Friday, May 06, 2022

Baudax Bio (BXRX)
First Quarter 2022 Reported; Slow But Steady Progress

Gregory Aurand, Senior Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.

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

1Q 2022 revenues reported. Late Wednesday the 4th, Baudax Bio released their 1Q 2022 results and held a conference call early on the 5th.  Revenues of $.422 million, somewhat due to timing differences, missed our estimate of $.577 million.  Covid still muted Q1 procedures, but procedure growth showed signs of life late in the quarter.

Company reduced headcount and expenses. In March, the Company implemented a reduction in force (RIF) workplace plan to curtail expenses and reduce need for capital. Intended to reduce operating costs in connection with ANJESO commercialization, the company expects approximately $4.0 million in severance and related costs to be taken by end of 2Q 2022, of which approximately $1.7 million was taken late in Q1. Cash burn will be substantially reduced in 2H 2022 and beyond. The company is evaluating possible partnering options which will further reduce cash needs.

…

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. 

PsyBio Therapeutics Initiates Blood-Brain Barrier Permeability Testing

Research, News, and Market Data on PsyBio

OXFORD, Ohio and COCONUT CREEK, Fla., May 6, 2022  /CNW/ – PsyBio Therapeutics Corp. (TSXV: PSYB) (OTCQB: PSYBF) (”
PsyBio” or the “Company“), a fully integrated and intellectual property driven biotechnology company developing novel, bespoke psychoactive medicinal candidates targeting the potential treatment of mental health challenges, neurological disorders and other human health conditions, today reported that it has expanded its pre-clinical pipeline activities by initiating blood-brain barrier permeability testing. This additional testing procedure is expected to provide a comprehensive, state of the art research methodology with which PsyBio’s ever expanding portfolio of compounds can be more readily and rapidly developed. Favorable results from this type of testing are anticipated to expediate compound selection and progress toward the filing of Investigational New Drug (“IND”) applications with the US Food and Drug Administration (“FDA”).

“Establishing this type of testing is critically important for PsyBio to allow candidate selection to proceed more rapidly,” stated Michael Spigarelli, MD, PhD, MBA, PsyBio’s Chief Medical Officer. “We expect that this will allow us to effectively screen and select compounds that are active both in vitro and in
vivo to more efficiently produce psycho-targeted therapeutics intended to potentially improve mental and neurological health.”

PsyBio retains the global, exclusive, and perpetual right to license platform technology enabling rapid generation of tryptamines and related compounds through a biosynthetic process using genetically modified bacteria and has demonstrated the ability to manufacture one of its first promising therapeutic candidates at commercial scale.  This type of permeability testing is expected to improve the selection ability for compounds able to cross the blood-brain barrier and ultimately produce a brain-based effect.

“We continue to apply and utilize state-of-the-art research methodology to further our ability to produce a variety of psycho-targeted therapeutic candidates as one of the only biotechnology companies in the psychoactive therapeutic industry developing their own compounds,” stated Evan Levine, PsyBio’s Chief Executive Officer. “The initiation of this type of pre-clinical laboratory testing is expected to improve PsyBio’s ability to develop novel therapeutic agents.”

The Company is not proceeding with its previously announced proposed acquisition of Everest Pharma (Pty) Ltd., a Lesotho Company in Southern Africa, as described in the Company’s press release dated March 15, 2022.

About PsyBio Therapeutics Corp.

PsyBio is an intellectual property driven biotechnology company developing new, bespoke, fully approved, psycho-targeted therapeutics to potentially improve mental and neurological health. The team has extensive experience in drug discovery based on synthetic biology and metabolic engineering as well as clinical and regulatory expertise progressing drugs through human studies and regulatory protocols. Research and development is currently ongoing for naturally occurring psychoactive tryptamines originally discovered in different varieties of hallucinogenic mushrooms, other tryptamines and phenethylamines and combinations thereof. The Company utilizes a bio-medicinal chemistry approach to therapeutic development, in which psychoactive compounds can be utilized as a template upon which to develop precursors and analogs, both naturally and non-naturally occurring, specifically because they are already known to have an effect within the brain.

