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Dec 13
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Levicept Completes Recruitment in Phase II Clinical Trial of Neurotrophin Modulator, LEVI-04

By Levicept, Press Release, Private Companies
Press Release.

 

Levicept Completes Recruitment in Phase II Clinical Trial of Neurotrophin Modulator, LEVI-04
Study of Novel Biological Agent for Chronic Pain in Patients with Osteoarthritis

Sandwich, UK – 13 December 2023 – Levicept Ltd, a biotechnology company focused on the development of LEVI-04, a first-in-class treatment for chronic pain indications, today announces it has completed recruitment in its Phase II clinical trial of LEVI-04, a novel neurotrophin-modulating biological agent. Top line data are expected to be announced in late first half of 2024.

The multiple arm, multicentre, prospective, randomized, double-blind, placebo-controlled, Phase II study enrolled more than 500 participants with pain due to osteoarthritis of the knee (ClinicalTrials.gov ID: NCT05618782). The trial was designed to evaluate the efficacy, safety and tolerability of five, monthly, infusions of LEVI-04 as compared to placebo in participants with radiographic and symptomatic knee osteoarthritis. The trial is being conducted at sites across Europe and in Hong Kong, led by Dr Philip Conaghan at The School of Medicine, University of Leeds, UK.

LEVI-04 modulates the neurotrophin pathway that is clinically proven to provide effective analgesia in a range of indications and is designed to minimise use-limiting side effects of anti-NGF antibodies, including rapid progression of osteoarthritis. It is a proprietary p75 neurotrophin receptor fusion protein (p75NTR-Fc).

LEVI-04 is designed to deliver neurotrophin homeostasis by providing a reversible binding protein which scavenges excess neurotrophins present in chronic pain states. It operates across four receptor pathways in the neurotrophin system and is designed to supplement the existing endogenous p75NTR binding protein and returning normal neurotrophin function. Importantly, unlike anti-NGF antibodies which ablate NGF signalling as they bind irreversibly to NGF, LEVI-04 provides analgesia while maintaining the neurotrophin function required for cartilage and bone repair.

Phase I data on LEVI-04 demonstrated a favourable safety, tolerability and pharmacokinetic profile. Analysis of biomarkers indicated target engagement and provided early supporting evidence of efficacy / proof-of-concept.

Dr Philip Conaghan, Principal Investigator said, “Safe and effective pain management is a critical need in arthritis with existing treatments limited by addiction-liabilities, adverse effects and poor efficacy. While the potential of the neurotrophin pathway to provide effective pain relief is recognised, safety issues have significantly hampered development. LEVI-04 offers a highly promising new approach to safely harness the analgesic potential of neurotrophin modulation and we look forward to the trial results next year.”

Simon Westbrook, founder and CSO of Levicept, and study director said, “The fact we completed recruitment in this large-scale Phase II in under a year is testament to physicians’ interest in our programme and patients’ pressing need for new treatment options for their chronic pain.”

LEVI-04 was originally discovered at Pfizer UK by Levicept’s founder, Simon Westbrook who subsequently acquired the asset. Levicept recently announced the appointment of experienced biotech executive Eliot Forster as CEO to help steer the best path for the further development of LEVI-04 and its ultimate commercialisation.
ENDS

Levicept
Eliot Forster, CEO – info@levicept.com

Media Enquiries
Charles Consultants
Sue Charles – Sue@charles-consultants.com +44 (0)7968 726585
Chris Gardner – Chris@CGComms.onmicrosoft.com +44 (0)7956 031077

About Levicept – www.levicept.com

Levicept Ltd is a UK-based biotechnology company developing a novel, safe and efficacious biological therapy (LEVI-04 [p75NTR-Fc]) for the treatment of chronic pain. LEVI-04 is currently in phase II in patients with osteoarthritis of the knee. LEVI-04 modulates the neurotrophin pathway, clinically proven to provide effective analgesia in a range of indications and is designed to minimise use-limiting side effects of anti-NGF antibodies, including rapid progression of osteoarthritis. It is estimated that the market opportunity for drugs that modulate neurotrophins, developed to a high safety standard, is worth in excess of $10 billion. LEVI-04 was originally discovered at Pfizer by Levicept’s founder, Simon Westbrook who subsequently acquired the asset. Levicept’s investors include Medicxi, Advent Life Sciences, Gilde Healthcare and Pfizer Ventures.

Follow us on LinkedIn – https://www.linkedin.com/company/levicept-ltd

Dec 07
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Aura Biosciences Announces First Patient Dosed in Global Phase 3 CoMpass Trial

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Aura Biosciences Announces First Patient Dosed in Global Phase 3 CoMpass Trial Evaluating the Safety and Efficacy of Belzupacap Sarotalocan (Bel-sar) for First-Line Treatment of Early-Stage Choroidal Melanoma

 

BOSTON–(BUSINESS WIRE)– Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the first patient has been dosed in the global Phase 3 CoMpass trial evaluating the safety and efficacy of bel-sar for the first-line treatment of adult patients with early-stage choroidal melanoma.

“Dosing of the first patient in this global Phase 3 trial is a major milestone for Aura, and for patients with early-stage choroidal melanoma, a life-threatening rare disease with no approved targeted therapies,” said Elisabet de los Pinos, Ph.D., Chief Executive Officer of Aura. “With the SPA agreement from the FDA, and a strong endorsement from the ocular oncology community, we continue to move bel-sar closer to potentially being approved as a first-line vision preserving treatment for patients living with this disease.”

