Tag: Gene Therapy

Ocugen (OCGN) – 2Q24 Reported With Summary Of Progress In Clinical Trials

Posted on by slightbourne@noblecapitalmarkets.com


Friday, August 09, 2024

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

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

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

Ocugen reported a 2Q24 loss of $10.3 million or $(0.04) per share. During the quarter, the first patient was treated in the Phase 3 liMeliGhT (pronounced “limelight”) trial for OCU400 in retinitis pigmentosa (RP). Separately, the Phase 1/2 OCU410 trial for geographic atrophy in dry AMD completed dosing of its third cohort and began Phase 2. Cash on June 30 was $15.7 million, excluding $32.6 million raised in a public offering on August 2. Based on our quarterly estimates, we project cash to last until 2H25 with about $40 million in cash at the end of 3Q24.

OCU400 Began The Phase 3 liMeliGht Trial and Expanded Access. During the quarter, the first patient in the Phase 3 liMeliGhT trial testing OCU400 in retinitis pigmentosa (RP) was treated. The trial has one arm testing OCU400 in patients that have the RHO mutation and another arm with any of several gene mutations associated with RP. Each arm will have 75 patients for a total target enrollment of 150 patients.


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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. 

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

Posted on by slightbourne@noblecapitalmarkets.com

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.

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

Posted on by slightbourne@noblecapitalmarkets.com

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.

Cargo Therapeutics Positions for One of 2023’s Largest Biotech IPOs

Posted on by slightbourne@noblecapitalmarkets.com

Cargo Therapeutics is gearing up for an initial public offering (IPO) that could be one of the biggest biotech listings in 2023. The cancer-focused gene therapy startup aims to raise around $300 million through the sale of 18.75 million shares priced between $15 to $17.

If successful, it would be a rare bright spot in an otherwise dreary IPO market for life science companies this year. Cargo’s offering comes at a time when biotech IPOs have slowed to a trickle amid volatile market conditions.

The company is developing CRG-022, an experimental CD22 CAR-T therapy for certain blood cancers. Cargo’s candidate takes a patient’s own T-cells and engineers them to target and kill cancerous B-cells expressing the CD22 antigen.

Cargo hopes CRG-022 can benefit patients with large B-cell lymphoma who have failed previous CD19 CAR-T treatment. It initiated a potentially pivotal Phase 2 trial for this population in September. Data from the study could support regulatory approval in 2025.

Beyond blood cancers, Cargo intends to study CRG-022 in solid tumors expressing CD22. This includes some forms of breast, lung, colorectal and liver cancers. The company believes its therapy may demonstrate activity in a wider range of advanced cancers than existing CAR-Ts.

Proceeds from the IPO will help fund Cargo’s clinical programs and earlier R&D. According to its SEC filing, the company had $42.4 million in cash at the end of June 2022 but accumulated losses exceeding $77 million. The capital infusion will provide runway through the expected interim Phase 2 data readout.

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

Cargo’s offering will be a key test of investor appetite for preclinical biotech IPOs. These platform companies developing multiple experimental drugs based on a core technology have fallen out of favor recently.

However, Cargo could attract more interest with CRG-022 already in mid-stage testing and potential for near-term commercialization. The FDA has approved several CAR-T cell therapies over the past five years, providing a regulatory pathway for followers like Cargo.

But biotech IPOs in general face challenges in the current environment. Volatility, rising interest rates, and recession fears have rocked stock markets in 2022. Biotech has been among the hardest hit sectors, with the Nasdaq Biotech Index down over 30% year-to-date.

Companies pursuing IPOs have been forced to scale back valuations and offering sizes. Those that do list are often trading below issue price. So far in 2022, only around 15 biotechs have braved public markets compared to 60+ in recent years.

Yet some experts believe companies with innovative therapies and strong data can still obtain IPO financing. Cargo will provide a barometer of latent investor demand for biotech offerings amid the downturn.

A successful IPO could potentially reinvigorate biotech’s depressed financing environment. It may encourage other firms contemplating IPOs to move forward with planned deals.

Conversely, a lackluster response would signal biotech IPOs remain out-of-favor for now. This could lead companies to instead pursue private financing to advance programs and extend runways.

In any case, Cargo’s listing will generate insight into the health of biotech capital markets. The deal’s performance could significantly influence investment decisions and sentiment around the battered sector heading into 2023.

