Skip to main content
All Posts By

Site Content Team

Aleta Biotherapeutics and Cancer Research UK’s Centre for Drug Development Announce First Patient Dosed in ALETA-001 Phase 1/2 Clinical Trial

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

 

Aleta Biotherapeutics and Cancer Research UK’s Centre for Drug
Development Announce First Patient Dosed in ALETA-001 Phase 1/2 Clinical
Trial in Patients with Relapsed/Refractory B-Cell Malignancies

 

  •  The Phase 1/2 clinical trial will evaluate safety, dose, PK/PD, and early efficacy
    signals of Aleta’s first-in-class biologic CAR T-Cell Engager, ALETA-001
  • ALETA-001 entering the clinic is a critical milestone toward treatment availability to
    improve CAR T-cell patient response when a complete response is not achieved or
    maintained

NATICK, Mass., and LONDON, UK, February 21, 2024 – Aleta Biotherapeutics (Aleta),
a clinical stage, immuno-oncology company with a CAR T-Cell Engager (CTE) platform
that enables cell cancer therapies to work more effectively, and Cancer Research UK’s
Centre for Drug Development, today announce the first patient was dosed in a Phase 1/2
clinical trial. This trial is evaluating the Company’s first-in-class biologic CAR T-Cell
Engager, ALETA-001, for the treatment of patients with B-cell malignancies who are
relapsed/refractory to CD19-targeting CAR T-cell therapy.

Cancer Research UK’s Centre for Drug Development is sponsoring and conducting the Phase
1/2 clinical trial of ALETA-001, the lead agent in Aleta’s portfolio.

“It is very exciting and meaningful to have ALETA-001 now in the clinic. Clinical
evaluation of ALETA-001 is a key milestone toward a much-needed treatment option for the
many cancer patients whose CD19-targeted CAR T-cell therapies ultimately stop working.
ALETA-001 restores and increases the effectiveness with which CAR T-cells can kill cancer
cells, and we believe that it will enable more patients to successfully benefit from cell
therapies,” stated Dr. Paul Rennert, President and Chief Scientific Officer, Aleta
Biotherapeutics.

The ALETA-001 Phase 1/2 clinical trial is an open-label, dose-expansion trial which will
evaluate safety and tolerability, pharmacokinetic and pharmacodynamic effects, and early
signals of clinical efficacy of ALETA-001 as a single agent. Chief Investigator Dr. Sridhar
Chaganti is leading the trial from University Hospital Birmingham NHS Foundation Trust in
Birmingham, UK, and will enroll patients who are relapsed/refractory following treatment
with available CD19-targeting CAR T-cell therapies. For more information on this trial,
please visit clinicaltrials.gov (NCT06045910)

Dr. Lars Erwig, Cancer Research UK’s Director of Drug Development, said, “A
significant number of patients with blood cancers unfortunately relapse following CD19-
directed CAR T-cell therapy. ALETA-001 is being developed to provide patients with these
cancers a better chance for a successful treatment outcome. At Cancer Research UK, we are
very excited to study the clinical potential of ALETA-001 to transform a patient’s treatment
journey – which can possibly be lifesaving in many cases.”

“Aleta-001 is a uniquely designed new molecule with a novel mechanism of action, coating
tumor cells densely with the target antigen, thereby stimulating the tumor killing ability of
activated CAR T-cells,” commented Dr. Sridhar Chaganti, Chief Investigator, University
Hospital Birmingham NHS Foundation Trust, Birmingham, UK. “This is an important
new strategy being investigated to improve outcomes for patients with relapsed or refractory
lymphomas who experience disease progression after CAR T-cell therapy and have a poor
prognosis.”