The CoMpass trial is designed as a superiority trial comparing bel-sar versus a sham control. The trial is a global Phase 3, randomized, multi-center, masked study, intended to enroll approximately 100 patients randomized 2:1:2 to receive high dose regimen of bel-sar, low dose regimen of bel-sar with suprachoroidal (SC) administration or a sham control. Aura received written agreement from the U.S. Food and Drug Administration (FDA) under a Special Protocol Assessment (SPA) for the overall design of the CoMpass trial.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing VDCs, a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting, anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura is enrolling patients in the global Phase 3 trial evaluating first-line treatment of early-stage choroidal melanoma, a vision- and life-threatening form of eye cancer where the standard of care with radiotherapy leaves patients with severe comorbidities, including significant vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive and muscle invasive bladder cancer. Aura is headquartered in Boston, MA.

For more information, visit aurabiosciences.com, or follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, choroidal metastasis, muscle invasive bladder cancer and non-muscle invasive bladder cancer; any express or implied statements regarding Aura’s expectations for the Phase 3 clinical trial of bel-sar for early-stage choroidal melanoma; and the potential approvability of bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

View source version on businesswire.com: https://www.businesswire.com/news/home/20231207348086/en/

Contacts

Investor and Media Contact:

Alex Dasalla
Head of Investor Relations and Corporate Communications
adasalla@aurabiosciences.com

Nov 30
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Levicept Appoints Eliot Forster as CEO

By Levicept, Press Release, Private Companies
Press Release.

 

Levicept Appoints Eliot Forster as CEO

Leadership Team Strengthened as Clinical Development of LEVI-04 a Novel Neurotrophin Modulator for Osteoarthritis and Chronic Pain Advances

Sandwich, UK – 30 November 2023 – Levicept Ltd, a biotechnology company focused on the development of LEVI-04, a first-in-class treatment for chronic pain indications, today announces the appointment of Eliot Forster as CEO. Founder and inventor of LEVI-04, Simon Westbrook, is to take the role of CSO.
Eliot brings more than thirty years of experience in the biotech and pharmaceutical sectors, with considerable experience in deal making and finance. His appointment strengthens the Levicept leadership team as the company matures through clinical development of LEVI-04 towards the future advancement and its ultimate commercialisation.

LEVI-04 modulates the neurotrophin pathway, that is clinically proven to provide effective analgesia in a range of indications and is designed to minimise use-limiting side effects of anti-NGF antibodies, including rapid progression of osteoarthritis. Having successfully completed a phase I trial in 2021, Levicept is advancing LEVI-04, a proprietary p75 neurotrophin receptor fusion protein (p75NTR-Fc), a once-a-month injectable for the treatment of osteoarthritis and chronic pain, through a 500+ patient phase II clinical trial.

Eliot was most recently CEO of F-star (NASDAQ:FSTX), a clinical-stage, NASDAQ-listed biotech that was acquired by inovX Pharma limited in March 2023. Prior to this he was CEO of Immunocore (NASDAQ:IMCR), and previously led the clinical development of several pain medicines, including the COX-2 inhibitors. His early career was at GSK and Pfizer. Alongside his role at Levicept, currently he serves on the Boards of Tessellate BIO, Avacta Group (AIM:AVCT), Ochre Bio, Protalix Biotherapeutics (AMEX:PLX), and Immatics NV (NASDAQ:IMTX).

Eliot Forster, CEO of Levicept, said, “The safe and effective management of chronic pain remains a critical need in medicine. While existing treatments can be effective their use is often limited by adverse effects, addiction-liabilities, or poor pain control. LEVI-04 has the potential to effectively target the clinically proven neurotrophin pain pathways, but with a superior safety profile. I very much look forward to working with Simon and his team to advance this important programme to its completion.”

Kevin Johnson, Chairman of Levicept and Partner at investor Medicxi said, “As a highly focused single asset company, Levicept has made excellent progress with LEVI-04 though phase I and ongoing phase II studies. With data to date looking very promising, I am delighted to welcome Eliot to the company at an important time for the company, to work with Simon and the team to help steer the best path for the further development of LEVI-04.”

Simon Westbrook, founder and CSO of Levicept said, “Eliot’s appointment underlines the potential we see in LEVI-04 and the near-term opportunities on the back of the pre-clinical and clinical work we have undertaken. With our phase II trial nearing completion, we can now benefit from Eliot’s experience and networks to ultimately maximise the chances of LEVI-04 reaching patients and offering a new, safe treatment for chronic pain.”

It is estimated that the market opportunity for drugs that modulate neurotrophins, developed to a high safety standard, will be worth in excess of $10 billion by 2033.