All eyes will be on whether one of biotech’s most promising young companies can buck the prevailing IPO trends. Cargo’s offering will help determine if the window for issuance might finally be opening back up.

Kyowa Kirin Bets on Gene Therapy With $477M Orchard Therapeutics Acquisition

Posted on by slightbourne@noblecapitalmarkets.com

Japan-based pharma Kyowa Kirin has agreed to acquire gene therapy specialist Orchard Therapeutics in a deal worth up to $477.6 million. The buyout aims to strengthen Kyowa Kirin’s emerging presence in the high-potential genetic medicine field.

Under the terms, Kyowa Kirin will pay $16 per Orchard ADS in cash upfront, representing a 144% premium to Orchard’s recent share price. Orchard shareholders will also receive a contingent value right worth an additional $1 per ADS if certain regulatory milestones are met.

The total potential payout values the deal at $477.6 million. Kyowa Kirin expects the acquisition to close in Q1 2024 pending approvals.

Orchard focuses on developing therapies using genetically modified hematopoietic stem cells (HSCs) taken from patients themselves. Its treatments aim to correct the underlying genetic cause of diseases in a single administration.

The company’s lead asset is Libmeldy, approved in Europe for treating a rare metabolic disorder called MLD. It also has two other programs for pediatric neurological conditions in late-stage testing.

Beyond the commercial and near-term pipeline assets, Kyowa Kirin gains Orchard’s HSC gene therapy platform. This technology can be leveraged to develop new treatments for diseases in Kyowa Kirin’s wheelhouse like oncology, autoimmune disorders, and others.

Kyowa Kirin has made gene and cell therapy a priority as part of its vision to deliver transformative new medicines. Orchard’s proven development capabilities and leadership position in HSC gene therapy make it an ideal fit for this strategy.

The high premium paid reflects Orchard’s status as a pioneer in the burgeoning field of genetic medicine. The deal provides Kyowa Kirin immediate scale and expertise in leveraging gene therapy.

Kyowa Kirin also gains commercial infrastructure to support the global launch of Libmeldy. The FDA is currently reviewing Libmeldy for approval in the U.S. with a decision date in March 2024.

Orchard’s two other clinical programs in development also address rare pediatric neurological disorders with immense unmet need. Additional earlier stage preclinical assets add further upside to the pipeline.

The deal continues biotech industry consolidation as large players acquire innovators to reinforce their drug development pipelines. The competition among pharmas for gene therapy assets has intensified as the field matures.

For Orchard investors, the buyout represents a significant premium after a long stretch of the stock languishing. But with cash running low, the company faced challenges transitioning its pipeline programs to commercial status alone.

The deal provides ample resources to continue advancing Orchard’s mission of tackling rare genetic diseases. Kyowa Kirin expects to hit $1 billion in sales from the MLD treatment alone if approved in the U.S.

Gene therapy has disrupted drug development over the past decade with its potential to deliver curative, lifelong treatment through a single administration. As technology improves, dealmaking and R&D in the space continues gaining steam.

Kyowa Kirin is the latest pharma to bet big on gene therapy’s possibilities. If it can successfully harness Orchard’s specialized platform and assets, the deal may pave the way to developing life-changing genetic medicines while delivering solid returns to shareholders.

Regeneron Strengthens Gene Therapy Pipeline Through Acquisition of Decibel Therapeutics

Posted on by slightbourne@noblecapitalmarkets.com

Regeneron Pharmaceuticals has expanded its gene therapy programs by acquiring Decibel Therapeutics, a biotech company focused on developing treatments for hearing loss. The $1.1 billion deal provides Regeneron with three promising gene therapy candidates that use adeno-associated virus (AAV) vectors to restore hearing.

The most advanced asset is DB-OTO, an AAV-based gene therapy designed to provide long-term hearing to individuals with profound congenital hearing loss caused by otoferlin gene mutations. DB-OTO is currently being evaluated in a Phase 1/2 clinical trial known as CHORD. The gene therapy aims to deliver a functional copy of the otoferlin gene to inner ear hair cells, potentially enabling hearing restoration.

The acquisition also includes two earlier-stage gene therapies, AAV.103 and AAV.104, targeting other genetic forms of hearing loss – GJB2 and STRC respectively. Both utilize a similar AAV gene delivery approach to DB-OTO.

