Benitec Biopharma Lands $40M Lifeline to Advance Gene Therapy Pipeline

In an oversubscribed private placement deal, clinical-stage biotech Benitec Biopharma (NASDAQ: BNTC) has secured $40 million in fresh capital to propel its lead gene therapy program into human trials. The financing provides an essential lifeline for the company as it aims to validate its novel “Silence and Replace” platform through clinical data readouts.

Benitec sold 5.7 million shares of its common stock at $4.80 per share, while also issuing 2.6 million pre-funded warrants in the transaction. The deal was led by healthcare investment firm Suvretta Capital Management, with participation from an investor syndicate including Adage Capital Partners, Nantahala Capital, multiple specialist healthcare funds, and a large mutual fund.

The $40 million gross proceeds dwarf Benitec’s $8.5 million cash balance exiting 2023 and strengthen the biotech’s financial runway considerably. Executives stated the capital will primarily fund development of BB-301, Benitec’s lead therapeutic candidate for Oculopharyngeal Muscular Dystrophy (OPMD).

Specifically, Benitec plans to kick off a natural history lead-in study and then initiate a Phase 1b/2a clinical trial evaluating BB-301 in OPMD patients. A portion will also support general operations as the company works to build out its pipeline leveraging the next-generation “Silence and Replace” platform.

For Benitec, scoring this level of financing commitment represents a major endorsement from the investment community. The company has been touting the promise of its dual RNA interference and gene therapy technology for years, but has leaned on equity injections and partnerships to keep the lights on.

Now, with $40 million from a blue-chip investor group, Benitec will have resources to prove its bold scientific vision can translate into real-world results for patients. Delivering clinical validation would be a game-changer in unlocking the plethora of therapeutic opportunities the “Silence and Replace” platform could potentially address.

As part of the deal terms, Benitec has agreed to consider adding Suvretta portfolio manager Kishen Mehta to its board of directors. Having greater oversight and alignment with the lead investor could tighten Benitec’s focus on prudent capital allocation and strategic execution going forward.

The financing did require issuing shares at a discount to the $4.80 prior closing price as well as warrant coverage for investors to get the deal done. But scoring that magnitude of capital from high-quality funds suggests belief in the innovative science and upcoming data milestones.

For a pre-revenue biotech still in clinical development stages, continual cash raises remain the norm. Yet this latest $40 million haul buys Benitec significant runway to produce human proof-of-concept results and hit major value-inflection points, without being forced to give away the farm through onerous dilution or a cut-rate M&A exit.

Of course, as is the case with all cutting-edge technologies, execution risk remains. Benitec and its investors are betting big on the “Silence and Replace” platform living up to its game-changing gene therapy potential. Success would be transformative, but fai lures are all too common in the high-risk, high-reward biotech realm.

With its coffers newly replenished, Benitec is approaching a make-or-break inflection point. This $40 million lifeline paves the way for the pioneering gene therapy firm to generate pivotal clinical data that could vindicate its ambitious “Silence and Replace” platform. The road ahead is unforgiving, with little margin for error against the high bar set for regulatory approval and commercial success in the cutthroat biotech sphere. But if Benitec can deliver validating evidence that its dual RNA interference and gene replacement approach translates into meaningful therapeutic benefits, it could spark a tectonic shift in how the industry tackles genetic diseases. Benitec is staring down its chance to forever change the landscape of modern medicine.

Take a moment to take a look at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage universe.

Release – QuantaSing Group Accelerates Global Growth with Strategic Initiatives

Research News and Market Data on QSG

BEIJING, April 17, 2024 (GLOBE NEWSWIRE) — QuantaSing Group Limited (NASDAQ: QSG) (“QuantaSing” or the “Company”), a leading online learning service provider in China, today announced several strategic initiatives integral to the Company’s global growth acceleration. QuantaSing is strengthening its presence in the United States and in Hong Kong as part of this plan.

