Tag: Biotechnology

Release – GeoVax Announces Issuance of Malaria Vaccine Patent

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Research News and Market Data on GOVX

 

Patent Covers Multiple Component Vaccine for Both Prevention and Treatment

Atlanta, GA, January 3, 2024 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that the U.S. Patent and Trademark Office has issued Patent No. 11,857,611 to GeoVax, pursuant to the Company’s patent application No. 17/726,254 titled “Compositions and Methods for Generating an Immune Response to Treat or Prevent Malaria.”

The claims granted by the patent generally cover compositions comprising GeoVax’s modified vaccinia Ankara (MVA) vector expressing Plasmodium antigens and methods of inducing an immune response to malaria utilizing the compositions. The compositions and methods covered in the claims are useful both prophylactically and therapeutically and may be used to prevent and/or treat malaria.

David Dodd, GeoVax President and CEO, commented, “During 2023, there were the first cases of locally transmitted malaria reported in the United States in 20 years, with cases reported in Florida, Maryland and Texas. Worldwide, there are over 600,000 deaths annually attributed to malaria. This patent represents a potentially significant advance against a most critical deadly threat worldwide. While our focus and priority is to support our clinical-stage programs, we also remain strongly committed to improving public health worldwide and developing vaccines against global public health threats, such as malaria, as a part of our long-term vision for the company.”

According to data from the World Health Organization, globally, malaria causes 227 million infections and 619,000 deaths annually. Despite decades of vaccine research, vaccine candidates have failed to induce substantial protection. Most of these vaccines are based on individual proteins that induce immune responses targeting only one stage of the malaria parasite’s life cycle. GeoVax’s MVA-VLP malaria vaccine candidates incorporate antigens derived from multiple stages of the parasite’s life cycle and are designed to induce an immune response with durable functional antibodies and CD4+ and CD8+ T cell responses, all hallmarks of an ideal vaccine-induced immune response.

About GeoVax

GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation COVID-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information, visit our website: www.geovax.com.

Forward-Looking Statements

This release contains forward-looking statements regarding GeoVax’s business plans. The words “believe,” “look forward to,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax is able to obtain acceptable results from ongoing or future clinical trials of its investigational products, GeoVax’s immuno-oncology products and preventative vaccines can provoke the desired responses, and those products or vaccines can be used effectively, GeoVax’s viral vector technology adequately amplifies immune responses to cancer antigens, GeoVax can develop and manufacture its immuno-oncology products and preventative vaccines with the desired characteristics in a timely manner, GeoVax’s immuno-oncology products and preventative vaccines will be safe for human use, GeoVax’s vaccines will effectively prevent targeted infections in humans, GeoVax’s immuno-oncology products and preventative vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete development, there is development of competitive products that may be more effective or easier to use than GeoVax’s products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control.

Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law. 

Company Contact:             Investor Relations Contact:             Media Contact:
[email protected] [email protected] [email protected] 
678-384-7220 212-698-8699 202-779-0929

ZyVersa Therapeutics, Inc. (ZVSA) – Novel Therapies For Renal Diseases and Inflammation

Posted on by [email protected]


Wednesday, January 03, 2024

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.

Initiating Coverage With An Outperform Rating. ZyVersa Therapeutics is a biotechnology company focused on renal and inflammatory diseases. Its lead product, VAR 200, is in development to reduce renal cholesterol and lipid accumulation in the kidney. These accumulations damage the kidney and lead to loss of filtration in kidney diseases. Its second product in development, IC 100, is an inflammasome inhibitor to inhibit the inflammation that contributes to many chronic diseases.

Renal Diseases. Accumulation of renal cholesterol and lipids in the kidney leads to cellular damage that starts a decline in filtration function and leakage of protein into the urine. VAR 200 was developed to reduce levels of cholesterol and lipids in renal cells to reduce the damage, scarring, and disease progression. The first clinical trial in diabetic kidney disease (DKD) is planned for early 2024.


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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.

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*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. 

Bristol Myers Squibb $4.1B RayzeBio Buyout

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Pharma giant Bristol Myers Squibb (BMY) announced Tuesday that it will acquire clinical-stage biotech RayzeBio for $4.1 billion, continuing Bristol’s strategy of deals to refresh its drug pipeline amid upcoming patent expirations.

