Category: Health

MustGrow Biologics Corp. (MGROF) – A Progress Update

Posted on by cpatches


Friday, September 16, 2022

Joe Gomes, Senior Research Analyst, Noble Capital Markets, Inc.

Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.

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

A Look-in on Programs. Yesterday, MustGrow announced the update on the Company’s biological programs with various partners Sumitomo, Bayer, Janssen PMP, and NexusBioAg. The update includes the soil biofumigation, postharvest food preservation, and bioherbicide of the Company’s mustard-derived technology.

Soil Biofumigation. Data regarding efficacy continue to remain positive, as the Company recently extended the program with Sumitomo that we highlighted in August. Sumitomo is working with MustGrow and the EPA in the registration approval process in the U.S. along with registration in Mexico. In Chile, the country approved an Experimental Use permit and registration work has commenced. Bayer has shown positive results in laboratory and greenhouse studies, and has new trials underway and further studies planned for Europe, Asia, and Africa.


Get the Full Report

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. 

With New Health Research Mandate Billions To Be Made Available

Posted on by cpatches
Image: Public Domain (Pexels)

ARPA-H: High-Risk, High-Reward Health Research is the Mandate of New, Billion-Dollar US Agency

A new multibillion-dollar federal agency was created with a goal of supporting “the next generation of moonshots for health” in science, logistics, diversity and equality. And the agency now has it’s first leader, as President Joe Biden announced Renee Wegrzyn as director of the Advanced Research Projects Agency for Health, or ARPA-H, on Sept. 12, 2022.

Since the announcement of the intention to establish ARPA-H two years ago, this new agency has sparked interest and questions within both academia and industry.

This article was republished  with permission from The Conversation, a news site dedicated to sharing ideas from academic experts. It represents the research-based findings and thoughts of Tong Sun, Assistant Dean of Translational Health Sciences, University of Washington.

I have been a director of innovation-driven health institutes for decades and have worked with many of the government agencies that fund science. I and many of my colleagues hope ARPA-H will become an agency that can quickly turn scientific discoveries into real-world advances to detect, prevent and treat diseases like cancer, diabetes and Alzheimer’s. But questions still remain surrounding how it will work and what makes it different from other government-funded agencies such as the National Institutes of Health and the National Science Foundation.

What is ARPA-H?

ARPA-H is the newest entity established within the National Institutes of Health. ARPA-H was explicitly set up as an independent agency within NIH, in theory allowing it to benefit from the NIH’s vast scientific and administrative expertise and resources while still being nimble and forward-thinking.

ARPA-H was inspired by and modeled after the Defense Advanced Research Projects Agency, or DARPA, to rapidly develop cutting-edge technologies. DARPA is small compared to other federal research and development agencies, but has long been hugely successful. It played a critical role in spawning many technologies ranging from the internet to GPS, and even funded Moderna to develop mRNA vaccine technology in 2013.

ARPA-H is not the only DARPA spinoff. In 2006, the federal government created the Intelligence Advanced Research Projects Activity to tackle difficult challenges in the intelligence community, and in 2009, the Advanced Research Projects Agency for Energy was launched. Though its budget is small compared to the Department of Energy, ARPA-E has been incredibly effective in funding ambitious research into fighting climate change. By funding ambitious mid- and long-term projects, IARPA, ARPA-E and now ARPA-H are meant to operate in between slow, government-funded basic research and short-term, profit-driven private sector venture capital.

ARPA-H is modeled after the Defense Advanced Research Projects Agency, which played a key role in developing many modern technologies, including personal computers. Tim Colegrove (Wikimedia Commons)

How Will the Agency Function?

Biden wants ARPA-H to replicate the success of DARPA, but in the health care realm, by providing “leadership for high-risk, high-reward biomedical and health research to speed application and implementation of health breakthroughs equitably.”

Established federal agencies like the NIH and the National Science Foundation prefer to fund more basic research and less risky projects compared to the high-risk, high-reward, applied science approach of DARPA. If ARPA-H wants to achieve the success of its predecessor, it will need to operate differently from NIH and NSF.

DARPA employs about 100 program managers who are “borrowed” from academia or industry for three to five years. These managers travel across the nation to meet with scientists and experts in different fields in order to generate ideas and start projects. These managers make funding decisions and work closely with their funded teams to overcome problems, but will cut funding if teams cannot deliver promised milestones on time. Many DARPA projects don’t produce spectacular successes, yet they pushed the boundaries of science and technology.

Many years ago, I had the privilege of working with a DARPA program manager alongside numerous experts in various scientific and medical fields. After several months of meetings, the program manager came up with the idea to develop “fracture putty” – a puttylike material that could be applied to the shattered bones of a wounded soldier in the battlefield. The material would support weight, prevent infection, expedite healing and bone regeneration and eventually dissolve away. The program launched in 2008, and our team of chemists, nanomaterials experts, bioengineers, mathematical modelers and surgeons was one of the funded teams in this program.

President Joe Biden appointed Renee Wegrzyn, a former DARPA project manager and biotech industry expert, to lead ARPA-H. Image: Renee Wegrzyn (@rwegrzyn)

Who is the New Director?

Wegrzyn holds a Ph.D. in molecular biology and bioengineering from Georgia Tech. She is currently a vice president of business development at Ginkgo Bioworks, a U.S. biotech company. Wegrzyn spent four and half years as a program manager in DARPA’s Biological Technologies Office, where she managed projects that focused on using genetic engineering and gene editing for biosecurity and public health. She also worked for another DARPA-inspired agency, Intelligence Advanced Research Projects Activity.

At this moment, we don’t know yet the exact plan and progress in hiring APPA-H program managers and where APAR-H’s headquarters will be located. Several cities have expressed interest.

What Should People Look for as ARPA-H Gets Started?

DARPA is driven by talented, ambitious and risk-taking program managers. They are the ones who generate ideas and turn lofty goals into executable projects. It will be interesting to see how many and what kind of program managers ARPA-H hires in its early days, as these decisions will give an indication of which areas within health care the agency will be prioritizing.

I’ll also be watching to see how well ARPA-H and its program managers work within the NIH, which has an unbelievable depth of resources and expertise in all health care related fields that ARPA-H can tap into. But the NIH has very different funding mechanisms and culture from DARPA.

The final question is money. Biden wants US$6.5 billion in funding for ARPA-H, and he’s only gotten $1 billion from Congress so far. This is its first, biggest challenge. Finding political unity for funding may have to be this new agency’s first big breakthrough if it is to reach its goals.