The Company’s executive team, led by Tim (Dong) Xie, Chief Financial Officer of QuantaSing Group, is currently in the United States engaged in dozens of meetings with investors, potential partners and other key stakeholders in the market. These exchanges are focused on the company’s development, establishing and strengthening new and existing relationships, and shaping next steps for QuantaSing’s growth.

“Our recent visit to the United States has opened up a wealth of opportunities. We look forward to announcing new partnerships in due course,” said Tim Xie, CFO of QuantaSing. “We also anticipate the opening of our new office in Hong Kong this summer and extend a warm welcome to our friends and partners worldwide to meet us when in market.”

QuantaSing returns to the ASU + GSV Summit

QuantaSing has returned to the ASU + GSV Summit in San Diego this year as a sponsor and continues to contribute to the discussion on the future of online learning, adult education, and education technology. Ken Chau, Chief Executive Officer of Kelly’s Education (a QuantaSing company), joined a panel discussion on “Responsible AI for Kids” highlighting how Kelly’s Education and QuantaSing have harnessed AI tools to break down barriers, reduce stigma, and empower both children and elderly adults to lead more fulfilling lives.

Kelly’s Education shares key business updates

Kelly’s Education recently announced partnerships with two major authorities in English learning courses: Disney World of English and National Geographic Learning. These partnerships aim to provide children with a higher quality teaching environment and learning materials, further enhancing their learning outcomes and building Kelly’s Education as the children’s English learning platform of choice in Hong Kong.

Moreover, Kelly’s Education will announce the opening of its first offline school in May. The school will serve as an interactive, innovative learning center, offering various educational resources and high-quality teaching services, which can effectively combine the advantages of both online and traditional learning methods, providing children with an innovative and engaging learning experience.

“I am very excited to have the opportunity to engage in a discussion about the delicate dynamics of AI and children’s education,” said Ken Chau, CEO of Kelly’s Education. “During the summit, we were also able to share how we apply AI to our work to better the customer experience while guarding children’s wellbeing. Since becoming a part of the QuantaSing Group, Kelly’s Education has seen remarkable growth. Our newly established partnerships will propel us forward in our mission to establish ourselves as the premier children’s education brand in Hong Kong.”

QuantaSing to open Hong Kong office in summer

QuantaSing announces the intention to open an office in Hong Kong, the Company’s first office outside Mainland China. The new office will be situated in a prime location close to investors and business decisionmakers. With an opening planned for early-summer, the office will serve as a hub for innovation, collaboration, and further expansion into global markets. This marks an exciting new chapter in QuantaSing’s international growth journey.

About QuantaSing Group Limited

QuantaSing is a leading online service provider in China dedicated to improving people’s quality of life and well-being by providing lifelong personal learning and development opportunities. The Company is the largest service provider in China’s online adult learning market and China’s adult personal interest learning market in terms of revenue, according to a report by Frost & Sullivan based on data from 2022. By leveraging its proprietary tools and technology, QuantaSing offers easy-to-understand, affordable, and accessible online courses to adult learners, empowering users to pursue personal development. Leveraging its extensive experience in individual online learning services and its robust technology infrastructure, the Company has expanded its services to corporate clients, and diversified its operations into its e-commerce business and its AI and technology business.

For more information, please visit: https://ir.quantasing.com

Contact

Investor Relations

Leah Guo

QuantaSing Group Limited

Email: ir@quantasing.com

Tel: +86 (10) 6493-7857

Robin Yang, Partner

ICR, LLC

Email: QuantaSing.IR@icrinc.com

Tel: +1 (212) 537-0429

Public Relations

Brad Burgess, Senior Vice President

ICR, LLC

Email: Brad.Burgess@icrinc.com

Small Biotech Cullinan Goes All-In on Autoimmune CAR-T in $280M Pivot

In a bold strategic move, small-cap biotech Cullinan Oncology is transforming into an autoimmune disease company and rebranding as Cullinan Therapeutics. The Massachusetts company announced the major pivot alongside a $280 million private placement financing that extends its cash runway into 2028.