According to Regeneron, the addition of Decibel’s pipeline and capabilities will strengthen its genetic medicines portfolio. Gene therapy has become a major focus for Regeneron beyond its foundational expertise in antibodies. The company is exploring gene silencing, gene editing and gene therapy technologies across a range of therapeutic areas.

Take a look at Ocugen Inc., a biotechnology company focused on discovering, developing and commercializing novel gene and cell therapies and vaccines.

Hearing loss represents a new area for Regeneron, building on an existing collaboration with Decibel. Integration of Decibel’s team and experience in inner ear biology and AAV gene therapy for hearing disorders will be invaluable as Regeneron advances the acquired programs.

Gene therapy aims to address disease at its genetic root cause by introducing functional genes into cells. The goal is to durably restore protein expression and correct the downstream impacts of gene mutations. Gene therapy has shown promise for treating rare monogenic disorders like certain forms of inherited hearing loss.

Both Regeneron and Decibel have utilized AAV vectors to deliver gene therapy payloads. AAV is considered one of the most effective vehicles for gene delivery and has an established safety profile. The viruses can be engineered to target specific cell types following injection into the body.

For DB-OTO, the AAV vector carries a functional copy of the otoferlin gene. Inner ear hair cells are the targets for gene transduction. Otoferlin protein is critical for hearing signal transduction, but mutations in the encoding gene cause profound congenital deafness. Gene therapy aims to restore otoferlin expression and regain hearing function.

Regeneron’s push into gene therapy aligns with its mission of tackling serious diseases with novel technologies. Gene-based treatments have potential for one-time curative therapies. The acquisition of Decibel’s pipeline further diversifies Regeneron’s genetic medicine capabilities as it aims to help patients worldwide.

Release – Ocugen, Inc. Announces Connie Collingsworth Joins Business Advisory Board

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

September 21, 2023

MALVERN, Pa., Sept. 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, biologics, and vaccines, today announced that Connie Collingsworth, former Chief Operating Officer of the Bill and Melinda Gates Foundation, will join the Company’s Business Advisory Board (BAB). The BAB was established in June 2023 to assist in driving public/private partnerships with governments around the world; pursuing business collaborations, partnerships, and licensing opportunities; creating awareness of the Company’s differentiated capabilities; and promoting access to the Company’s therapies around the world.

“Connie brings value-added expertise in all aspects of strategic growth and organizational governance—across a variety of industries—that will offer unique insights to the BAB,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “Connie’s deep financial background at a global level will provide meaningful counsel to Ocugen as we pursue new business development opportunities to support and evolve our clinical programs.”

Ms. Collingsworth has been a primary advisor to Bill Gates, Melinda French Gates, and Warren Buffett regarding governance. During her tenure with the Bill and Melinda Gates Foundation, she designed and implemented the core operational infrastructure and created the intellectual property strategy known as “Global Access,” as well as advising on the creation of a $2.5 billion strategic investment fund.

“I am inspired by Ocugen’s commitment to not only developing novel therapies that address unmet medical needs for patients with serious conditions, but their passion to ensure access to all patients around the world,” said Ms. Collingsworth. “I look forward to sharing my diverse experiences as an executive and a board member so that patients can potentially benefit.”

Ms. Collingsworth joins Senator Pat Toomey, Ambassador Joseph W. Westphal, Ph.D., and Dennis Carey, Ph.D. on the BAB. These advisors will work alongside the Executive Leadership Team to offer guidance to enable the Company to fulfill its mission and achieve its short- and long-term strategy.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
Head of Corporate Communications
Tiffany.Hamilton@ocugen.com

Source: Ocugen

Ocugen (OCGN) – OCU400 Update Shows Improvement In Three Vision Measures

Posted on by cpereira@noblefcm.com


Thursday, September 14, 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.

Data Shows Clinically Meaningful Improvements. Ocugen provided an update to the Phase 1/2 clinical trial data for OCU400 in retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA), two rare diseases in which genetic mutations cause loss of vision and progress to blindness. We see these data as clinically meaningful for these indications as well as providing proof-of-concept for Ocugen’s method of targeting a “master control gene” that has downstream effects on other genes.

We See Proof-of-Concept For The “Master Control Gene” Approach. RP is a group of genetic disorders with over 100 possible genetic mutations that may be present and contributing to loss of vision. This makes the course of disease highly variable and difficult to target with a single therapy. Ocugen has been developing OCU400 to target the RHO and NR2E3 genes that control pathways and regulate other genes that act later in those pathways. The data show that OCU400 can affect several genetic pathways in eye, with improvements in three measures of vision.


