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Jan 11
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Amgen and Arrakis Therapeutics Announce Multi-Target Collaboration to Identify Novel RNA Degrader Small Molecule Therapeutics

By Arrakis Therapeutics, Press Release, Private Companies
Press Release.

 

Amgen and Arrakis Therapeutics Announce Multi-Target Collaboration to Identify Novel RNA Degrader Small Molecule Therapeutics

Collaboration Creates “Targeted RNA Degraders” by Bringing Together Amgen’s Induced Proximity Platform Discovery Expertise and Arrakis’ Leading-edge RNA-targeted Drug Discovery Platform

THOUSAND OAKS, Calif. and WALTHAM, Mass., Jan. 11, 2022 – Amgen (NASDAQ:AMGN) and Arrakis Therapeutics today announced a research collaboration focused on the discovery and development of RNA degrader therapeutics against a range of difficult-to-drug targets in multiple therapeutic areas. This new class of “targeted RNA degraders” consists of small molecule drugs that selectively destroy RNAs encoding disease-causing proteins by inducing their proximity to nucleases.

Under the terms of the agreement, Arrakis will lead research activities for the identification of RNA-targeted small molecule (rSM) binders against a broad set of targets nominated by Amgen. Both parties will collaboratively design and functionalize these molecules to specifically degrade targeted RNAs, and Amgen will lead further preclinical and clinical development activities. Amgen will pay $75 million upfront to Arrakis for five initial programs and will have the option to nominate additional programs. For each program, Arrakis will be eligible for additional payments from Amgen for preclinical, clinical, regulatory and sales milestones, and royalties up to low double digits. Arrakis could potentially receive several billion dollars in future payments if all milestones are met and future program options are exercised.

“Targeted RNA degradation is an exciting area that is pushing the boundaries of drug discovery and design,” said Raymond Deshaies, Ph.D., senior vice president of Global Research at Amgen. “The collaboration with Arrakis combines Amgen’s induced proximity expertise in discovering multispecific molecules to target the biologic mechanisms of disease and Arrakis’ pioneering discovery platform to predict RNA structures and identify small molecules that bind to them, significantly broadening the possibilities of addressing difficult protein targets considered undruggable because they may not have binding sites needed for conventional medicines. Combining this approach with Amgen’s targeted protein degradation induced proximity research already underway has the potential to significantly expand the druggable genome.”

By integrating the capabilities of the two innovative discovery platforms from Amgen and Arrakis, the collaboration creates an opportunity to design and engineer targeted RNA degraders. Amgen has built its Induced Proximity Platform to identify multispecific molecules that harness the power of cell biology by forming novel connections between natural effectors and targets. One end of the molecule binds to the target to be altered (inhibited, activated or destroyed) and the other end binds to a cellular effector that acts on the target, offering the potential to engage a broad range of cellular mechanisms to treat disease. With targeted RNA degraders, the effector, such as a ribonuclease or other RNA modulator, is brought into proximity of the RNA to degrade or otherwise modify the disease-causing RNA of interest. This complements Amgen’s existing efforts to target RNA with siRNA. In this collaboration, Arrakis’ rSM platform will be applied as a drug discovery engine to identify small molecules that bind target RNA. These rSMs will then be functionalized with nuclease recruiters to create heterobifunctional molecules that trigger degradation of disease-relevant RNA targets.

“We are excited to partner with Amgen’s strong research team to pursue a shared goal of creating a new class of medicines that induce degradation of disease-causing RNAs. This collaboration further demonstrates the utility of our proprietary rSM discovery platform for targeting RNA with small molecules and paves the way for creating powerful new treatments for patients,” said Michael Gilman, Ph.D., chief executive officer of Arrakis. “Based on our long-term goal to build a broad and industry-leading platform that adapts state-of-the-art drug discovery tools to target RNA biology, we have enabled a range of different applications and collaborations to leverage our science, a strong capital position and the ability to grow our business and our impact to advance RNA-targeted drug programs for diseases unaddressed by today’s medicines.”

About Amgen

Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people’s lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world’s leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

Amgen is one of the 30 companies that comprise the Dow Jones Industrial Average and is also part of the Nasdaq-100 index. In 2021, Amgen was named one of the 25 World’s Best Workplaces™ by Fortune and Great Place to Work™ and one of the 100 most sustainable companies in the world by Barron’s.

For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.

About Arrakis Therapeutics

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on cancer and genetically validated targets in other disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life science investors. The company is located in Waltham, Massachusetts.

For more information, please visit www.arrakistx.com and engage with us on Twitter @ArrakisTx or on LinkedIn.

Dec 15
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Artax Biopharma Announces First-in-Human Dosing in Phase 1 Clinical Trial for AX-158, Company’s First-in-Class Oral Immunomodulator to Treat T Cell-Mediated Diseases

By Artax Biopharma, Press Release, Private Companies
Press Release.

 

The Phase 1 Trial, Expected to Conclude in 2022, Will Evaluate Safety, Exposure, and Pharmacokinetics of AX-158 in Healthy Volunteers

AX-158, a Novel, Oral Small Molecule Immunomodulating Agent for the Treatment of T Cell-Mediated Diseases, Selectively Modulates T Cell Responses without Suppression of Immune System Function

Cambridge, Mass., December 15, 2021 – Artax Biopharma, Inc., a clinical-stage biotechnology company focused on transforming the treatment of T Cell-mediated diseases, today announces first-in-human dosing in a Phase 1 clinical trial for AX-158, the Company’s first-in-class oral small molecule immunomodulator. AX-158 is the Company’s first oral small molecule immunomodulating agent to enter clinical development for the treatment of T Cell-mediated diseases.