Cullinan is staking its future on the emerging potential of CAR-T cell therapies to treat autoimmune conditions like systemic lupus erythematosus (SLE). The company plans to advance its lead candidate CLN-978, a bispecific T cell engager originally developed for lymphoma, into SLE. An Investigational New Drug (IND) filing is targeted for the third quarter of 2024, with additional autoimmune indications likely to follow.

The strategic refocusing comes as preliminary data from small academic studies hint that CAR-T cells could induce durable remissions in autoimmune patients by depleting pathogenic B cells and modulating the immune system. In February, researchers reported that 8 out of 15 SLE patients achieved remission for over 1 year after CAR-T treatment, allowing them to discontinue all other medications.

“The unmet need in autoimmune diseases is vast, with most patients cycling through treatment after treatment without achieving remission,” said Cullinan CEO Alejandra Carvajal. “CAR-T cell therapy represents a potential paradigm shift, with an entirely novel mechanism to re-educate the immune system.”

Cullinan is one of the first movers in the autoimmune CAR-T space, but it won’t be alone for long. Peers like Kyverna, Cabaletta, Allogene and Arbor have all initiated programs or partnerships in the last year to develop similar cell therapies.

Cullinan has stopped enrolling patients in CLN-978’s lymphoma study to fully transition to autoimmune disorders. But the company is retaining its existing oncology pipeline, including lead asset zipalertinib in non-small cell lung cancer.

To fund the new autoimmune endeavors, Cullinan raised $280 million through the sale of shares and convertible securities to institutional investors. The private placement was led by venBio Partners and included Cullinan’s existing investors.

“This successful financing provides Cullinan with the resources to rapidly advance our CLN-978 program in SLE and beyond,” stated Carvajal. “We’re excited to lead the way into this new frontier at the intersection of cell therapy and autoimmune disease.”

Cullinan is making a high-risk, high-reward bet on still-unproven science. CAR-T’s efficacy in autoimmune conditions has only been explored in small patient numbers so far. But if the approach proves transformative, Cullinan could be at the vanguard of disrupting the large autoimmune drug market.

The hefty $280 million raise buys Cullinan plenty of runway to generate data from larger trials evaluating CLN-978 and shaping its future autoimmune portfolio. For autoimmune disease patients in need of new options, all eyes will be on Cullinan’s pioneering role in the promising CAR-T space.

Release – YS Biopharma to Report First Nine Months of Fiscal Year 2024 Financial Results on April 19, 2024

Research News and Market Data on YS

Download this Press Release

GAITHERSBURG, Md., April 16, 2024 /PRNewswire/ — YS Biopharma Co., Ltd. (NASDAQ: YS) (“YS Biopharma” or the “Company”), a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and delivering new generations of vaccines and therapeutic biologics for infectious diseases and cancer, today announced that it plans to release its financial results for the first nine months of the fiscal year ended March 31, 2024, before the U.S. market opens on Friday, April 19, 2024.

(PRNewsfoto/YishengBio Co., Ltd)

   

The Company’s management will hold an earnings conference call at 8:00 A.M. Eastern Time on Friday, April 19, 2024 to discuss the financial results. Listeners may access the call by dialing the following numbers:

United States Toll Free:           1-888-346-8982
International:                             1-412-902-4272
Mainland China Toll Free:       4001-201203
Canada Toll Free:                   1-855-669-9657
Hong Kong:                               852-301-84992

Upon dialing-in, participants should ask to be joined into the YS Biopharma Co., Ltd. call.

The replay will be accessible through April 26, 2024 by dialing the following numbers:

United States Toll Free:             1-877-344-7529
International:                     1-412-317-0088
Canada Toll Free:                 1-855-669-9658
Access Code:                       2468327

A live and archived webcast of the conference call will also be available at the Company’s investor relations website at https://investor.ysbiopharm.com/.

About YS Biopharma 

YS Biopharma is a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and commercializing new generations of vaccines and therapeutic biologics for infectious diseases and cancer. It has developed a proprietary PIKA® immunomodulating technology platform and a series of preventive and therapeutic biologics with a potential for improved Rabies, Coronavirus, Hepatitis B, Influenza, and Shingles vaccines. YS Biopharma operates in China, the United States, Singapore and the Philippines, and is led by a management team that combines rich local expertise and global experience in the bio-pharmaceutical industry. For more information, please visit www.ysbiopharma.com.