Levicept
Eliot Forester, CEO – info@levicept.com

Media Enquiries
Charles Consultants
Sue Charles – Sue@charles-consultants.com +44 (0)7968 726585
Chris Gardner – Chris@CGComms.onmicrosoft.com +44 (0)7956 031077

About Levicept – www.levicept.com

Levicept Ltd is a UK-based biotechnology company developing a novel, safe and efficacious biological therapy (LEVI-04 [p75NTR-Fc]) for the treatment of chronic pain. LEVI-04 is currently in phase II in patients with osteoarthritis of the knee. LEVI-04 modulates the neurotrophin pathway, clinically proven to provide effective analgesia in a range of indications and is designed to minimise use-limiting side effects of anti-NGF antibodies, including rapid progression of osteoarthritis. It is estimated that the market opportunity for drugs that modulate neurotrophins, developed to a high safety standard, will be worth in excess of $10 billion by 2033. LEVI-04 was originally discovered at Pfizer by Levicept’s
founder, Simon Westbrook who subsequently acquired the asset. Levicept’s investors include Medicxi, Advent Life Sciences, Gilde Healthcare and Pfizer Ventures.

Follow us on LinkedIn – https://www.linkedin.com/company/levicept-ltd

Nov 10
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Artax Granted Clinical Trial Authorization for AX-158 Phase 2a Psoriasis Study

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

 

Patient Enrollment Expected to Begin by Year End

Phase 2a Study Seeks to Demonstrate the Safety and Efficacy of AX-158 in Patients with Mild-to-Moderate Psoriasis, Further Advancing a New Approach to Address Autoimmune Diseases

Globally Nearly 125 Million People Live with Psoriasis

CAMBRIDGE, Mass., Nov. 9, 2023 /PRNewswire/ — Artax Biopharma, Inc., a clinical-stage biotechnology company focused on transforming the treatment of T Cell-mediated diseases, today announces that the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) has granted clinical trial authorization (CTA) to evaluate AX-158 in a Phase 2a psoriasis study.

This authorization marks the initiation of the clinical trial process, allowing initial patient enrollment by the end of 2023.  The clinical trial sites are located throughout the United Kingdom.

The study, A Phase 2a, Proof of Concept Study Evaluating the Safety and Tolerability of AX-158 in Patients With Mild to Moderate Plaque Psoriasis (NCT-05725057) will evaluate the safety and efficacy of AX-158 versus placebo.

“We are proud to announce the clinical progress of AX-158 and the potential that it may provide a novel treatment to patients managing psoriasis,” stated Artax Biopharma Chief Executive Officer Joseph Lobacki. “Additionally, we are excited by the promise of AX-158 as a new standard of care in treating a broad range of autoimmune diseases.  In particular, we believe that AX-158 will offer the opportunity to provide a convenient once daily oral therapy that will treat disease while not impairing the ability of the patient’s immune system to function properly.”

Prof. Menno de Rie, MD, Department of Dermatology, Amsterdam UMC, The Netherlands, and an Artax Scientific Advisory Board member, commented: “As a physician and as a researcher treating psoriasis for decades, I can attest to the dire need for safe, effective, and tolerable therapies that will truly help patients manage their disease. I am excited about the innovation and tremendous science AX-158 represents for the many people with psoriasis. Managing autoimmune disease without inducing the profound immunosuppression associated with current conventional or biological therapies will be transformative for the immunology field.”

About AX-158
Immunomodulation assists the immune system in maintaining healthy control and addresses the underlying driver of T Cell-mediated diseases. AX-158 is an investigational oral, small molecule immunomodulator with a novel first-in-class mechanism that selectively modulates, or adjusts, T cell responses which play a critical role in healthy immune system function. By acting at the T Cell Receptor (TCR), AX-158 modulates multiple disease-causing cytokines without immunosuppression. It can be taken with or without food, and may be used as a monotherapy, or in combination.

About Psoriasis
Psoriasis is a common, immune-mediated disease that affects 125 million people globally.  In the US 3.0% of the adult population, or more than 7.5 million adults, are affected.  Psoriasis patients may exhibit visible signs of inflammation anywhere on the body as raised plaques and scales on the skin. Plaques may appear as few small patches or can affect large areas. It is possible to have psoriasis plaques and scales on more than one location on the body at a time.  Treating psoriasis can help improve symptoms as well as lower the risk of developing other health conditions such as psoriatic arthritis, heart disease, obesity, diabetes, and depression.

About Artax Biopharma and Immunomodulation
Artax Biopharma is a clinical-stage biotechnology company transforming the treatment of T Cell-driven autoimmune diseases through innovative small molecules delivering immune system modulation without immunosuppression. Immunomodulation maintains healthy control of the immune system and addresses the underlying source of T Cell-driven diseases. Central to a well-functioning immune system is the T Cell Receptor (TCR).  When TCR signaling becomes dysregulated, it causes T Cell-driven conditions, including autoimmune diseases, and induced T Cell pathologies where medical treatments result in immune reactions (such as stem cell transplants resulting in acute graft-versus-host-disease or immuno-oncology treatments resulting in immune related-adverse events). We believe the immunomodulation mechanism offered by our investigational agents holds broad potential to revolutionize how these T Cell-driven autoimmune diseases are addressed, while not impairing the ability of a patient’s immune system to function properly. artaxbiopharma.com

For Phase 2a study inquiries, please contact: ax-158-1011@artaxbiopharma.com

Nov 06
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AviadoBio Announces FDA IND Clearance and Fast Track Designation for AVB-101 for the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio Announces FDA IND Clearance and Fast Track Designation for AVB-101 for the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations

• AVB-101 is an investigational gene therapy delivered as a one-time infusion directly to the brain, enabling distribution to brain areas affected by FTD while limiting systemic exposure

• The Phase 1/2 ASPIRE-FTD global clinical trial is currently open in Europe and will open in the U.S. in 2024

• FTD is a leading cause of dementia in people under the age of 65

London, UK; November 6, 2023 — AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AVB-101 to be studied in people with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene. In addition, the FDA has granted Fast Track designation to AVB-101 for the treatment of FTD-GRN to slow disease progression. Fast Track is a process designed to facilitate the development and expedite the review of new drugs to treat serious conditions and fill an unmet medical need. Fast Track designation is intended to bring new medicines to patients earlier.