RayzeBio is developing a novel targeted radiotherapy called RYZ101 to treat multiple types of cancer. The company’s technology combines tumor-targeting antibodies with radioactive isotope payloads that selectively damage cancer cells’ DNA when delivered.

RYZ101 is currently in Phase 3 testing for treating metastatic castration-resistant prostate cancer. Early clinical data showed promising results with the drug demonstrating tumor response rates of 44-55%.

Bristol gains full rights to RYZ101 and RayzeBio’s broader platform for linking radioisotopes to cancer-fighting proteins. The deal gives Bristol a potential new blockbuster cancer treatment as competition intensifies in the immuno-oncology space.

Shoring Up the Cancer Business

Bristol already markets leading cancer immunotherapies Opdivo and Yervoy. However, Opdivo faces patent expiration in 2028/2031, forcing Bristol to find new long-term growth drivers.

The RayzeBio deal comes right after Bristol announced the $13.1 billion acquisition of schizophrenia drug developer Karuna Therapeutics last Friday. Karuna’s lead drug KarXT could generate peak annual sales of over $3 billion, analysts project.

These acquisitions help future-proof Bristol’s business as its top-selling drugs face new competition. Blood thinner Eliquis, which makes up over 30% of Bristol’s revenue, will see biosimilar rivals by 2026. Cancer drug Revlimid, acquired in Bristol’s 2019 buyout of Celgene, faces generics soon too.

“We are focused on strengthening our portfolio through a combination of internal programs and targeted business development,” said Bristol Myers CEO Giovanni Caforio. The RayzeBio and Karuna deals “complement our existing pillars of growth,” he added.

Betting Big on Radio-Pharmaceuticals

In addition to RYZ101’s potential, Bristol gains RayzeBio’s expertise with radio-pharmaceuticals. Attaching radioactive particles to antibodies allows them to precisely pinpoint tumor cells and kill them via DNA damage.

RayzeBio’s technology overcomes past challenges with radio-drugs such as lack of tumor specificity and rapid decay of radioisotopes. Linking radioisotopes to robust antibodies circumvents these issues and improves the drugs’ efficacy.

Analysts see radio-pharmaceuticals as an emerging trend in oncology. Radio-immunotherapies like RayzeBio’s could complement immuno-oncology drugs that activate the immune system against cancer.

By acquiring RayzeBio’s platform, Bristol can expand development of new radio-drug conjugates across its oncology pipeline. Bristol may also look to license out the technology to other companies given the heightened industry interest.

An Expensive Acquisition

Bristol is paying a huge premium to acquire RayzeBio before the biotech can prove RYZ101’s efficacy in late-stage testing. The $4.1 billion price tag works out to $62.50 per share, more than double RayzeBio’s prior closing price.

But Bristol likely wanted to preempt competition for the promising biotech asset. Amgen and Novartis are also developing radio-pharmaceutical drugs for cancer. And RayzeBio would have commanded an even higher valuation had RYZ101 succeeded in Phase 3.

Bristol expects the acquisition will reduce its adjusted earnings by about 13 cents per share in 2024. But Bristol maintained its existing profit guidance for 2022 and 2023, implying confidence the long-term benefits outweigh the near-term costs.

The company plans to finance the purchase using new debt. Bristol’s strong cash flows should allow it to service the additional debt load as it waits for RYZ101 to potentially reach the market around 2025.

Conclusion: Bolstering Its Firepower

The back-to-back deals for Karuna Therapeutics and RayzeBio showcase Bristol Myers Squibb’s strategy to acquire new therapies and drug platforms that can drive growth over the next decade. While expensive, these acquisitions reduce Bristol’s reliance on aging blockbuster drugs facing patent cliffs.

Gaining Karuna’s potential multi-billion dollar schizophrenia medicine and RayzeBio’s cutting-edge radio-pharmaceutical technology gives Bristol valuable new firepower to deploy in the fiercely competitive pharma market. If successful, the deals will ensure Bristol Myers remains an industry leader as it confronts upcoming challenges from biosimilar and generic competition.

Take a moment to take a look at emerging growth biotechnology companies by looking at Noble Capital Markets Senior Research Analyst Robert LeBoyer’s coverage universe.

