Category Archives: Biotech Companies

Orbis Medicines Launches with €26 Million Seed Financing to Transform Macrocycle Drug Development through Next-Generation Orally Dosable ‘nCycles’

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Financing led by Novo Holdings and Forbion

Orbis’ platform is first to systematically explore macrocycle chemical space using unique combination of high-throughput chemical synthesis and large-scale assaying, supported by machine learning

Pipeline to initially focus on high-value oral alternatives to blockbuster biologic drugs and opening new targets to therapeutic intervention

COPENHAGEN, Denmark–(BUSINESS WIRE)–Orbis Medicines, a leader in oral macrocycle drug discovery, today announces its launch with a €26 million financing led by global life sciences investor Novo Holdings and European life sciences venture firm Forbion. The funding will support Orbis’ expansion and advancement of its portfolio of next-generation macrocycle drugs it calls ‘nCycles’. Macrocycles are a large and diverse family of compounds with highly desirable therapeutic properties, defined by the presence of a cyclic structure. nCycles are systematically designed by Orbis’ nGen1 platform to be orally bioavailable and membrane permeable, solving decades-long challenges in macrocycle drug design.

The Orbis pipeline includes programs against targets validated by blockbuster biologic drugs, with the goal of providing oral alternatives that will enable the treatment of many more patients. In addition, Orbis is developing a pipeline of nCycles for other attractive target classes, both intra- and extra-cellular. Oral macrocycle drugs can potentially address a very wide range of diseases based on their ability to target a majority of the potential molecular targets in the body.

“After decades of challenging de novo synthesis, the universe of potential macrocycle targets is now available for exploration. Not only can Orbis reach a wealth of new targets, both inside and outside cells, but we can do this while preserving an oral format to pioneer a new therapeutic class of oral macrocycles. It’s a milestone for drug development,” said Morten Døssing, Chair of the Orbis Board and Partner at Novo Holdings. “We believe Orbis has the premier chemistry-led approach in the field, which gives us a distinct data advantage. We can generate hundreds of thousands of diverse compounds in record time and immediately apply a range of functional assays in a high-throughput fashion to each individual compound to home in on winners. Establishing this robust, reliable engine for candidate design was crucial for us as we look to bring macrocycle drugs to major patient populations.”

Orbis was founded in 2021 by the Seeds Investment team of Novo Holdings, with Mr. Døssing as Executive Chair and João Ribas as interim CBO establishing the company’s team and building its corporate strategy. The scientific foundation of Orbis was developed by Prof. Christian Heinis and Sevan Habeshian at the Swiss Federal Institute of Technology in Lausanne (EPFL).

“The broad potential of oral macrocycle drugs to address challenging targets untouchable by small molecules, coupled with recent momentum in the field, makes this an incredibly exciting development,” said Marco Boorsma, Ph.D., General Partner at Forbion. “With a compelling AI-enabled platform technology, scientific expertise and a clear strategy for growth, Orbis is attracting strong interest for its approach to finally delivering on the promise of this class for medicine. We believe Orbis’ combinatorial approach and ability to continuously generate big data from real compounds gives them an undeniable opportunity to unlock the potential of orally available macrocycle drugs for significantly larger patient populations.”

The chemical methods by which Orbis rapidly generates vast libraries of diverse macrocycle compounds ready for immediate assay were previously published in Nature Communications. Authors of the paper, which included Orbis’ founders Professor Heinis and Dr. Habeshian, detail how the methods draw on a large range of components to build compounds, including both natural and synthetic amino acids, creating an extremely large chemical space for exploration. Additionally, research recently published in Nature Chemical Biology demonstrates nGen’s ability to deliver nCycles that are orally bioavailable, marking a new era for macrocycle drug discovery.

About Orbis Medicines

Orbis Medicines is a biotechnology company focused on pioneering a new era for oral macrocycle drug discovery. Macrocycles are a large and diverse family of compounds with highly desirable therapeutic properties, defined by the presence of a cyclic structure. Orbis’ nGen platform is designed to systematically explore the macrocycle chemical space using a highly automated chemistry platform and deliver oral macrocycle drug candidates – nCycles – suitable for both intra- and extracellular targets. Orbis’ programs are focused on high-value oral alternatives to blockbuster biologic drugs and targets challenging for established modalities. Orbis was founded by Novo Holdings. For more information, please visit: www.orbismedicines.com

1About nGen

nGen is Orbis’ technology platform for generating oral macrocycle drug candidates, which it calls nCycles. It consists of multiple proprietary integrated elements starting with hit finding libraries of 100 billion compounds. Hits identified are progressed using Orbis’ highly automated chemistry-based platform that rapidly creates and tests hundreds of thousands of individual analogues with a full suite of assays to identify the most desirable ones. The scale and quality of the data produced from these real compounds, paired with machine learning, creates an industry-leading platform that de-risks and accelerates development. Recently published research in Nature Chemical Biology demonstrates nGen’s ability to deliver nCycles that are orally bioavailable, marking a new era for macrocycle drug discovery.

About Novo Holdings A/S

Novo Holdings is a holding and investment company that is responsible for managing the assets and the wealth of the Novo Nordisk Foundation. The purpose of Novo Holdings is to improve people’s health and the sustainability of society and the planet by generating attractive long-term returns on the assets of the Novo Nordisk Foundation.

Wholly owned by the Novo Nordisk Foundation, Novo Holdings is the controlling shareholder of Novo Nordisk A/S and Novozymes A/S and manages an investment portfolio with a long-term return perspective. In addition to managing a broad portfolio of equities, bonds, real estate, infrastructure and private equity assets, Novo Holdings is a world-leading life sciences investor. Through its Seeds, Venture, Growth, and Principal Investments teams, Novo Holdings invests in life science companies at all stages of development. As of year-end 2022, Novo Holdings had total assets of EUR 108 billion. www.novoholdings.dk

About Forbion

Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands and Germany. Forbion invests in life sciences companies that are active in the biopharmaceutical space, managing €3 billion across multiple fund strategies that cover all stages of biopharmaceutical drug development. The firm’s current team consists of more than 30 life sciences investment professionals that have built an impressive performance track record since the late nineties with investments in over 100 companies across 8 funds. Forbion’s record of sourcing, building and guiding life sciences companies has resulted in many breakthrough therapies and valuable exits. In addition to its financial return objectives, Forbion selects investments that will positively affect the health and well-being of patients. The firm is a signatory to the United Nations Principles for Responsible Investment. More information can be found at www.forbion.com

Contacts

Media:
Kit Rodophele

Ten Bridge Communications

617-999-9620

krodophele@tenbridgecommunications.com

Promising Results: TGR-63 Enhances Memory in Alzheimer’s Mouse Model

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IGC Pharma today announced the results of preclinical studies investigating its drug candidate TGR-63 as a treatment for Alzheimer’s disease. The data demonstrated enhanced memory function in an Alzheimer’s mouse model, including improved memory acquisition, consolidation, and retrieval.

TGR-63 is a therapeutic candidate designed to disrupt the structure of the amyloid-β (Aβ) peptide, mitigating symptoms such as memory loss and disrupted learning ability, signs of cognitive impairment. TGR-63 targets a critical neuropathological hallmark of Alzheimer’s Disease by addressing the misfolding and aggregation of Aβ peptides, specifically the toxic Aβ42 aggregation species associated with neuronal toxicity and cognitive decline. The cognitive impact of TGR-63 was assessed using two renowned behavioral tests, the Novel Object Recognition (“NOR”) Test and the Morris Water Maze (“MWM”), conducted on APP/PS1 genetically modified Alzheimer’s mice.