AVB-101 has been designed as a potential one-time therapy to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to areas of the brain affected by FTD. Both the U.S. FDA and the European Commission (EC) granted orphan designation to AVB-101 for the treatment of FTD in 2022. The company recently announced enrollment is open in European countries for ASPIRE-FTD, an open-label, multi-center dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN.

“The IND clearance and Fast Track designation of AVB-101 are important milestones for the FTD community and for our company. Sadly, there are currently no disease-modifying therapies approved for the treatment of people living with frontotemporal dementia with progranulin mutations. Receiving Fast Track designation underscores the significant need for treatment options for these patients, and we are eager to soon open U.S. clinical trial sites and offer an innovative option for eligible patients with FTD-GRN,” said Lisa Deschamps, Chief Executive Officer.

“We are using adeno-associated virus (AAV) technology and taking a novel approach to deliver a functional copy of the GRN gene directly to the brain via bilateral, MRI-guided intrathalamic infusion with the aim to restore normal levels of the progranulin protein,” said David Cooper, M.D., Chief Medical Officer. “Our preclinical program shows robust biodistribution to the brain areas where it is needed to restore levels of progranulin, potentially slowing or stopping the progression of FTD-GRN, with little to no progranulin in the rest of the body where it may have adverse effects.”

FTD is a devastating form of early-onset dementia that typically leads to death within seven to 13 years of symptom onset and three to 10 years from diagnosis.¹,² People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.³ FTD is a leading cause of dementia in people under the age of 65⁴ with an estimated prevalence at any one time of up to 4.6 cases per 1,000 of people.⁵ Genetic FTD cases account for about one-third of cases and are most frequently associated with autosomal dominant mutations in three genes, including the progranulin gene.⁶

 

1. Onyike CU. Neuroepidemiology. 2011;37:166–167;
2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310;
3. Pressman P and Miller BL. Biol Psychiatry. 2014;75(7):574–581;
4. Hendriks S, Peetoom K, Bakker C, et al. Global Prevalence of Young-Onset Dementia: A Systematic Review and Meta-analysis. JAMA Neurol. 2021;78(9):1080–1090. doi:10.1001/jamaneurol.2021.216;
5. Hogan DB et al. Can J Neurol Sci. 2016;43 (Suppl 1):S96–S109;
6. Young JJ et al. Ther Adv Psychopharmacol. 2018;8(1):33–48.

ABOUT ASPIRE-FTD

ASPIRE-FTD is an open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, AVB-101 will be delivered as a one-time treatment into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.

More information about the ASPIRE-FTD study can be found at https://clinicaltrials.gov/study/NCT06064890.

 

ABOUT AVB-101

An experimental gene therapy, AVB-101, contains a correct (non-mutated) version of the GRN gene. It is designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. AVB-101 will be delivered as a ‘one-time dose’ directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center.

 

ABOUT AVIADOBIO

AviadoBio’s mission is to develop and deliver potentially transformative gene therapies for people living with devastating neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working relentlessly to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the Company for success in bringing transformative medicines to patients.

AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Lifesciences, and LifeArc.

For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.

LEARN MORE ABOUT US

CONTACT

FOR MEDIA ENQUIRIES

Farah Speer
SVP, Head of Communications and External Relations
Fspeer@aviadobio.com
+1-312-543-2881

Jenna Kane
Health+Commerce
jennakane@healthandcommerce.com
+1-480-388-9587

Nov 06
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Aura Biosciences Receives FDA Agreement for CoMpass Phase 3 Clinical Trial

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Aura Biosciences Receives FDA Agreement Under Special Protocol Assessment (SPA) for CoMpass Phase 3 Clinical Trial of Belzupacap Sarotalocan (Bel-sar) in Early-stage Choroidal Melanoma
November 6, 2023

Positive Clinical Efficacy Updates of Bel-sar for Early-Stage Choroidal Melanoma from the Ongoing Phase 2 Clinical Trial with Suprachoroidal Administration Presented at AAO 2023

Preliminary Data from Phase 1 Trial in Bladder Cancer – First Patient Utilizing a Single Dose of Bel-sar with Light Activation Demonstrated a Clinical Complete Response

BOSTON–(BUSINESS WIRE)–Nov. 6, 2023– Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced it has received agreement from the U.S. Food and Drug Administration (FDA) under an SPA for the design and planned analysis of CoMpass, the Company’s global Phase 3 clinical trial of bel-sar for the first-line treatment of adult patients with early-stage choroidal melanoma (CM). The Company also announced the presentation of positive Phase 2 safety and efficacy data of bel-sar with 90% of patients at twelve months of follow-up evaluating two key clinical endpoints: tumor control and visual acuity preservation using suprachoroidal (SC) route of administration for the first-line treatment of adult patients with early-stage CM. The results were presented at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting in San Francisco, California.