Cautionary Note Regarding Forward-Looking
Statements

This press release contains statements that constitute “forward-looking information” (“forward-looking
information“) within the meaning of applicable Canadian securities legislation. All statements, other than statements of historical fact, are forward-looking information and are based on expectations, estimates and projections as at the date of this news release. Any statement that discusses predictions, expectations, beliefs, plans, projections, objectives, assumptions, future events or performance (often but not always using phrases such as “expects”, or “does not expect”, “is expected”, “anticipates” or “does not anticipate”, “plans”, “budget”, “scheduled”, “forecasts”, “estimates”, “believes” or “intends” or variations of such words and phrases or stating that certain actions, events or results “may” or “could”, “would”, “might” or “will” be taken to occur or be achieved) are not statements of historical fact and may be forward-looking information. Forward looking-statements in this press release include statements regarding: PsyBio’s plans and ability to conduct successful blood-brain barrier permeability testing; the impact of favorable results from blood-brain barrier permeability testing on compound selection and progress toward the filing of IND applications with the FDA; PsyBio’s plans for filing IND applications with the FDA; PsyBio’s ability to develop novel therapeutic agents; PsyBio’s ability to develop novel formulations to potentially treat neurologic and psychologic conditions and other disorders; PsyBio’s ability to launch clinical trials; PsyBio’s ability to build its intellectual property portfolio of novel drug candidates; PsyBio’s ability to achieve cost competitive synthesis with reduced environmental impact over current production methods; and PsyBio’s ability to move target candidates into scaled commercial manufacturing and regulatory application.

In disclosing the forward-looking information contained in this press release, the Company has made certain assumptions, including that: blood-brain barrier permeability testing will yield favorable results; favorable results from blood-brain barrier permeability testing will have a positive impact on compound selection and progress toward the filing of IND applications with the FDA; PsyBio will be successful in protecting its intellectual property; PsyBio will be successful in discovering new valuable target molecules; PsyBio will be successful in obtaining IND applications and will be able to obtain all necessary approvals for clinical trials; PsyBio will be successful in launching clinical trials; the results of preclinical safety and efficacy testing will be favorable; PsyBio’s technology will be safe and effective; a confirmed signal will be identified in PsyBio’s selected indications; and that drug development involves long lead times, is very expensive and involves many variables of uncertainty. Although the Company believes that the expectations reflected in such forward-looking information are reasonable, it can give no assurance that the expectations of any forward-looking information will prove to be correct. Known and unknown risks, uncertainties, and other factors which may cause the actual results and future events to differ materially from those expressed or implied by such forward-looking information. Such factors include, but are not limited to: compliance with extensive government regulations; domestic and foreign laws and regulations adversely affecting PsyBio’s business and results of operations; decreases in the prevailing process for psilocybin and nutraceutical products in the markets in which PsyBio operates; the impact of COVID-19; and general business, economic, competitive, political and social uncertainties. Accordingly, readers should not place undue reliance on the forward-looking information contained in this press release. Except as required by law, the Company disclaims any intention and assumes no obligation to update or revise any forward-looking information to reflect actual results, whether as a result of new information, future events, changes in assumptions, changes in factors affecting such forward-looking information or otherwise.

PsyBio makes no medical, treatment or health benefit claims about PsyBio’s proposed products. The FDA or other similar regulatory authorities have not evaluated claims regarding psilocybin and other next generation psychoactive compounds. The efficacy of such products has not been confirmed by FDA-approved research. There is no assurance that the use of psilocybin and other psychoactive compounds can diagnose, treat, cure, or prevent any disease or condition. Vigorous scientific research and clinical trials are needed. PsyBio has not conducted clinical trials for the use of its intellectual property. Any references to quality, consistency, efficacy and safety of potential products do not imply that PsyBio verified such in clinical trials or that PsyBio will complete such trials. If PsyBio cannot obtain the approvals or research necessary to commercialize its business, it may have a material adverse effect on the PsyBio’s performance and operations.

The TSX Venture Exchange (the “TSXV“)
has neither approved nor disapproved the contents of this news release. Neither
the TSXV nor its Regulation Services Provider (as that term is defined in the
policies of the TSXV) accepts responsibility for the adequacy or accuracy of
this release.

Lineage Cell Therapeutics to Report First Quarter 2022 Financial Results and Provide Business Update on May 12, 2022

CARLSBAD, Calif.–(BUSINESS WIRE)–Lineage Cell
Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced that it will report its first quarter 2022 financial and operating results on Thursday, May 12, 2022, following the close of the U.S. financial markets. Lineage management will also host a conference call and webcast on Thursday, May 12, 2022, at 4:30 p.m. Eastern Time/1:30 p.m. Pacific Time to discuss its first quarter 2022 financial and operating results and to provide a business update.