Ram Mukunda, CEO, commented, “These results underscore the efficacy of TGR-63 in enhancing spatial memory function in an Alzheimer’s disease model, offering promising implications for potential therapeutic interventions in Alzheimer’s and related neurodegenerative conditions. The improvements in memory acquisition, consolidation, spatial memory formation, and retrieval highlight its potential in addressing cognitive impairment in Alzheimer’s Disease. We are optimistic about TGR-63’s potential and anticipate advancing it towards a Phase 1 trial, enriching our clinical portfolio.”

The NOR Test, a cornerstone of preclinical research, evaluates recognition memory, a component of declarative memory responsible for recognizing previously encountered stimuli. Widely applicable across neuropharmacology and neurobiology, this test provides crucial insights into memory function and potential interventions for memory-related disorders such as Alzheimer’s. During the NOR Test, Alzheimer’s mice displayed a significantly diminished Discrimination Index (“DI”) compared to normal mice, indicating impaired long-term memory. TGR-63-treated Alzheimer’s mice showed a 51% improvement in DI, signifying promising therapeutic efficacy. After 48 hours, while untreated Alzheimer’s mice experienced a 100% decline in memory, those treated with TGR-63 demonstrated an 86% increase in DI, suggesting robust long-term potentiation (“LTP”) formation and successful memory retrieval.

The Morris Water Maze (“MWM”) is a pivotal tool in cognitive research, offering deep insights into spatial learning and memory processes. In this test, mice are trained to find a hidden platform in a pool over four days. Particularly crucial in neurodegenerative diseases like Alzheimer’s, the MWM allows researchers to explore spatial navigation and memory formation mechanisms. Over four days of training, TGR-63-treated mice exhibited a statistically significant reduction in the time required to locate the hidden platform, reflecting improved spatial memory acquisition. Additionally, when the platform was removed, the untreated AD mice spent 87% of their time away from the target area, while TGR-63-treated mice spent only 66% of their time away. TGR-63-treated mice also spent more time in the target area, showing improved spatial memory retrieval.

IGC Pharma is currently underway with a Phase 2b trial in the United States and Canada evaluating IGC-AD1 for agitation in Alzheimer’s. The trial encompasses 146 patients, with half assigned to the treated group receiving IGC-AD1, while the other half, the control group, receiving a placebo.

About IGC Pharma Inc. (dba IGC):

IGC Pharma is pursuing innovative solutions to fight Alzheimer’s disease and related challenges. IGC Pharma’s portfolio comprises five assets, all with a singular mission – to transform the landscape of Alzheimer’s treatment. IGC-AD1 and LMP target neuroinflammation, Aβ plaques, and neurofibrillary tangles. IGC-AD1 is currently in a Phase 2b clinical trial for agitation in dementia due to Alzheimer’s (clinicaltrials.gov, NCT05543681). TGR-63 targets Aβ plaque to disrupt the progression of Alzheimer’s disease. IGC-M3 targets the inhibition of Aβ plaque aggregation with the potential to create a profound impact on early-stage Alzheimer’s. IGC-1C targets tau and neurofibrillary tangles in a forward-thinking approach to Alzheimer’s therapy. In parallel, IGC Pharma is at the forefront of Generative AI development, with projects including clinical trials, early detection of Alzheimer’s, and drug interactions with cannabinoids.

Forward-Looking Statements:

This press release contains forward-looking statements. These forward-looking statements are based largely on IGC Pharma’s expectations and are subject to several risks and uncertainties, certain of which are beyond IGC Pharma’s control. Actual results could differ materially from these forward-looking statements as a result of, among other factors, the Company’s failure or inability to commercialize one or more of the Company’s products or technologies, including the products or formulations described in this release, or failure to obtain regulatory approval for the products or formulations, where required, or government regulations affecting AI or the AI algorithms not working as intended or producing accurate predictions; general economic conditions that are less favorable than expected; the FDA’s general position regarding cannabis- and hemp-based products; and other factors, many of which are discussed in IGC Pharma’s U.S. Securities and Exchange Commission (“SEC”) filings. IGC Pharma incorporates by reference the human trial disclosures and Risk Factors identified in its Annual Report on Form 10-K filed with the SEC on July 7, 2023, and Quarterly Report on Form 10-Q filed with the SEC on February 14, 2024, as if fully incorporated and restated herein. Considering these risks and uncertainties, there can be no assurance that the forward-looking information contained in this release will occur.

Contacts

Investors
IMS Investor Relations

Rosalyn Christian

igc@imsinvestorrelations.com
(203) 972-9200

Media
JVPRNY

Janet Vasquez

jvasquez@jvprny.com
(212) 645-5498

VERAXA Biotech AG acquires Synimmune GmbH to expand cancer pipeline

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Zurich, 29th of December 2023: VERAXA Biotech AG, a pioneering company in the development of superior antibody-based cancer therapies and a portfolio company of Xlife Sciences AG (SIX: XLS), is proud to announce the acquisition of Synimmune GmbH and its innovative Phase I antibody (FLYSYN) for the treatment of Acute Myeloid Leukemia (AML). This marks a major milestone in the company’s history and with the expansion of VERAXA’s cancer pipeline it is hope for patients battling AML – one of the most common types of acute leukemia in adults.

The FLYSYN antibody was acquired by VERAXA Biotech AG from Xlife Sciences AG portfolio company Synimmune GmbH via a share deal. The deal included an upfront payment as well as several milestones and will reach a total deal value of up to 32 million EUR. With this clinical asset, VERAXA strengthens its internal cancer pipeline of innovative antibodies and antibody-drug conjugates (ADCs).

AML, characterized by its prevalence in individuals over the age of 68 and accounting for approximately 1% of all cancer occurrences, has long presented a challenge in the medical community, particularly for those under 45 where it is less common. The successful First-in-Man multicenter clinical study of FLYSYN focused on patients with AML who had measurable residual disease (MRD). The introduction of FLYSYN, is a testament to VERAXA Biotech AG’s commitment to advancing healthcare and providing innovative solutions for serious health conditions.

Oliver R. Baumann, CEO of Xlife Sciences AG comments: «Securing the innovative Phase I antibody, FLYSYN, for Acute Myeloid Leukemia (AML) treatment marks a significant step forward. This milestone reflects our commitment to advancing healthcare and sets the stage for a promising future in reshaping AML treatment. »

Dr. Christoph Antz, CEO of VERAXA Biotech AG, stated: «With FLYSYN, we are one step closer to offering better, more effective treatments for those affected by Acute Myeloid Leukemia. »

As we continue to navigate the complexities of AML treatment, VERAXA Biotech AG remains steadfast in its mission to transform the landscape of medical care. For more information about VERAXA Biotech AG and our work in AML treatment, please visit www.veraxa.com.

 

Financial calendar

Annual Report 2023 23 April 2024
Annual Shareholders Meeting 2024 20 June 2024
Half-Year Report 2024 19 September 2024

Contact 
Information for investors and journalists: Xlife Sciences AG, Dr. Dennis Fink, dennis.fink@xlifesciences.ch
Information related to VERAXA Biotech AG: Dr. Christoph Antz, antz@veraxa.com

Xlife Sciences AG 
Talacker 35, 
8001 Zurich, Switzerland,
Phone +41 44 385 84 60
info@xlifesciences.ch, www.xlifesciences.ch 
Commercial Register Zurich CHE-330.279.788 
Stock Exchange: SIX Swiss Exchange

End of Inside Information

Language: English
Company: Xlife Sciences AG
Talacker 35
8001 Zürich
Switzerland
Phone: +41 44 385 84 60
E-mail: info@xlifesciences.ch
Internet: www.xlifesciences.ch
ISIN: CH0461929603
Valor: A2PK6Z
Listed: SIX Swiss Exchange
EQS News ID: 1806049

 
End of Announcement EQS News Service

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A new clinical study highlights efficiency in spine surgery of NextAR Spine, augmented reality surgical application

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A new clinical study highlights efficiency in spine surgery of NextAR Spine, augmented reality surgical application

CASTEL SAN PIETRO, 14 December 2023 – Medacta Group SA (“Medacta”, SIX:MOVE), a Swiss company specializing in the design, production, and distribution of innovative, personalized, and sustainable solutions for joint replacement, sports medicine, and spine surgeries, announces the publication of a new study in the European Spine Journal which demonstrates that NextAR Spine seamlessly integrates into the OR workflow, enabling the surgeon to enhance efficiency, accuracy, and versatility in spine surgery.