“This written agreement from the FDA is consistent with our regulatory strategy and reaffirms that the design and planned analysis of the CoMpass Phase 3 clinical trial, if successful, adequately address the objectives necessary to support a biologics license application submission for bel-sar for the treatment of early-stage CM,” said Dr. Jill Hopkins, Chief Medical Officer and President of R&D of Aura Biosciences. “We are excited with the momentum in the ocular oncology community to participate in what would be the first ever registration-enabling clinical trial for early-stage CM.”

“The Phase 2 data presented today, with 90% of patients at twelve months follow-up, show results that are highly consistent with and strongly support the assumptions for the design of the CoMpass trial. We have observed 80% tumor control and 90% visual acuity preservation for patients that have been treated with the therapeutic regimen of bel-sar and meet the Phase 3 enrollment criteria. The safety profile continues to be favorable with no treatment-related serious adverse events or significant adverse events. This is very encouraging as most of these patients had tumors close to the fovea or optic disk and would have likely experienced severe and irreversible vision loss with the current standard of care with radiotherapy,” said Dr. Carol Shields, Chief of the Ocular Oncology Service at Wills Eye Hospital and Professor of Ophthalmology at Thomas Jefferson University (USA). “We are excited for the possibility to have for the first time a vision preserving therapy, expanding the possibility to treat patients earlier than we do today.”

Agreement from the FDA Under an SPA for the Global Phase 3 CoMpass Trial:

The Company received written agreement from the FDA under an SPA for the design and planned analysis of the Global Phase 3 CoMpass trial indicating concurrence by the FDA with the adequacy of the study, if successful, to address the objectives necessary to support the Company’s planned biologics license application submission. The Phase 3 trial is designed as a superiority trial comparing bel-sar versus sham. The trial is a global, multi-center, masked study, and it is intended to enroll approximately 100 patients randomized 2:1:2 to receive high dose regimen of bel-sar with SC administration, low dose regimen of bel-sar with SC administration, or a sham control. The primary endpoint is time to tumor progression, and the first key secondary endpoint is a composite time to event analysis that will compare the tumor control and visual acuity of the bel-sar high dose regimen to sham when the last patient completes their 15 months of follow up. The trial is powered at greater than 90%. The Company is on track to dose the first patient in Q4 2023.

Updated Safety and Efficacy Data from the Phase 2 Trial with SC Administration

The Phase 2 trial (NCT04417530) is assessing the safety and preliminary efficacy of single- and multiple ascending-doses of bel-sar up to three cycles of treatment via SC administration for the first-line treatment of early-stage CM. A total of 22 adult patients have been enrolled in the trial including the single dose Cohorts 1-3 (n=6) and multiple dose escalation Cohorts 4-6 (n=16). Cohorts 5 and 6 (n=13) received up to three cycles of therapy, which was considered the therapeutic regimen for evaluation. One patient in Cohort 5 (n=3) received two cycles of therapy and two patients in Cohort 5 received three cycles of therapy (40 μg/dose). All patients from Cohort 6 (n=10) were assigned to receive three cycles of therapy at the highest dose (80 μg/dose). One patient from Cohort 6, who discontinued after one cycle due to unrelated serious adverse events (SAEs), is not included in the analysis. All patients in Cohorts 5 and 6 had active tumor growth at study entry, as an enrichment strategy to evaluate preliminary efficacy. This group of patients with active growth treated at the therapeutic regimen of three cycles was evaluated for tumor control, visual acuity preservation and tumor growth rate as the defined clinical endpoints to evaluate preliminary efficacy. The results, with 90% of patients at twelve months of follow-up who received three cycles of therapy in Cohorts 5 and 6, and who match the criteria for the global Phase 3 trial, showed a tumor control rate of 80% (8/10) and the visual acuity preservation rate was 90% (9/10). The majority of patients being at high-risk for vision loss with tumors close to the fovea or optic disk. For the 80% of patients that responded, data showed a statistically significant reduction in tumor growth rate (-0.382 mm/yr, p = <0.0001) compared to each patient’s documented growth rate at study entry. The overall tolerability profile of bel-sar was favorable, with no dose-limiting toxicities, treatment-related SAEs or significant AEs reported as of August 3, 2023. There was no posterior inflammation and only mild anterior inflammation (Grade 1) in ~18% of the patients which was self-limited or resolved with a short course of topical steroids. Treatment-related AEs were predominantly mild and resolved without sequalae. We believe these updated results indicate that bel-sar may offer a targeted, vision preserving therapy for the first-line treatment of primary CM, where 80% of patients are diagnosed at an early stage and have no approved therapies to date.

The presentation can be accessed on the Company’s website here.

Preliminary Data from Phase 1 Trial in Bladder Cancer

The trial has completed enrollment of the cohort that received bel-sar injection without light activation. Protocol mandated safety review found no safety issues and the study has proceeded to the bel-sar injection plus light activation cohorts. Preliminary data from the first patient in the light activated cohort of the trial, utilizing a single dose of bel-sar with light activation, demonstrated a clinical complete response demonstrated by absence of cancer cells on histopathology with evidence of extensive necrosis and immune activation.