Interested parties may access the conference call by dialing (866) 888-8633 from the U.S. and Canada and (636) 812-6629 from elsewhere outside the U.S. and Canada and should request the “Lineage Cell Therapeutics Call”. A live webcast of the conference call will be available online in the Investors section of Lineage’s website. A replay of the webcast will be available on Lineage’s website for 30 days and a telephone replay will be available through May 20, 2022, by dialing (855) 859-2056 from the U.S. and Canada and (404) 537-3406 from elsewhere outside the U.S. and Canada and entering conference ID number 1875641.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelial cell therapy in Phase 1/2a development for the treatment of geographic atrophy secondary to age-related macular degeneration, which is being developed under a worldwide collaboration with Roche and Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy, and (v) PNC1, a photoreceptor neural cell therapy for the treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit www.lineagecell.com or follow the company on Twitter @LineageCell.

Contacts

Lineage Cell Therapeutics, Inc. IR
Ioana C. Hone
(
ir@lineagecell.com)
(442) 287-8963

Solebury Trout IR
Justin Frantz
(
jfrantz@soleburytrout.com )
(617) 221-9100

Russo Partners – Media Relations
Nic Johnson or David Schull
Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com
(212) 845-4242

Image Credit: Fantastic Voyage (1966)

Nanoparticles are the Future of Medicine – Researchers are Experimenting with New Ways to Design Tiny Particle Treatments for Cancer

When you hear the word “nanomedicine,” it might call to mind scenarios like those in the 1966 movie “Fantastic Voyage.” The film portrays a medical team shrunken down to ride a microscopic robotic ship through a man’s body to clear a blood clot in his brain.

Nanomedicine has not reached that level of sophistication yet. Although scientists can generate nanomaterials smaller then several nanometers – the “nano” indicating one-billionth of a meter – today’s nanotechnology has not been able to generate functional electronic robotics tiny enough to inject safely into the bloodstream. But since the concept of nanotechnology was first introduced in the 1970s, it has made its mark in many everyday products, including electronics, fabrics, food, water and air treatment processes, cosmetics and drugs. Given these successes across different fields, many medical researchers were eager to use nanotechnology to diagnose and treat disease.

This article was republished with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It was written by and represents the research-based opinions of Duxin Sun, Professor of Pharmaceutical Sciences, University of Michigan.

I am a pharmaceutical scientist who was inspired by the promise of nanomedicine. My lab has worked on developing cancer treatments using nanomaterials over the past 20 years. While nanomedicine has seen many successes, some researchers like me have been disappointed by its underwhelming overall performance in cancer. To better translate success in the lab to treatments in the clinic, we proposed a new way to design cancer drugs using nanomaterials. Using this strategy, we developed a treatment that was able to achieve full remission in mice with metastatic breast cancer.

What is Nanomedicine?

Nanomedicine refers to the use of materials at the nanoscale to diagnose and treat disease. Some researchers define nanomedicine as encompassing any medical products using nanomaterials smaller than 1,000 nanometers. Others more narrowly use the term to refer to injectable drugs using nanoparticles smaller than 200 nanometers. Anything larger may not be safe to inject into the bloodstream.

Analysis of the World, from Experts

Several nanomaterials have been successfully used in vaccines. The most well-known examples today are the Pfizer-BioNTech and Moderna COVID-19 mRNA vaccines. These vaccines used a nanoparticle made of of lipids, or fatty acids, that helps carry the mRNA to where it needs to go in the body to trigger an immune response.

Researchers have also successfully used nanomaterials in diagnostics and medical imaging. Rapid COVID-19 tests and pregnancy tests use gold nanoparticles to form the colored band that designates a positive result. Magnetic resonance imaging, or MRI, often uses nanoparticles as contrast agents that help make an image more visible.

Several nanoparticle-based drugs have been approved for cancer treatment. Doxil (doxorubicin) and Abraxane (paclitaxel) are chemotherapy drugs that use nanomaterials as a delivery mechanism to improve treatment efficacy and reduce side effects.