Investor Relations Contact

Alyssa Li
Director of Investor Relations
Email: ir@yishengbio.com 

Robin Yang
Partner, ICR, LLC
Tel: +1 (212) 537-4035
Email: YSBiopharma.IR@icrinc.com

Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/ys-biopharma-to-report-first-nine-months-of-fiscal-year-2024-financial-results-on-april-19-2024-302118047.html

SOURCE YS Biopharma Co., Ltd.

Eliem Bets Big on Autoimmune Pipeline with $210M Tenet Buyout

The biotech deal scene is heating up, with Eliem Therapeutics becoming the latest to double down on an emerging pipeline through M&A. The clinical-stage company announced it is acquiring private biotech Tenet Medicines for $120 million in stock, while simultaneously raising $120 million in a private placement to fund Tenet’s lead autoimmune disease program.

The transaction allows Eliem to pivot from its previous focus on neurological disorders to prioritize TNT119 – an anti-CD19 antibody that Tenet was developing across multiple autoimmune indications like lupus, immune thrombocytopenia, and membranous nephropathy. With $210 million in projected cash reserves after closing, the combined company plans to rapidly advance TNT119 into Phase 2 studies for systemic lupus erythematosus and immune thrombocytopenia in the second half of 2024.

“TNT119 represents a promising clinical asset across autoimmune diseases where there is a clear need for improved treatments,” said Andrew Levin, Eliem’s Executive Chairman. “This deal allows us to accelerate development of a potential best-in-class therapy while creating value for our shareholders.”

The acquisition continues the torrid pace of deal-making in biotech as companies look to revamp pipelines and build out emerging focus areas through M&A. Just in the first few months of 2024, over $30 billion in biotech acquisitions have been announced according to Bloomberg data.

Major deals included Vertex’s $4.9 billion buyout of Alpine Immune Sciences to gain a promising IgA nephropathy drug, Pfizer’s $43 billion acquisition of Seagen to bolster its oncology portfolio, and Amgen’s $27.8 billion deal for Horizon Therapeutics’ rare disease pipeline. Now Eliem is the latest to join the fray, making a significant bet on the autoimmune space.

“We believe TNT119 has best-in-class potential and could transform treatment for many autoimmune patients,” said Stephen Thomas, who will become interim CEO of the combined company after previously leading Tenet.

In addition to systemic lupus erythematosus and immune thrombocytopenia, TNT119 has also shown preliminary efficacy in treating membranous nephropathy – an autoimmune kidney disorder. By depleting B cells that produce autoantibodies driving the diseases, the therapy could provide a novel approach across this set of serious inflammatory conditions.

To fund TNT119’s ambitious development program, Eliem secured backing from a syndicate of major healthcare investors including RA Capital, Deep Track Capital, Boxer Capital and Janus Henderson in the $120 million private placement.

The wave of biotech deals has been driven by larger players looking to rebuild pipelines, while smaller companies seek resources to push programs forward amid a challenging economic environment and tight funding markets. With its Tenet acquisition, Eliem is aiming to thread that needle – gaining a promising clinical asset while raising enough capital to rapidly usher it through key studies.

For Eliem shareholders, the risky pivot to autoimmune therapies represents a major strategic shift. But if TNT119 can live up to its blockbuster aspirations, it could allow the company to go from an underdog in neurological disorders to a standout in the hot autoimmune space. That big “if” appears to be a gamble Eliem and its investors are willing to take amid biotech’s current deal-making renaissance.

Release – Ocugen To Participate In Fireside Chat At The Emerging Growth Virtual Healthcare Equity Conference, Presented By Noble Capital Markets

Research News and Market Data on OCGN

April 12, 2024

MALVERN, Pa., April 12, 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 Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder will participate in a fireside chat during the Emerging Growth Virtual Healthcare Equity Conference, presented by Noble Capital Markets, taking place from April 17 – 18, 2024.