AX-158 employs a first-in-class mechanism of action that selectively modulates T cell responses that play a critical role in healthy immune system function. AX-158 has the potential to treat T Cell mediated diseases without the risk of immunosuppression.

The start of the Phase 1 clinical trial, which marks the beginning of Artax’s AX-158 clinical trial program, is expected to conclude in 2022. The Phase 1 clinical trial will assess the safety, exposure, and pharmacokinetics of AX-158 in healthy volunteers and will include ex-vivo stimulated measures of its pharmacodynamic activity. In preclinical studies, AX-158 demonstrated potential to treat T Cell mediated diseases by decreasing key cytokines including INFγ, TNFα and IL-2 in whole human blood samples.

“Artax has a unique opportunity to make a transformative difference for patients with T Cell-driven conditions, including autoimmune diseases, T Cell malignancies and induced T Cell pathologies – while not impacting the immune system’s ability to mount a strong response to foreign pathogens and infections,”

commented Artax Biopharma Chief Executive Officer Joseph Lobacki.

“We look forward to the results of this Phase 1 clinical study to inform our future clinical studies evaluating patients managing T Cell-mediated diseases.”

About Artax Science and Immunomodulation
A healthy immune system eliminates harmful foreign pathogens, while being tolerant of self-tissues and organs. The T Cell Receptor (TCR) is central to healthy T Cell function and a
well-functioning immune system. When TCR signaling becomes dysregulated, T Cells behave abnormally. This behavior results in T Cell-driven conditions, including autoimmune diseases, T Cell malignancies (lymphomas), and induced T Cell pathologies in which medical treatments result in immune reactions (such as stem cell transplants resulting in acute graft-versus-hostdisease or immuno-oncology treatments resulting in immune related-adverse events). Artax believes immunomodulation – a mechanism through which our investigational agents assist in rebalancing the immune system and eliminating a cause of T Cell-mediated diseases while not impacting patients’ ability to properly fight foreign pathogens – holds great potential.

About Artax-158
AX-158 is a first-in-class, oral small molecule immunomodulating agent in clinical development for the treatment of T Cell-mediated diseases. AX-158 employs a novel mechanism
of action that selectively modulates, or adjusts, T Cell responses that play a critical role in immune system function. Nck is a protein that naturally amplifies T Cell signaling directly at the TCR, contributing to T Cell mediated disease. AX-158 is a Nck SH3.1 domain inhibitor which selectively counteracts the role of Nck in T Cells. This process of immunomodulation assists the immune system to maintain healthy control and eliminates a direct contributor to T Cell-mediated diseases. Importantly, Artax believes that preclinical data suggests AX-158 will not be immunosuppressive and so will not impact the immune system’s ability to mount a strong response to foreign pathogens and infections.

About Artax Biopharma
Artax Biopharma is a clinical-stage biotechnology company transforming T Cell-mediated disease treatment by developing innovative small molecules that modulate the immune system.
Artax science holds broad potential to treat T Cell-mediated diseases such as autoimmune diseases, induced T cell pathologies (such as acute graft versus host disease and immuneoncology treatment-related adverse events) and T Cell malignancies, while simultaneously allowing the body to fight foreign pathogens. For more information, please visit
www.artaxbiopharma.com.

Contacts:
Karen LaRochelle
Chief Business Officer
Artax Biopharma
klarochelle@artaxbiopharma.com

Media:
Linda Phelan Dyson
+1 973-986-5873
ldyson@artaxbiopharma.com

Dec 06
Love0

Amphista Therapeutics Appoints Dr Martin Pass as CDO

By Amphista Therapeutics, Press Release, Private Companies
Press Release.

 

Amphista Therapeutics Appoints Dr Martin Pass as CDO

Cambridge, England, 6 December 2021 – Amphista Therapeutics, a leader in next generation targeted protein degradation (TPD) approaches, today announced the appointment of Dr Martin Pass, as Chief Development Officer (CDO).

Martin joins Amphista from AstraZeneca, where he was Vice President, Head Early Oncology Projects and led successful clinical development efforts. Martin has over 30 years’ experience in the pharmaceutical industry with many successes spanning early drug discovery through to Phase 3 clinical development.

Previously, Martin was a medicinal chemist at GlaxoSmithKline where he helped progress multiple candidates into clinical development in the cardiovascular, CNS and gastrointestinal therapeutic areas. He has a PhD in Organic Chemistry from Imperial College London and worked in post-doctoral research at the University of Virginia, USA. He is a co-author on over 60 patents and peer-reviewed publications.

Amphista’s CEO Dr Nicola Thompson said,

“On behalf of the Amphista team, I am delighted to welcome Martin as our CDO. Martin brings a wealth of strategic clinical development expertise from an impressive global career as a leader in clinical trials, in two world leading pharmaceutical companies. Martin’s leadership will support the realisation of the full potential of our proprietary next generation TPD technology across multiple therapy areas in order to bring transformative medicines to patients.”

Amphista’s new CDO, Dr Martin Pass, commented on his appointment,

“I’m delighted to join Amphista’s dynamic, world class team. Amphista is founded on transformational science and I am looking forward to driving the Company’s clinical development programmes and research as we pursue our mission of building the world’s leading TPD company and ultimately bringing benefit to patients.”

– Ends –

Media contacts:

Amphista Therapeutics
CEO NicolaThompson
+44 7464 974714
nicki@amphista.com

Scius Communications
Katja Stout
+44 7789 435990
katja@sciuscommunications.com

About Amphista Therapeutics
Amphista Therapeutics is focused on transforming the lives of patients with severe diseases including cancer. The company is applying its proprietary Amphista degrader platform to advance new approaches in targeted protein degradation (TPD), which address the challenges faced by the field and to realise the full therapeutic potential of this transformational approach. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s labs at the University of Dundee. The company has raised over $60M to date and is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, BioMotiv and Eli Lilly & Company.