While nanomedicine isn’t “Fantastic Voyage,” it shares the film’s treatment goal of delivering a drug exactly where it needs to go.

Cancer and Nanomedicine

The potential of nanomedicine to improve a drug’s effectiveness and reduce its toxicity is attractive for cancer researchers working with anti-cancer drugs that often have strong side effects. Indeed, 65% of clinical trials using nanoparticles are focused on cancer.

The idea is that nanoparticle cancer drugs could act like biological missiles that destroy tumors while minimizing damage to healthy organs. Because tumors have leaky blood vessels, researchers believe this would allow nanoparticles to accumulate in tumors. Conversely, because nanoparticles can circulate in the bloodstream longer than traditional cancer treatments, they could accumulate less in healthy organs and reduce toxicity.

Although these design strategies have been successful in mouse models, most nanoparticle cancer drugs have not been shown to be more effective than other cancer drugs. Furthermore, while some nanoparticle-based drugs can reduce toxicity to certain organs, they may increase toxicity in others. For example, while the nanoparticle-based Doxil decreases damage to the heart compared with other chemotherapy options, it can increase the risk of developing hand-foot syndrome.

Improving Nanoparticle-Based Cancer Drugs

To investigate ways to improve how nanoparticle-based cancer drugs are designed, my research team and I examined how well five approved nanoparticle-based cancer drugs accumulate in tumors and avoid healthy cells compared with the same cancer drugs without nanoparticles. Based on the findings of our lab study, we proposed that designing nanoparticles to be more specific to their intended target could improve their translation from animal models to people. This includes creating nanoparticles that address the shortcomings of a particular drug – such as common side effects – and home in on the types of cells they should be targeting in each particular cancer type.

Using these criteria, we designed a nanoparticle-based immunotherapy for metastatic breast cancer. We first identified that breast cancer has a type of immune cell that suppresses immune response, helping the cancer become resistant to treatments that stimulate the immune system to attack tumors. We hypothesized that while drugs could overcome this resistance, they are unable to sufficiently accumulate in these cells to succeed. So we designed nanoparticles made of a common protein called albumin that could deliver cancer drugs directly to where these immune-suppressing cells are located.

When we tested our nanoparticle-based treatment on mice genetically modified to have breast cancer, we were able to eliminate the tumor and achieve complete remission. All of the mice were still alive 200 days after birth. We’re hopeful it will eventually translate from animal models to cancer patients.

Nanomedicine’s Bright but Realistic Future

The success of some drugs that use nanoparticles, such as the COVID-19 mRNA vaccines, has prompted excitement among researchers and the public about their potential use in treating various other diseases, including talks about a future cancer vaccine. However, a vaccine for an infectious disease is not the same as a vaccine for cancer. Cancer vaccines may require different strategies to overcome treatment resistance. Injecting a nanoparticle-based vaccine into the bloodstream also has different design challenges than injecting into muscle.

While the field of nanomedicine has made good progress in getting drugs or diagnostics out of the lab and into the clinic, it still has a long road ahead. Learning from past successes and failures can help researchers develop breakthroughs that allow nanomedicine to live up to its promise.

Suggested Reading

Attacking Tumors by Returning Cancer Cells to the Body

Measuring Cancer Cells to Tailor Treatments

Ocugen (OCGN) NobleCon18 Presentation Replay

Cocrystal Pharma (COCP) NobleCon18 Presentation Replay

 

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Genprex Issues Shareholder Letter and Provides 2022 Corporate Update

Research, News, and Market Data on Genprex

Company achieves major
milestones in clinical development programs in 2022

Patient
treatment in Acclaim-2 clinical trial commences

 

AUSTIN, Texas — (May 5, 2022) — Genprex,
Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that it has issued a shareholder letter and corporate update outlining the Company’s recent progress in its clinical development programs and key milestones and achievements for 2022 and beyond.

“We have had a strong start to this year, achieving many key milestones for our Company,” said Rodney Varner, President and Chief Executive Officer of Genprex. “We announced our second Fast Track Designation for REQORSA™ Immunogene Therapy, we commenced patient treatment in our Phase 1/2 Acclaim-1 clinical trial for non-small cell lung cancer (NSCLC), we opened our Phase 1/2 Acclaim-2 clinical trial for NSCLC and commenced patient treatment in that trial (having treated the first patient in April 2022), and we expanded our clinical development pipeline to include small cell lung cancer (SCLC).  I am enthusiastic about our future as we continue to advance our leading edge gene therapy programs with the goal of extending hope and life to patients with serious disease and unmet need.”