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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. 

Rocket Shares Soar as FDA Aligns on Danon Disease Gene Therapy Trial

Posted on by slightbourne@noblecapitalmarkets.com

Rocket Pharmaceuticals (NASDAQ: RCKT) announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the design of a global pivotal Phase 2 trial for its gene therapy RP-A501 in Danon Disease. This marks an important milestone on the path to delivering a potentially transformative treatment for this devastating inherited cardiomyopathy.

Danon Disease is caused by genetic mutations in the LAMP2 gene leading to fatal heart failure. There are currently no approved therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe, taking the lives of most males by age 20 and females by 40. RP-A501 aims to be the first-ever therapy to change the course of Danon Disease.

The news sent Rocket’s stock price soaring 34% to $20.46 in after-hours trading on Tuesday, as investors welcomed the positive regulatory update.

The planned Phase 2 pivotal trial will be a global, single-arm, open label study enrolling 12 Danon Disease patients. This includes a pediatric safety lead-in of 2 patients. All participants will receive a dose of 6.7 x 1013 GC/kg of RP-A501 delivered through intravenous infusion.

To support potential accelerated approval by the FDA, the co-primary efficacy endpoints at 12 months are LAMP2 protein expression and reduction in left ventricular mass, a key measure of heart damage. Expression of LAMP2, which is deficient in Danon patients, and decreased cardiac hypertrophy would signal RP-A501 is restoring cardiac function at its root genetic cause.

Take a moment to look at Ocugen Inc., a biotechnology company focused on discovering, developing and commercializing novel gene and cell therapies and vaccines.

Secondary trial endpoints consist of the biomarker troponin, heart failure questionnaires, functional classification, event-free survival, and safety. These could support eventual full approval. A concurrent global natural history study will provide an external control arm for comparison.

According to Rocket CEO Dr. Gaurav Shah, “RP-A501 has the potential to restore normal cardiac function and provide a lifetime of benefit to patients with Danon Disease who have no other viable treatment options.” The company believes this pivotal trial design sets the most rapid path to deliver RP-A501 to patients in dire need.

Rocket’s gene therapy approach involves using an engineered virus called AAV9 to deliver a functional LAMP2 gene into patients’ heart cells. The gene insert encodes LAMP2B, a key protein involved in the cellular recycling process called autophagy. Restoring LAMP2B in the heart could potentially halt the accumulation of cellular debris and improve cardiac structure and function.

The company has already manufactured sufficient high-quality RP-A501 drug product at its in-house cGMP facility to supply the full pivotal Phase 2 trial. Qualified potency assays are also in place to support quality control and regulatory compliance.

Looking ahead, Rocket plans to file a Clinical Trial Application in the EU in Q3 2023 to initiate study activities abroad. The company also recently secured an ICD-10 code from the Centers for Medicare and Medicaid Services for LAMP2 deficiency, which will support diagnostic efforts.

This alignment with the FDA represents a major achievement for both Rocket and the Danon Disease community. RP-A501 would be the first-ever approved treatment for this deadly cardiovascular condition. The gene therapy aims to be a one-time curative infusion that could provide transformative and lifelong benefits to affected patients.

Beyond Danon Disease, Rocket believes this program paves the way for developing genetic medicines against other inherited heart diseases. Cardiac gene therapy has long faced hurdles, but the company is forging a new path for treating genetic cardiovascular conditions at their root.

With the pivotal Phase 2 trial design now locked in, Rocket can move full speed ahead on enrolling patients and gathering data to support potential approval. The company expects to release initial results from the trial evaluating RP-A501 in 2024. This could lead to a approved treatment for Danon Disease in the not too distant future—bringing tremendous hope to patients and families affected by this devastating illness.

Tonix Pharmaceuticals announces poster presentation involving TNX-1700 in preclinical colorectal cancer models at the 7th International Cancer Immunotherapy Conference 2023.

Ocugen (OCGN) – Moving Past Covaxin, Focusing On Gene Therapy and Pipeline Products

Posted on by cpereira@noblefcm.com


Monday, May 08, 2023

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product, Covaxin, is a killed-virus vaccine for COVID-19 in-licensed from Bharat Biotech (India). The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

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

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

Financial Report Includes Discontinuation Of Covaxin. Ocugen reported 1Q23 financial results of a loss of $16.5 million or $(0.07) per share, beating our estimate of a loss of $23.9 million or $(0.11) per share.  The difference was largely due to lower Research and Development spending at $9.6 million compared to our estimate of $15.9 million, attributed to the discontinuation of Covaxin development. Cash on hand at March 31 was $76.7 million, with the reduction in R&D expected to extend the cash runway into 1Q24.

