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Jul 25
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Beacon Therapeutics Appoints Lance Baldo, MD as Chief Executive Officer and Thomas Biancardi as Chief Financial Officer

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

 

CEO Dave Fellows named non-Executive Chairman of the Board

Executive appointments follow $170 million Series B fundraise

London, UK and Cambridge, Mass., July 24, 2024 – Beacon Therapeutics Holdings Limited (‘Beacon’ or ‘the Company’), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced the appointment of Lance Baldo, MD as Chief Executive Officer, effective August 12, 2024, and Thomas Biancardi as Chief Financial Officer, effective August 1, 2024.

Dr. Baldo brings more than 20 years of experience in biopharmaceuticals including the successful launch of two new indications and a new formulation for Lucentis while at Genentech. Most recently, he served as Chief Medical Officer at Freenome, an early cancer detection company, where he led the design and execution of the Company’s medical strategy to support its pipeline, from clinical trials through registration and commercialization. Prior to Freenome, Dr. Baldo was the Chief Medical Officer at Adaptive Biotechnologies, serving as a member of the senior leadership team through the transition from a private to publicly traded company. He has also held numerous roles within the Roche Group and its affiliates, including Senior Vice President and Head of U.S. Medical Affairs of Genentech and Franchise Head for Ophthalmology.

Dr. Baldo succeeds David Fellows, who has served as CEO since Syncona Limited’s acquisition of AGTC in November 2022. Mr. Fellows will assist during the transition and assume the role of non-Executive Chairman of the Board in January 2025. During his tenure, Mr. Fellows oversaw the launch of Beacon Therapeutics in June 2023, initiation of the Phase II DAWN trial and registrational VISTA trial for AGTC-501 for the treatment of X-Linked Retinitis Pigmentosa (XLRP), and the recent closing of Beacon’s $170 million Series B.

“I am honored and incredibly excited to join Beacon at this important juncture for the Company. Dave and the team have guided Beacon to the forefront of ophthalmic gene therapy with a robust clinical pipeline of candidates poised to meaningfully improve the treatment paradigms for patients with both rare and prevalent blinding diseases,” said Dr. Baldo. “With AGTC-501 in a registrational study, world class science and incredible financial backing, we are poised to make a run at a devastating inherited retinal disease.”

Beacon also appointed Thomas Biancardi as the Chief Financial Officer, who assumes the role from interim CFO Andrew Prosser, effective August 1, 2024. Mr. Biancardi is a biopharmaceutical industry veteran with over 25 years of financial and operational leadership experience, predominantly within ophthalmology. During his career, he has assisted numerous companies in raising capital, and establishing clinical and commercial operations. As one of the first employees of Ophthotech Corporation, he helped the Company evolve from a pre-clinical venture backed startup to a publicly traded biotech company. He also played a crucial role in the successful launch of the first pharmacologic treatment for macular degeneration at Eyetech Pharmaceuticals.

“I am excited to serve as Beacon’s CFO and collaborate with Lance, the Board and Beacon’s experienced team to support the Company’s mission to fight blinding diseases,” shared Mr.
Biancardi. “Beacon has a strong operational foundation and the partnership of a global investor syndicate; I am looking forward to supporting the Company’s growth and development through market entry.”

“We are fortunate to bring in two experienced executives who share our passion to develop treatments for sight-threatening diseases. The addition of Lance and Tom will accelerate Beacon’s growth as a leading ocular gene therapy company focused on bringing transformative gene therapies to patients,” said Mr. Fellows.

Beacon has raised approximately $290 million in funding to date. In July, Beacon announced a $170 million Series B fundraise following several clinical milestones, including the first patient dosed in the VISTA registrational trial for AGTC-501, the initiation of the Phase II DAWN trial and the presentation of positive 12-month interim results of the Phase 2 SKYLINE trial at the 47th Annual Macula Society Meeting demonstrating the precision, effectiveness and safety of Beacon’s lead development candidate, AGTC-501.

About Beacon Therapeutics

Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD).

Lead development candidate AGTC-501, is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

The Company is supported by funds from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences, and additional investors.

Find out more about Beacon Therapeutics at beacontx.com.

Contact:

info@beacontx.com

Media:

beacontherapeutics@edelman.com

Jul 03
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Beacon Therapeutics Raises $170 Million in Series B Funding to Advance Development of Ophthalmic Gene Therapies

By Press Release, Private Companies
Press Release.

 

  • Financing led by life sciences venture capital firm Forbion, alongside existing investors Syncona and Oxford Science Enterprises, as well as new investors TCGX and Advent Life Sciences
  • Round will accelerate the development of lead asset AGTC-501 and broader pipeline
  • Beacon appoints Dmitrij Hristodorov, Wouter Joustra, and Cariad Chester to Board of Directors; Dominic Schmidt joins as a Board Observer

London, UK and Cambridge, Mass., July 3, 2024 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a mission to save and restore the vision of patients with blinding retinal diseases, today announced it has raised $170 million in Series B funding.