About CAR T-Cell Therapy Engager (CTE) ALETA-001
Aleta’s lead clinical stage program, ALETA-001, was designed specifically for the treatment
of B-cell malignancies in patients who have received a CD19-directed CAR T-cell therapy
and are at risk of treatment failure. Developed to improve the effectiveness of CD19-directed
CAR T-cell therapies by increasing CD19 antigen density and restoring lost CD19
expression on the cancer cell, ALETA-001 contains the CD19 target protein which is further
linked to an CD20 antibody domain. 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 1/2 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 Biotherapeutics is pioneering a transformation of cancer treatment by enabling CAR Tcell
cancer therapies to work more effectively. The Company’s unique portfolio of multiantigen
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 clinical stage biologic, ALETA-001 was designed specifically for the treatment
of B-cell malignancies in patients who have received a CD19-directed CAR T-cell therapy
and are at risk of treatment failure. Aleta is further developing its CTE platform technology
to address acute myeloid leukemia, multiple myeloma, and solid tumors including breast
cancer, gastric cancer, and pediatric brain cancers. aletabio.com

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 30 years, taking over 160 potential new anticancer
agents into clinical trials in patients. It currently has a portfolio of over 20 new anticancer
agents in preclinical development, Phase 1, or early Phase 2a 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. 13 other drugs are still
undergoing active development. www.cruk.org.uk/cdd

Contacts:
Media:
Linda Phelan Dyson, MPH
Corporate Communications
+1 973-986-5973
lpdyson@verizon.net

Amphista Therapeutics announces new data demonstrating in vivo efficacy and CNS activity of its mechanistically differentiated targeted protein degraders

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

 

Amphista Therapeutics announces new data demonstrating in vivo efficacy and CNS activity of its mechanistically differentiated targeted protein degraders

 

  • First demonstration of efficacy in disease-relevant in vivo models for an orally delivered bifunctional non-cereblon / non-VHL-based protein degrader
  • Deep degradation of target protein is achieved in a rapid, sustained, and highly selective manner
  • Successful achievement of efficient CNS delivery for multiple degraders and demonstration of significant degradation of target protein in the brain

Cambridge, UK, January 24th, 2024 – Amphista Therapeutics (“the Company” or “Amphista”), a leader in next generation targeted protein degradation (TPD) approaches, today announces the achievement of two compelling new data sets with its next generation bifunctional degraders demonstrating in vivo efficacy and the ability to target and degrade proteins in the central nervous system (CNS).

Amphista’s degraders, which leverage a novel protein degrading mechanism, with advantages beyond first generation cereblon or VHL-based approaches, have demonstrated:

–  Sustained degradation of target protein and anti-tumor efficacy: once daily oral dosing led to statistically significant reduction in tumor burden and survival advantage in multiple in vivo disease models.

–  Rapid degradation of target protein: significant levels of degradation were induced rapidly after dosing.

Highly selective degradation of target protein: statistically significant degradation of target protein vs >8000 other proteins, including closely related homologs, when dosed at 100x DC50.

Amphista also discloses significant progress in the advancement of its technology for the treatment of neurodegenerative diseases, a key focus of the Company. These data show:

  • Amphista degraders can be rationally designed to achieve CNS penetrance with examples achieved across multiple targets.
  • Rapid degradation of target protein achieved in the brain in vivo (dog and non-human primate, and in multiple brain regions) when dosed intravenously.

Louise Modis, Chief Scientific Officer of Amphista Therapeutics, said: “We are delighted by the progress we have made in advancing our pipeline and demonstrating the potential of our unique TPD technology. To our knowledge, this is the first time an orally delivered bifunctional non-cereblon / non-VHL-based protein degrader has shown efficacy in disease-relevant in vivo models. Combined with our progress in demonstrating in vivo degradation of targets within the CNS, we are extremely excited to have achieved these key preclinical development milestones and we look forward to providing additional updates across our pipeline throughout

the year.”

Beverley Carr, Interim Chief Executive Officer of Amphista Therapeutics, said: “The last six months have been

transformative for Amphista and these new compelling in vivo data strengthen our belief that our technology has the potential to deliver differentiated protein degrader molecules with class-leading physicochemical properties, enabling us to target a wider tissue and indication scope than traditional protein degrader approaches. These data are testament to the strength of our scientific team and we are on track with our portfolio priorities, exploring the tremendous potential of our platform across multiple indications in CNS and

oncology.”