“Precision and accuracy in spine surgery is critical. As stated in our recent study, NextAR, the new AR-assisted navigation system, greatly simplifies pedicle screw placement, ensuring effectiveness and accuracy. It’s a reliable tool, straightforward to use, and it elevates our ability to provide safe and precise navigation for various spinal conditions,” says Professor Dr. med. Bernhard Meyer, Director of the Neurosurgical Clinic and Policlinic at the University Hospital Rechts der Isar, Munich.

The study “Evaluating a cutting‑edge augmented reality‑supported navigation system for spinal instrumentation”1 led by Prof. Dr. med. Bernhard Meyer, evaluates the efficiency, accuracy, and versatility of NextAR Spine, a navigation system that incorporates augmented reality to provide unique real-time surgical guidance, superimposed onto the operative field, enhancing precision and enabling data-driven decision-making in the placement of pedicle screws, and addressing different types of spine indications, such as trauma, degeneration, infection, and tumor.

The placement of pedicle screws has been achieved with precision using both open and percutaneous approaches in both long and short constructs, indicating a lower intraoperative revision rate (1.7%)1 compared to the rate documented in the scientific literature with other navigation systems (3.4% by Ille at al2; 4.7% by Ryang at al3). This further highlights that NextAR Spine proves to be a reliable and safe tool for 3D imaging-based pedicle screw placement while requiring minimal setup during surgery, in line with Medacta’s philosophy of healthcare sustainability. The setup is reduced to a minimum by integrating the cameras into the surgical instruments and establishing a flexible platform which includes the preoperative planning.

The NextAR platform is offered as a hardware system with limited capital investment and single-use instrumentation at a low cost per case and offers the ability to host software for multiple applications (NextAR Knee, NextAR Shoulder, NextAR Spine, and Hip applications). The platform represents an optimal solution worldwide, particularly for US Ambulatory Surgery Centers (ASCs).

The NextAR surgical platform is part of Medacta’s MySolutions Personalized Ecosystem, a network of advanced digital solutions designed to improve patient outcomes and healthcare efficiency. MySolutions embodies Medacta’s holistic approach to personalized medicine, aiming at bringing value to every step of the patient’s journey, from preoperative through postoperative care.

NextAR is also supported by the comprehensive, tailored educational offerings provided by the M.O.R.E. Institute. With an international network of expert surgeons, the M.O.R.E. Institute is at the forefront of education on spine procedures and products with personalized high-level educational pathways. With Medacta, the surgeon is never alone.

REFERENCES
[1] Schwendner M, Ille S, Wostrack M, Meyer B. Evaluating a cutting-edge augmented reality-supported navigation system for spinal instrumentation. Eur Spine J. 2023 Nov 14. doi: 10.1007/s00586-023-08011-w. Epub ahead of print. PMID: 37962688.
[2] Ille S, Baumgart L, Obermueller T, Meyer B, Krieg SM (2021) Clinical efficiency of operating room-based sliding gantry CT as compared to mobile cone-beam CT-based navigated pedicle screw placement in 853 patients and 6733 screws. Eur Spine J 30(12):3720–3730. https:// doi. org/ 10. 1007/ s00586- 021- 06981-3
[3] Ryang YM, Villard J, Obermuller T et al (2015) Learning curve of 3D fluoroscopy image-guided pedicle screw placement in the thoracolumbar spine. Spine J 15(3):467–476. https:// doi. org/ 10. 1016/j. spinee. 2014. 10. 003    
 

Contact

Medacta International SA
Gianluca Olgiati     
Senior Director Global Marketing  
Phone: +41 91 696 60 60
media@medacta.ch

 

About Medacta

Medacta is an international company specializing in the design, production, and distribution of innovative orthopaedic products, as well as in the development of accompanying surgical techniques. Established in 1999 in Switzerland, Medacta is active in joint replacement, spine surgery, and sports medicine. Medacta is committed to improving the care and well-being of patients and maintains a strong focus on healthcare sustainability. Medacta’s innovation, forged by close collaboration with surgeon experts globally, began with minimally invasive surgical techniques and has evolved into personalized solutions for every patient. Through the M.O.R.E. Institute, Medacta supports surgeons with a comprehensive and tailored program dedicated to the advancement of medical education. Medacta is headquartered in Castel San Pietro, Switzerland, and operates in over 50 countries. Follow us on Medacta TV, YouTube, LinkedIn, and X.

 

 

 

Additional features:

File: MEDIA RELEASE – A new clinical study highlights efficiency in spine surgery of NextAR Spine, augmented reality surgical application

End of Media Release

Language: English
Company: Medacta Group SA
Strada Regina
6874 Castel San Pietro
Switzerland
Phone: +41 91 696 6060
E-mail: info@medacta.ch
Internet: www.medacta.com
ISIN: CH0468525222
Listed: SIX Swiss Exchange
EQS News ID: 1796547

 
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Tryp Therapeutics Inc. Announces Signing of Definitive Agreement with Exopharm Limited

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, 12/11/2023 / 04:00, EST/EDT – EQS Newswire – Tryp Therapeutics Inc. (Regulated Unofficial Market in Berlin, Frankfurt, Munich, Stuttgart, Tradegate Exchange; Canadian Stock Exchange, Nasdaq OTC)

KELOWNA, BC / ACCESSWIRE / December 11, 2023 / Tryp Therapeutics Inc. (“Tryp” or the “Company“) (CSE:TRYP), a clinical-stage biotechnology company focused on developing intravenous-infused psilocin (the active metabolite of psilocybin) for diseases with high unmet medical needs, is pleased to announce that the Company has entered into an arrangement agreement (the “Arrangement Agreement“) with Exopharm Limited ACN 163 765 991 (“Exopharm” or the “Purchaser“) dated as of December 8, 2023, pursuant to which Exopharm has agreed to acquire all of the issued and outstanding common shares in the capital of Tryp (the “Tryp Shares“) in consideration of the issuance of 4.52 ordinary shares in the capital of Exopharm (the “Exopharm Shares“) for each one (1) Tryp Share. The arm’s length transaction will be completed by way of a statutory plan of arrangement under the Business Corporations Act (British Columbia) (the “Arrangement“).

Key Transaction Highlights

  • The purchase price of CAD$0.08 represents a 78% premium to the closing price of $0.045 and a 112% premium to the 20-day volume weighted price of $0.0378 per Tryp Share on December 8, 2023
  • Tryp shareholders expected to receive 4.52 Exopharm Shares for each one (1) Tryp Share held, representing an aggregate transaction value of approximately $12.8 million.
  • Both the Board and the Special Committee unanimously recommend that Tryp securityholders vote in favor of the Arrangement.
  • The majority of the directors of the combined entity to be appointed by Tryp.
  • Tryp’s clinical programs and development strategy will remain as the foundation of the combined entity.
  • The combined entity is expected to relist on the Australian Securities Exchange (the “ASX“) in Q1 2024 subject to, among other conditions, receipt of the requisite approval of Exopharm shareholders and raising a minimum of AUD$6,000,000 under a public offering.
  • The Arrangement is subject to customary closing conditions, including approvals from Tryp securityholders and Exopharm shareholders, from the ASX and the Supreme Court of British Columbia (the “Court“).