Bel-sar is a novel investigational agent designed with a dual mechanism of action that includes targeted cytotoxicity and immune activation. The ongoing Phase 1 multi-center, open-label clinical trial is expected to enroll approximately 19 adult patients. The trial is designed to assess the safety and tolerability of bel-sar as a single agent. The trial will provide histopathological evaluation after the local treatment to assess bel-sar’s biological activity. In addition, the FDA has allowed an amendment to the protocol of the Company’s ongoing Phase 1 trial evaluating bel-sar, allowing the inclusion of adult patients with muscle invasive bladder cancer in addition to non-muscle invasive bladder cancer. The Company expects to provide more data mid-2024.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing VDCs, a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting, anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura has initiated activities for the global Phase 3 trial evaluating first-line treatment of early-stage choroidal melanoma, a vision- and life-threatening form of eye cancer where the standard of care with radiotherapy leaves patients with severe comorbidities, including significant vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive and muscle invasive bladder cancer. Aura is headquartered in Boston, MA.

For more information, visit aurabiosciences.com, or follow us on Twitter and LinkedIn

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, muscle invasive bladder cancer, non-muscle invasive bladder cancer and choroidal metastasis; any express or implied statements regarding the Company’s expectations for the Phase 2 and Phase 3 clinical trials of bel-sar for early-stage choroidal melanoma and the Phase 1 trial of bel-sar for muscle invasive bladder cancer and non-muscle invasive bladder cancer; and the potential approvability of bel-sar; the Phase 2 trial of bel-sar for choroidal metastasis.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 SPA agreement with FDA; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

ACTIVE/125869873.9

Investor and Media Contact:
Alex Dasalla
Head of Investor Relations and Corporate Communications
adasalla@aurabiosciences.com

Oct 10
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AviadoBio Announces Initiation of Phase 1/2 Clinical Trial

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio Announces Initiation of Phase 1/2 Clinical Trial, ASPIRE-FTD,

Evaluating AVB-101 in People with Frontotemporal Dementia with GRN Mutations

 

  • AVB-101 is an investigational gene therapy designed to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels in the brain

 

  • AVB-101 is delivered as a one-time treatment into the thalamus to facilitate targeted biodistribution in order to reach broad areas of the brain affected by FTD, while limiting treatment exposure to only the brain itself

 

  • FTD is a leading cause of dementia in people under the age of 65

 

London, UK; October 10, 2023 — AviadoBio, a pioneering gene therapy company developing and delivering transformative medicines for neurodegenerative disorders, today announced that it has initiated the Phase 1/2 ASPIRE-FTD clinical study of its investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene.

 

ASPIRE-FTD is an open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, AVB-101 will be delivered as a one-time treatment into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.

 

“FTD is a devastating form of early-onset dementia and there are currently no disease-modifying treatments available,” said Lisa Deschamps, CEO. “The initiation of our company’s first clinical trial demonstrates our commitment to developing and delivering potential transformative gene therapies for those living with neurodegenerative disorders such as FTD.”

 

“While it is known that progranulin protein supplementation is possible using gene therapy, effective brain distribution remains challenging primarily because of the brain’s anatomy,” said David Cooper, M.D., Chief Medical Officer. “Our aim is to use intrathalamic delivery to facilitate biodistribution of the PGRN protein to the areas of the cortex impacted by FTD. AVB-101 has already shown great promise in preclinical studies, and we now look forward to this important next step in its clinical development.”

 

Preclinical safety and biodistribution data following intrathalamic delivery to non-human primates demonstrate good tolerability and widespread progranulin expression in brain tissues. These data will be presented at the 30th Annual European Society of Gene & Cell Therapy (ESGCT) meeting in Brussels, Belgium Oct. 24-27, 2023.

 

People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 has been designed as a potential one-time therapy to halt disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to the affected areas of the brain.

 

AVB-101 is delivered using a minimally-invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families. This targeted delivery method aims to limit the treatment to the brain where it is needed most, thereby reducing the dose required and the potential systemic exposure.

 

More information about the ASPIRE-FTD study can be found at https://clinicaltrials.gov/study/NCT06064890.

 

# # #

 

About Frontotemporal Dementia (FTD) and FTD with GRN mutations (FTD-GRN)

FTD is a devastating form of early-onset dementia that typically leads to death within 7 to 13 years of symptom onset and 3 to 10 years from diagnosis. People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.

 

FTD is a leading cause of dementia in people under the age of 65 with an estimated prevalence at any one time of up to 4.6 cases per 1,000 of people. FTD typically strikes younger than Alzheimer’s disease and the majority of FTD cases occur between 45 and 68 years of age. Given the early onset, FTD has a substantially greater impact on work, family, and finances than Alzheimer’s disease. Genetic FTD cases account for about one-third of cases and are associated with autosomal dominant mutations in three genes, including the GRN (progranulin) gene. Approximately 11,000 people in the U.S. and EU5 are living with FTD-GRN with approximately 2,200 new cases per year. Some FTD cases may be misidentified, and diagnostic delay is common. As disease education, genetics knowledge, and research and treatment options grow, these numbers are expected to increase.

 

About AviadoBio

AviadoBio’s mission is to develop and deliver potentially transformative gene therapies for people living with devastating neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working relentlessly to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the Company for success in bringing transformative medicines to patients.

 

AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Lifesciences, and LifeArc.

 

For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.