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

Amphista and the Amphista logo are all trademarks of Amphista Therapeutics Limited.

For more information please contact:

Amphista Therapeutics

Beverley Carr, Interim CEO info@amphista.com

 

ICR Consilium

Amber Fennell, Namrata Taak

Email: Amphista@consilium-comms.com

Tel: +44 (0)20 3709 5813

Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease

By Orphalan, Press Release, Private Companies
Press Release.

 

Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease

Paris, France 18 January, 2024 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the approval of its trientine tetrahydrochloride product by China’s National Medical Products Administration (NMPA). This product, already marketed as Cuprior® in the EU, the UK, Saudi Arabia, Switzerland, and Colombia, and as Cuvrior® in the US, represents the first trientine approved by the NMPA. It offers a new treatment option for Wilson disease in children aged five years and older and adults intolerant to penicillamine therapy.
China is home to an estimated 80,000 individuals with Wilson disease, of whom approximately half have their diagnosis confirmed, and 25,000 are receiving regular treatment. The introduction of new treatment alternatives such as Orphalan’s trientine tetrahydrochloride, particularly for patients who are intolerant to currently available therapies, presents a significant opportunity to meet the needs of a broader segment of the Wilson disease population in China. Orphalan anticipates its product to be available in the country in the coming months.

Dr Naseem Amin, Chief Executive Officer at Orphalan, said: “We are pleased with the marketing authorisation approval of our trientine tetrahydrochloride product in China, which offers a well-tolerated and effective treatment option for patients with Wilson disease. This approval is a crucial step towards providing a much-needed treatment option for the Wilson disease community in China and reaffirms our commitment to improving the experience for patients living with rare diseases worldwide.”

Yan Qin, Director of Wuhan Tongxin (Wilson disease patient group in China) added: “We were thrilled when we heard that NMPA approved Orphalan’s trientine tetrahydrochloride in China. It is a significant step for those of us living with Wilson disease, representing a much-needed alternative treatment option. This approval, a result of Orphalan’s patient-focused approach, offers us a chance to better manage our condition with a new, effective treatment.”

Wilson disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Early treatment with legacy agents such as D-penicillamine (DPA), trientine HCI, and zinc salts can effectively slow the progression of the disease, however these options may not be suitable for long-term care due to adverse events associated with DPA and the need for close monitoring during treatment with zinc salts and DPA.

About Orphalan
Orphalan is an international orphan drug development and commercialization company headquartered in Paris. Founded in 2011, the Company develops and deliver innovative therapies for people living with rare and debilitating diseases. Our trientine tetrahydrochloride product has been approved for the treatment of Wilson disease and is available in more than 20 countries, branded as Cuprior® in EU, the UK, Saudi Arabia, Switzerland and Columbia, and as Cuvrior® in the United States. For more information visit www.orphalan.com and follow us on LinkedIn.

For more information, please contact:
Orphalan:
Géraldine van den Broek, Head of Corporate & BD
Tel: +33 (0)1 42 49 82 64
info@orphalan.com
ICR Consilium:
Tracy Cheung, Sukaina Virji, Davide Salvi
Tel: +44 (0) 203 709 5700
orphalan@consilium-comms.com

Bayer’s elinzanetant meets all primary and key secondary endpoints in pivotal OASIS 1 and 2 Phase III studies

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

 

Bayer’s elinzanetant meets all primary and key secondary endpoints in pivotal OASIS 1 and 2 Phase III studies

 

OASIS 1 and 2 pivotal studies evaluating investigational compound elinzanetant met all primary endpoints demonstrating a statistically significant reduction in frequency and severity of moderate to severe vasomotor symptoms (VMS) compared to placebo in postmenopausal women / Both studies also showed superiority over placebo for all key secondary endpoints with a statistically significant reduction in frequency of VMS at week 1, as well as improvement of sleep disturbances and menopause-related quality of life / The safety profile observed in both studies is overall consistent with previously published data on elinzanetant / OASIS 1 and 2 are two of three Phase III clinical studies investigating the efficacy and safety of elinzanetant, a first dual neurokinin-1,3 (NK-1,3) receptor antagonist, as a non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily

Berlin, January 8, 2024 – Bayer today announced positive top-line results of the pivotal Phase III studies OASIS 1 and 2 evaluating the efficacy and safety of the investigational compound elinzanetant versus placebo. Elinzanetant successfully met all four primary endpoints in both studies demonstrating statistically significant reductions in the frequency and severity of moderate to severe vasomotor symptoms (VMS, also known as hot flashes) from baseline to week 4 and 12 compared to placebo.

Both studies also achieved all three key secondary endpoints showing a statistically significant reduction in the frequency of VMS from baseline to week 1, as well as statistically significant improvements in sleep disturbances and menopause-related quality of life compared to placebo. The safety profile observed in the OASIS 1 and 2 studies is overall consistent with previously published data1,2 on elinzanetant.

“We are excited about the positive results of these two pivotal Phase III studies for elinzanetant, reinforcing its potential as a non-hormonal treatment option in menopause management,” said Dr. Christian Rommel, member of the Executive Committee of Bayer AG’s Pharmaceutical Division and Global Head of Research and Development. “We want to thank the women participating in the OASIS studies, their families, and all study investigators as well as their clinical and nursing staff for their time and commitment to advance menopause research.”

Elinzanetant is a first dual neurokinin-1,3 (NK-1,3) receptor antagonist, in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily.

“Menopausal symptoms such as hot flashes and sleep disturbances can be highly disruptive and broadly impact health and quality of life, still many women cope in silence and remain untreated,” said JoAnn Pinkerton, M.D., Professor and Director of Midlife Health at UVA Health. “It’s important that we continue to research for solutions that address the unmet needs of women, and I am looking forward to the unveiling of the full results.”

OASIS 1 and 2 (NCT05042362 and NCT05099159) are the first two Phase III studies in the OASIS clinical development program with results, and the details are planned to be presented at upcoming scientific congresses. The results of the third Phase III study OASIS 3 (NCT05030584) are expected in the coming months. Bayer plans to submit data from the OASIS 1, 2 and 3 studies to health authorities for approval of marketing authorizations for treatment of moderate to severe VMS associated with menopause.

About the OASIS 1 and 2 studies
OASIS 1 and 2 are double-blind, randomized, placebo-controlled multicenter studies investigating the efficacy and safety of elinzanetant administered orally once daily in women with moderate to severe VMS associated with menopause. OASIS 1 and 2 randomized 396 and 400 postmenopausal women between 40 and 65 years across 184 sites in 15 countries.

About the OASIS Clinical Development Program
The Phase III clinical development program of elinzanetant, OASIS, currently comprises four Phase III studies: OASIS 1, 2, 3 and 4. The OASIS 1, 2 and 3 studies investigate the efficacy and safety of elinzanetant 120 mg in women with moderate to severe VMS associated with menopause. The OASIS 4 study is an expansion of the clinical phase III program and investigates the efficacy and safety of elinzanetant in women with moderate to severe VMS caused by endocrine therapy for treatment or prevention of breast cancer.

The design and dosing of the Phase III clinical development program is based on the positive data from two Phase II studies (RELENT-1 and SWITCH-1). RELENT-1 was a Phase Ib/IIa study investigating the safety, pharmacokinetics and preliminary efficacy of elinzanetant. SWITCH-1 was a Phase IIb study investigating the efficacy and safety of four different doses of elinzanetant compared to placebo in women with VMS.