Jason Carroll, Chief Executive officer of Tryp, said “joining forces with Exopharm heralds a new era for Tryp, where we expect our combined strength and shared vision to elevate us to the prominent platform of the ASX. With enhanced access to capital and the evolving landscape of the Therapeutic Goods Administration (TGA) regulations in Australia for select psychedelics, we see even more paths forward to innovate and grow. I am thrilled to emphasize that our ongoing clinical commitments and our TRP-8803 R&D program remain unchanged and will be pursued with the same vigor and dedication. It is not expected that Exopharm’s current R&D program will be pursued following completion of the transaction. With this merger, we’re even better positioned to serve our stakeholders.”

The Arrangement Agreement is the culmination of an extensive and robust review of strategic alternatives available to maximize shareholder value that was conducted by the Company’s board of directors (the “Board“) and a special committee of the Board (the “Special Committee“).

Pursuant to the terms and conditions of the Arrangement Agreement, Exopharm has agreed to acquire 100% of the issued and outstanding Tryp Shares in consideration of 4.52 Exopharm Shares per one (1) Tryp Share. The consideration reflects a 78% premium to the closing price of the Shares on the Canadian Securities Exchange (the “CSE“) of $0.045 on December 8, 2023, the last trading day of the Tryp Shares prior to the announcement of the Arrangement. Pursuant to the Arrangement Agreement, holders of convertible securities of Tryp, including stock options, common share purchase warrants, secured convertible debentures and unsecured convertible notes (collectively, the “Tryp Convertible Securities“) will receive replacement securities of Exopharm having substantially similar economic terms in accordance with the rules of the ASX.

The Board, after receiving the recommendation of the Special Committee, has determined that the Arrangement, including the transactions contemplated thereunder, is fair to holders of Tryp securities and is in the best interests of the Company. Accordingly, the Board approved the Arrangement Agreement and recommends that securityholders vote their securities in favour of the Arrangement.

Each of the directors and executive officers of Tryp have entered into voting and support agreements with the Purchaser and have agreed to, among other things, vote their securities in favour of the Arrangement.

The Arrangement Agreement

Pursuant to the Arrangement, each Tryp Share outstanding immediately prior to the effective time of the Arrangement will be transferred to and purchased by the Purchaser in consideration of 4.52 Exopharm Shares and each Tryp Convertible Security outstanding immediately prior to the effective time of the Arrangement will be exchanged for securities of Exopharm having substantially similar economic terms, in accordance with the rules of the ASX.

The Arrangement is subject to a number of closing conditions, including: the approval of the Court; the approval of the ASX and all other applicable third party and regulatory consents for the Arrangement; the Company obtaining the requisite approval of its securityholders; Exopharm obtaining the requisite approval of its shareholders; no more than 10% of the Company’s shareholders exercising their rights of dissent in connection with the Arrangement, and the satisfaction of certain other closing conditions customary for a transaction of this nature.

The Arrangement Agreement includes customary provisions, including non-solicitation, right to match, and fiduciary out provisions, as well as certain representations, covenants and conditions which are customary for a transaction of this nature. The Arrangement Agreement provides for a termination fee payable by either party in certain circumstances in the event the Arrangement does not close.

Further information regarding the Arrangement will be contained in a management information circular that Tryp will prepare, file and mail to the Tryp securityholders (the “Circular“) in connection with an annual and special meeting of the Tryp securityholders to be held to consider the Arrangement (the “Meeting“). All securityholders are urged to read the Circular once available as it will contain important additional important information concerning the Arrangement. The Arrangement Agreement will also be filed on SEDAR+.

Only the holders of Tryp Shares, Tryp option holders and Tryp warrant holders at the close of business on the record date will be entitled to vote at the Meeting. The Arrangement will require the approval of (i) at least 66⅔% of the votes cast by Tryp shareholders; and (ii) at least 66⅔% of the votes cast by holders of Tryp Convertible Securities, voting as a class.

Advisors and Counsel

Pushor Mitchell LLP is acting as legal counsel to Tryp in Canada and K&L Gates LLP is acting as legal counsel to Tryp in Australia in connection with the Arrangement.

Osler, Hoskin & Harcourt LLP is acting as legal counsel to Exopharm in Canada and Hamilton Locke Pty Ltd is acting as legal counsel to Exopharm in Australia.

ACNS Capital Markets Pty Ltd trading as Alto Capital acted as Tryp’s advisor to Tryp’s recently closed private placement of unsecured convertible notes which raised approximately AUD$3,390,000, and is acting Exopharm’s corporate advisor in connection with the Arrangements.

About Tryp

Tryp Therapeutics is a clinical-stage biotechnology company focused on developing proprietary, novel formulations for the administration of psilocin in combination with psychotherapy to treat diseases with unmet medical needs. Tryp’s lead program, TRP-8803, is a proprietary formulation of IV-infused psilocin (the active metabolite of psilocybin) that alleviates numerous shortcomings of oral psilocybin including: significantly reducing the time to onset of the psychedelic state, controlling the depth and duration of the psychedelic experience, and reducing the overall duration of the intervention to a commercially feasible timeframe. The Company has completed a Phase 2a clinical trial for the treatment of Binge Eating Disorder at the University of Florida, which demonstrated an average reduction in binge eating episodes of greater than 80%. The Company has also started a Phase 2a clinical trial with the University of Michigan for the treatment of fibromyalgia and is preparing to initiate a Phase 2a clinical trial (IND has been cleared to proceed) with Massachusetts General Hospital for the treatment of abdominal pain and visceral tenderness in patients suffering from IBS. Each of the studies are utilizing TRP-8802 (synthetic, oral psilocybin) to demonstrate clinical benefit in these indications. Where a positive clinical response has been demonstrated, subsequent studies are expected to utilize TRP-8803 (IV-infused psilocin), which has the potential to further improve efficacy, safety and patient experience. For more information, please visit www.tryptherapeutics.com.

About Exopharm

Exopharm (EX1) is a leader in advancing and manufacturing technologies for exosome-based medicines using exosomes or extracellular vesicles (EVs) as a chassis for improved and non- viral drug-delivery.
Exosomes can be loaded with a variety of active pharmaceutical ingredients (APIs) and can be targeted to selected cell-types and tissue types, improving the safety-profile of the APIs and providing better treatments. Exosomes can be used to deliver small molecule drugs, mRNA, DNA and other types of APIs. Currently Exopharm is executing a strategy to maximize the significant value of the company’s IP position in the exosome field.

The Exopharm Shares to be issued under the Arrangement have not been and will not be registered under the U.S. Securities Act of 1933 and may not be offered or sold in the United States absent registration or applicable exemption from registration requirements. It is anticipated that any securities to be issued under the Arrangement will be offered and issued in reliance upon the exemption from the registration requirements of the U.S. Securities Act of 1933 provided by Section 3(a)(10) thereof.

Completion of the Arrangement is subject to a number of conditions, including but not limited to, all requisite regulatory, court and exchange approvals, the approval of the Court as well as the approvals of the securityholders of Tryp and shareholders of Exopharm. The Arrangement cannot close until the required approvals are obtained. There can be no assurance that the Arrangement will be completed as proposed or at all.

Investors are cautioned that, except as disclosed in the Circular to be prepared in connection with the Arrangement, any information released or received with respect to the Arrangement may not be accurate or complete and should not be relied upon. Trading in the securities of the Company should be considered highly speculative.

Neither the CSE nor the Market Regulator (as that term is defined in the policies of the CSE) has in any way passed upon the merits of the Arrangement and associated transactions and neither of the foregoing entities accepts responsibility for the adequacy or accuracy of this release or has in any way approved or disapproved of the contents of this press release.