For more information, please visit: http://www.amphista.com/

Dec 02
Love0

Highlight Therapeutics & Cima announce positive results from pre-clinical study combining BO-112 + STING agonist published in Journal for ImmunoTherapy of Cancer

By Highlight Therapeutics, Press Release, Private Companies
Press Release.

 

Intratumoral co-injection of BO-112 and a STING agonist synergize to achieve local and distant anti-tumor efficacy

Pre-clinical study data follow publication of positive Phase 2 results of BO-112 + pembrolizumab in melanoma at SITC

Madrid, Spain, 2 December, 2021 – Highlight Therapeutics, (“Highlight”), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, and the Spanish research institute Cima Universidad de Navarra today announced that positive results from a pre-clinical study of intratumoral administration of BO-112 with a STING agonist have been published in the Journal for ImmunoTherapy of Cancer (JITC, Impact Factor 13.75). 1

BO-112 is a dsRNA agonist which is in development to target anti-PD1 resistance. Positive preliminary results of a phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with advanced melanoma that have progressive disease on anti-PD-1-based therapy were presented at The Society for Immunotherapy of Cancer (SITC) in Washington, D.C. on Nov. 12.

Results of a pre-clinical in vivo study of intra-tumoral co-injections of BO-112 and the DXMAA STING agonist conducted by Cima demonstrated synergistic efficacy with the ability to eradicate distant, uninjected tumor lesions. The combination of BO-112 and DMXAA was chosen because of its excellent efficacy, and the requirements for antitumor effects were studied on selective depletion of immune cell types and in gene-modified murine strains lacking BATF3, IFNAR or STING.

“These results, following the positive Phase 2 data on the combination of BO-112 and anti-PD1 therapies, are very encouraging,”

said Dr. Marisol Quintero, CEO of Highlight Therapeutics.

“We have been collaborating with Dr. Melero to better understand BO-112’s Mode of Action. The main objective has been to achieve both local control of the disease and, more importantly, efficacy against distant tumor lesions. The work published in the Journal for ImmunoTherapy of Cancer shows that co-injections of BO-112 and a STING agonist attain synergistic efficacy, enabling distant uninjected tumor lesions to be eradicated. There may also be further opportunities involving irradiation of locally injected lesions and we look forward to continuing this work.”

“We believe this combined intratumoral approach should be feasible in the clinic, since both BO-112 and several STING agonists have been injected into patients’ tumors in clinical trials. We have consistently observed synergistic therapeutic effects on a variety of transplantable murine tumor models, showing complete regressions of the injected and non-injected concomitant tumor lesions”

commented Dr. Ignacio Melero, senior researcher in immunotherapy at Cima and co-director of the Department of Immunology at Clínica Universidad de Navarra.

Abstract published in JITC
Background BO-112 is a nanoplexed form of polyinosinic:polycytidylic acid that acting on TLR3, MDA5 and PKR elicits rejection of directly injected transplanted tumors, but has only modest efficacy against distant untreated tumors. Its clinical activity has also been documented in early phase clinical trials. The DMXAA STING agonist shows a comparable pattern of efficacy when used via intratumoral injections.
Methods Mice subcutaneously engrafted with bilateral MC38 and B16.OVA-derived tumors were treated with proinflammatory immunotherapy agents known to be active when intratumorally delivered. The combination of BO-112 and DMXAA was chosen given its excellent efficacy and the requirements for antitumor effects were studied on selective depletion of immune cell types and in gene-modified mouse strains lacking BATF3, IFNAR or STING. Spatial requirements for the injections were studied in mice bearing three tumor lesions.
Results BO-112 and DMXAA when co-injected in one of the lesions of mice bearing concomitant bilateral tumors frequently achieved complete local and distant antitumor efficacy. Synergistic effects were
contingent on CD8 T cell lymphocytes and dependent on conventional type 1 dendritic cells, responsiveness to type I IFN and STING function in the tumor-bearing host. Efficacy was preserved even if BO-112 and DMXAA were injected in separate lesions in a manner able to control another untreated third-party tumor. Efficacy could be further enhanced on concurrent PD-1 blockade.

Conclusion Clinically feasible co-injections of BO-112 and a STING agonist attain synergistic efficacy able to eradicate distant untreated tumor lesions.

Next steps in the development of BO-112 include:

  • Initiation of a pivotal Phase 3 study in 2nd-line melanoma is planned in 2022 following discussions with regulatory agencies in the US and Europe
  • Highlight Therapeutics has initiated strategic partnerships discussions with anti-PD1 companies interested in enhancing their anti-PD1 market potential
  • Initial data from a sponsor-initiated Phase 1B trial by UCLA evaluating BO-112 + pembrolizumab in anti-PD1 resistant hepatocellular carcinoma currently recruiting patients is expected in 2022

 

1. Alvarez M, Molina C, De Andrea CE, et al Intratumoral co-injection of the poly I:C-derivative BO-112 and a STING agonist synergize to achieve local and distant anti-tumor efficacy Journal for ImmunoTherapy of Cancer 2021;9:e002953. doi: 10.1136/jitc-2021-002953
https://jitc.bmj.com/content/9/11/e002953.info

For more information, please contact:

Highlight Therapeutics S. L.
info@highlighttherapeutics.com
Marisol Quintero, CEO
Mo PR Advisory
Tel: +44 (0) 7876 444977 / 07860 361746

Mo Noonan/Jonathan Birt
Cima Universidad de Navarra
mphuarte@unav.es
Maria Pilar Huarte
Notes to editors

About Highlight Therapeutics

Highlight Therapeutics, formerly known as Bioncotech Therapeutics, is a private, clinical-stage company dedicated to unlocking the full potential of immuno-oncology. Our lead drug candidate BO-112 is a best-in-class RNA-based therapy which has been demonstrated to initiate a powerful immune response, leveraging a unique multi-target approach to turn ‘cold’ tumors ‘hot’ and therefore visible to the immune system. It has the potential to rescue patients who are resistant to current checkpoint inhibitor therapy, a very large market opportunity. BO-112 is currently being investigated in a range of clinical trials as a monotherapy and in combination with checkpoint inhibitors. In addition to in-house research, Highlight Therapeutics has a number of external collaborators, including Merck & Co and UCLA.