Release – Tonix Pharmaceuticals Announces Data Presentations Involving TNX-1500 (Fc-Modified Anti-CD40L mAb) for the Prevention of Rejection in Allograft and Xenograft Transplantation in Animal Models at the International Congress of The Transplantation Society (TTS 2022)

Posted on by [email protected]

Research, News, and Market Dat on TNXP

September 15, 2022 7:00am EDT

TNX-1500 Treatment Prevents Organ Rejection and Preserves Function for Both Allograft and Xenograft Transplants in Animal Studies

CHATHAM, N.J., Sept. 15, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced data from three oral presentations by faculty at the Center for Transplantation Sciences, Massachusetts General Hospital Center at the 29th International Congress of The Transplantation Society (TTS 2022) held September 10-14, 2022 in Buenos Aires, Argentina. The data involve studies of Tonix’s TNX-1500 (Fc modified anti-CD40L monoclonal antibody) product candidate in development for the prevention of organ transplant rejection. The molecular target of TNX-1500 is CD40-ligand (CD40L), which is also known as CD154. Copies of the presentations are available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com.

The presentations titled, “Long-term rejection-free renal allograft survival with Fc-modified anti-CD154 antibody monotherapy in nonhuman primates,” and “Monotherapy with TNX-1500, a Fc-modified anti-CD154mAb, prolongs cardiac allograft survival in cynomolgus monkeys,” include data demonstrating that TNX-1500 treatment showed activity in preventing organ rejection and was well tolerated in non-human primates. Blockade of CD40L with TNX-1500 monotherapy consistently and safely prevented pathologic alloimmunity in non-human primate cardiac and kidney allograft models without clinical thrombosis.

The presentation titled, “Long-term (>1 year) rejection-free survival of kidney xenografts with triple xenoantigen knockout and multiple human transgenes in nonhuman primates,” includes data demonstrating that TNX-1500 treatment showed activity in preventing xenograft kidney rejection and was well tolerated in non-human primates. Xenografts are transplanted organs from donors of a different species from the recipient, and in this study, the xenografts originated from genetically engineered pigs. Blockade of CD40L with TNX-1500 monotherapy consistently and safely prevented pathologic xenoimmunity in non-human primate kidney xenograft models without clinical thrombosis.

“There remains a significant need for new treatments with improved activity and tolerability to prevent organ transplant rejection,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “To date, there has not been a humanized anti-CD40L antibody that can effectively prevent transplant rejections with an acceptable level of tolerability. TNX-1500 is a third generation anti-CD40L mAb that has been designed by protein engineering to decrease FcγRII binding and to reduce the potential for thrombosis. The animal studies found that TNX-1500 retains activity to prevent rejection and preserve graft function. We believe TNX-1500 has the potential for treating and preventing organ transplant rejection in both allograft and xenograft transplants. Tonix expects to initiate a Phase 1 trial of TNX-1500 in the first half of 2023. Preventing transplant rejection is the first indication we are pursuing, but based on results of anti-CD40L in numerous animal models, we believe that TNX-1500 has the potential for treating a number of autoimmune conditions.”

Tonix Pharmaceuticals Holding Corp.*

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’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 (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022 and interim data expected in the second quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022 and expects interim data in the first half of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the first quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is expected to enter the clinic with a Phase 2 study in the fourth quarter of 2022. Tonix’s rare disease portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s infectious disease pipeline consists of a vaccine in development to prevent smallpox and monkeypox, next-generation vaccines to prevent COVID-19, and a platform to make fully human monoclonal antibodies to treat COVID-19. TNX-801, Tonix’s vaccine in development to prevent smallpox and monkeypox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in Kenya in the first half of 2023. Tonix’s lead vaccine candidate for COVID-19 is TNX-1850, a live virus vaccines based on Tonix’s recombinant pox live virus vector vaccine platform. A Phase 1 study of the COVID-19 vaccine is expected to be initiated in the second half of 2023.

*All of Tonix’s product candidates are investigational new drugs or biologics and have not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
[email protected]
(862) 904-8182

Olipriya Das, Ph.D. (media)
Russo Partners
[email protected]
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
[email protected]
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.

Released September 15, 2022

Release – MustGrow and Global Partners Drive Biological Programs Forward

Posted on by [email protected]

Research, News, and Market Data on MGROF

  • Soil biofumigation platform continues to show promising results across multiple continents.
  • Postharvest activities in potatoes, fruits and vegetables have demonstrated initial efficacy and work is now focused on application, timing, dose rates and residues.
  • Bioherbicide optimization and formulation work advances.

Saskatoon, Saskatchewan–(Newsfile Corp. – September 15, 2022) – MustGrow Biologics Corp. (CSE: MGRO) (OTCQB: MGROF) (FSE: 0C0) (the “Company” or “MustGrow“), and its global agriculture partners continue to drive MustGrow’s organic mustard-derived crop protection and food preservation technology forward through field trials, development work and regulatory approval strategies in key global agricultural regions. Publicly-disclosed programs include:

  • Sumitomo Corporation

Exclusive evaluation agreement in the Americas for preplant soil fumigation, bioherbicide, postharvest and food preservation for potatoes and bananas. Sumitomo Corporation is funding and driving all field development, including toxicology, safety, efficacy, and regulatory work necessary for commercialization. (Press release link)

  • Bayer

Exclusive evaluation agreement in Europe, Asia Pacific, Middle East and Africa for preplant soil fumigation, bioherbicide applications and postharvest food preservation of potatoes including sprout inhibition. The first year of the evaluation is focused on the pre-plant soil fumigant application. Bayer is funding and driving all laboratory, field, regulatory, and market assessments in the target regions. (Press release link)

  • Janssen PMP, a division of Johnson & Johnson-owned Janssen Pharmaceutica NV

Global exclusive evaluation agreement for postharvest storage preservation of fruits and vegetables, excluding grains, potatoes, bananas and shipping container fumigation. Janssen PMP is funding and driving all application testing and development work. (Press release link)

  • NexusBioAg, a division of Univar Solutions

Exclusive marketing and distribution agreement in Canada for canola (clubroot disease) and pulse crops (aphanomyces disease). NexusBioAg is funding and driving all field development and regulatory work necessary for commercialization. (Press release link)

———

GLOBAL PROGRAM ADVANCEMENTS:

Soil Biofumigation
Sumitomo Corporation has continued to generate data across multiple crops, evaluating application timing, methods, and dose rates. Efficacy data generated across multiple countries and programs remains positive and has already resulted in new country expansions and program extension. MustGrow and Sumitomo Corporation are continually assessing market opportunities for further regional and application expansions. Utilizing MustGrow’s platform technology, Sumitomo Corporation currently has field trials and regulatory development work ongoing in the US, Mexico, and Chile.