 

Media Contacts:

Farah Speer

SVP, Head of Communications and External Relations

Fspeer@aviadobio.com

+1-312-543-2881

 

Jenna Kane

Health+Commerce

jennakane@healthandcommerce.com

+1-480-388-9587

Sep 26
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Amphista Therapeutics Achieves First Milestone in Second Discovery Programme with Bristol Myers Squibb

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

 

PRESS RELEASE

 

Amphista Therapeutics Achieves First Milestone in Second Discovery Programme with Bristol Myers Squibb

 

Cambridge, UK, September 26th, 2023 – Amphista Therapeutics (“the Company” or “Amphista”), a leader in next generation targeted protein degradation (TPD) approaches, today announces it has achieved the first milestone in the second active discovery programme under its strategic collaboration and license agreement with Bristol Myers Squibb (NYSE: BMY), triggering a payment for achieving the milestone.

This  follows the announcement of the first milestone achieved in the first discovery programme  on May 4, 2023. Both programmes are part of the original agreement with Bristol Myers Squibb, which was announced in May 4, 2022 and included a $30 million upfront payment and the potential for up to $1.25 billion in performance-based milestone payments and payment for a limited expansion of the collaboration, as well as royalties on global net sales of products.

Louise Modis, Chief Scientific Officer of Amphista Therapeutics said: “The achievement of this first milestone in the second discovery programme under Amphista’s strategic collaboration with Bristol Myers Squibb is a testament to our innovative science and ability to discover and progress at pace differentiated, novel therapeutic molecules for our pharmaceutical partners. We have now delivered milestones on both active discovery programmes in our ongoing collaboration with the company and we look forward to continuing this strong momentum. Such collaborations further validate our unique approach and ambition to overcome challenges faced by other target protein degradation modalities.  With potential for CNS penetration, we continue to expand the range of potential applications for Amphista’s therapeutic modality.”

Under the agreement, Amphista is responsible for the discovery and development of small molecule protein degraders using EclipsysTM, its next-generation TPD platform. Bristol Myers Squibb is granted a global exclusive license to the resulting degraders and will be responsible for further development and commercialization activities.

ENDS

 

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). They aim to address the challenges faced by earlier stage TPD research 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 laboratories 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, Eli Lilly & Company and The Dementia Discovery Fund. For more information, please visit: www.amphista.com  

 

For more information please contact:

Consilium Strategic Communications

Amber Fennell, Namrata Taak, Stella Lempidaki

Email: Amphista@consilium-comms.com

Tel: +44 (0)20 3709 5813

 

 

Aug 09
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Aleta Biotherapeutics Granted MHRA Clinical Trial Authorization for Biologic CAR T-Cell Engager ALETA-001

By Aleta Biotherapeutics, Press Release, Private Companies
Press Release.

 

Aleta Biotherapeutics Granted MHRA Clinical Trial Authorization for Biologic CAR T-Cell Engager ALETA-001

 

  • Aleta’s lead program, ALETA-001 has clinical support from collaborator Cancer Research UK, which will initiate the Phase 1/2 Clinical Trial during H2 2023

 

  • ALETA-001, previously granted a UK Innovation Passport, was developed to address the urgent unmet patient need of relapse after CD19 CAR T-Cell Cancer Treatment

 

 

NATICK, Mass., August 8, 2023 – Aleta Biotherapeutics (Aleta), a privately-held immuno-oncology company with a platform of CAR T-Cell Engagers (CTE) which enable cell therapies to improve outcomes for patients with cancer, announces that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted clinical trial authorization (CTA) to evaluate investigational biologic ALETA-001 in a Phase 1/2 clinical trial in the treatment of patients with B-cell malignancies who are relapsed/refractory to CD19 CAR T-cell therapy.

 

Aleta’s lead program, ALETA-001 has clinical support and funding from its collaboration partner, Cancer Research UK’s Centre for Drug Development, which has sponsored and will conduct the Phase 1/2 clinical trial.

 

“The authorization to clinically evaluate, in collaboration with Cancer Research UK, the potent activity of ALETA-001, our lead biologic CAR T-Cell Therapy Engager, marks important progress for the entire global oncology community – especially patients,” commented Paul Rennert, Ph.D., Chief Executive Officer and Chief Scientific Officer, Aleta Biotherapeutics. “Aleta’s CTEs act by transforming the expression of any cancer tumor cell to match the CAR T-cell therapies circulating in a patient’s blood, importantly restoring and increasing the effectiveness with which CAR T-Cells can kill cancer cells,” continued Dr. Rennert.

 

Dr. Nigel Blackburn, Cancer Research UK’s Director of Drug Development, stated,

“CAR-T cell therapy has been transformative for some cancer patients but there remains a critical need to ensure that this therapy is an option for all. ALETA-001 is working to address this treatment gap for people with blood cancers by advancing a potentially life-saving CAR-T cell therapy into the clinic. We are delighted to play a key role in this trial and look forward to seeing this treatment become available for more patients in the future.”

 

 

 

About CAR T-Cell Therapy Engager (CTE) ALETA-001

Aleta’s lead development program, ALETA-001, contains the CD19 target protein which is further linked to an CD20 antibody domain. ALETA-001 is designed to improve the effectiveness of CD19-directed CAR T therapies by increasing CD19 antigen density and restoring lost CD19 expression on the cancer cell. This allows CD19+/CD20+ cancer cells to be easily recognized and killed by CD19-directed CAR T-cells that were previously administered and are already circulating within a patient.