For more information, please visit www.highlighttherapeutics.com

About Cima Universidad de Navarra

Cima Universidad de Navarra is a biomedical research institution of the Universidad de Navarra. Our mission is to conduct translational research of excellence, based on novel biological knowledge and aimed at finding therapeutic solutions to patients’ needs. To translate the results of basic research into clinical applications, Cima has a Translation and Transfer unit that seeks to establish collaborations with biotechnology and pharmaceutical companies to ensure patients can benefit from scientific innovation.

We specialize in cancer, heart and liver diseases, immunotherapy and gene therapy research.

More information: www.cima.unav.edu/en

Dec 02
Love0

AviadoBio™ Raises $80 million in Series A Financing to Advance Neurodegenerative Gene Therapy Platform

By AviadoBio, Press Release, Private Companies
Press Release.

 

  • Series A led by New Enterprise Associates (NEA) and co-led by Monograph Capital follows seeding by Advent Life Sciences, Dementia Discovery Fund (DDF), F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), and LifeArc
  • AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery
  • Co-founded by leading neurologist and neuroscientist Professor Christopher Shaw, molecular neurobiologist Dr Youn Bok Lee and vector biologist Dr Do Young Lee from King’s College London and the UK Dementia Research Institute
  • Lisa Deschamps appointed Chief Executive Officer, bringing 25 years of industry and extensive gene therapy experience

London, UK, December 2, 2021 — AviadoBio Ltd, a pioneering gene therapy company focused on developing and delivering transformative medicines for people living with neurodegenerative disorders, announced today the successful completion of an $80 million (£58.6 million) Series A financing round, following an initial $16.5 million (£12 million) seed financing. The funds will be used to advance AviadoBio’s lead program in frontotemporal dementia (FTD) into the clinic, progress its preclinical assets, including for amyotrophic lateral sclerosis (ALS), whilst continuing to expand its industry-leading team.

The financing was led by New Enterprise Associates (NEA) and co-led by Monograph Capital, with participation from LSP, as well as seed investors Advent Life Sciences, Dementia Discovery Fund (DDF), F-Prime Capital, JJDCand medical research charity LifeArc.

AviadoBio’s technology was developed in the laboratory of Christopher Shaw, Professor of Neurology and Neurogenetics at King’s College London’s Institute of Psychiatry, Psychology & Neuroscience, with the support of the UK Dementia Research Institute, the My Name’5 Doddie Foundation and the Van Geest Foundation and, incubated by F-Prime Capital and JJDC.

“Whilst neurodegenerative conditions are focal at onset, the pathology eventually spreads throughout the nervous system. We have seen that modifying gene expression can be curative, but achieving widespread distribution is the greatest challenge. We have shown that precision micro dosing to neural networks will deliver broad CNS expression, providing safe and effective treatments,”

said Prof. Christopher Shaw, Co-founder and Chief Scientific and Clinical Advisor of AviadoBio.

“We are directly exploiting insights into the causes of diseases to design therapies that have the potential to cure patients for whom there are no effective treatments. I believe that AviadoBio has the potential to move neurodegeneration from palliation to prevention.”

Shaw added:

“AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery. Precision micro dosing achieves extensive gene expression throughout the nervous system, maximizing the therapeutic potential for patients living with devastating neurological diseases.”

The Company is led by recently appointed Chief Executive Officer and Board member Lisa Deschamps, former Senior Vice President and Chief Business Officer at Novartis Gene Therapies. Lisa brings more than 25 years of biopharma experience including leading the global neuroscience business of Novartis and the worldwide commercialization of its gene therapy for spinal muscular atrophy.

“I am excited about the promise we can bring to people living with neurodegenerative disease, where a significant unmet medical need remains with only symptomatic therapies currently available. Our differentiated approach has the potential to transform the treatment landscape and we have the opportunity with our group of experienced investors, our advisors and founders, to change the lives of people living with these debilitating conditions,”

said Lisa Deschamps, Chief Executive Officer.

“The expansion of our team and investor syndicate, positions AviadoBio to make this mission a reality while advancing the field.”

As part of the Series A financing, Matt McAviney, Partner at NEA and Tim Funnell, Partner at Monograph Capital have been appointed to the Company’s Board of Directors. They are joined by Amber Salzman and Prof. Philip Scheltens as an independent Board Director and a Board Observer for LSP, respectively. Amber has been a pioneering entrepreneur in leading companies and not-for-profit organizations focused on gene therapies and rare diseases for more than 20 years, while Prof. Sheltens is a world-renowned dementia scientist and thought leader.

“Traditionally, there have been very few options to combat the devastating course of neurodegenerative disorders,”

said Matt McAviney, Partner, NEA and AviadoBio Board member.

“AviadoBio’s gene therapy platform introduces a revolutionary approach, supported by world-renowned neuroscience expertise and strong preclinical data. The Company is poised to make a meaningful impact, and we are thrilled to partner with the team to introduce a new portfolio of treatments to patients suffering from a range of debilitating diseases.”