Bristol Myers Drops $14 Billion to Acquire Karuna Therapeutics, Gaining Schizophrenia Drug

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Pharmaceutical giant Bristol Myers Squibb made a bold move into neuroscience today, announcing the $14 billion acquisition of clinical-stage biotech Karuna Therapeutics. The massive deal provides Bristol Myers with Karuna’s lead drug candidate, KarXT, a potential new treatment for schizophrenia and other psychiatric disorders.

KarXT could be the first drug in its class approved for schizophrenia in decades. The market for schizophrenia drugs is estimated at over $7 billion globally. If approved, KarXT is projected to achieve multi-billion dollar peak sales. Bristol Myers is betting the experimental medicine could transform treatment for millions struggling with serious mental illness.

This acquisition is the latest in a wave of big pharma interest in the emerging neuroscience space. Companies are eager to find new approaches to historically hard-to-treat psychiatric conditions like schizophrenia, depression and Alzheimer’s disease.

Smaller biotechs like Karuna have led the charge, developing novel therapies targeting neurological mechanisms of psychiatric disorders. But larger players like Bristol Myers have taken notice of the promise of these new technologies.

Karuna’s KarXT combines xanomeline, a novel muscarinic receptor agonist, with trospium chloride, an FDA-approved muscarinic receptor antagonist. Early clinical results show this approach reduces side effects and improves efficacy compared to current schizophrenia drugs.

Take a look at other emerging growth biotechnology companies by taking a look at Noble Capital Markets’ Senior Research Analyst Robert Leboyer’s coverage list.

In late-stage clinical trials, KarXT demonstrated statistically significant and clinically meaningful improvements in schizophrenia symptoms. Patients experienced rapid reductions in hallucinations and delusions with far fewer problematic side effects like sedation.

Based on positive Phase 3 data, Karuna submitted a New Drug Application for KarXT in schizophrenia in mid-2022. The FDA accepted the application and set a PDUFA goal date of September 2023 for a potential approval.

Clearly Bristol Myers feels confident about KarXT’s chances, agreeing to pay $28.5 billion upfront in cash to finalize the acquisition. Karuna shareholders will also be eligible for up to $3.5 billion in milestone payments if KarXT reaches certain commercial goals.

For Bristol Myers, the move signals a push into neuroscience and psychiatric disease, an area it has not traditionally emphasized. But the company likely sees major growth potential, given the prevalence of mental illness and the need for better treatments.

Almost 3% of the U.S. population suffers from schizophrenia. Another 17% experience some other mental illness like depression, bipolar disorder or PTSD. Existing drugs fail to adequately manage symptoms for many patients and carry tolerability issues that lead to poor compliance.

Doctors and patients are eagerly awaiting novel therapies like KarXT that balance safety and efficacy. Karuna is also exploring KarXT’s potential in dementia-related psychosis and other indications beyond schizophrenia.

The lucrative deal builds on other recent big-ticket acquisitions for Bristol Myers as the company looks to expand its portfolio. Earlier this year, Bristol Myers acquired cancer biotech Turning Point Therapeutics for $3.2 billion and the oncology company MyoKardia for $13 billion.

But the Karuna purchase represents Bristol Myers’ biggest bet yet on the emerging neuroscience space. It’s the second largest biopharma acquisition announced in 2022 after Pfizer’s $43 billion buyout of cancer drugmaker Seagen.

Other large pharmaceutical companies have also signed deals to access neuropsychiatric drug candidates. AbbVie recently acquired an option to purchase Alector’s experimental Alzheimer’s therapy for up to $2.2 billion. And Eli Lilly collaborated with NextCure on novel immuno-oncology approaches for treating mental illness.

As more novel mechanisms like KarXT arrive, expect growing competition among pharma giants to capture market share. Bristol Myers struck first with today’s monumental acquisition, but likely won’t be the last looking to neuroscience for future growth.

Release – Ocugen Gains FDA Alignment on Key Aspects of OCU400—Modifier Gene Therapy—Pivotal Phase 3 Study Design

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Research News and Market Data on OCGN

December 21, 2023

MALVERN, Pa., Dec. 21, 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 and vaccines, today announced that the Company received alignment from FDA on key aspects of the Phase 3 clinical trial design to assess the safety and efficacy of OCU400 in patients with RHO and other gene mutations associated with Retinitis Pigmentosa (RP).