Details of the fireside chat are as follows:

Date: Wednesday, April 17, 2024

Time: 10 a.m. ET

Location: Presentation Track 1

Webcast: Available in the Events section of Ocugen website

During the fireside chat and one-on-one sessions, Dr. Musunuri will showcase the Company’s recent business and clinical development progress including FDA clearance to initiate a Phase 3 clinical trial for OCU400—a modifier gene therapy for patients battling retinitis pigmentosa (RP). OCU400 is the first gene therapy to enter a late-stage trial with a broad RP indication.

A live webcast of the fireside chat will be available in the Events section of the Company’s website and as part of a complete catalog of presentations available on Channelchek—the investor portal created by Noble. The webcast will be archived on the Company’s website for 90 days following the event. 

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
Tiffany.Hamilton@ocugen.com

Cadrenal Therapeutics (CVKD) – Tecarfarin Receives Orphan Drug Designation For Second Indication


Friday, April 12, 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.

Orphan Designation Received For Circulatory Assist Devices. Cadrenal Therapeutics has received Orphan Drug designation for tecarfarin as an anticoagulant in patients with circulatory-assist devices, including ventricular assist devices (VADs) and total artificial hearts. This is the second Orphan designation for tecarfarin. The first indication, atrial fibrillation associated with end stage renal disease (AFib with ESRD), is expected to begin a Phase 3 trial in 2Q24.

Circulatory Assist Device Patients Do Not Have Effective Anticoagulants. Mechanical devices to assist blood circulation can activate the clotting cascade, putting patients who receive them at risk of clots or thromboembolisms. Commonly used anticoagulants in the direct oral anticoagulant (DOAC) category have not shown efficacy in these patients and have been contraindicated. This leaves the patients at high risk for thromboembolic events and hospitalizations.


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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. 

Vertex Banks on Autoimmune Therapy in $4.9 Billion Alpine Acquisition

Boston-based biotech giant Vertex Pharmaceuticals announced today that it has agreed to acquire Alpine Immune Sciences for $4.9 billion in cash, placing a major bet on the smaller company’s promising drug candidate for treating serious autoimmune diseases.

The crown jewel of the acquisition is Alpine’s lead molecule povetacicept, a dual antagonist of the BAFF and APRIL proteins that have been implicated in driving several autoimmune and inflammatory conditions. Through Phase 2 trials, povetacicept has demonstrated potentially best-in-class efficacy for treating IgA nephropathy (IgAN), a serious progressive kidney disease caused by autoimmune complexes.

IgAN is the most common cause of primary glomerulonephritis (inflammation of the kidney’s filtering units) worldwide, affecting approximately 130,000 people in the U.S. alone. The disease frequently leads to end-stage renal failure, yet there are currently no approved treatments that target the underlying causes of IgAN. Povetacicept is slated to enter pivotal Phase 3 clinical trials in the second half of 2024.

“Alpine is a compelling strategic fit that furthers our ambition of creating transformative medicines for serious diseases with high unmet need,” said Reshma Kewalramani, Vertex’s CEO and President. “We look forward to bringing povetacicept, a potential best-in-class treatment for IgAN, to patients faster.”

But Vertex is betting big that povetacicept’s impact could extend far beyond just IgAN. Due to its dual mechanism targeting BAFF and APRIL, the drug candidate holds promise as a potential “pipeline-in-a-product” for treating other autoimmune diseases affecting the kidneys like membranous nephropathy and lupus nephritis. Clinical trials are also evaluating povetacicept’s utility for autoimmune cytopenias that destroy blood cells.

The $4.9 billion acquisition allows Vertex, a leader in cystic fibrosis treatments, to expand into autoimmune and inflammatory diseases – one of the hottest areas of drug development. It also provides Vertex with Alpine’s protein engineering expertise that could unlock new therapeutic modalities.

“Povetacicept has demonstrated potential best-in-class attributes and has broad development potential across autoimmune conditions with significant unmet need,” said Mitchell Gold, Alpine’s CEO. “We’re excited for the opportunity to make a meaningful difference as part of Vertex.”