ABOUT AVIADOBIO

At AviadoBio our mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute, co-founded by Prof. Christopher Shaw, Dr. Youn Bok Lee and Dr. Do Young Lee, with support from leading life science investors and Dr. Jonathan Jones and Dr. Graeme Fielder, as the Company’s founding management team. AviadoBio is building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products.

For more information, please visit www.aviadobio.com and follow us at Twitter @AviadoBio and LinkedIn AviadoBio.
LEARN MORE ABOUT US

CONTACT
For media enquiries:

Consilium Strategic Communications
Chris Gardner, Angela Gray, Isabelle Abdou
+44 (0) 20 3709 5700
AviadoBio@consilium-comms.com

Nov 12
Love0

Highlight Therapeutics announces positive preliminary results from Phase 2b study of BO-112 + anti-PD1 in confirmed anti-PD1 progressor melanoma patients at SITC

By Highlight Therapeutics, Press Release, Private Companies
Press Release.

 

Highlight Therapeutics announces positive preliminary results from Phase 2b study of BO-112 + anti-PD1 in confirmed anti-PD1 progressor melanoma patients at SITC

Breakthrough cancer treatment shows best-in-class potential to open up 2nd-line immunotherapy market to anti-PD1 resistant patients

  • BO-112 demonstrates potential as best-in-class therapy to overcome anti-PD1 resistance in melanoma patients whose disease has progressed on prior anti-PD-1 treatment
  • Primary endpoint met with a 27% Overall Response Rate (ORR), 8% Complete Responses (CR) & 65% Disease Control Rate (DCR) substantially exceeding current standard of care; further improvements anticipated over one year follow up
  • Hard-to-treat mucosal melanoma patients achieved 66% ORR and 100% DCR
  • Durable responses and manageable safety profile, with no patients discontinuing due to adverse events
  • Preparation for pivotal trial based on FDA guidance underway; potential for use in multiple solid cancers resistant to anti-PD1 inhibitors, and with different anti-PD1 combinations

Madrid, Spain, 12 November, 2021 – Highlight Therapeutics, (“Highlight”), a clinical-stage biopharmaceutical company developing RNA-based therapies against cancer, today announced positive preliminary results of a Phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with advanced melanoma whose disease had progressed on first-line anti-PD1-based therapy. BO-112 is a dsRNA agonist targeting anti-PD1 resistance, which has been successfully tested in several previous Phase 1b studies.

Initial results of the study were presented today (12th November) in a late-breaking session at The Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting in Washington DC, Nov. 10–14, 2021. (LBA Poster Abstract (961): Preliminary results of a phase 2 study of intratumoral administration of BO-112 with pembrolizumab in patients with advanced melanoma that have progressive disease on anti-PD-1-based therapy, presented in the Poster Hall in Washington, D.C. and the virtual ePoster Hall beginning Friday, Nov. 12 at 7 a.m. EST).

“These are potentially game-changing results showing that BO-112 can rescue melanoma patients who have failed first-line immune-therapy with anti-PD1,”

said Dr Carlos Paya, Executive Chairman of Highlight Therapeutics.

“Anti-PD1 based immunotherapy has revolutionized oncology treatments, but only a fraction of patients initially respond and many of these patients progress thereafter. These initial Phase 2 results show that BO-112 combined with a leading PD1 inhibitor rescue around 65% of anti-PD1 failing patients, making many of them respond to the combined treatment. These much-anticipated outcomes demonstrate BO-112’s potential as a best-in-class therapy for melanoma patients whose disease has progressed on prior anti-PD1 treatment, and we now look forward to further clinical studies, not only in melanoma but in other major tumor types in which anti-PD1 resistance is also an issue.”

Anti-PD1 therapies are valued at approximately $24 billion1 and are used across most solid tumors but currently fewer than 20% of all cancer patients benefit from first-line anti-PD1 treatment. BO-112 in combination with anti-PD1 therapy is designed to resensitize tumors to anti-PD1 treatment through improved antigen presentation, enhanced T-cell infiltration and increased MHC-1 and PDL1 expression by the tumor itself.

“This initial data is very encouraging and has the potential to change medical practice,”

said Dr. Marisol Quintero, CEO of Highlight Therapeutics.

“The preliminary ORR of 27%, including 8% Complete Responses, already exceeds current standard of care, such as the use of anti-CTLA-4. Based on the mode of action and experience from previous BO-112 Phase 1 studies, additional follow-up of these patients is expected to deliver further improvements in ORR. We are also encouraged by the excellent safety profile, with no patients discontinuing the study due to adverse events. Highlight Therapeutics is in the planning stage of a pivotal Phase 3 trial, due to begin in 2022, and we look forward to opening discussions with potential partners to explore combinations with anti-PD1s.”

Highlight Therapeutics and Merck, known as MSD outside the United States and Canada, conducted an open-label, single arm study to evaluate the efficacy & safety of intra-tumoral administration of BO-112 + pembrolizumab in mucosal, acral and cutaneous melanoma patients whose disease had progressed, confirmed by two consecutive CT scans. The study recruited 42 patients in France and Spain, with recruitment completed by August 24, 2021. Patients included those with high LDH levels, which are often associated with poor response rates and have been excluded from comparable clinical trials.