“This news brings us even closer to fulfilling our mission to bring our first-in-class, gene-agnostic therapies to market and provide access to patients globally,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We look forward to beginning the Phase 3 clinical trial, which we plan to initiate in early 2024.”

During a multidisciplinary meeting with FDA, based on preliminary results from an ongoing Phase 1/2 study, Ocugen received alignment on key aspects of the Phase 3 study design—including the study endpoint, patient enrollment strategy, and study duration of one year. The Phase 3 clinical trial will enroll a broader group of RP patients, including patients with the most common RHO gene mutation, based on OCU400’s potentially gene-agnostic mechanism of action.

With orphan drug and RMAT designations in place for OCU400, FDA’s alignment on key aspects of the Phase 3 study design positions Ocugen to confidently move forward in pursuing product development and licensure for OCU400.

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. Advancing OCU400 to Phase 3 clinical development will be an important step toward addressing unmet needs in the RP patient community.

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, including, but not limited to, statements regarding qualitative assessments of available data, potential therapeutic and clinical benefits of our product candidates, expectations for clinical trial timing and results, anticipated timing of clinical trial updates and expectations for timing and outcome of regulatory interactions, 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; that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities; that receipt of orphan drug and RMAT designations may not lead to faster development or regulatory review; and that regulatory authorities may disagree with additional aspects of our clinical trial designs or may not approve our future IND applications on the anticipated timeline or at all. 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]

Tonix Pharmaceuticals (TNXP) – Fibromyalgia Trial Meets All Endpoints

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Thursday, December 21, 2023

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

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

RESILIENT Study Shows Strong Results In Fibromyalgia. Tonix announced that the Phase 3 RESILIENT study testing TNX-102 SL in fibromyalgia met its primary endpoint of pain reduction and its six secondary endpoints. The fibromyalgia population is estimated at 6 to 12 million patients, making this a significant market for Tonix. The company plans to submit an application for FDA approval in 2H2024, consistent with our projected product launch in 2025.

Tonix Raised Funds With Stock and Warrants. The company also announced a $144 million Registered Direct offering that will raise $30 million upon closing and a potential $114 million through warrant exercise. We believe the offering offset the positive news from the RESILIENT clinical trial, causing the stock to decline despite good clinical news.


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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) – OCU400 Receives RMAT Designation, OCU410 Treated First Patient

Posted on by [email protected]


Wednesday, December 20, 2023

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.

OCU400 Received RMAT Designation. The FDA has designated OCU400 as a Regenerative Medicine Advanced Therapy (RMAT) for treating retinitis pigmentosa associated with RHO mutations. RMAT designation is intended to speed development of regenerative medicines, and is awarded to therapies that have potential treat, reverse, or modify a life-threatening disease. We see this as a validation of Ocugen’s clinical trial data. Additional data submissions to expand the designation to other gene mutations in RP and Leber congenital amaurosis (LCA) are planned.

Phase 3 Trial Should Benefit From RMAT Designation. RMAT designation includes the benefits of Fast Track and Breakthrough designations, with increased guidance from the FDA. It also allows the use of surrogate markers as endpoints in clinical trials, and accelerated review after the application is filed. Ocugen is currently finalizing the design of the Phase 3 trial for OCU400. We expect the trial to begin in early 2024 with data available in 2H24.


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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 Announces OCU400 Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for Treatment Of Retinitis Pigmentosa Associated With Rho Mutations

Posted on by [email protected]

Research News and Market Data on OCGN

December 19, 2023

  • RMAT designation intended to help expedite development of new regenerative medicines

MALVERN, Pa., Dec. 19, 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 and vaccines, today announced that the FDA has granted RMAT designation to Ocugen’s investigational product OCU400 for the treatment of retinitis pigmentosa (RP) associated with RHO mutations.

“RMAT designation is a significant accomplishment for the OCU400 clinical development program, as it validates the potential for our game-changing gene therapy approach to fulfill an unmet medical need for people who are facing blindness due to RP,” said Arun Upadhyay, PhD, Chief Scientific Officer and Head of R&D at Ocugen. “FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”

RMAT designation for OCU400 was based on preliminary clinical data supporting the maintenance and improvement of visual acuity and function in RP patients in the OCU400 -101 Phase 1/2 clinical trial as measured by Best Corrected Visual Acuity (BCVA), Low Luminance Visual Acuity (LLVA), and Multi-Luminance Mobility Test (MLMT).