The deal is structured as an all-cash tender offer, with Vertex paying $65 per share for Alpine’s outstanding stock – a substantial 92% premium over Alpine’s closing price on April 9th. Vertex expects to finance the $4.6 billion net transaction cost through a combination of existing cash on hand and new debt financing.

The acquisition, which was unanimously approved by both companies’ boards, is expected to close in the second quarter of 2024 pending regulatory approval and other customary closing conditions. It marks Vertex’s second acquisition in the autoimmune disease space in recent years, having purchased protein therapeutics firm Semma Therapeutics in 2019 for $950 million.

With povetacicept’s promising data and Vertex’s resources behind it, the combined company will be well-positioned to rapidly advance a potentially transformative new class of autoimmune therapies. But at a lofty price tag nearing $5 billion, the deal places a major bet that the Alpine drug can live up to its blockbuster aspirations.

Tonix Pharmaceuticals (TNXP) – Increasing The Focus on Fibromyalgia


Thursday, April 11, 2024

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.

We Expect Tonmya To Drive TNXP Forward. Since announcing successful Phase 3 RESILIENT trial data in late December, Tonix has been shifting its development and financial strategy to focus on filing the NDA for Tonmya approval during 2H24. The company has raised capital and plans lower R&D expenses to preserve cash. We have revised our earnings estimates and price target while maintaining our Outperform rating.

Tonix Will Focus On Products For CNS Disorders and Reduce Expenses. Tonix has developed broad pipeline of products in infectious disease, biodefense, and immunology that grew from its original work in neurology and immunology. In the coming year, it will focus on the NDA filing and premarketing activities for Tonmya. We have reduced our clinical expenses estimates, since the large trials have been completed and no late-stage trials are scheduled.


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Century Therapeutics Makes Bold Move in Autoimmune Disease and Cell Therapy

Century Therapeutics, a pioneering biotech company developing induced pluripotent stem cell (iPSC)-derived cell therapies, announced a transformative set of initiatives that could reshape the landscape of cell therapy in cancer and autoimmune diseases.

The centerpiece is an ambitious expansion of Century’s lead program CNTY-101, a novel CD19-targeting immune cell therapy, into multiple autoimmune indications beyond the previously planned systemic lupus erythematosus (SLE) trial. This strategic pivot is backed by a $60 million private financing round and bolstered by the acquisition of Clade Therapeutics.

CNTY-101 is an allogeneic, iPSC-derived natural killer (iNK) cell therapy that has shown promising potential in eradicating cancerous B-cells in early clinical trials. Century believes its unique design, including gene edits to enable repeat dosing without lymphodepletion, could make it an ideal therapy for autoimmune diseases driven by B-cell dysregulation.

“We’ve seen compelling translational data pointing to CNTY-101’s potential in diseases like lupus,” said Century CEO Brent Pfeiffenberger. “This financing allows us to aggressively pursue that opportunity across multiple autoimmune indications with high unmet need.”

While the SLE trial remains on track for 2024, Century plans additional regulatory filings for CNTY-101 in autoimmune diseases in the second half of this year, supported by the $60 million raise from investors like Bain Capital Life Sciences and Adage Capital.

But the company didn’t stop there. Century also acquired Clade Therapeutics and its innovative iPSC-derived alpha beta T-cell platform for $35 million upfront, with potential future milestones. The deal adds three promising preclinical cancer and autoimmune programs to Century’s pipeline.

More importantly, it provides Century with next-generation capabilities to manufacture highly-functional, engineered T-cell therapies from iPSCs, something the field has long sought after.

“Clade’s groundbreaking platform replicates the natural T-cell development process, overcoming key limitations of current therapies,” said Century R&D President Hy Levitsky, M.D. “Combined with our iPSC-derived NK and gamma delta T-cells, this gives us unparalleled ability to create potential cures across a wide range of diseases.”