In the US, EPA product registration work continues to advance, with Sumitomo Corporation playing a pivotal role in the registration approval process. In addition, Mexico registration work is progressing forward, as there have now been two consecutive years of successful field trials with Sumitomo Corporation. Mexico is a key global agriculture region both in size and strategic location. In Chile, registration work has commenced with the country approving an Experimental Use Permit to Sumitomo Corporation and MustGrow for field testing and registration activities.

Bayer’s initial laboratory and greenhouse studies utilizing MustGrow’s mustard-derived technology have been promising. Building on these positive results, Bayer and MustGrow have new trials underway and further studies planned across Europe, Asia and Africa. Bayer is evaluating registration pathways for multiple regions.

Building on their 2021 field trial program for treatment of canola (clubroot disease) and pulse crops (aphanomyces disease), NexusBioAg and MustGrow signed an Exclusive Marketing and Distribution Agreement in Canada. NexusBioAg intends to trial MustGrow’s technology in its 2023 BioAg Advantage program (“BAT“), which are wide-scale field trials conducted at the farmer level. NexusBioAg leverages its BAT program to introduce new and exciting potential ‘portfolio’ products to large Canadian farmers.

Postharvest Food Preservation
Harnessing MustGrow’s mustard-derived biological agent as a postharvest fruit and vegetable application, Janssen PMP has demonstrated initial success in decay, fungal, and bacterial disease efficacy. Advancing to the next stage, Janssen PMP’s testing program will focus on phytotoxicity, residues, application rates, application methods and dose timing. As food preservation is a critical global issue, reducing postharvest fruit and vegetable losses could substantially increase worldwide food availability.

MustGrow had previously tested its organic mustard-based technology and outperformed leading synthetic chemical standards for treatment of stored potatoes for both Fusarium dry rot disease and sprouting. No disease/sprouting combination solutions currently exist in the marketplace, making MustGrow’s application unique in addressing both issues simultaneously. Leading sprout inhibiter chlorpropham (CIPC) had been banned by the European Union as of Oct. 8, 2020. Sumitomo Corporation is advancing this program in the US with trials and data review ongoing.

Bioherbicide
MustGrow’s bioherbicide program is now focused on extraction optimization and formulation work. Perfomance and efficacy knowledge utilizing the mustard-derived Thiocyanate molecule, both internally and externally, has demonstrated potential impact as both a stand alone and combination herbicide in multiple markets. MustGrow is focused on production parameters, improving formulations for application flexibility, and the potential to combine MustGrow’s bioherbicide with leading chemicals.

———

About MustGrow
MustGrow is an agriculture biotech company developing organic biopesticides and bioherbicides by harnessing the natural defense mechanism of the mustard plant to protect the global food supply from diseases, insect pests, and weeds. MustGrow and its leading global partners — Janssen PMP (pharmaceutical division of Johnson & Johnson), Bayer, Sumitomo Corporation, and Univar Solutions’ NexusBioAg — are developing mustard-based organic solutions to potentially replace harmful synthetic chemicals. Over 100 independent tests have been completed, validating MustGrow’s safe and effective approach to crop and food protection. Pending regulatory approval, MustGrow’s patented liquid products could be applied through injection, standard drip or spray equipment, improving functionality and performance features. Now a platform technology, MustGrow and its global partners are pursuing applications in several different industries from preplant soil treatment and weed control, to postharvest disease control and food preservation. MustGrow has approximately 49.2 million basic common shares issued and outstanding and 55.6 million shares fully diluted. For further details, please visit www.mustgrow.ca.

ON BEHALF OF THE BOARD

“Corey Giasson”

Director & CEO
Phone: +1-306-668-2652
[email protected]

MustGrow Forward-Looking Statements

Certain statements included in this news release constitute “forward-looking statements” which involve known and unknown risks, uncertainties and other factors that may affect the results, performance or achievements of MustGrow.

Generally, forward-looking information can be identified by the use of forward-looking terminology such as “plans”, “expects”, “is expected”, “budget”, “estimates”, “intends”, “anticipates” or “does not anticipate”, or “believes”, or variations of such words and phrases or statements that certain actions, events or results “may”, “could”, “would”, “might”, “occur” or “be achieved”. Examples of forward-looking statements in this news release include, among others, statements MustGrow makes regarding: (i) potential product approvals; (ii) anticipated actions by partners to drive field development work including dose rates, application frequency, application methods, and the regulatory work necessary for commercialization; (iii) expected product efficacy of MustGrow’s mustard-based technologies; and (iv) expected outcomes from collaborations with commercial partners.

Forward-looking statements are subject to a number of risks and uncertainties that may cause the actual results of MustGrow to differ materially from those discussed in such forward-looking statements, and even if such actual results are realized or substantially realized, there can be no assurance that they will have the expected consequences to, or effects on, MustGrow. Important factors that could cause MustGrow’s actual results and financial condition to differ materially from those indicated in the forward-looking statements include, among others, the following: (i) the preferences and choices of agricultural regulators with respect to product approval timelines; (ii) the ability of MustGrow’s partners to meet obligations under their respective agreements; and (iii) other risks described in more detail in MustGrow’s Annual Information Form for the year ended December 31, 2021 and other continuous disclosure documents filed by MustGrow with the applicable securities regulatory authorities which are available at www.sedar.com. Readers are referred to such documents for more detailed information about MustGrow, which is subject to the qualifications, assumptions and notes set forth therein.

This release does not constitute an offer for sale of, nor a solicitation for offers to buy, any securities in the United States.

Neither the CSE nor its Regulation Services Provider (as that term is defined in the policies of the CSE) accepts responsibility for the adequacy or accuracy of this release.

© 2022 MustGrow Biologics Corp. All rights reserved.

Release – Schwazze Signs Definitive Documents To Acquire Certain Assets Of Lightshade Labs LLC

Posted on by [email protected]

Research, News, and Market Data on SHWZ

Continues to Go Deep, Adding to Retail Footprint in Colorado

DENVER, CO – September 14, 2022 – Schwazze, (OTCQX:SHWZ NEO:SHWZ) (“Schwazze” or the “Company”), announced that it has signed definitive documents to acquire certain assets of Lightshade Labs LLC (“Lightshade”). The proposed transaction includes the adult use Lightshade dispensaries located at 503 Havana St. in Aurora, as well as 2215 E. Mississippi Ave. in Denver’s vibrant Washington Park neighborhood. This acquisition continues Schwazze’s aggressive expansion in Colorado and upon close will bring the Company’s total number of Colorado dispensaries to 25.