Forbion led the financing, which included existing investors Syncona Limited, Oxford Science Enterprises and the University of Oxford, as well as initial investments from TCGX and Advent Life Sciences. The funds will be used to support the continued clinical development of Beacon’s lead asset, AGTC-501 for X-Linked Retinitis Pigmentosa (XLRP) and generate Phase 1/2 clinical trial data for the Company’s Dry Age-related Macular Degeneration (dAMD) program.

Beacon Therapeutics also appointed Dmitrij Hristodorov and Wouter Joustra, General Partners of Forbion, and Cariad Chester, Managing Partner of TCGX, to its Board of Directors. Dominic Schmidt, General Partner of Advent Life Sciences, joins as a Board Observer.

Beacon is focused on both orphan and prevalent diseases, including XLRP, a blinding orphan disease for which there is no available treatment, as well as dAMD. AGTC-501, the Company’s lead asset, is currently in a registrational clinical trial for the treatment of XLRP. AGTC-501 expresses the full length RPGR protein, thereby addressing all photoreceptor damage caused by XLRP, including both rod and cone loss.

David Fellows, Chief Executive Officer of Beacon Therapeutics, said, “We are focused on progressing our pipeline of ophthalmic gene therapies to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness. I am confident that along with the addition of Dmitrij Hristodorov, Wouter Joustra and Cariad Chester to the Beacon Board, these funds will support the ongoing development of our late-stage and pre-clinical pipeline and enable acceleration of the development of AGTC-501 as we progress through the clinic and toward commercialization.”

“Forbion is pleased to support Beacon Therapeutics at this critical juncture in the development of its pipeline of pioneering ophthalmic gene therapies. Beacon’s deep expertise and renowned management bench give us confidence in the plan to build a leading retinal gene therapy company,” shared Dmitrij Hristodorov, General Partner at Forbion.

Elisa Petris, Lead Partner at Syncona Investment Management Limited and Board Director of Beacon Therapeutics, said, “Syncona’s continued backing of Beacon Therapeutics is a testament to the Company’s proven leadership team and innovative approach to developing gene therapies for retinal diseases. This financing and the partnership of this high-quality syndicate will propel Beacon’s pipeline and enable the Company to advance its programs for both rare and prevalent ophthalmic diseases.”

Beacon has raised approximately $290 million in funding to date. This funding round follows several clinical milestones for the company, including the first patient dosed in the VISTA registrational trial for AGTC-501, the initiation of the Phase II DAWN trial and the presentation of positive 12-month interim
results of the Phase 2 SKYLINE trial at the 47th Annual Macula Society Meeting demonstrating the precision, effectiveness and safety of Beacon’s therapeutic interventions.

J.P. Morgan acted as sole placement agent to Beacon Therapeutics for this transaction.

About Beacon Therapeutics
Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP), as well as two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting cone-rod dystrophy (CRD), an inherited retinal disease.

Lead development candidate AGTC-501, is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

Find out more about Beacon Therapeutics at beacontx.com.

Contact:
info@beacontx.com

Media:
beacontherapeutics@edelman.com

May 24
Love0

Amphista Therapeutics unveils new differentiated mechanism of action for BRD9 degradation at 2024 Protein Degradation in Focus Symposium

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

 

Amphista Therapeutics unveils new differentiated mechanism of action for BRD9 degradation at 2024 Protein Degradation in Focus Symposium

  • First presentation of a novel mechanism of action for the degradation of BRD9 – completely differentiated from cereblon- or VHL-based PROTACs
  • Amphista’s bifunctional BRD9 degraders selectively induce BRD9 to DCAF16 proximity and serve as molecular glues, leading to strong and rapid degradation of BRD9
  • Discovery further underscores Amphista’s proprietary chemistry and high-quality science which is being applied to the discovery and development of additional targeted glues beyond BRD9

Cambridge, UK, May 23, 2024 – Amphista Therapeutics (“Amphista”), a leader in next generation targeted protein degradation (TPD) approaches, today announces the unveiling of a new mechanism of action for the degradation of BRD9, an emerging target in oncology, that is differentiated from cereblon- or VHL-based PROTACs, during an oral presentation at the 2024 Protein Degradation in Focus Symposium held in Dundee, UK.