RMAT designation is part of the 21st Century Cures Act. The program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition. Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA. Ocugen is working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development for OCU400 to support an application for marketing authorization.

Current data support the gene-agnostic mechanism of action for OCU400, which suggests that it may be able to provide treatment benefit to a broader group of RP and Leber congenital amaurosis (LCA) patients. Ocugen intends to submit additional efficacy and safety data for OCU400 in RP and LCA patients to the FDA in the future to potentially expand this RMAT designation to broader RP and LCA patient populations.

RHO mutations affect more than 10,000 of the 110,000 people in the United States diagnosed with RP. In the latest clinical study update from the Phase 1/2 trial of OCU400, 86% (6/7) of RHO mutation subjects experienced either stabilization or improvement in MLMT scores from baseline, among which 29% (2/7) demonstrated 3 Lux luminance level improvement. There are currently no treatment options available for RP patients with RHO gene mutations.

OCU400 represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions, such as homeostasis, reproduction, development, and metabolism to potentially improve retinal health and function.

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, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, the expected benefits of RMAT designation, and anticipated regulatory interactions, 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 Announces Dose Selection In Thio-101 Phase 2 Clinical Trial For Non-Small Cell Lung Cancer

Posted on by [email protected]

Research News and Market Data on MAIA

December 19, 2023 7:00am EST

  • Selected dose shows unprecedented disease control and overall response rates in a NSCLC clinical trial

CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA” or the “Company”), a clinical-stage biopharmaceutical company developing telomere-targeting immunotherapies for cancer, today announced dose selection for THIO-101, a Phase 2 clinical trial evaluating its lead asset, THIO, in sequential combination with Regeneron’s anti-PD-1 cemiplimab (Libtayo®) in patients with advanced non-small cell lung cancer (NSCLC).

During the dose-finding stage of THIO-101, patients were administered either 60mg, 180mg, or 360mg of THIO per cycle, followed by 350mg of cemiplimab (Libtayo®). The selected dose, 180mg/cycle, presented better safety profile and outperformed the other doses in the key measures of efficacy for NSCLC trials. Subsequently, all future trial participants will be treated with THIO 180mg/cycle.

“All THIO dose levels tested exceeded the disease control rate (DCR) thresholds in Stage 1 of the THIO-101 Phase 2 trial. We observed disease control in the first 8 to 9 patients with a post baseline scan in each arm, beating our goal of disease control in 8 out of 19 patients per arm. Among the three studied doses, the 180mg dose showed stronger DCR and preliminary response rates compared to other doses,” said Vlad Vitoc, M.D., MAIA’s Chairman and Chief Executive Officer.

“These results are particularly impressive in this pool of patients who were heavily pre-treated and resistant to prior treatments with immune checkpoint inhibitors, a group that does not yet have standard of care treatment. We are highly encouraged by the unprecedented clinical data generated thus far in our Phase 2 trial, and as we move forward, we plan to pursue accelerated approval for THIO in the U.S. for the treatment of patients with advanced NSCLC. We believe THIO’s DCRs and ORRs in second line treatment suggest the drug’s potential to define the standard of care for this NSCLC patient population.”

THIO is the only direct telomere targeting agent currently undergoing clinical development in the field of cancer drug discovery and treatment.

About THIO

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

About THIO-101, a Phase 2 Clinical Trial

THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate THIO’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of THIO administered prior to cemiplimab (Libtayo®) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of THIO administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of THIO using Overall Response Rate (ORR) as the primary clinical endpoint. Treatment with cemiplimab (Libtayo®) followed by THIO has been generally well-tolerated to date in a heavily pre-treated population. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.

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/20231219972050/en/

Investor Relations Contact
+1 (872) 270-3518
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Source: MAIA Biotechnology, Inc.

Released December 19, 2023

Bluebird Bio Announces $150 Million Public Offering to Fund Approved Gene Therapies

Posted on by [email protected]

Cambridge-based gene therapy developer Bluebird Bio announced a public offering of $150 million in common stock to raise capital supporting its three approved treatments and provide working capital.