The consideration for the proposed acquisition is US$2.75 million and will be paid as all cash. The acquisition is expected to close in the first quarter of 2023 after Colorado Marijuana Enforcement Division and local licensing approval.

“Schwazze is excited to add to our retail footprint in the greater Denver area, providing two additional retail locations to our existing 23 throughout Colorado. We look forward to extending our exceptional customer service and wide product selection to both new and existing customers in these new locations.” said Collin Lodge, Division President of Colorado.

Since April 2020, Schwazze has acquired or announced the planned acquisition of 35 cannabis dispensaries as well as seven cultivation facilities and two manufacturing assets in Colorado and New Mexico. In May 2021, Schwazze announced its BioSciences division and in August 2021 it commenced home delivery services in Colorado.

About Schwazze

Schwazze (OTCQX: SHWZ NEO: SHWZ) is building a premier vertically integrated regional cannabis company with assets in Colorado and New Mexico and will continue to take its operating system to other states where it can develop a differentiated regional leadership position. Schwazze is the parent company of a portfolio of leading cannabis businesses and brands spanning seed to sale. The Company is committed to unlocking the full potential of the cannabis plant to improve the human condition. Schwazze is anchored by a high- performance culture that combines customer-centric thinking and data science to test, measure, and drive decisions and outcomes. The Company’s leadership team has deep expertise in retailing, wholesaling, and building consumer brands at Fortune 500 companies as well as in the cannabis sector. Schwazze is passionate about making a difference in our communities, promoting diversity and inclusion, and doing our part to incorporate climate-conscious best practices. Medicine Man Technologies, Inc. was Schwazze’s former operating trade name. The corporate entity continues to be named Medicine Man Technologies, Inc. Schwazze derives its name from the pruning technique of a cannabis plant to enhance plant structure and promote healthy growth.

Forward-Looking Statements

This press release contains “forward-looking statements.” Such statements may be preceded by the words “plan,” “will,” “may,” “continue,” “predicts,” or similar words. Forward-looking statements are not guarantees of future events or performance, are based on certain assumptions, and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control and cannot be predicted or quantified. Consequently, actual events and results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) our inability to manufacture our products and product candidates on a commercial scale on our own or in collaboration with third parties; (ii) difficulties in obtaining financing on commercially reasonable terms; (iii) changes in the size and nature of our competition; (iv) loss of one or more key executives or scientists; (v) difficulties in securing regulatory approval to market our products and product candidates; (vi) our ability to successfully execute our growth strategy in Colorado and outside the state, (vii) our ability to consummate the acquisition described in this press release or to identify and consummate future acquisitions that meet our criteria, (viii) our ability to successfully integrate acquired businesses, including the acquisition described in this press release, and realize synergies therefrom, (ix) the ongoing COVID-19 pandemic, (x) the timing and extent of governmental stimulus programs, and (xi) the uncertainty in the application of federal, state and local laws to our business, and any changes in such laws. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company’s filings with the Securities and Exchange Commission (SEC), including the Company’s Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC’s website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise except as required by law.
 

Investors
Joanne Jobin
Investor Relations
[email protected]
647 964 0292		Media
Julie Suntrup, Schwazze
Vice President | Marketing & Merchandising
[email protected]
303 371 0387

Release – Ocugen Announces Publication of a Comprehensive Review of BBV152 in Frontiers in Immunology

Posted on by [email protected]

Research, News, and Market Data on OCGN

September 14, 2022

  • Data include the persistence of immune responses and protection against variants of concern, especially Delta and Omicron
  • Ocugen has North American commercialization rights for BBV152, commercialized as COVAXIN™

MALVERN, Pa., Sept. 14, 2022 (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 the publication “A comprehensive review of BBV152 vaccine development, effectiveness, safety, challenges, and prospects” appeared in Frontiers in Immunology. BBV152, commercialized as COVAXIN™, is developed and manufactured by Ocugen’s partner Bharat Biotech, a global leader in vaccine innovation based in Hyderabad, India. BBV152 is currently authorized by the World Health Organization, authorized under Emergency Use Authorization in 28 countries, and accepted as a COVID-19 vaccine to travel into over 85 countries. It is under clinical investigation by Ocugen in the United States for use in adults aged 18 years and older.

This review by Dotiwala and Upadhyay provides a detailed analysis of the immunogenicity, safety, and efficacy of BBV152—a whole virus inactivated vaccine and an important tool in the fight to control the COVID-19 pandemic. Additionally, BBV152 has a broader impact on public health, as it induces high neutralization efficacy against different SARS-CoV-2 variants of concern.

“Unfortunately, the COVID-19 pandemic is not yet over, despite the introduction of effective vaccines and a greater understanding of COVID-19 pathogenesis and transmission dynamics,” said David Fajgenbaum, MD, MBA, MSc, Assistant Professor of Medicine, Translational Medicine & Human Genetics, University of Pennsylvania, and Ocugen Vaccine Scientific Advisory Board member. “This study demonstrates durability through immune memory and a broader immune response with BBV152 and provides further evidence that additional vaccine options—including those built on a traditional vaccine platform—are needed.”

Findings include:

  • 77.8% and 93.4% protection from symptomatic COVID-19 disease and severe symptomatic COVID-19 disease respectively.
  • Studies in pediatric populations show superior immunogenicity (geometric mean titer ratio of 1.76 compared to an adult) with a seroconversion rate of >95%.
  • Reactogenicity and safety profiles were comparable across all pediatric age groups between 2-18 yrs.

The study concludes that BBV152 is a suitable alternative to mRNA vaccines.

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

Onconova Therapeutics (ONTX) – Rigosertib Phase 1/2a Trial Data Reported At ESMO

Posted on by cpatches


Tuesday, September 13, 2022

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 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.

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.

Data Shows Efficacy After Treatment Failures.  Onconova presented positive interim data from its Phase 1/2a trial testing rigosertib at the European Society for Medical Oncology (ESMO).  The Investigator-sponsored trial tested the combination of rigosertib with nivolumab, an anti-PD1 checkpoint inhibitor, in non-small cell lung cancer patients that had progressive non-small cell lung cancer (NSCLC) following treatment with checkpoint inhibitor therapies.