To read the letter in its entirety, a digital copy of the Company’s Shareholder Letter can be found on the Company’s website here.

 

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex’s oncology program utilizes its unique, proprietary, non-viral ONCOPREX® Nanoparticle Delivery System, which the Company believes is the first systemic gene therapy delivery platform used for cancer in humans. ONCOPREX encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is then taken up by tumor cells that express proteins that are deficient. The Company’s lead product candidate, REQORSA™ (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance. In 2020, the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for REQORSA for NSCLC in combination therapy with AstraZeneca’s Tagrisso® (osimertinib) for patients with EFGR mutations whose tumors progressed after treatment with Tagrisso. In 2021, the FDA granted Fast Track Designation for REQORSA for NSCLC in combination therapy with Merck & Co’s Keytruda® (pembrolizumab) for patients whose disease progressed after treatment with Keytruda.

For more information, please visit the Company’s web site at 
www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

 

Cautionary Language Concerning
Forward-Looking Statements 

Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made on the basis of the current beliefs, expectations and assumptions of management, are not guarantees of performance and are subject to significant risks and uncertainty. These forward-looking statements should, therefore, be considered in light of various important factors, including those set forth in Genprex’s reports that it files from time to time with the Securities and Exchange Commission and which you should review, including those statements under “Item 1A – Risk Factors” in Genprex’s Annual Report on Form 10-K for the year ended December 31, 2021.

Because forward-looking statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: the timing and success of Genprex’s clinical trials and regulatory approvals, including the extent and impact of the COVID-19 pandemic; the effect of Genprex’s product candidates, alone and in combination with other therapies, on cancer and diabetes; Genprex’s future growth and financial status; Genprex’s commercial and strategic partnerships, including those with its third party manufacturers and their ability to successfully perform and scale up the manufacture of its product candidates; and Genprex’s intellectual property and licenses. 

These forward-looking statements should not be relied upon as predictions of future events and Genprex cannot assure you that the events or circumstances discussed or reflected in these statements will be achieved or will occur. If such forward-looking statements prove to be inaccurate, the inaccuracy may be material. You should not regard these statements as a representation or warranty by Genprex or any other person that Genprex will achieve its objectives and plans in any specified timeframe, or at all. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Genprex disclaims any obligation to publicly update or release any revisions to these forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this press release or to reflect the occurrence of unanticipated events, except as required by law.

Genprex,
Inc.

(877) 774-GNPX (4679)

GNPX
Investor Relations

investors@genprex.com

GNPX
Media Contact

Kalyn Dabbs

media@genprex.com

Wednesday, May 04, 2022

Lineage Cell Therapeutics (LCTX)
Additional Data Presented Confirms Our OpRegen Expectations

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelial cell therapy in Phase 1/2a development for the treatment of geographic atrophy secondary to age-related macular degeneration; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy, and (v) PNC1, a photoreceptor neural cell therapy for the treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit www.lineagecell.com or follow the company on Twitter @LineageCell.

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.

OpRegen Data Presented At ARVO.  Lineage Cell Therapeutics presented data from the Phase 1/2a trial testing OpRegen, its retinal pigment epithelial cell transplantation for treating age-related macular degeneration (dry AMD).  The data was presented at the Annual Meeting of the Association for Research in Vison and Ophthalmology, ARVO.  The presentation added additional patient data from its last cohort, demonstrating safety, tolerability, and improvements in visual function and cellular structure.

Additional Data From The Efficacy Cohort Was Presented.   The Phase 1/2a trial is an open-label trial with three cohorts of 4 patients each receiving ascending doses to establish safety, then a fourth cohort of 12 patients to test efficacy.  Patients in the updated presentation have been assessed at 12 months following OpRegen treatment, with some patients followed for up to 5 years….

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. 

Onconova Therapeutics To Provide Corporate Update And Announce First Quarter Financial Results On May 11, 2022

News and Market Data on Onconova Therapeutics

Company to host conference call and webcast at 4:30 p.m. Eastern Time on Wednesday, May 11, 2022

NEWTOWN, Pa., May 04, 2022 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that the Company intends to release its first quarter 2022 financial results on Wednesday, May 11, 2022. Management plans to host a conference call and live webcast at 4:30 p.m. ET on the same day to discuss these results and provide an update on its pipeline programs.