The pioneer in gene therapies will offer shares on the NASDAQ under the ticker symbol BLUE, with underwriters granted a 30-day option to purchase an additional $22.5 million in stock. Bluebird stated the final size and terms remain subject to market conditions.

Goldman Sachs and J.P. Morgan are serving as joint book runners on the deal, with Raymond James as co-manager on the offering. All shares sold will come directly from Bluebird Bio.

Proceeds from the public stock sale will specifically further commercialization, manufacturing, and launch efforts behind the company’s newly approved gene therapies – Zynteglo for beta thalassemia, Skysona for cerebral adrenoleukodystrophy, and Lyfgenia for sickle cell disease.

The capital raise also provides balance sheet support as Bluebird continues its transition into a fully-integrated commercial biotech selling proprietary therapies targeting rare diseases.

Analysts see the offering as a move to seize current investor enthusiasm and strengthen Bluebird’s financial position after a turbulent few years adjusting to regulatory setbacks.

With three potent gene therapies now approved since August 2022, Bluebird looks to ride accelerating momentum as its treatments reach more patients globally. But the specialized nature of gene therapy production and administration constrains rapid scaling despite massive market opportunities.

Hefty expenses can also accrue during the early stages of drug launches pending insurance coverage and reimbursement decisions country by country.

Tuesday’s proposed $150 million offering suggests management sees room to accelerate growth in 2024 while demand runs hot for novel gene therapies.

Gene Therapies Target Root Causes of Diseases by Manipulating Genes

The permanent gene corrections from one-time gene therapy represent potential cures promising to revolutionize treatment for blood disorders, cancers, inherited disorders and degenerative diseases.

After gene therapy showed immense promise in the 2010s, developmental and safety hurdles caused temporary setbacks for the emerging category.

But breakthrough approvals over the past 18 months from Bluebird and others have reinvigorated investor appetite to fund the next generation of radical genetic medicines now reaching patients in need.

While small in patient size, the market chances to generate multi-billion sales treating high unmet needs in rare diseases with no other solutions for the underlying condition.

Goldman Sachs and JPMorgan’s involvement arranging Bluebird’s latest stock sale reflects rising investor intrigue and renewed confidence in realizing gene therapy’s paradigm-changing potential after past stumbles.

Still Long Road Ahead as Gene Therapies Slowly Build Adoption

However, analysts caution the road remains long translating hype into real revenues as gene therapy faces entrenched barriers preventing mass adoption anytime soon.

Priced at over $2 million per treatment, gene therapies today dispense more hope than profit for developers. Reimbursement pushback from insurers and intense medical limitations temper growth projections.

Bluebird’s approved drugs currently treat tiny populations measured in the single digit thousands globally. But success establishing coverage helps pave the way for expanding into wider therapeutic indications in time.

With fresh financing now on tap, Bluebird Bio stock offers a investment into a maturing gene therapy leader well-positioned to ride coming decades of medical advancements illuminating genetics’ role beating back disease.

Yet expectations likely stay muted near-term for all gene therapy plays absent key inflection events bringing more treatments past global regulatory gates.

Cadrenal Therapeutics (CVKD) – Addressing Unmet Anticoagulation Needs In Orphan Populations

Posted on by [email protected]


Monday, December 18, 2023

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.

Initiating Coverage Of CVKD.  Cadrenal Therapeutics is developing tecarfarin, an oral anticoagulant for prevention of systemic thromboembolism (blood clots) in rare medical conditions where patients cannot take the commonly prescribed oral anticoagulants. The only available therapy is warfarin, a drug with wide variations in bloodstream levels that requires frequent monitoring to prevent side effects including excessive bleeding risk.

Phase 3 Trial Is Expected In 2024. The lead orphan indication for tecarfarin is in patients with end-stage kidney disease (ESKD) with atrial fibrillation (AFib, irregular heartbeat). The ACTOR-AF Phase 3 trial has been designed as a randomized, double-blind, placebo-controlled study testing tecarfarin against placebo. The amended protocol is expected to be submitted in 1H24 to allow patient treatment to begin in 2H24.