Patients Showed Both Complete and Partial Responses.  The interim data reported was for 14 out of 19 patients enrolled in the study.  Four of the 14 (29%) showed disease control responses, including 1 complete response, 2 partial responses, and 1 stable disease.  Mean survival for these responders was 6.75 months, compared with 1-2 months for the non-responders.  Each responding patient had a different mutation in the KRAS gene, showing activity against multiple variants.


Get the Full Report

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. 

Ayala Pharmaceuticals (AYLA) – Phase 2/3 RINGSIDE Data Presented, Dose Selected For Next Stage

Posted on by cpatches


Tuesday, September 13, 2022

Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations. Ayala’s approach is focused on predicating, identifying and addressing tumorigenic drivers of cancer through a combination of its bioinformatics platform and next-generation sequencing to deliver targeted therapies to underserved patient populations. The company has two product candidates under development, AL101 and AL102, targeting the aberrant activation of the Notch pathway with gamma secretase inhibitors to treat a variety of tumors including Adenoid Cystic Carcinoma, Triple Negative Breast Cancer (TNBC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM) (in collaboration with Novartis). AL101, has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and is currently in a Phase 2 clinical trial for patients with ACC (ACCURACY) bearing Notch activating mutations. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE) and is being evaluated in a Phase 1 clinical trial in combination with Novartis’ BMCA targeting agent, WVT078, in Patients with relapsed/refractory Multiple Myeloma. For more information, visit www.ayalapharma.com.

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.

First AL012 RINGSIDE Data Presented At ESMO.  Ayala presented first data the from the Phase 2/3 RINGSIDE trial testing AL102 in desmoid tumors, a rare tumor of the connective tissue.  The data was from Part A, designed to test three dosing intervals for tolerability, safety, and select a dosing schedule for the double-blind, placebo-controlled Part B.  Patients receiving drug at intervals of either 1.2 mg daily, 2 mg for 2 days then 5 days rest, or 4 mg for 2 days then 5 days rest.  The 1.2mg daily dose was chosen for Part B.

The Stock Fell On The News.  We attribute the stock price decline to the selection of the lower dose given daily rather than one of the higher doses given in cycles.  The data from the daily dosing arm also had partial responses, rather than complete responses, although the effect improved over time.  We point out that Part A was designed to determine tolerability and select the dose for Part B, and the upcoming Part B with 156 patients will determine efficacy and approvability.


Get the Full Report

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. 

Why Biotech May Finish out 2022 Very Strong

Posted on by cpatches
Image Credit: Jason Mrachina (Flickr)

The Reasons Biotech is Gaining Ground on the Field

Similar to their watching a horse race, with a sense that their horse is starting to come from behind and may even be moving toward the front of the pack, biotech investors are leaning over the rail, watching their sector’s increased pace. This week biotechs, as measured by the ETF $XBI, crossed above its 200-day moving average – only last week the biotech sector’s momentum took it above its 50-day moving average. Does this technical indicator demonstrate the growing strength will continue, or does this indicate that it may be approaching overbought?

Technical analysis is not usually clear on this; below, we look for clues in the sector’s fundamentals to better handicap its chances.

Source: Koyfin

Pace of Deals

In 2021, the overall healthcare sector experienced record merger and acquisition activity. The first half of 2022 also had significant activity; however, at $92.4 billion in value and 481 deals announced, the pace of activity was down 51% from the same period in 2021. This may feel slow but is well ahead of the pre-pandemic pace for these companies.  For example, it’s a 37% increase from the first half of 2020. This could be seen as fundamentally positive for a sector that is trading below its 2021 levels and even below the second half of 2020. 

One catalyst for this continued high pace of deals which may even help accelerate it, is that big pharmacies are flush with cash. This cash serves them best if invested in the next generation of medicine or valuable patent. Fortunately for big pharma, small and mid-sized biotech companies are more likely now to form financial partnerships, agree to merger arrangements, or be outright purchased in order to help with their need for cash to continue operations.

This dynamic is easy to understand; there is less money flowing into the smaller incubator-type companies than the big pharmaceutical companies that have had money pouring in from generous pandemic-related government contracts. These small companies, many working on what may be life-changing science, rarely have sizeable sales. Sales and revenue come after the final phase of testing, FDA approval, and marketing. A small biotech company that sees its research and development possibly making a difference a few years from now, but is currently burning through capital at a pace where it may only last another 12 months, might welcome partnership or acquisition talks with a cash-rich suitor.

News of any injection of cash or capital in these smaller biotechs is usually an event that pushes the price up by percentage points in a short period of time. A full buyout can do much more.

XBI provides exposure to US biotech stocks, as defined by GICS, from a universe that invests across the market-cap spectrum. The fund equal-weights its portfolio, which in turn emphasizes small- and micro-caps and greatly reduces single-name risk. Thus, the weighted-average market-cap is much smaller than some competitors. Unlike other funds in this segment, XBI is a pure biotech play, with relatively small pharma overlap. The index is rebalanced quarterly.  – FactSet

Put yourself in the position of big pharma in 2022 into 2023. Your firm may now be sitting on a huge war chest thanks to the pandemic. This is now being eroded by high inflation. Management’s role is to use resources to provide value to shareholders. In the meantime, biotech is well-priced and motivated to talk.

And then the clock is always ticking on patent cliffs for big pharma. They may be very amenable to shop for acquisition targets as they look out at the expiration of patent rights and the exclusive protection those patents provide. Analysts estimate the top-ten pharmaceutical manufacturers have more than 46 percent of their revenues at risk between 2022 and 2030. Behemoths like Bristol Myers Squibb, Pfizer and Merck will be among the most exposed over the next decade.

Take Away

Watching a thoroughbred that was lagging behind the pack find an opening and begin to gain ground on those around it is exciting. When you have money on the horse, it’s even more enjoyable. The performance of the biotech sector was far behind other industries for much of this year. Moving into fall it has been outperforming its own average and many other investment areas.

There are fundamentals in play that could keep this strength going. What’s more is that these factors have little to do with the overall market, which many now fear

This challenge may catalyze M&A in the industry as large firms look to recoup lost revenue streams and invest in patents.

Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.nasdaq.com/articles/consolidation-case-for-biotech-etf-bbh

https://www.jdsupra.com/legalnews/big-pharma-firms-return-to-the-deal-6580128/

Release – Axcella Therapeutics to Participate in the H.C. Wainwright Global Investment Conference

Posted on by [email protected]

Research, News, and Market Data on AXLA

September 12, 2022 at 8:00 AM EDT

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sep. 12, 2022– Axcella Therapeutics (Nasdaq: AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, today announced that management will participate in a fireside chat and one-on-one meetings at the H.C. Wainwright Global Investment Conference taking place September 12 – 14, 2022 both virtually and in-person in New York City.

Details for the fireside chat are as follows:

Date:Tuesday, September 13, 2022
Time:10:30 am Eastern Time
Webcast:https://journey.ct.events/view/6604fa89-69be-476a-b518-b615e1ac7fb6

The conference call webcast will be accessible in the Investors & News section on the company’s website at www.axcellatx.com. An archive of the webcast replay will be available on the Company’s website for up to 90 days.

To request a one-on-one in-person or virtual meeting, please register here.

Internet Posting of Information

Axcella uses the “Investors and News” section of its website, www.axcellatx.com, as a means of disclosing material nonpublic information, to communicate with investors and the public, and for complying with its disclosure obligations under Regulation FD. Such disclosures include, but may not be limited to, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, and public conference calls and webcasts. The information that we post on our website could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.

About Axcella Therapeutics (Nasdaq: AXLA)

Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using compositions of endogenous metabolic modulators (EMMs). The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to reset multiple biological pathways, improve cellular energetics, and restore homeostasis. Axcella’s pipeline includes lead therapeutic candidates in Phase 2 development for the treatment of Long COVID and NASH. The company’s unique model allows for the evaluation of its EMM compositions through non-IND clinical studies or IND clinical trials. For more information, please visit www.axcellatx.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220912005400/en/

Ashley Robinson
[email protected]
(617) 430-7577

Source: Axcella Therapeutics

Release – Ayala Pharmaceuticals Presents Positive Interim Data from RINGSIDE Pivotal Phase 2-3 Trial of AL102 in Desmoid Tumors at ESMO Congress 2022

Posted on by [email protected]

Research, News, and Market Data on AYLA

September 12, 2022

– Updated interim results from Part A of RINGSIDE show first confirmed partial response (PR) achieved at week 16 and 3 additional unconfirmed PRs over the follow-up period –

– Consistent early tumor shrinkage with measures deepening over time –

– AL102 was well tolerated at all doses evaluated –

– Part B and Open-Label Extension being initiated with the selected dose of 1.2mg once daily –

– Company to host Key Opinion Leader event on September 28 to discuss results –

REHOVOT, Israel and WILMINGTON, Del., Sept. 12, 2022 (GLOBE NEWSWIRE) — Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers today announces updated, positive interim results from Part A of the ongoing RINGSIDE Pivotal Phase 2/3 clinical trial evaluating investigational new drug AL102 in desmoid tumors. AL102 is a potent, selective, oral gamma-secretase inhibitor.

The data are being featured in an oral presentation today at the European Society for Medical Oncology (ESMO) Congress 2022. The presentation entitled, “Initial Results of Phase 2/3 Trial of AL102 for Treatment of Desmoid Tumors” is being delivered by Prof. Robin Jones, M.D., Head of the Sarcoma Unit at The Royal Marsden, London, UK.

“The results presented at ESMO from the RINGSIDE study are very encouraging. AL102 demonstrated an early and meaningful effect on tumors within a 16-week period and was well tolerated, which could allow for long term treatment of patients. AL102 has the potential to significantly improve the lives of patients suffering from desmoid tumors who currently have no approved therapy,” said Prof. Robin Jones.

Jeanne Whiting, Executive Director & Co-Founder of the Desmoid Tumor Research Foundation stated, “Desmoid tumors are rare, connective tissue tumors that can have aggressive infiltrative growth and high risk of local recurrence. Patients and physicians struggle with the fact that there are no FDA-approved therapies. We are very encouraged by the results presented by Ayala and the opportunity that AL102 holds in improving outcomes for patients with this rare condition.”

“We are excited to share strong interim results from the AL102 RINGSIDE study at this year’s ESMO Congress which continue to demonstrate early and meaningful anti-tumor activity as monotherapy in patients with desmoid tumors,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “Efficacy was demonstrated across all cohorts of Part A of the study, with early responses that deepened over time. The results also showed that AL102 was well-tolerated across all doses. We are advancing to Part B of RINGSIDE with a selected dose of 1.2mg once daily, as well as enrolling patients in the open label extension study. The results presented today give us increased confidence in the potential for AL102 to improve the lives of patients with desmoid tumors.”

Results as of the Cut-Off Date of July 14, 2022
Part A Interim Efficacy Results:

  • Patient enrollment in Part A of RINGSIDE was completed in February 2022. Patients were dosed in AL102 monotherapy cohorts of 1.2mg (once daily), 2mg (2 days on, 5 days off), or 4mg (2 days on, 5 days off).
  • The activity of AL102 is being evaluated by change in tumor volume (central MRI readings) and response (per RECIST 1.1) determined by blinded independent central review.   At data cut, 28 patients were evaluable for tumor volume and 29 were evaluable for RECIST with a scan at base line and at least one additional scan at week 16.
  • 12 subjects had follow up MRI scans at week 28 and one patient had a scan at week 40.
  • One patient had a partial response (PR) per RECIST at week 16, confirmed at week 28.
  • Three additional unconfirmed PRs were observed, two at week 28 and one at week 40.
  • Continuous tumor volume reduction was observed over time in all patients that underwent 2 or more MRI scans.

Part A Interim Efficacy Results of Selected Dose of 1.2mg daily:

  • At week 16 there were 9 evaluable patients for RECIST in the selected dose of 1.2mg once daily with one PR observed, confirmed at week 28. The remaining 8 patients had stable disease, of which 7 patients had a tumor reduction.
  • At week 28 there were three patients evaluable for RECIST in the selected dose of 1.2mg daily with one confirmed and one unconfirmed PR and one stable disease with all patients showing tumor reduction and deepening of tumor shrinkage since previous scan.
  • At the selected dose of 1.2mg once daily, at week 16 there were 9 evaluable patients for volume change with 7 patients experiencing tumor volume reduction. At week 28 there were three evaluable patients for volume change in the selected dose of 1.2mg once daily with all three patients experiencing continuous tumor shrinkage.