The preliminary analysis shows:

  • Primary endpoint (ORR by independent reviewer) has been met
  • With a median follow up of three months, there is a clear clinical benefit in patients with confirmed anti-PD1-resistant melanoma, with a 27% ORR and a 65% DCR, superior to 2nd line Standard of Care in stage III/IV melanoma of ~8% (continuing with anti-PD1 Ab) or 13% (second line ipilimumab).
  • Three hard-to-treat mucosal melanoma patients have achieved an ORR of 66% and DCR of 100%
  • High baseline LDH levels (>3xULN) predict progressive disease
  • Responses and SD are durable
  • Study treatment has a manageable safety profile, with no patients discontinuing due to adverse events

Next steps in the development of BO-112 include:

  • Initiation of a pivotal Phase 3 study in 2nd-line melanoma is planned in 2022 following discussions with regulatory agencies in the US and Europe
  • Highlight Therapeutics has initiated strategic partnerships discussions with anti-PD1 companies interested in enhancing their anti-PD1 market potential
  • Initial data from a sponsor-initiated Phase 1B trial by UCLA evaluating BO-112 + pembrolizumab in anti-PD1 resistant hepatocellular carcinoma currently recruiting patients is expected in 2022

1. IQVIA Global Oncology Report 2020

For more information, please contact:

Highlight Therapeutics S. L.                      info@highlighttherapeutics.com
Marisol Quintero, CEO

Mo PR Advisory                                       Tel: +44 (0) 7876 444977 / 07860 361746
Mo Noonan/Jonathan Birt

Consilium Strategic Communications     Tel: +44 7921 697654
Chris Gardner                                            cgardner@consilium-comms.com

Notes to editors

About Highlight Therapeutics

Highlight Therapeutics, formerly known as Bioncotech Therapeutics, is a private, clinical-stage company dedicated to unlocking the full potential of immuno-oncology. Our lead drug candidate BO-112 is a best-in-class RNA-based therapy which has been demonstrated to initiate a powerful immune response, leveraging a unique multi-target approach to turn ‘cold’ tumors ‘hot’ and therefore visible to the immune system. It has the potential to rescue patients who are resistant to current checkpoint inhibitor therapy, a very large market opportunity. BO-112 is currently being investigated in a range of clinical trials as a monotherapy and in combination with checkpoint inhibitors. In addition to in-house research, Highlight Therapeutics has a number of external collaborators, including Merck & Co and UCLA.

For more information, please visit www.highlighttherapeutics.com

Nov 01
Love0

Amphista Therapeutics Appoints CBO, Beverley Carr

By Amphista Therapeutics, Press Release, Private Companies
Press Release.

 

Amphista Therapeutics Appoints CBO, Beverley Carr

Cambridge, England, 1 November 2021 – Amphista Therapeutics, a leader in next generation targeted protein degradation (TPD) approaches, today announced the appointment of a Chief Business Officer (CBO), Beverley Carr.

Amphista’s CEO Dr Nicola Thompson said,

“On behalf of the Amphista team, I am thrilled to welcome Beverley as our CBO.  Beverley brings a wealth of business development expertise from an impressive career as a business leader, spanning both global Pharma and Biotech. Beverley’s leadership of this strategically important function will be key to ensuring that the full potential of our proprietary next generation TPD technology is captured across therapy areas in order to bring transformative medicines to patients.”

Amphista’s new CBO, Beverley Carr, commented on her appointment,

“I’m delighted to join Amphista’s world class team. Amphista is founded on transformational science and I’m looking forward to driving the Company’s business development strategy as we execute our mission of building the leading TPD company.”

Beverley joins Amphista from Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company, where she was CBO. Previously Beverley was Vice President Business Development for the Immunoinflammation Therapy Area at GSK, where she led over twenty transactions for the immune-inflammation therapy area. Beverley has negotiated and closed major deals across all stages of drug discovery and development. She is a scientist by training with an MA and DPhil in chemistry from Oxford University and has an MBA from Cambridge University.

– Ends –

Media contacts:

Amphista Therapeutics
CEO NicolaThompson
+447464974714
nicki@amphista.com

Scius Communications
Katja Stout
+447789435990
katja@sciuscommunications.com

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases including cancer. The company is applying its proprietary Amphista degrader platform to advance new approaches in targeted protein degradation (TPD), which address the challenges faced by the field and to realise the full therapeutic potential of this transformational approach. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s labs at the University of Dundee. The company has raised over $60M to date and is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, BioMotiv and Eli Lilly & Company.
For more information, please visit: http://www.amphista.com/

Sep 21
Love0

Amphista Therapeutics Appoints New Chairman, Joshua T. Brumm

By Amphista Therapeutics, Press Release, Private Companies
Press Release.

 

Glasgow, UK, 21 September 2021 – Amphista Therapeutics, a leader in next generation targeted protein degradation (TPD) approaches, today announced the appointment of Joshua T. Brumm as its new Independent Chairman, succeeding Satish Jindal.

Amphista’s CEO Dr Nicola Thompson said,

“On behalf of the Amphista team I am thrilled to welcome someone of Josh’s calibre to the Board. I look forward to working with Josh as we rapidly develop our pipeline of TPD therapeutics in oncology and beyond and unlock the full therapeutic potential of this exciting new modality. I would also like to thank our outgoing Chairman, Satish Jindal, who has tirelessly given his support as we have successfully advanced Amphista to this next stage of growth.”

Josh Brumm, Amphista’s new Chairman, commented on his appointment,

“I am delighted to join Amphista as its Chairman. The company, founded by leading pioneers in the field, has a unique, truly differentiated approach to targeted protein degradation and the opportunity to develop transformational therapies for patients. The potential of the technology is extremely exciting and together with Amphista’s world class team, I’m looking forward to executing on our mission of building the leading TPD company.”

Founding investor Raj Parekh, General Partner, Advent Life Sciences added,

“I am excited to welcome Josh as Amphista’s new Chairman. He brings a wealth of experience that will compliment the work that Nicki and the team have achieved and will support Amphista’s ambitious strategy”.