 

Aleta previously secured landmark clinical support and funding from Cancer Research UK for the ALETA-001 Phase I/II clinical trials, and ALETA-001 has received a UK Innovation Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). ILAP designation is granted to medicines that address life-threatening or seriously debilitating conditions, and where there exists a significant patient or public health need.

 

About Aleta Biotherapeutics

Aleta Therapeutics is pioneering a transformation of cancer treatment by enabling CAR-T cell cancer therapies to work more effectively. The Company’s unique portfolio of multi-antigen CAR-T Engagers (CTEs) are simple, potent biologic therapies designed to transform the expression of cancer tumor cells to match CAR-T cell therapies circulating in patient blood. These CTEs are being developed to produce deeper and more durable responses in patients receiving CAR-T cell therapies through increasing cancer target density, and preventing resistance and escape from therapy, thus increasing the speed and effectiveness with which CAR-T cells can kill cancer cells.

 

Aleta’s lead program, ALETA-001, is clinic-ready for the treatment of B-cell malignancies in patients who are relapsed/refractory to CD19-directed CAR T-cell therapy. Additional CTEs in the Aleta portfolio include:

  • ALETA-004: a preclinical, multi-antigen binding CTE designed to extend the cancer killing activity of CAR-T cells to treat advanced acute myeloid leukemia, and
  • ALETA-005: a discovery phase CTE that allows multiple myeloma tumors to present multiple antigen binding sites to B-cell maturation antigen (BCMA) CAR T-cells, stimulating optimal BCMA CAR-T cell killing of advanced multiple myeloma.

Aleta is further developing its CTE platform technology to address solid tumors including breast cancer, gastric cancer, and pediatric brain cancers.

 

About Cancer Research UK’s Centre for Drug Development 

Cancer Research UK has an impressive record of developing novel treatments for cancer. The Cancer Research UK Centre for Drug Development has been pioneering the development of new cancer treatments for 25 years, taking over 150 potential new anti-cancer agents into clinical trials in patients. It currently has a portfolio of 21 new anti-cancer agents in preclinical development, Phase I or early Phase II clinical trials. Six of these new agents have made it to market including temozolomide for brain cancer, abiraterone for prostate cancer and rucaparib for ovarian cancer. Two other drugs are in late development Phase III trials. www.cruk.org.uk/cdd

 

#  #  #

 

 

 

Contacts:

Media:

Linda Phelan Dyson, MPH

973-986-5973

lpdyson@verizon.net

Jun 02
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Artax Biopharma Announces Positive Phase 1 Results for AX-158, Company’s First-in-Class Oral Immunomodulator Developed to Treat T Cell-Mediated Diseases

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

 

Artax Biopharma Announces Positive Phase 1 Results for AX-158, Company’s First-in-Class Oral Immunomodulator Developed to Treat T Cell-Mediated Diseases

 

Phase 1 Findings Demonstrated that Investigational Agent AX-158 Was Well Tolerated at All Doses, with a Profile that Strongly Supports Further Clinical Development Efforts

AX-158 is Being Developed for Once Daily Oral Dosing to Address Autoimmune Diseases by Providing Immunomodulation without Immunosuppression

 

Cambridge, Mass., June 1, 2023 – Artax Biopharma, Inc., a clinical-stage biotechnology     company focused on transforming the treatment of T Cell-mediated diseases, today announces positive Phase 1 clinical trial results for AX-158, the Company’s first-in-class oral small molecule immunomodulator to enter clinical development to treat T Cell-mediated diseases.

 

The Phase 1 clinical trial findings demonstrated that AX-158:

  • had no related adverse events in the clinical trial;
  • was well tolerated at all doses, up to the maximum of 50mg QD in the single ascending dose cohorts and up to the maximum of 15mg QD for 10 days in the multiple ascending dose cohorts; and
  • had a consistent, predictable pharmacokinetic (PK) profile and bioavailability among healthy volunteers, which supports the use of AX-158 as a once daily oral dosing regimen, which can be dosed independently of food.

 

Importantly, these positive results support the further evaluation of AX-158 at a once daily dose of 10mg in a Phase 2 clinical trial. 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 through immunomodulation without the risk of immunosuppression.

 

“With these strong Phase 1 results suggesting AX-158’s clean safety profile, we are eager to further evaluate safety and efficacy in Phase 2 clinical studies, and to ultimately making AX-158 available to patients managing complex T Cell-driven conditions, including autoimmune diseases, and induced T Cell pathologies,” stated Artax Biopharma Chief Executive Officer Joseph Lobacki. “AX-158 has the potential   to positively impact health outcomes through rebalancing immune system function, while maintaining a strong response to foreign pathogens and infections – providing true immunomodulation without immunosuppression.”

 

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, and induced T Cell pathologies in which medical treatments result in immune reactions (such as stem cell transplants resulting in acute graft-versus-host- disease or immuno-oncology treatments resulting in immune related-adverse events). Artax believes immunomodulation – a mechanism through which our investigational agents assist in modulating the immune system to ameliorate 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 blocks the role of Nck in T Cells.  Immunomodulation assists the immune system to maintain healthy control and eliminates the underlying driver of 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 without causing immunosuppression. 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 immune- oncology 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:

Maria Nichol, DPhil, EPA, CPA

Vice President, Business Development

+44 7901 666564

mnichol@artaxbiopharma.com

Media:

Linda Phelan Dyson

+1 973-986-5873

lpdyson@verizon.net