Part A Safety:

  • AL102 was generally well tolerated at all doses
  • Most adverse events were grade 1 or 2 and included mainly diarrhea
  • No grade 4 or 5 events were observed and low rates of grade 3 events.
  • At the selected dose (1.2 mg once daily) 3 out of the 14 patients (21.4%) had grade 3 events.
  • Ovarian dysfunction was observed in about 22% of women with childbearing potential (N=23)

About the RINGSIDE study
The RINGSIDE pivotal Phase 2/3 study is a randomized global multi-center trial. Part A of the study is evaluating the efficacy, safety, tolerability, and tumor volume by MRI after 16 weeks of AL102 in patients with desmoid tumors. It enrolled 42 patients and is evaluating 3 doses of AL102. Patients who participated in Part A are eligible to enroll into an open-label extension study at the Part B selected dose of 1.2 mg daily, and long-term efficacy and safety will be monitored.

Part B of the study is a double-blind, placebo-controlled segment enrolling up to 156 patients with progressive disease, comparing AL102 at 1.2 mg once daily to placebo. The primary endpoint for Part B will be progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR), tumor volume reduction, and patient-reported Quality of Life (QOL) measures. For more information on the RINGSIDE Phase 2/3 study with AL102 for the treatment of desmoid tumors, please visit ClinicalTrials.gov and reference Identifier NCT04871282 (RINGSIDE).

About Desmoid Tumors
Desmoid tumors also called aggressive fibromatosis or desmoid-type fibromatosis, are rare connective tissue tumors that typically arise in the upper and lower extremities, abdominal wall, head and neck area, mesenteric root, and chest wall with the potential to arise in additional parts of the body. Desmoid tumors do not metastasize, but often aggressively infiltrate neurovascular structures and vital organs. People living with desmoid tumors are often limited in their daily life due to chronic pain, functional deficits, general decrease in their quality of life and organ dysfunction. Desmoid tumors have an annual incidence of approximately 1,700 patients in the United States and typically occur in patients between the ages of 15 and 60 years. They are most commonly diagnosed in young adults between 30-40 years of age and are more prevalent in females. Today, surgery is no longer regarded as the cornerstone treatment of desmoid tumors due to a high rate of recurrence post-surgery and there are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.

About Ayala Pharmaceuticals
Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare tumors and aggressive cancers. Ayala’s approach is focused on predicating, identifying and addressing tumorigenic drivers of cancer through a combination of its bioinformatics platform and next-generation sequencing to deliver targeted therapies to underserved patient populations. The company has two product candidates under development, AL101 and AL102, targeting the aberrant activation of the Notch pathway with gamma secretase inhibitors to treat a variety of tumors including Adenoid Cystic Carcinoma (ACC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM). AL101, has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and is currently in a Phase 2 clinical trial for patients with ACC (ACCURACY) bearing Notch activating mutations. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE). For more information, visit www.ayalapharma.com. 

Contacts:

Investors:
Joyce Allaire
LifeSci Advisors LLC
+1-617-435-6602
[email protected]

Ayala Pharmaceuticals:
+1-857-444-0553
[email protected] 

Media:
Tricia Persad-Bevil
JPA
+44-7792-524442

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements relating to our development of AL102, the promise and potential impact of AL102, the timing and results of our clinical trials or readouts, the prevalence of desmoid tumors and the treatment required to manage the disease, and the design of our clinical trials. These forward-looking statements are based on management’s current expectations. The words ”may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “estimate,” “believe,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words.

These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have incurred significant losses since inception and anticipate that we will continue to incur losses for the foreseeable future; we are not currently profitable, and we may never achieve or sustain profitability; we will require additional capital to fund our operations, and if we fail to obtain necessary financing, we may not be able to complete the development and commercialization of AL101 and AL102; we have a limited operating history and no history of commercializing pharmaceutical products, which may make it difficult to evaluate the prospects for our future viability; we are heavily dependent on the success of AL101 and AL102, our most advanced product candidates, which are still under clinical development, and if either AL101 or AL102 does not receive regulatory approval or is not successfully commercialized, our business may be harmed; due to our limited resources and access to capital, we must prioritize development of certain programs and product candidates; these decisions may prove to be wrong and may adversely affect our business; the outbreak of COVID-19, may adversely affect our business, including our clinical trials; our ability to use our net operating loss carry forwards to offset future taxable income may be subject to certain limitations; our product candidates are designed for patients with genetically defined cancers, which is a rapidly evolving area of science, and the approach we are taking to discover and develop product candidates is novel and may never lead to marketable products; we were not involved in the early development of our lead product candidates, therefore, we are dependent on third parties having accurately generated, collected and interpreted data from certain preclinical studies and clinical trials for our product candidates; enrollment and retention of patients in clinical trials is an expensive and time-consuming process and could be made more difficult or rendered impossible by multiple factors outside our control; if we do not achieve our projected development and commercialization goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and our business will be harmed; our product candidates may cause serious adverse events or undesirable side effects, which may delay or prevent marketing approval, or, if approved, require them to be taken off the market, require them to include safety warnings or otherwise limit their sales; the market opportunities for AL101 and AL102, if approved, may be smaller than we anticipate; we may not be successful in developing, or collaborating with others to develop, diagnostic tests to identify patients with Notch-activating mutations; we have never obtained marketing approval for a product candidate and we may be unable to obtain, or may be delayed in obtaining, marketing approval for any of our product candidates; even if we obtain FDA approval for our product candidates in the United States, we may never obtain approval for or commercialize them in any other jurisdiction, which would limit our ability to realize their full market potential; we have been granted Orphan Drug Designation for AL101 for the treatment of ACC and may seek Orphan Drug Designation for other indications or product candidates, and we may be unable to maintain the benefits associated with Orphan Drug Designation, including the potential for market exclusivity, and may not receive Orphan Drug Designation for other indications or for our other product candidates; although we have received Fast Track designation for AL101, and may seek Fast Track designation for our other product candidates, such designations may not actually lead to a faster development timeline, regulatory review or approval process; we face significant competition from other biotechnology and pharmaceutical companies and our operating results will suffer if we fail to compete effectively; we are dependent on a small number of suppliers for some of the materials used to manufacture our product candidates, and on one company for the manufacture of the active pharmaceutical ingredient for each of our product candidates; if we are unable to enter into new collaborations, or if these collaborations are not successful, our business could be adversely affected; enacted and future healthcare legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates, if approved, and may affect the prices we may set; if we are unable to obtain, maintain, protect and enforce patent and other intellectual property protection for our technology and products or if the scope of the patent or other intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and we may not be able to compete effectively in our markets; we may engage in acquisitions or in-licensing transactions that could disrupt our business, cause dilution to our stockholders or reduce our financial resources; and risks related to our operations in Israel could materially adversely impact our business, financial condition and results of operations.