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Biotech Dealmaking Heats Up as Private Capital Charges Back In

Posted on by [email protected]

A wave of multibillion dollar buyouts has swept the beaten-down biotech sector in recent months, marking a potential turning point for an industry hammered throughout 2022 – 2023.

With valuations of public companies still depressed, flush private investors have stepped up acquisitions of promising drug developers to bolster pipelines for the long-term. And in a bullish sign for the strategic direction of the space, therapeutics targeting high unmet needs and novel modalities remain key areas of focus amid dealmaking.

As macro gloom recedes, the renewed embrace of biotech M&A highlights a pivot back toward the innovation-driven spending required to sustain growth post-pandemic.

BMS Kicks Off Buying Spree With $13.2B Turning Point Deal

Bristol Myers Squibb fired the starting gun on big-ticket biopharma deals in October, announcing a $5.8 billion purchase of Mirati Therapeutics (MRTX). The buyout delivered a 122% premium in order to land Mirati’s promising portfolio of precision cancer medicines.

Market observers viewed the unsolicited, $58 per share bid as a credible benchmark of intrinsic value vigilantly researched by a strategic acquirer. Immediately in the deal aftermath, similar development-stage oncology names rallied sharply as traders priced in new takeout probabilities.

In fact, suitors moved swiftly to capitalize on improved biotech sentiment, with Horizon Therapeutics agreeing to a $26.4 billion around the same time. The transaction marked 2023’s largest healthcare buyout, further reinforcing peak valuations remain attainable for commercial-stage rare disease names.

Scaling Up to Compete in Gene Therapy

Gene therapy remains one especially alluring area for dealmaking despite lofty price tags. These ultra-rare disease medicines come with cure potential that commands premium sales and reimbursement pricing power.

Recognizing the imperative to bulk up gene therapy capabilities, Pfizer ponied up $5.4 billion to reinforce its genetic medicines pipeline through the acquisition of French outfit Vivet Therapeutics. The move added Vivet’s promising gene therapy for Wilson disease, along with manufacturing strengths across multiple delivery mechanisms.

And gene editing pioneer Sangamo Therapeutics is selling off its cell therapy assets to Sanofi for $700 million as it refocuses efforts around in vivo gene insertion. The deal hands Sanofi disruptive cell therapy technology utilizing precisely engineered zinc fingers to correct disease-causing mutations.

Analysts say more buyouts centered on next-gen platforms are likely on the horizon as drug developers vie for leadership in areas forecast to reshape therapeutic spaces.

Take a look at more biotechnology companies by looking at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage universe.

Private Capital Eagerly Steps in to Back Innovation

Beyond M&A from strategic acquirers, private equity firms have swooped in to capitalize on depressed biotech valuations. The robust dry powder levels built up during the boom years leave private investors eager to allocate while achieving advantageous cost bases.

Among notable deals, Angel Pond Capital teamed up with life science investor OrbiMed to take gene therapy biotech Generate Biomedicines private for $478 million. The transaction represented a 130% premium to ensure locking up Generate’s base editing technologies believed to be capable of correcting over 75% of known point mutations.

In cybersecurity and enterprise software, sponsor-led take privates had utterly dominated deal flow in 2022. But order books are now once again filling up with biotech buyouts from special purpose acquisition vehicles, highlighting a normalization in deal dynamics after last year’s freeze-out from rate-sensitive private market valuations.

Market Recovery Taking Shape

The fresh upswing in biotech M&A follows a wave of dip buying from some the world’s largest asset managers in shares of industry leaders like Vertex Pharmaceuticals and Regeneron Pharmaceuticals. Warren Buffett’s Berkshire Hathaway has been particularly aggressive stepping in to purchase stakes in key biopharma bluechips.

Meanwhile, the fund-raising backdrop continues improving for earlier stage biotechs as well after deal activity all but shuttered for much of 2023. Multiple debt offerings and venture rounds have successfully priced in recent months, ensuring the all-important continuity of innovation cycling.

With fundamentals stabilizing and access to capital normalizing, the environment for biopharma dealmaking has markedly improved. Expect the momentum to carry through 2024 as drug developers position through M&A for the next, post-pandemic leg higher while private capital readily supports compelling technologies at discounted prices. The long-term health of the biotech ecosystem depends on transactions advancing today’s high-potential assets, and the industry appears to have emerged from its lull ready to strike the necessary deals.