Conference Call and Webcast Information

Interested parties who wish to participate in the conference call may do so by dialing (855) 428-5741 for domestic and (210) 229-8823 for international callers and using conference ID 7369861.

Those interested in listening to the conference call via the internet may do so by visiting the investors and media page on the company’s website at www.onconova.com and clicking on the webcast link. In addition to the live webcast, a replay will be available on the Onconova website for 90 days following the call.

About Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose-escalation and expansion studies. These trials are currently underway in the United States and China.

Onconova’s product candidate rigosertib is being studied in an investigator-sponsored study program, including in a dose-escalation and expansion Phase 1/2a investigator-sponsored study with oral rigosertib in combination with nivolumab for patients with KRAS+ non-small cell lung cancer.

For more information, please visit www.onconova.com.

Company
Contact:
Avi Oler
Onconova Therapeutics, Inc.
267-759-3680

ir@onconova.us
https://www.onconova.com/contact/

Investor
Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200

bmackle@lifesciadvisors.com

 

 

RG6501 (OpRegen®) Phase 1/2a Clinical Results Support the Potential for OpRegen to Slow, Stop or Reverse Disease Progression in Geographic Atrophy Secondary to Age-Related Macular Degeneration

Research, News, and Market Data on Lineage Cell Therapeutics

• 12-Month Primary
  Endpoint Data Suggest OpRegen is Well Tolerated with an Acceptable Safety
  Profile
• Preliminary
  Evidence of Visual Function and Outer Retinal Structure Improvements
  Observed in Cohort 4 Patients with GA and Impaired Vision
• Data Reported at
  2022 ARVO Meeting by Allen C. Ho, M.D., FACS

CARLSBAD, Calif.–(BUSINESS WIRE)–Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced that results from the primary endpoint, safety and tolerability 1 year post-transplant, in the ongoing Phase 1/2a clinical study of RG6501 (OpRegen), a retinal pigment epithelial cell therapy currently in development for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD), were presented yesterday at the 2022 Association for Research in Vision and Ophthalmology
Annual Meeting (ARVO 2022). The presentation, “Safety and Efficacy of a Phase 1/2a Clinical Trial of Transplanted
Allogeneic Retinal Pigmented Epithelium (RPE, OpRegen) Cells in Advanced Dry
Age-Related Macular Degeneration (AMD)” was featured as part of the Retinal Prostheses and Transplantation Session, by Allen C. Ho, M.D., FACS, Wills Eye Hospital Attending Surgeon and Director of Retina Research, Professor of Ophthalmology, Thomas Jefferson University, Mid Atlantic Retina and President, The Retina Society (abstract number 3714956). RG6501 (OpRegen) is currently being developed under an exclusive worldwide collaboration between Lineage, Roche and Genentech, a member of the Roche Group.

“These data, though uncontrolled, offer the promising demonstration that OpRegen may be able to impact GA disease progression in a clinically meaningful manner, particularly when delivered on-target and in earlier disease, in patients afflicted with what was previously thought to be an inevitably progressive disease,” stated Dr. Ho. “Obviously, larger, controlled studies are required, but spontaneous disease regression does not occur in GA so these results suggest that OpRegen may be a potentially transformational therapy and strongly support further development.”

2022 ARVO Presentation
Highlights

Summary of Safety Results

• All 24 treated patients reported at least one adverse event (AE) and at least one ocular AE
• The majority of AEs reported with OpRegen were mild (Cohort 1-3, 87%; Cohort 4, 93%), and the immunosuppressive regimen was well tolerated
• Ocular AEs observed with OpRegen were mainly related to the surgical procedures used for subretinal delivery, with the most common being conjunctival hemorrhage/hyperemia (n=17) and epiretinal membrane (n=16)
• One patient discontinued the study due to an AE that was unrelated to treatment
• No cases of rejection, acute or delayed intraocular inflammation, or sustained increases in intraocular pressure following OpRegen subretinal delivery have been reported