This press release is not an offer to sell or the solicitation of an offer to buy the securities in the United States or in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to qualification or registration under the securities laws of such jurisdiction. The securities being offered have not been, nor will they be, registered under the United States Securities Act of 1933, as amended, and such securities may not be offered or sold within the United States or to, or for the account or benefit of, U.S. persons absent registration or an applicable exemption from U.S. registration requirements and applicable U.S. state securities laws.

Investor & Media Contact

Peter Molloy
Chief Business Officer
Tryp Therapeutics Inc.
pmolloy@tryptherapeutics.com

Forward-looking Statements:

Certain information in this news release is considered forward-looking within the meaning of certain securities laws and is subject to important risks, uncertainties and assumptions. This forward-looking information includes, among other things, information with respect to the Company’s beliefs, plans, expectations, anticipations, estimates and intentions. The words “may”, “could”, “should”, “would”, “suspect”, “outlook”, “believe”, “anticipate”, “estimate”, “expect”, “intend”, “plan”, “target” and similar words and expressions are used to identify forward-looking information. Forward-looking information in this news release relates to, among other things: anticipated benefits of the Arrangement to securityholders; the timing and receipt of required securityholder, Court, and regulatory approvals for the Arrangement; the ability of Exopharm to complete a prospectus offering and the amount to be raised thereunder, the composition of the board of directors of the combined entity, the ability of the Company and Exopharm to satisfy the other conditions to, and to complete, the Arrangement.

These statements reflect the Company’s current views with respect to future events and are necessarily based upon a number of assumptions and estimates that, while considered reasonable by the Company, are inherently subject to significant uncertainties and contingencies. Many factors, both known and unknown could cause actual results, performance or achievements to be materially different from the results, performance or achievements that are or may be expressed or implied by such forward-looking statements or information and the Company has made assumptions and estimates based on or related to many of these factors. Such factors include, without limitation, the satisfaction or waiver of all applicable conditions to closing of the Arrangement including, without limitation, receipt of all necessary securityholder, court and regulatory approvals or consents and lack of material changes with respect to the Company and its business, all as more particularly set forth in the Arrangement Agreement. In respect of the forward-looking statements and information concerning the anticipated completion of the proposed Arrangement and the anticipated timing for completion of the Arrangement, the Company has made certain assumptions that management believes are reasonable at this time, including assumptions as to the time required to prepare and mail Meeting materials and the satisfaction of all closing conditions. These dates may change for a number of reasons, including unforeseen delays in preparing Meeting materials; inability to secure necessary securityholder, court and regulatory approvals in the time anticipated or the need for additional time to satisfy the other conditions to the completion of the Arrangement. Accordingly, readers should not place undue reliance on the forward-looking statements and information contained in this news release concerning these times. In addition, in the event the Arrangement Agreement is terminated in certain circumstances, the Company may be required to pay a termination fee to the Purchaser, the result of which could have a material adverse effect on the Company’s financial position and results of operations and its ability to fund growth prospects and current operations.

The forward-looking information in this news release describes the Company’s expectations as of the date of this news release. Readers are cautioned against attributing undue certainty to forward-looking statements or information. Although the Company has attempted to identify important factors that could cause actual results to differ materially, there may be other factors that cause results not to be anticipated, estimated or intended. The Company does not intend, and does not assume any obligation, to update these forward-looking statements or information to reflect changes in assumptions or changes in circumstances or any other events affecting such statements or information, other than as required by applicable law.

SOURCE: Tryp Therapeutics

12/11/2023 EQS Newswire / EQS Group AG

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Ocedurenone: A Novel Therapy for Uncontrolled Hypertension in Advanced Chronic Kidney Disease

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EMJ Nephrology interviewed two World-renowned experts in nephrology and cardiology, George Bakris and Faiez Zannad, regarding the challenges of treating uncontrolled hypertension (uHTN) in patients with advanced chronic kidney disease (CKD), a large unmet need for safe, effective therapies, and the potential of one therapy, Ocedurenone, to address this need.

Original article: EMJ-8.1-2023-1.pdf (emg-health.com)

Large Unmet Need

Patients with advanced CKD have a high burden of disease, compounded by serious comorbidities, most commonly hypertension. Controlling hypertension is vital in these patients to reduce the associated risks of morbidity and mortality, as well as reducing cardiorenal risk, but treatment options are limited due to safety concerns with the use of existing agents.

Current Options

Bakris:”The PATHWAY-2 trial showed that in people with resistant hypertension without advanced CKD, spironolactone produced a greater reduction in blood pressure than a β-blocker and an α-blocker. However, it presents safety risks to patients when administered.”

“Then there is eplerenone, given twice a day, while safe, has a much weaker effect on blood pressure reduction than spironolactone. That’s all we’ve got, so that’s what people use.”

Dr Bakris also mentioned two existing nonsteroidal MRAs, esaxerenone which is “approved exclusively in Japan, for which there are no placebo-controlled comparisons”, and Finerenone which “reduces blood pressure (placebo subtracted) variously from 3 mmHg (ARTS-DN, FIGARO-DKD, and FIDELIO-DKD studies) to approximately 8 mmHg (subset analysis from ARTS-DN study, eGFR of approximately 68, though office blood pressure from the same study showed around 3 mmHg reduction)”.

Future Option – Ocedurenone

Bakris:”Now, Ocedurenone, a non-steroidal MRA, shows clear promise in patients whose standard of care includes two, three, or more drugs. Ocedurenone is the only drug that has consistently steadied resistant hypertension in people with advanced CKD.”

In the first dedicated study of patients with uncontrolled hypertension and advanced CKD (BLOCK-CKD), Ocedurenone demonstrated efficacy in lowering blood pressure, together with a favorable safety profile. The experts were optimistic that it could provide a much-needed treatment option for these patients, with the added potential for reduction in the risk of cardiorenal outcomes.

“This study showed that Ocedurenone reduces SBP by 11 mmHg (placebo-subtracted), a reduction that could lead to potential approval. No other agent to date has steadied blood pressure as well in this group of patients. And the point is that these are the patients who need the most help.”

Zannad reinforced the importance of the hyperkalemia data for the clinical safety of Ocedurenone:”With Ocedurenone, we may for the first time have an MRA that is both safe and efficacious.”

Bakris added:”A Phase 3 study (CLARION-CKD) of Ocedurenone in a similar patient population is ongoing. I remain optimistic that the signal seen in the BLOCK-CKD study will be seen in this larger study.”

“I remain excited about Ocedurenone. Its tolerability and efficacy in lowering blood pressure in a very high-risk group will have a significant impact on morbidity.”

“Mineralocorticoid receptors are so ubiquitous which may allow for indications beyond CKD and cardiovascular conditions. This is likely just the beginning for Ocedurenone.”

About Ocedurenone (KBP-5074)

KBP Biosciences is a global, clinical-stage biotechnology company, headquartered in Princeton, NJ, focused on discovering, developing, and commercializing innovative small-molecule therapeutics for the treatment of serious cardiorenal and infectious diseases with large unmet medical needs.

Ocedurenone (KBP-5074) is a non-steroidal MRA discovered and developed by KBP Biosciences. At present, the Phase 3 clinical trial of the first indication for Ocedurenone, advanced CKD and uncontrolled hypertension, is underway.

About KBP Biosciences, please visit https://www.kbpbiosciences.com/.