About Elinzanetant
Elinzanetant is a first dual neurokinin-1,3 (NK-1,3) receptor antagonist, in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily. Elinzanetant may address moderate to severe VMS by modulating a group of estrogen sensitive neurons in the hypothalamus region of the brain (the KNDy neurons) which, with the decrease of estrogen, become hypertrophic and lead to a hyperactivation of the thermoregulatory pathway, consequently disrupting body heat control mechanisms resulting in VMS. Elinzanetant may also decrease sleep disturbances associated with menopause.

About Vasomotor Symptoms
Vasomotor symptoms (VMS; also referred to as hot flashes) result from hyperactivation of the thermoregulatory pathway mediated by hypertrophy of the KNDy neurons. This is due to a decrease of estrogen, which can result from the progressive reduction of ovarian function due to natural menopause or medical intervention by bilateral oophorectomy or endocrine therapy.

VMS are reported by up to 80% of women at some point during the menopausal transition and are one of the leading causes for seeking medical attention during this phase of a woman’s life. Over one-third of menopausal women report severe symptoms, which can last 10 years or more after the last menstrual period, with relevant impact on quality of life.

VMS may also be caused by endocrine therapy, for the treatment or prevention of breast cancer, impacting quality of life and treatment adherence. For these women, there are currently no approved treatment options.

About Menopause
By 2030, the world population of women experiencing menopause is projected to increase to 1.2 billion, with 47 million new women entering this phase each year. Menopause is a transitional phase in women’s lives, related to the progressive decline of ovarian function, and which usually occurs in women during their 40s or early 50s. It can also be the result of surgical or medical treatment, for example breast cancer treatment. The hormonal decline can lead to various symptoms which can substantially affect a woman’s health, quality of life, healthcare utilization and work productivity. The most frequently reported and disruptive symptoms during the menopausal transition are VMS, sleep disturbances and mood changes. Addressing these symptoms is key to maintaining functional ability and quality of life in menopause which is highly relevant from both a healthcare and socio-economic perspective.

About Women’s Healthcare at Bayer
Women’s Health is in Bayer’s DNA and as a global leader in women’s healthcare Bayer has a long-standing commitment to delivering science for a better life by advancing a portfolio of innovative treatments. Bayer offers a wide range of effective short- and long-acting birth control methods as well as therapies for menopause management and gynecological diseases. Bayer is also focusing on innovative options to address the unmet medical needs of women worldwide and to broadening treatment choices such as in menopause. Additionally, Bayer intends to provide 100 million women per year in low-and-middle income countries by 2030 with access to family planning by funding multi-stakeholder aid programs for capacity building and by ensuring the supply of affordable modern contraceptives. This is part of the comprehensive sustainability measures and commitments from 2020 onwards and in line with the Sustainable Development Goals of the United Nations.

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com.

Find more information at https://pharma.bayer.com
Follow us on Facebook: http://www.facebook.com/bayer
Follow us on LinkedIn: Bayer | Pharmaceuticals

Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

References:

1 Simon JA, Anderson RA, Ballantyne E, Bolognese J, Caetano C, Joffe H, Kerr M, Panay N, Seitz C, Seymore S, Trower M, Zuurman L, Pawsey S. Efficacy and safety of elinzanetant, a selective neurokinin-1,3 receptor antagonist for vasomotor symptoms: a dose-finding clinical trial (SWITCH-1). Menopause. 2023 Mar 1;30(3):239-246.

2 Trower M, et al. Effects of NT-814, a dual neurokinin 1 and 3 receptor antagonist, on vasomotor symptoms in postmenopausal women: a placebo-controlled, randomized trial. Menopause: The Journal of The North American Menopause Society. 2020; 27 (5): 498-505.

Contact for global media inquiries:
Katja Wiggers, phone +49 30 221541614
Email: katja.wiggers@bayer.com

Contact for US media inquiries:
Courtney Ambrosi, phone 1 (908) 798-1107
Email: courtney.ambrosi@bayer.com

Contact for investor inquiries:
Bayer Investor Relations Team, phone +49 214 30-72704
Email: ir@bayer.com
www.bayer.com/en/investors/ir-team

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

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

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 Leviceptwww.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

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

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.

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

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