The move establishes Century as a preeminent player in allogeneic, off-the-shelf iPSC cell therapy, with an arsenal of NK cells, alpha beta T-cells, and gamma delta T-cells for oncology and autoimmune diseases. It diversifies the pipeline with complementary assets while providing a renewable cell source to manufacture consistent, high-quality therapies.

While still in early stages, some analysts view this as an aggressive and smart play by Century to stay ahead of the competition in this rapidly evolving space. By expanding into autoimmune diseases, acquiring transformative technology, and putting significant capital behind it all, Century is cementing its position as an iPSC cell therapy leader looking to deliver on the modality’s long-awaited promise.

Biotech Buzz: Adial and Skye Bioscience Deliver Promising Updates Amid Sector Momentum

The biotech and healthcare sectors have seen a flurry of activity in recent weeks, with companies making strides through drug developments, clinical trials, and corporate milestones. Two firms generating buzz today are Adial Pharmaceuticals (ADIL) and Skye Bioscience (SKYE), both reporting encouraging news that has fueled investor interest.

Adial Pharmaceuticals, a clinical-stage biopharmaceutical company focused on developing addiction therapies, announced the publication of a peer-reviewed article highlighting the promising safety data and high patient compliance observed with its lead investigational drug AD04 in a Phase 3 clinical trial for alcohol use disorder (AUD).

The study, published in the European Journal of Internal Medicine, comprehensively analyzed the liver safety profile of low-dose AD04 compared to a placebo in patients with AUD and a specific genetic profile. Notably, AD04 did not significantly impact biochemical markers of liver injury like ALT, AST, and bilirubin levels, underscoring its potential to address AUD while mitigating liver damage risks.

Moreover, AD04 demonstrated an impressive safety and tolerability profile, with low adverse event occurrence, high medication adherence, and minimal dropout rates – a rarity for AUD treatments. Adial’s CEO, Cary Claiborne, expressed enthusiasm about providing a precision treatment tailored to individuals with AUD, potentially offering a novel approach to managing alcohol consumption and liver harm.

Separately, Skye Bioscience (SKYE), a clinical-stage biotech focused on the endocannabinoid system, achieved a significant milestone by uplisting its common stock to the Nasdaq Global Market. Trading under the ticker “SKYE” is expected to commence on April 11th.

The Nasdaq uplisting is a testament to Skye’s recent accomplishments, including advancing its Phase 2 clinical programs, strengthening its financial position, and broadening its shareholder base. As CFO Kaitlyn Arsenault noted, the move aims to enhance visibility, liquidity, and ultimately drive long-term shareholder value.

Skye’s pipeline includes SBI-100 Ophthalmic Emulsion, a CB1 agonist being studied in a Phase 2 trial for glaucoma and ocular hypertension, with top-line data expected this quarter. Additionally, the company plans to launch a Phase 2 clinical trial in Q3 2024 for nimacimab, a peripheral CB1 inhibitor, targeting obesity through monotherapy and combination arms with a GLP-1R agonist.

These developments from Adial and Skye underscore the vibrant activity within the biotech and healthcare sectors, where companies are continuously striving to advance innovative therapies and achieve corporate milestones.

Contributing to the sector’s momentum is the upcoming Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference, taking place on April 17-18. This premier event will bring together industry leaders, investors, and emerging companies, providing a platform to showcase groundbreaking research, discuss market trends, and explore potential partnerships and collaborations.

With the biotech and healthcare industries consistently evolving, such conferences play a crucial role in fostering collaboration, facilitating knowledge-sharing, and driving progress towards improving patient outcomes and advancing healthcare solutions.

As companies like Adial and Skye continue to make strides, the broader biotech and healthcare sectors remain vibrant and poised for growth, fueled by scientific advancements, regulatory approvals, and investor confidence. The upcoming Noble Capital Markets Virtual Healthcare Conference promises to further catalyze innovation and propel the industry forward.

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

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, NR2E3NR2E3 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
Tiffany.Hamilton@ocugen.com

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

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:

ir@unicycive.com
(650) 543-5470

SOURCE: Unicycive Therapeutics, Inc.

Released April 10, 2024