MiNA Therapeutics Presents Late-Breaking Positive Phase 1b Data for MTL-CEBPA in Combination with an Anti-PD1 Checkpoint Inhibitor at the Annual American Association of Cancer Research (AACR) Meeting

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MiNA Therapeutics Presents Late-Breaking Positive Phase 1b Data for MTL-CEBPA in Combination with an Anti-PD1 Checkpoint Inhibitor at the Annual American Association of Cancer Research (AACR) Meeting




MiNA Therapeutics Presents Late-Breaking Positive Phase 1b Data for MTL-CEBPA in Combination with an Anti-PD1 Checkpoint Inhibitor at the Annual American Association of Cancer Research (AACR) Meeting

Findings validate clinical proof of mechanism of MTL-CEBPA as a combination therapy in solid tumor cancers

Company to initiate out-licensing activities of its immuno-oncology portfolio, including MTL-CEBPA

LONDON–(BUSINESS WIRE)–MiNA Therapeutics Limited, the pioneer in small activating RNA (RNAa) therapeutics, will present positive updated biomarker data from its Phase 1a/b TIMEPOINT study of MTL-CEBPA in combination with the anti-PD1 checkpoint inhibitor, pembrolizumab, in adults with advanced solid tumors at the annual meeting of the American Association of Cancer Research (AACR) in Orlando, Florida. Findings from the Phase 1b portion of the study validate the clinical proof of mechanism of MTL-CEBPA as a combination treatment and identify a novel predictive biomarker of clinical response. The data will be presented on Tuesday, April 18, during a late-breaking research session beginning at 9:00 am EDT.

MTL‑CEBPA is the first therapy that specifically up-regulates CCAAT/enhancer binding protein alpha (C/EBP-α), a transcription factor that acts as a master regulator of myeloid cells. Dysregulated myeloid cells are implicated in several diseases including solid tumor cancers. MTL-CEBPA is designed to improve the effectiveness of checkpoint inhibitors and other immunotherapies by enhancing the body’s immune response and ability to attack the cancerous cells. Interim data from an open-label, multi-center study demonstrated the safety, tolerability, immunological and clinical activity of MTL-CEBPA in combination with pembrolizumab.

“Anti-PD1 checkpoint inhibition has significantly advanced the treatment of cancer, but many patients are resistant to treatment and present with tumors that resemble an ‘immune-desert’,” said Robert Habib, CEO of MiNA Therapeutics. “The updated analysis of TIMEPOINT shows that MTL-CEBPA used in combination with anti-PD1 checkpoint inhibitors profoundly reprograms the tumor microenvironment, reducing resistance and enabling infiltration of disease-fighting T-cells.”

The TIMEPOINT study also identified a novel predictive biomarker of clinical response to the combination treatment, presenting an opportunity for further evaluation as a potential companion diagnostic. The novel biomarker predicted clinical response across multiple tumor types.

MiNA plans to explore out-licensing opportunities for its immuno-oncology portfolio, which uniquely combines the capability to specifically restore or boost any dysregulated gene target with clinically validated in vivo delivery to myeloid immune cells.

MiNA’s immuno-oncology portfolio includes the following programs:

  • MTL‑CEBPA, which has been cleared for evaluation in a global Phase 2 clinical trial in combination with the tyrosine kinase inhibitor, sorafenib, in advanced hepatocellular carcinoma (HCC or liver cancer) and in a Phase 1 clinical trial in pediatric patients with MPS1 Hurler Syndrome
  • MTL-STING currently in pre-clinical development
  • Additional programs currently in discovery

About TIMEPOINT

TIMEPOINT is a global Phase 1a/b clinical study in patients with solid tumor malignancies to assess the safety and tolerability of MTL‑CEBPA in combination with pembrolizumab in patients who are ineligible or resistant to standard therapies. The study has received clearance from the U.S. Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA). To learn more about the TIMEPOINT clinical study, visit ClinicalTrials.gov (NCT04105335).

The poster presentation at AACR entitled, “MTL-CEBPA in combination with pembrolizumab converts an immune desert to an inflamed TME in solid tumors resistant to checkpoint blockade,” will be available on MiNA’s website in the Publications section under “RNA Activation”.

About MTL-CEBPA

MTL-CEBPA is the first therapy that specifically up-regulates CCAAT/enhancer binding protein alpha (C/EBP-α), a transcription factor that acts as a master regulator of myeloid cell lineage determination and differentiation. Dysregulated myeloid cells have been implicated in several diseases, and in solid tumor cancers these cells have been identified as a critical barrier to induction of clinical response for many therapies. In pre-clinical studies, MTL-CEBPA has been shown to improve the anti-tumor activity of cancer therapies by targeting dysregulated myeloid cells and reducing or eliminating their suppressive effect on immune response and therapies in the tumor microenvironment.

About MiNA Therapeutics

MiNA Therapeutics is the global leader in small activating RNA therapeutics or RNAa. Harnessing innate mechanisms of gene activation, RNAa therapeutics are a revolutionary new class of medicines that can restore or boost normal function of genes and thereby protein-modulated pathways in patients’ cells. We are advancing a proprietary pipeline of new medicines with an initial focus on genetic medicine, while collaborating with leading pharmaceutical companies to apply our technology platform across a broad range of other therapeutic areas. Based on our unique know-how in RNA activation, we are expanding the possibilities of RNA-based medicine for patients. www.minatx.com

Contacts

MiNA Therapeutics

Peter Bains, CBO

+44 208 811 6700

bd@minatx.com

Media:
Mike Beyer

Sam Brown, Inc.

mikebeyer@sambrown.com
+1 312-961-2502

Epic Sciences Raises $24M in Series G Financing to Continue Funding Commercialization of DefineMBC

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Epic Sciences Raises $24M in Series G Financing to Continue Funding Commercialization of DefineMBC




Epic Sciences Raises $24M in Series G Financing to Continue Funding Commercialization of DefineMBC

Funding will accelerate commercial launch of DefineMBC™

SAN DIEGO–(BUSINESS WIRE)–Epic Sciences, Inc. (“Epic” or “Epic Sciences”), a privately held diagnostics company, has completed a $24 million Series G which together with its $43 million Series F financing, totals $67 million raised in the last twelve months. The company will use the capital from this additional round of private investment to fund the commercial infrastructure required to commercialize the three component tests of DefineMBC, which together provide a comprehensive blood-based biopsy result for patients with metastatic breast cancer.

The investment was co-led by Deerfield Management and Arsenal Capital Partners, and included broad participation from the company’s shareholders including Blue Ox Healthcare Partners, Domain Ventures, and Labcorp.

“I’m excited to announce that Epic’s pre-launch activities related to DefineMBC have exceeded our expectations. We have delivered comprehensive test results to 700 patients and their oncologists in our Clinical Experience Program,” said Lloyd Sanders, President and CEO, Epic Sciences. “We have surveyed the physicians participating in the Clinical Experience Program and their responses indicate that oncologists see the value in DefineMBC’s ability to provide actionable information that allows them to better care for their patients.”

DefineMBC™ provides the most comprehensive blood-based biopsy results available to patients with metastatic breast cancer by delivering a report which includes:

  • detection of circulating tumor cells (CTCs)
  • assessment of HER-2 cellular protein expression coupled with

    • single-cell sequencing of identified cells to assess amplification of the ERBB2 gene locus
  • assessment of ER cellular protein expression
  • a plasma-based, 56-gene panel of cell-free DNA including

    • ERBB2 and PIK3CA
    • tumor mutational burden (TMB)

The new investment will enable the company’s efforts related to the commercialization of the DefineMBC tests, including the continued expansion of the company’s new market-facing departments: sales, product marketing, customer service, medical affairs, payer markets, and billing. The investment also continues to fund the development of additional concordance and outcomes data for the DefineMBC tests.

Andrew El Bardissi, a member of the company’s board of directors and Partner at Deerfield Management, said, “Deerfield is pleased to support Epic Sciences at this critical stage of commercialization. We believe DefineMBC is poised to change the management of metastatic breast cancer.”

