans and goals for Traws’ product candidates and other statements that are not historical fact. All statements other than statements of historical fact contained in this release are forward-looking statements. These forward-looking statements are made as of the date they were first issued, and were based on the then-current expectations, estimates, forecasts, and projections, as well as the beliefs and assumptions of management.

Forward-looking statements are subject to a number of risks and uncertainties, many of which involve factors or circumstances that are beyond Traws’ control. Traws’ actual results could differ materially from those stated or implied in forward-looking statements due to a number of factors, including but not limited to (i) failure to timely obtain stockholder approval for the preferred stock issued in the Merger and private financing; (ii) uncertainties as to the timing and funding of the private financing; (iii) risks related to Traws’ ability to manage its operating expenses and its expenses associated with the Merger; (iv) unexpected costs, charges or expenses resulting from the transactions; (v) potential adverse reactions or changes to business relationships resulting from the completion of the Merger; (vi) the uncertainties associated with Traws’ product candidates, as well as risks associated with the clinical development and regulatory approval of product candidates, including potential delays in the commencement and completion of clinical trials, studies and evaluations; (vii) risks related to the inability of Traws to obtain sufficient additional capital to continue to advance their product candidates; (viii) uncertainties in obtaining successful clinical results for product candidates and unexpected costs that may result therefrom; (ix) risks related to the failure to realize any value from product candidates currently being developed and anticipated to be developed in light of inherent risks and difficulties involved in successfully bringing product candidates to market; and (x) risks associated with the possible failure to realize certain anticipated benefits of the Merger, including with respect to future financial and operating results. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties. These and other risks and uncertainties are more fully described in periodic filings with the U.S. Securities and Exchange Commission (the “SEC”), including the factors described in the section titled “Risk Factors” in Onconova’s Annual Report on Form 10-K for the year ended December 31, 2023, which was filed with the SEC on April 1 2024, subsequent Quarterly Reports on Form 10-Q filed with the SEC, and in other filings that Traws makes and will make with the SEC. You should not place undue reliance on these forward-looking statements, which are made only as of the date hereof or as of the dates indicated in the forward-looking statements. Traws expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in its expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
[email protected]