Moving Beyond Covaxin. In view of recent changes in FDA guidance for monovalent COVID-19 vaccines, Ocugen has determined that Covaxin is no longer commercially viable and has discontinued development. We see this as a neutral to mild positive since the company had already announced that it would not continue development without outside funding. This should complete the shift to the gene therapy platform, NeoCart, and the inhaled vaccines.


Get the Full Report

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. 

Ocugen (OCGN) – Positive Preliminary Data Shows Proof-Of-Concept For OCU400

Posted on by cpereira@noblefcm.com


Monday, April 17, 2023

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product, Covaxin, is a killed-virus vaccine for COVID-19 in-licensed from Bharat Biotech (India). The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

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

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

Preliminary Data Shows Proof-Of-Concept. Ocugen reported preliminary data from its Phase 1/2 trial testing OCU400 gene therapy in retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA).  The study was designed to establish safety and  test increasing dosage using trial endpoints designed to measure vision in low light and visual acuity. Patient responses showed improvements in function, with safety and tolerability. We see this as an important proof-of-concept for OCU400 and Ocugen’s gene therapy technology.

Data Showed Functional Improvements In Vision. The data presented was from a preliminary analysis of 7 patients in the first two cohorts who received the low and medium doses. These patients had either of two retinitis pigmentosa mutations, RHO or NR2E3. The outcome measures presented were the multi-luminance mobility test (MLMT), a measure of the ability to detect levels of light, and BCVA (best corrected visual acuity).


Get the Full Report

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. 

Ocugen (OCGN) – FY2022 Reported With Pipeline Updates

Posted on by cpereira@noblefcm.com


Wednesday, March 01, 2023

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product, Covaxin, is a killed-virus vaccine for COVID-19 in-licensed from Bharat Biotech (India). The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.

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

Financial Results Were Within Expectations.  Ocugen reported 4Q22 and FY2022 Net Loss of $21.9 million or $(0.10) per share, and $81.4 million or $(0.38) per share for FY2022. Cash on hand as of December 31 was $90.9 million or $0.41 per share, which management projects will fund operations through 1Q24. On its quarterly conference call, management discussed recent milestones and development programs in gene therapy, cellular therapy, and vaccines.

Ophthalmology Programs Are Advancing.  Ocugen filed an IND for OCU200 to begin Phase 1 clinical trial in diabetic macular edema (DME), meeting the expected timeframe. Trial enrollment is expected to begin in 2Q23. A second IND filing is expected during 2Q23 for OCU410 in dry AMD and OCU410ST in Stargardt disease. The Phase 1/2 dose testing OCU400 in retinitis pigmentosa (RP) escalation phase previously completed enrollment for the RP cohort, with enrollment continuing in the Leber congenital amaurosis (LCA) patient cohort. Phase 3 is expected to start by year-end 2023.


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Ocugen (OCGN) – Phase 2/3 Immuno-Bridging Study Meets Co-Endpoints

Posted on by cpereira@noblefcm.com


Tuesday, January 17, 2023

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product, Covaxin, is a killed-virus vaccine for COVID-19 in-licensed from Bharat Biotech (India). The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.

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

Bridging Study Shows Equivalence. Ocugen announced that its Phase 2/3 immuno-bridging study for Covaxin met both its co-primary immunogenicity endpoints.  The study was designed to demonstrate that vaccination with Covaxin in US patients stimulated immunogenicity that is equivalent to the Phase 3 study conducted by Bharat Biotech in India in 2020.  The study also assessed the boosting effect of Covaxin in patients previously receiving an mRNA or viral vector vaccine.

Study Design.  The OCU-002 immuno-bridging study was a randomized, placebo-controlled study that enrolled 419 patients at 8 clinical sites. This was intended to test Covaxin in a demographically diverse population of healthy adults in the US with current SARS-CoV-2 strains. Participants received a first dose of Covaxin or placebo followed by a second dose 28 days later. The immune response was evaluated using microneutralization test (MNT) assay by a blinded observer.


Get the Full Report

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.