About Epic Sciences

Epic Sciences, Inc. is developing and marketing novel diagnostics to guide therapy selection and monitor disease progression, personalizing and advancing the treatment and management of prostate and breast cancers. The company’s liquid biopsy platform leverages proven, proprietary cell analysis capabilities as well as cell-free analysis to provide more complete, efficient analysis and clearer insights – Comprehensive Cancer Profiling. Using its full-service CAP/CLIA-accredited laboratory and research support services, Epic Sciences partners with leading pharmaceutical companies and major cancer centers around the world to improve patient outcomes.

For more information, visit www.epicsciences.com or follow us on LinkedIn, Facebook or Twitter.

About Arsenal Capital Partners

Arsenal is a leading private equity firm that specializes in investments in healthcare and industrial growth companies. Since its inception in 2000, Arsenal has raised institutional equity investment funds of more than $10 billion, has completed more than 250 platform and add-on investments, and achieved more than 30 realizations. Arsenal invests in industry sectors in which the firm has significant prior knowledge and experience. The firm works with management teams to build strategically important companies with leading market positions, high growth, and high value-add. Visit www.arsenalcapital.com for more information.

Contacts

Brian.Strike@EpicSciences.com

Alpha Cancer Technologies (ACT) Publishes Pre-Clinical Study Results in Cancer Cell International Demonstrating Excellent Safety and Significant Anti-Tumor Activity from the Company’s Next Generation ADC, ACT-903

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Alpha Cancer Technologies (ACT) Publishes Pre-Clinical Study Results in Cancer Cell International Demonstrating Excellent Safety and Significant Anti-Tumor Activity from the Company’s Next Generation ADC, ACT-903




Alpha Cancer Technologies (ACT) Publishes Pre-Clinical Study Results in Cancer Cell International Demonstrating Excellent Safety and Significant Anti-Tumor Activity from the Company’s Next Generation ADC, ACT-903

ACT-903 conjugate demonstrated significant anti-tumor activity, with tumors becoming undetectable in 9 of 10 mice, and all 10 mice surviving through Day 60 with no obvious signs of toxicity

ACT’s technology highlights the potential to use the natural function of AFP as a carrier protein to deliver a payload uniquely targeted to cancer cells, while reducing risk of immune reactions or anti-drug antibody accumulation

Alpha fetoprotein conjugates had a very low percentage of free toxin detectable in blood after administration, and did not show any bone marrow accumulation of cytotoxic payload, suggesting limited off-target toxicity

TORONTO–(BUSINESS WIRE)–#ACT903–Alpha Cancer Technologies Inc. (ACT), a biopharmaceutical company focused on developing and commercializing targeted immuno-oncology and immunomodulation therapies based on its proprietary recombinant human Alpha Fetoprotein (AFP) platform, today announced the publication of pre-clinical study results in Cancer Cell International highlighting the company’s recombinant human AFP (ACT-101) conjugate with maytansine in mouse xenograft models of colorectal cancer. Results from the study demonstrate the exceptional safety profile and potent anti-tumor activity of multiple ACT-101 conjugates through the successful targeting of the alpha fetoprotein receptor uniquely located on cancerous cells and myeloid-derived suppressor cells (MDSCs).

The AFP receptor has been found on the surface of cancer cells and MDSCs exclusively, making it an attractive target for the delivery of toxins selectively to tumor cells and MDSCs. Human AFP acts as a shuttle protein, transporting a variety of molecules including targeted anti-cancer agents inside the cell via the AFP receptor. ACT-101 is a recombinantly produced non-glycosylated form of human AFP, which will carry and deliver maytansine and other chemotherapy payloads to tumor cells via the AFP receptor. The selection of maytansine was based on its prior efficacy in clinical studies when incorporated into ADCs.

The AFP receptor has emerged as a novel target for cancer therapeutics given the expression of AFP on most cancers and MDSCs, and lack of presence on normal tissues. Studies were performed to investigate the use of ACT-101 conjugated with maytansine for targeted toxin delivery to cancer. Four structurally different ACT-101-maytansinoid conjugates containing cleavable glutathione sensitive linkers were initially investigated in a mouse xenograft model of colorectal cancer (COLO-205). Reduction in tumor volume was seen for all four conjugates compared to control (p < 0.05). The anti-tumor effects of the conjugate selected for further development, ACT-903, persisted after treatment discontinuation, with tumors becoming undetectable in 9 of 10 mice, and all 10 mice surviving through Day 60 with no obvious signs of toxicity. A follow-up study performed in the same model compared the effects of single intravenous doses of ACT-903 (10–50 mg/kg) to control groups receiving vehicle or ACT-101. A significant reduction of tumor burden compared to control was achieved in the 40 and 50 mg/kg dose groups. Survival was also significantly prolonged in the 40 mg/kg and 50 mg/kg cohorts. Free maytansine blood levels at 4 hours were 0.008% of the dose, indicating stability in circulation as was expected based on in vitro plasma stability studies. No obvious signs of toxicity were seen in any of the treated groups. The observed efficacy and excellent tolerability of ACT-903 in these xenograft models support advancing the development of ACT-903 into clinical studies.

“Based on the evidence we continue to observe in our pre-clinical studies, we believe ACT-903 has the potential to address the limitations of current-generation antibody drug conjugates through a targeted delivery exclusively to cancer and immune suppressor cells, allowing for greater efficacy with minimal toxicity,” said Dr. Igor Sherman, CEO of ACT. “Given the broad range of cancer cells expressing the AFP receptor, we believe our technology offers a potential pan-cancer opportunity and we look forward to continuing the development of this program to bring it to cancer patients in the shortest possible time.”

The manuscript, titled “High anti-tumor activity of a novel alpha-fetoprotein-maytansinoid conjugate targeting alpha-fetoprotein receptors in colorectal cancer xenograft model,” was published in Cancer Cell International on April 5, 2023 and the full manuscript can be accessed here.

About Alpha Cancer Technologies, Inc.

Alpha Cancer Technologies Inc., (ACT) is a private clinical stage biotechnology company with immuno-oncology, theranostics and immunomodulation platforms under development. The company’s drug products use ACT’s patented recombinant human alpha fetoprotein (AFP or ACT-101). ACT’s immuno-oncology products target AFP receptors uniquely expressed on almost all solid and liquid tumors and immune suppressor cells but are absent on normal cells. This approach utilizes next-generation ADC technology and offers the benefits of much lower toxicity and greater efficacy compared to conventional chemotherapy and other targeted therapies. ACT is based in Toronto, Ontario, Canada. For more information, please visit www.alpha-cancer.com

Contacts

Richard Potts, Chair

Alpha Cancer Technologies Inc.
Tel: +1 (416) 464-2678

Email: rpotts@alpha-cancer.com

Igor Sherman, Ph. D., President & CEO

Alpha Cancer Technologies Inc.
Tel: +1 (416) 826-6626

Email: isherman@alpha-cancer.com

For investors, please contact:
Stephen Jasper

Gilmartin Group
Tel: +1 (858) 525-2047

Email: stephen@gilmartinir.com

Rallybio Announces Clinical Proof-of-Concept Results for RLYB211, an Anti-HPA-1a Polyclonal Antibody for the Prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT), Published in the Journal of Thrombosis and Haemostasis

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Rallybio Announces Clinical Proof-of-Concept Results for RLYB211, an Anti-HPA-1a Polyclonal Antibody for the Prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT), Published in the Journal of Thrombosis and Haemostasis




Rallybio Announces Clinical Proof-of-Concept Results for RLYB211, an Anti-HPA-1a Polyclonal Antibody for the Prevention of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT), Published in the Journal of Thrombosis and Haemostasis

— Data Support Potential Use of an Anti-HPA-1a Antibody as Prophylaxis for FNAIT —

— In March 2023, Rallybio Announced Clinical Proof-of-Concept Achieved for its Lead Product Candidate, RLYB212, a Monoclonal Anti-HPA-1a Antibody, in the First Quarter of 2023 —

NEW HAVEN, Conn.–(BUSINESS WIRE)–Rallybio Corporation (Nasdaq: RLYB) today announced publication of Phase 1 / 2 proof-of-concept results for RLYB211, a polyclonal anti-HPA-1a antibody derived from human plasma for the prevention of FNAIT. FNAIT is a rare and life-threatening bleeding disorder in which maternal alloantibodies directed against fetal platelets can lead to devastating outcomes for the fetus/neonate. There are currently no approved therapies for the prevention or treatment of FNAIT. The findings were published in the Journal of Thrombosis and Haemostasis, the official journal of the International Society on Thrombosis and Haemostasis.