Josh Brumm is the president and CEO of Dyne Therapeutics, Inc. (NASDAQ: DYN), a leading muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. Before joining Dyne, he served as COO and CFO of Kaleido Biosciences, leading the company’s IPO, and helped bring the lead program into Phase 2 development.  Prior to this he was COO and CFO at Versartis, where he oversaw the company’s financial strategy including the successful completion of its IPO. Brumm has also held senior positions at Pharmacyclics, ZELTIQ Aesthetics and Proteolix, Inc as well as investment banking positions at Citigroup Global Markets, Inc. and Morgan Stanley. Over the course of his career, he has raised nearly $2 billion in capital. He holds a B.A. in business administration from the University of Notre Dame.

– Ends –

Media contacts:

Amphista Therapeutics
CEO NicolaThompson
+447464974714
nicki@amphista.com

Scius Communications
Katja Stout
+447789435990
katja@sciuscommunications.com

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases including cancer. The company is applying its proprietary Amphista degrader platform to advance new approaches in targeted protein degradation (TPD), which address the challenges faced by the field and to realise the full therapeutic potential of this transformational approach. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s labs at the University of Dundee. The company has raised over $60M to date and is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, BioMotiv and Eli Lilly & Company.

For more information, please visit: http://www.amphista.com

Sep 15
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Rappta Therapeutics Appoints Sunjeet Sawhney as Chief Executive Officer

By Press Release, Private Companies, Rappta Therapeutics
Press Release.

 

  • Experienced industry leader with successful track-record in oncology to lead Rappta through its next stages of growth
  • Rappta’s industry-leading phosphatase technology platform offers a new paradigm in oncology
  • Lead compounds on track to enter IND/CTA-enabling studies in the first half of 2022

Helsinki, Finland and London, UK, 15 September, 2021: Rappta Therapeutics (“Rappta”), which is focused on developing first-in-class anti-cancer drugs reactivating protein phosphatase 2A (PP2A), announces today the appointment of Sunjeet Sawhney as Chief Executive Officer (CEO) with immediate effect. Rappta’s Co-founder and Founding CEO Mikko Mannerkoski will move to be Chief Financial Officer (CFO) as planned.

With over 20 years of experience in the industry, Sunjeet joins Rappta from Ipsen where he has most recently been the Global Head of the Oncology Franchise. Prior to this Sunjeet led businesses across a range of geographies both in biotech and major pharmaceutical companies. Sunjeet holds a MSc in Immunology from the University of London and will be based in London, UK.

In October 2020, Rappta raised its first round of financing from blue-chip investors Advent Life Sciences, Novartis Bioventures, Novo Holdings, and a family office. Under the scientific leadership of Dr. Goutham Narla, Rappta’s Co-Founder and Chief Scientific Officer, the Company is developing first-in-class anti-cancer drugs reactivating PP2A – a critical enzyme regulating protein de-phosphorylation and a key tumor suppressor which to date has been very difficult to target pharmaceutically. Rappta’s strengthened leadership team is well-positioned to take its first-in-class program of PP2A-reactivating anti-cancer molecules to IND/CTA-enabling studies in the first half of 2022.

Sunjeet Sawhney, CEO of Rappta Therapeutics, commented:

“I am honored to have been given the opportunity to lead Rappta. The Company’s robust first-in-class program of anti-cancer drugs that reactivate PP2A represents an exciting clinical and commercial opportunity, much like kinase inhibitors did more than two decades ago. I am particularly impressed with the rigorous science and the swift progress that the team has made in moving the program forward from discovery towards development. I look forward to working together with the Rappta team in progressing our lead compounds while continuing to leverage our proprietary tools and unique understanding of PP2A to build a platform of a new class of anti-cancer drugs.”

Mikko Mannerkoski, CFO and Co-Founder of Rappta Therapeutics, commented:

“I look forward to working with Sunjeet, an experienced industry leader, to guide the Company through the next important stages of growth. As an entrepreneur and co-founder, it gives me great pride that Rappta has in a very short period of time built a robust drug development program that is ready to enter IND/CTA-enabling studies and subsequent first-in-human clinical trials, which has the potential to offer real benefit to patients while creating value for our shareholders.”

Goutham Narla, Rappta’s CSO and Co-Founder, commented:

“I am thrilled to work with Sunjeet on this opportunity to build a new platform and a novel class of pharmaceuticals to treat cancer. We have a unique team whose deep understanding of the PP2A biochemistry, structural biology, biogenesis, medicinal chemistry, and drug development represent a great combination of expertise to translate these discoveries into meaningful outcomes for patients. Sunjeet’s vast industry experience will add a new dimension to our competencies and accelerate the growth of the Company.”

ENDS

 

For more information please contact:

Rappta Therapeutics
Sunjeet Sawhney, CEO
+44 74 7244 6005
sunjeet.sawhney@rappta-therapeutics.com

Optimum Strategic Communications
Mary Clark, Manel Mateus, Vici Rabbetts
+44 (0) 20 3922 1906
rappta@optimumcomms.com

About Rappta Therapeutics

Rappta Therapeutics, a private biotech with operations in Finland, UK and the US, is developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A). It has developed proprietary tools and a unique understanding of PP2A which allows it to therapeutically reactivate PP2A, a critical enzyme regulating protein de-phosphorylation and tumor growth, with the potential to create a new class of anti-cancer drugs. Rappta has a strong scientific, management and commercial team. Its scientific team, led by CSO and co-founder, Professor Goutham Narla, Division Chief of Genetic Medicine at the University of Michigan, represents world-leading expertise in PP2A. The team is supported by a Scientific Advisory Board led by Dr. William Hahn, Professor of Medicine at the Harvard Medical School and the Chief Operating Officer of the Dana-Farber Cancer Institute and a Clinical Advisory Board lead by Dr. Mathew Galsky, Professor of Medicine and Director of Genitourinary Medical Oncology at the Icahn School of Medicine at Mount Sinai. The Company is backed by blue-chip investors Advent Life Sciences, Novartis Bioventures, Novo Holdings and a family office. For more information, go to www.rappta-therapeutics.com.