These and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2021 filed with the U.S. Securities and Exchange Commission (SEC) on March 28, 2022 and our other filings with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. New risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Release – Onconova Therapeutics Announces Updated Data from Investigator-sponsored Phase 1-2a Trial Evaluating Rigosertib in Combination with Nivolumab in Advanced KRAS-mutated Non-Small Cell Lung Cancer at the ESMO Congress 2022

Posted on by [email protected]

Research, News, and Market Data on ONTX

  • Data show an early signal of efficacy in an extensively pre-treated population with 1 complete response and 2 partial responses achieved in 14 evaluable patients

  • Responses achieved in patients with 3 distinct and different KRAS mutations, confirming the MOA of rigosertib being KRAS+ agnostic

  • 4 of 14 (29%) evaluable patients demonstrated disease control

  • The combination of rigosertib and nivolumab has been well tolerated with no synergistic toxicities observed to-date

NEWTOWN, Pa., Sept. 12, 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 updated data from an investigator-sponsored Phase 1/2a trial of oral rigosertib plus the anti-PD-1 immune checkpoint inhibitor (ICI) nivolumab in advanced KRAS-mutated (KRAS+) non-small cell lung cancer (NSCLC). The data, which are featured in a poster at the European Society for Medical Oncology (ESMO) Congress 2022, show an early and encouraging signal of efficacy in the trial’s extensively pre-treated population. The studied doublet has been well tolerated to-date.

“The emerging data being presented at ESMO are encouraging, as treatment with rigosertib plus nivolumab led to both complete and partial responses in patients with KRAS-mutated lung cancers who failed prior ICI therapy,” said Dr Rajwanth Veluswamy, the principal investigator of the study. “Objective responses showcased rigosertib’s KRAS mutation-agnostic mechanism of action, as each responding patient had a tumor with a different underlying variant. This differentiates rigosertib from agents targeting a single KRAS mutation variant, and positions it to potentially address the unmet needs of a much broader patient population. In addition, the ESMO data demonstrated activity in multiple patients with both low PD-L1 expression at diagnosis and STK11/LKB1 co-mutations, both poor predictive features for current lung cancer treatments.”

Key data from the presentation include:

Demographics:

  • All enrolled patients failed at least one line of prior therapy with a PD-1 checkpoint inhibitor (includes evaluable and non-evaluable patients)
  • 80% of enrolled patients failed at least two lines of prior therapy

Response results (as of August 15th, 2022-data cutoff date):

  • 3 of 14 evaluable patients achieved an objective response
    • 1 patient achieved a complete response (CR) as per RECIST Criteria, with complete resolution of the primary lung tumor as well as sites of metastatic disease.
    • 2 patients achieved a partial response (PR)
    • Responses were achieved in patients with 3 distinct KRAS mutations (CR: KRAS G12V; PRs: KRAS G12C/STK11 and Q61H/STK11)
  • The mean duration of response is 6.75 months
  • 4 of 14 evaluable patients achieved disease control (CR, PR, or stable disease)

Safety results:

  • The studied doublet has been generally well tolerated. Treatment-related adverse events (TRAE) have been mostly mild and manageable.
  • One dose limiting toxicity of grade 3 hyponatremia has been observed (previously documented with rigosertib)
  • Urinary toxicities well documented with rigosertib are the most common TRAE
  • No unexpected safety events or synergistic toxicities have been observed

Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova, commented, “The evidence of efficacy observed in the trial’s highly challenging population suggests rigosertib may synergize with ICI and potentially provide clinical benefit to patients with limited therapeutic options. This hypothesis is supported both by these latest clinical data and the results of preclinical studies in multiple indications. Looking forward, we expect the maturation of the trial’s current results, as well as the new data we expect to collect by enrolling additional patients, to provide key insights that will inform the next steps for rigosertib’s current investigator-sponsored study program.”   

The ESMO poster (#1018P) is titled “Phase 1/2 Trial of Rigosertib and Nivolumab for KRAS Mutated Non-Small Cell Lung Cancer (NSCLC) Patients.” It is currently available for viewing on the congress’s virtual platform and is being presented by the trial’s principal investigator, Rajwanth Veluswamy, M.D., Assistant Professor, Medicine, Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, today during Poster Session 14. The poster is available on the “Scientific Presentations” section of the Onconova website.

About the Investigator-sponsored Phase 1/2a Trial

This Phase 1/2a trial is designed to evaluate the combination of rigosertib and nivolumab in advanced KRAS+ metastatic NSCLC patients who have progressed on standard-of-care with anti-PD-1 monotherapy or anti-PD-1 in combination with chemotherapy. It includes a dose-escalating Phase 1 portion followed by a Phase 2a dose-expansion portion. Patients in the trial receive oral rigosertib twice daily on days 1-21, and intravenous nivolumab on days 1 and 15 of 28-day cycles. The primary endpoints of the trial are safety assessments to determine maximum tolerated dose, and overall response rate. Secondary endpoints include progression-free survival and overall survival. For more information on the trial, see ClinicalTrials.gov Identifier: NCT04263090.

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.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding the timing of Onconova’s and investigator-initiated clinical development and trial data, and the mechanisms, therapeutic effects, and indications for Onconova’s product candidates. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Company Contact:
Mark Guerin
Onconova Therapeutics, Inc.
267-759-3680
[email protected] 
https://www.onconova.com/contact/

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

Cocrystal Pharma (COCP) – Influenza Program Update

Posted on by cpatches


Monday, September 12, 2022

Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

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.

Cocrystal Has Reported Progress In Its Influenza Program.  Cocrystal has reported Phase 1 data and plans for a Phase 2a  trial for CC-42344, its oral drug for seasonal and pandemic influenza.  The drug is an inhibitor of the enzyme PB2 (polymerase basic 2), a subunit of the RNA polymerase required for the replication cycle of the virus.  This early point of action could make it effective against all strains of influenza.

Phase 1 Data Confirmed Once-Daily Dosing.  In July 2022, Cocrystal announced pharmacokinetic data from its Phase 1 trial. The single ascending-dose stage met its goals, confirming administration on a once-daily basis.  Patient enrollment continues in the multiple ascending dose portion.  The company plans to present the study data at a medical meeting later in the year.


Get the Full Report

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.