Integra Bets on Ear, Nose, and Throat Growth With $280 Million Acclarent Purchase

Posted on by [email protected]

Medical device maker Integra LifeSciences announced today it will purchase Acclarent, a leader in ear, nose and throat (ENT) technologies, from Johnson & Johnson’s Ethicon division for $275 million upfront plus future regulatory milestones. The deal values Acclarent at approximately 2.5 times sales, with the company generating $110 million in revenues during 2022.

For Integra, the acquisition provides an opportunity to significantly expand its footprint beyond neurosurgery and establish the company as a major player in the attractive ENT specialty devices segment. The global ENT market is projected to grow at a 5-6% clip annually, adding an estimated $1 billion in addressable market opportunity for Integra.

Acclarent brings to Integra pioneered balloon dilation platforms for treating chronic sinusitis as well as novel treatments for Eustachian tube dilations. Its flagship products are the only FDA-approved stents for maintaining sinus openings after surgery. Acclarent also provides image guidance systems to assist surgeons with minimally invasive procedures.

The company maintains strong brand awareness and deep clinical relationships after rebuilding its commercial presence following a period of declining sales between 2017-2020.

Integra management sees substantial room for additional share gains in ENT given Acclarent’s leadership in balloon dilation and the generally fragmented supplier landscape in ENT today. The global sinus dilation devices market alone is projected to reach $3.5 billion by 2030, providing a sizable growth pipeline for Acclarent’s portfolio.

Strategic and Financial Benefits

The acquisition furthers Integra’s strategy to complement its legacy strength in neurosurgery with scaled positions across faster-growth clinical applications adjacent to its core.

Integra aims to replicate its #1 share in dural repair for neuro procedures by becoming one of few dominant players in ENT. The company believes the combination of its commercial infrastructure and Acclarent’s innovative portfolio can support above-market growth for the foreseeable future.

Financially, Acclarent is being acquired at an attractive upfront valuation of 2.5 times sales. Integra management expects the deal will be immediately accretive to earnings per share after closing.

Acclarent generated gross margins in line with Integra’s overall company average in 2022, providing opportunities for further margin expansion from operating leverage as the business scales.

The transaction also comes at a time when medtech valuations have declined from their pandemic peaks, enabling Integra to obtain Acclarent at what it believes to be an opportunistic price.

Cultural and Portfolio Fit

Integra CEO Jan De Witte highlighted the cultural alignment between both organizations and focus on restoring patient lives as key rationales behind the deal.

De Witte said, “Acclarent’s culture of pioneering technologies aligns with Integra’s legacy of innovation to transform care and restore patients’ lives. We are looking forward to welcoming the Acclarent employees to the Integra team. Together, we can make a profound impact on the future of ENT and neurosurgery.”

Acclarent will operate as part of Integra’s $1.3 billion Codman Specialty Surgical division focused on neurosurgery. Integra sees substantial opportunities for its neurosurgery and ENT sales teams to collaborate on treating certain brain tumors by leveraging skull base surgical approaches.

Integra also gains access to a robust ENT product development pipeline, including next-generation surgical staplers, powered sinus surgery technologies, and potential new indications for Acclarent’s balloon dilation platforms.

Acclarent’s R&D and regulatory expertise will help accelerate Integra’s internal efforts to bring new generations of minimally invasive surgery products to market.

Smooth Post-Close Integration

Integra expects to retain Acclarent’s entire workforce as part of ensuring a smooth organizational transition after the deal closes. The company aims to operate Acclarent as an independent business unit during the near-term while integrating back-office functions.

Manufacturing operations will continue to be outsourced to third parties and Integra anticipates no supply chain disruptions to Acclarent’s product availability.

The transaction is projected to close by the second quarter of 2024, subject to customary antitrust and regulatory clearances globally. Transition services agreements will provide additional support for up to four years following deal closure.

By maintaining continuity of strategy, personnel and manufacturing, Integra hopes to achieve targeted revenue and cost synergies from the integration of Acclarent, while continuing its above-market growth trajectory in the ENT segment. The addition of Acclarent’s portfolio and innovative roadmap makes this transaction an important step forward in Integra’s strategy to complement leadership in neurosurgery with scaled positions in some of medtech’s most attractive and fastest-growing markets.