Rallybio’s randomized, single-blind, placebo-controlled, study investigated the ability of a single dose of intravenous RLYB211 to eliminate HPA-1a-positive platelets transfused into HPA-1a-negative healthy subjects. The platelet transfusion in this study was selected based on the precedent of historical clinical trial designs to assess the efficacy of anti-D prophylaxis, in which the transfusion of antigen positive cells is designed to mimic a catastrophic fetal maternal hemorrhage. As has been shown with anti-D prophylactic agents, the ability of RLYB211 to drive the rapid and complete elimination of antigen positive cells is a surrogate indicator of its potential to prevent maternal alloimmunization, and therefore FNAIT. In Cohort 1, subjects received RLYB211 or placebo one hour after transfusion of HPA-1a positive platelets and in Cohort 1B subjects received RLYB211 or placebo followed by platelet transfusion one week later. Proof-of-concept was defined as ≥90% reduction in platelet elimination half-life compared to placebo.

The publication stated:

  • RLYB211 accelerated the elimination of HPA-1a positive platelets versus placebo, with all subjects meeting proof-of-concept criteria for greater than 90% reduction in platelet elimination half-life compared to placebo.
  • Rapid elimination of transfused platelets was evident 7 days after RLYB211 administration, simulating prophylactic administration of an anti-HPA-1a antibody prior to a fetal-maternal bleed.
  • RLYB211 was well tolerated and no serious adverse events were reported.

“We are pleased that these clinical results were published in the Journal of Thrombosis and Haemostasis, indicating that RLYB211 markedly accelerated the elimination of HPA-1a-positive platelets. We believe that these data demonstrate the prophylactic potential of anti-HPA-1a antibodies to prevent HPA-1a alloimmunization and occurrence of FNAIT,” said Róisín Armstrong, Ph.D., Rallybio’s FNAIT Program Lead. “Our FNAIT program continues to advance and as previously announced, we are pleased that an abstract reporting results from the Phase 1b proof-of-concept study for RLYB212, our monoclonal candidate, has been accepted for presentation at the upcoming 31st Congress of International Society on Thrombosis and Haemostasis in June.”

Dr. Christof Geisen, Institute of Transfusion Medicine and Immunohaematology, German Red Cross Blood Transfusion Service, and a key collaborator in the RLYB211 study, stated, “In addition to the rapid elimination of transfused platelets in all subjects, the effect of RLYB211 was also demonstrated when it was administered one week prior to platelet transfusion, suggesting a durable potential treatment effect for HPA-1a-specific antibodies and that FNAIT might safely be prevented by rapidly eliminating platelet antigen from maternal circulation before alloimmunization can occur.”

The RLYB211 Phase 1 / 2 results were published in the April issue (Volume 21, Issue 4, Pages 838-849) of the Journal of Thrombosis and Haemostasis, https://doi.org/10.1016/j.jtha.2022.11.041.

Given the advantages of small volume subcutaneous dosing of RLYB212 as compared to RLYB211 and the expected manufacturing and supply efficiencies for RLYB212, Rallybio announced in March 2023 that the Company will not continue development of RLYB211.

About FNAIT

Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) is a potentially life-threatening rare disease that can cause uncontrolled bleeding in fetuses and newborns. FNAIT can arise during pregnancy due to an immune incompatibility between an expectant mother and her fetus in a specific platelet antigen called human platelet antigen 1, or HPA-1. There are two predominant forms of HPA-1, known as HPA-1a and HPA-1b, which are expressed on the surface of platelets. Individuals who are homozygous for HPA-1b, meaning that they have two copies of the HPA-1b allele and no copies of the HPA-1a allele, are also known as HPA-1a negative. Upon exposure to the HPA-1a antigen, these individuals can develop antibodies to that antigen in a process known as alloimmunization. In expectant mothers, alloimmunization can occur upon mixing of fetal blood with maternal blood. When alloimmunization occurs in an expectant mother, the anti-HPA-1a antibodies that develop in the mother can cross the placenta and destroy platelets in the fetus. The destruction of platelets in the fetus can result in severely low platelet counts, or thrombocytopenia, and potentially lead to devastating consequences including miscarriage, stillbirth, death of the newborn, or severe lifelong neurological disability in those babies who survive. There is currently no approved therapy for the prevention or treatment of FNAIT.

About Rallybio

Rallybio (NASDAQ: RLYB) is a clinical-stage biotechnology company with a mission to develop and commercialize life-transforming therapies for patients with severe and rare diseases. Rallybio has built a broad pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of maternal fetal health, complement dysregulation, hematology, and metabolic disorders. The company has two clinical stage programs: RLYB212, an anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and RLYB116, a C5 complement inhibitor with the potential to treat several diseases of complement dysregulation, as well as additional programs in preclinical development.

Rallybio is headquartered in New Haven, Connecticut with an additional facility at the University of Connecticut’s Technology Incubation Program in Farmington, Connecticut. For more information, please visit www.rallybio.com and follow us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements that are based on our management’s beliefs and assumptions and on information currently available to management. In some cases, forward-looking statements can be identified by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements concerning the ability of RLYB212 and RLYB211, and the prophylactic administration of HPA-1a-specific antibodies, to clear platelets and prevent alloimmunization in pregnant women, and the anticipated threshold effect for rapid and complete elimination of HPA-1a positive platelets, the expected progress, results, and plans for RLYB212 and RLYB211, the initiation, substance, design and timing of our planned or ongoing studies for RLYB212, the timing of the availability of data from such studies, our expectations regarding reporting of data from such studies, and our expectations regarding the usefulness of data from such studies. The forward-looking statements in this press release are only predictions and are based largely on management’s current expectations and projections about future events and financial trends that management believes may affect Rallybio’s business, financial condition and results of operations. These forward-looking statements speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions, including, but not limited to, our ability to successfully initiate and conduct our planned clinical trials, including the ongoing and future clinical trials for RLYB212 for the prevention of FNAIT, and complete such clinical trials and obtain results on our expected timelines, or at all, whether our cash resources will be sufficient to fund our operating expenses and capital expenditure requirements and whether we will be successful raising additional capital, competition from other biotechnology and pharmaceutical companies, and those risks and uncertainties described in Rallybio’s filings with the U.S. Securities and Exchange Commission (SEC), including Rallybio’s Annual Report on Form 10-K for the period ended December 31, 2022, and subsequent filings with the SEC. The events and circumstances reflected in our forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Moreover, we operate in an evolving environment. New risks and uncertainties may emerge from time to time, and it is not possible for management to predict all risks and uncertainties. Except as required by applicable law, we are not obligated to publicly update or revise any forward-looking statements contained in this press release, whether as a result of any new information, future events, changed circumstances or otherwise.

Contacts

Investors
Ami Bavishi

Head of Investor Relations and Corporate Communications

475-47-RALLY (Ext. 282)

abavishi@rallybio.com

Hannah Deresiewicz

Stern Investor Relations, Inc.

212-362-1200

hannah.deresiewicz@sternir.com

Media
Tara DiMilia

908-369-7168

Tara.dimilia@tmstrat.com