Summary of Activity Results

• Preliminary evidence of improvement in visual function was observed in patients with GA and impaired vision at baseline (Cohort 4 [n=12])
• Patients in Cohort 4 had an average 7.6 letter gain in visual acuity at 12 months in the study eye
• Three patients in Cohort 4 (25%) had a 15 letter or greater gain in visual acuity at 12 months in the study eye

• Five patients in Cohort 4 with OpRegen delivered to most or all of the GA area, including the fovea, had greater gains in visual function (average 12.8 letter gain), with evidence for regions of apparent improvement of outer retinal structure as assessed by SD-OCT
• The SD-OCT imaging analysis is ongoing

These data support the potential for OpRegen to slow, stop or reverse disease progression in GA. Further assessment of the optimal disease stage for intervention, surgical procedure for subretinal delivery and target delivery location of OpRegen in a larger, controlled clinical study is needed to confirm these findings.

Dr. Ho’s presentation is available on the Events and Presentations section of Lineage’s website.

The Association for Research in Vision and Ophthalmology, Inc. (ARVO) was founded in 1928 in Washington, DC by a group of 73 ophthalmologists. ARVO is the largest and most respected eye and vision research organization in the world. ARVO members include nearly 11,000 researchers from over 75 countries. ARVO advances research worldwide into understanding the visual system and preventing, treating and curing its disorders. For more information, please visit https://www.arvo.org/ or follow the association on Twitter @ARVOInfo.

About OpRegen

OpRegen^® is a retinal pigment epithelial cell therapy in development for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration. Following subretinal delivery, OpRegen has the potential to counteract RPE cell loss in areas of GA lesions by supporting retinal structure and function. OpRegen is being developed under a worldwide collaboration between Lineage, Roche and Genentech, a member of the Roche Group.

About the Phase 1/2a Study

The Phase 1/2a study is an open-label, single-arm, multi-center, dose-escalation trial evaluating a single administration of OpRegen delivered subretinally in patients with bilateral GA. Twenty-four patients were enrolled into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with a best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 patients with impaired vision (BCVA from 20/65 to 20/250 with smaller mean areas of GA). Cohort 4 also included patients treated with a new “thaw-and-inject” formulation of OpRegen, which can be shipped directly to sites and used immediately upon thawing, removing the complications and logistics of having to use a dose preparation facility. The primary objective of the study was to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment-emergent adverse events. Secondary objectives are to evaluate the preliminary activity of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance.

About Geographic Atrophy

Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) characterized by severe loss of visual function. GA is a leading cause of adult blindness in the developed world, affecting at least 5 million people globally. There are two forms of advanced AMD: neovascular AMD and GA. GA and neovascular AMD can occur simultaneously in the same eye, and patients treated for neovascular AMD may still go on to develop GA. GA typically affects both eyes. There are currently no U.S. Food and Drug Administration (FDA) or European Medicines Agency (EMA) approved treatment options available for patients with GA.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelial cell therapy in Phase 1/2a development for the treatment of geographic atrophy secondary to age-related macular degeneration; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy, and (v) PNC1, a photoreceptor neural cell therapy for the treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit www.lineagecell.com or follow the company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “aim,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “can,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” “project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the collaboration and license agreement with Roche and Genentech and activities expected to occur thereunder; and the potential benefits of treatment with OpRegen and that OpRegen may be a potential transformational therapy. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, but not limited to, the risk that positive findings in early clinical and/or nonclinical studies of a product candidate may not be predictive of success in subsequent clinical and/or nonclinical studies of that candidate; the risk that competing alternative therapies may adversely impact the commercial potential of OpRegen; the risk that Roche and Genentech may not be successful in completing further clinical trials for OpRegen and/or obtaining regulatory approval for OpRegen in any particular jurisdiction; the risk that Lineage may not be able to manufacture sufficient clinical quantities of its product candidates in accordance with current good manufacturing practice; risks and uncertainties inherent in Lineage’s business and other risks discussed in Lineage’s filings with the Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the SEC, including Lineage’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC and its other reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Contacts

Lineage Cell Therapeutics, Inc. IR
Ioana C. Hone
(
ir@lineagecell.com)
(442) 287-8963

Solebury Trout IR
Mike Biega
(
Mbiega@soleburytrout.com)
(617) 221-9660

Russo Partners – Media Relations
Nic Johnson or David Schull
Nic.johnson@russopartnersllc.com
David.schull@russopartnersllc.com
(212) 845-4242

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