About PP2A

Reversible phosphorylation is a fundamental mechanism controlling all cell signaling and communication and this process is regulated through the opposing actions of phosphatases (which remove phosphate groups from proteins) and kinases (which add phosphate groups to proteins). Altered cellular signaling as a result of protein hyperphosphorylation, results in the sustained growth of malignant cells and is a hallmark of human cancer development and progression.

Protein Phosphatase 2A (PP2A) is a serine/threonine phosphatase that functions as a tumor suppressor by negatively regulating multiple oncogenic signaling pathways responsible for driving cancer progression. PP2A is made up of three subunits, that form a complete and active enzyme when bound together. The active enzyme is comprised of a scaffolding subunit (A), serving as the structural platform for the assembly of the catalytic (C) subunit and one substrate directing regulatory (B) subunit. In cancer, the tumor-suppressive activity of PP2A is often disrupted as a result of the inability of the three subunits to bind together correctly, rendering the PP2A enzyme inactive. This inactivation of PP2A, leads to increased oncogenic signaling, driving cancer progression and growth. Therefore, the reactivation of PP2A affords a unique therapeutic strategy to restore PP2A activity and cellular homeostasis, that can be used for the treatment of cancer and a broad range of other diseases.

Sep 02
Love0

Orphalan announces FDA acceptance for filing of New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s Disease

By Orphalan, Press Release, Private Companies
Press Release.

 

  • NDA supported by positive data from Phase 3 CHELATE clinical trial
  • TETA 4-HCl previously granted Orphan Drug Designation for first-line treatment of Wilson’s Disease

Paris, France – 02 September 2021 – Orphalan SA, a Company that identifies, develops and delivers worldwide therapies for orphan diseases, today announces that the US Food and Drug Administration (FDA) has accepted for review the Company’s New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the first-line treatment of Wilson’s Disease.

Wilson’s Disease is a rare inherited disorder of copper transport primarily affecting the liver and brain, affecting about 1 in every 30,000 people worldwide. For the last 70 years d- Penicillamine has been the only approved first-line treatment of Wilson’s Disease and to which about a third of patients develop intolerance.

TETA 4HCl is proposed as an alternative copper chelating agent to d-Penicillamine as a first line treatment. The Company’s NDA submission follows its previous Orphan Drug Designation by the FDA. The filing is based upon positive data from the CHELATE Phase 3 clinical trial that met its primary efficacy endpoint by demonstrating that TETA 4HCl was non-inferior to d-Penicillamine as measured by copper speciation evaluation of nonceruloplasmin copper (NCC).

Treatment with TETA 4HCl is supported by Orphalan’s novel NCC assay, for which the Company plans to file for FDA approval as a companion diagnostic. The in vitro NCC assay has the potential to provide an important additional tool to support physicians in identifying patients and monitoring their treatment with TETA 4HCl.

Dr Naseem Amin, Chief Executive Officer at Orphalan, commented:

“The FDA’s acceptance of the NDA brings us one step closer to providing first-line treatment to patients with Wilson’s Disease, who currently do not benefit from alternative methods of treatment, beyond D-Penicillamine”.

Notes to Editors

About the CHELATE Study

CHELATE was a Phase 3, multicentre, randomised, open label, active-controlled, noninferiority study conducted in 9 countries at 15 centres designed to evaluate efficacy and safety of trientine tetrahydrochloride compared to d-Penicillamine in patients with stable Wilson’s Disease. Fifty-three adult Wilson’s Disease patients with clinically stable disease for over one year and who met specific inclusion criteria, including laboratory measures of serum non-ceruloplasmin copper (NCC), 24-hour urinary copper excretion (UCE) and liver function tests, were followed for a baseline period for 12 weeks before being randomised 1:1 to either trientine tetrahydrochloride or d-Penicillamine twice daily. The study’s primary endpoint was serum NCC as measured using Orphalan’s proprietary method using copper speciation at 24-weeks post-randomisation. A secondary composite efficacy endpoint was NCC and 24-hour UCE.

Additional secondary endpoints included were: clinical Global Impression of Change (CGIC) score; serum copper and ceruloplasmin levels; the unified Wilson’s Disease Rating Scale (UWDRS); modified Nazer score; cognitive assessments and standard safety assessments. In addition, an independent adjudication committee blinded to the allocated treatment, and study centres assessed key efficacy and safety parameters to determine clinical stability of the patient. Trientine tetrahydrochloride was well tolerated and during treatment, more patients achieved the pre-specified composite endpoint of NCC and 24-hour Urinary Copper Excretion (UCE) within therapeutic target ranges, compared to patients treated with d- Penicillamine, 50% versus 24%. Data from the trial was presented during an oral presentation at EASL’s The International Liver Congress™ 2021.

About Orphalan

At Orphalan, our mission is clear: we are pioneers in orphan diseases. Orphalan identifies, develops and delivers worldwide innovative therapies for people living with Orphan diseases. Orphalan was founded in 2011 and has launched Cuprior™ across Europe with its own commercial organisation. For more information get in touch or follow us on LinkedIn.

For more information, please contact:

Orphalan
Tel: +33 (0)1 42 49 82 64
Email: info@orphalan.health

Consilium Strategic Communications:
Mary-Jane Elliott, Allison Connolly, Genevieve Wilson
Tel: +44 (0) 203 709 5700
Email: orphalan@consilium-comms.com