This significant news for the TPD field builds upon an earlier announcement by Amphista of two compelling data sets demonstrating in vivo efficacy and central nervous system (CNS) activity of its mechanistically differentiated protein degraders. At today’s presentation titled “Degradation of BRD9 by a novel targeted glue”, Dr Andrea Testa, Scientific Co-Founder and Senior Director, Discovery Chemistry showed data from proteomic and genetic validation studies which demonstrate that Amphista’s bifunctional degraders induced degradation of BRD9 by target-assisted E3 ligase recruitment. This novel mechanism selectively induces the proximity of BRD9 to DCAF16 and serves as a molecular glue. DCAF16 is a cullin-RING E3 ligase which can facilitate degradation of proteins of interest via ternary complex formation. Induction of this complex leads to deep and rapid degradation of BRD9 in vivo.

Louise Modis, Chief Scientific Officer of Amphista Therapeutics, said: “The discovery of a novel mechanism for BRD9 degradation, differentiated from cereblon- or VHL-based PROTACs, is a testament to Amphista’s proprietary chemistry and know-how. Our ability to translate this novel approach into high-quality, drug-like compounds with oral bioavailability and activity in vivo is truly exciting. As we continue to spearhead our efforts to advance new TPD approaches, our goal remains clear – to expand the offering of TPD medicines beyond CRBN and VHL-based agents and bring effective treatments to patients in areas of high unmet need.”

In the results presented, Amphista’s sub-nanomolar BRD9 degraders showed activity in both solid and liquid cancer cell lines and demonstrated a high degree of selectivity over 8,000 proteins quantified by proteomics, including the closely related proteins BRD7 and BRD4. Notably, these compounds induced BRD9 degradation in a mouse xenograft model illustrating Amphista’s BRD9 degraders are orally bioavailable and active in vivo. This is the first-time in vivo degradation of BRD9 has been demonstrated via DCAF16, underscoring the potential therapeutic application of Amphista’s bifunctional degraders.

Building on this knowledge, Amphista is applying its proprietary scientific know-how and expertise to the development of targeted glues which induce degradation of different targets, expanding the opportunity beyond BRD9. Future announcements will provide details of additional pipeline targets as part of Amphista’s ambitions to develop a first- and/or best-in-class portfolio of degraders with leading physicochemical properties.

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases, including cancer and neurodegenerative disease, through the advancement of next generation targeted protein degradation (TPD) medicines. Amphista is applying its proprietary warhead chemistry and mechanistic know-how to generate bifunctional targeted glues with a differentiated mechanism and leading physicochemical properties. Its portfolio offers the potential for first- and/or best-in-class assets and expanding the offering of TPD medicines beyond CRBN and VHL-based agents. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s laboratories at the University of Dundee. Amphista 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, and also has strategic collaborations with Bristol Myers Squibb and Merck. For more information, please visit: www.amphista.com

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

 

For more information please contact:

ICR Consilium
Amber Fennell, Namrata Taak
Email: Amphista@consilium-comms.com
Tel: +44 (0)20 3709 5813

May 08
Love0

Ventus Medical announces the submission of a novel electronic nicotine replacement therapy in the UK

By Press Release, Private Companies, Ventus Medical
Press Release.

 

A novel NRT for quitting smoking and vaping has been submitted to the UK medicines regulator for approval

LIVERPOOL, UNITED KINGDOM, May 7, 2024 /EINPresswire.com/ — Ventus Medical LTD announces that it has submitted a UK Marketing Authorisation Application for ENHALE™, a next-generation smoking cessation and harm reduction product which will give tobacco product users a safer alternative to both smoking and vaping.

ENHALE™ features a certified medical device and easily replaceable cartridges with novel and proprietary electronic inhalation technology. ENHALE™ meets the rigorous safety standards required of a medicinal product.
• ENHALE™’s proprietary formulation and low-temperature vaporisation technology ensure no thermal degradants and safer nicotine delivery than traditional vapes, an important differentiator from every other product.
• Obtaining UK MHRA approval is the first step in enabling healthcare professionals to prescribe for the first time, an effective vape-like product that is regulated and certified to be safe.
• When approved, ENHALE™ will be available without prescription in the UK.

Commenting on this submission, Kaasim Mahmood, Chairman of Ventus Medical and General Partner at Advent Life Sciences, said, “Smoking remains the most preventable cause of premature death globally. There is an urgent need for regulated, safer alternatives to vaping products and cigarettes. Advent Life Sciences is proud to have supported Ventus Medical, a UK company, on its journey to this important milestone. ENHALE™ has been designed and developed following extensive consultation with physicians, healthcare professionals and smokers who require a medicinal alternative to tobacco products.”

David Lawson, co-founder of Ventus Medical, added, “After nine years of rigorous research and development, we have successfully developed aerosolization technology to the strictest standards meeting pharmaceutical and medical device requirements globally. Clinical data on ENHALE™ confirm it’s ability to reduce the urge to smoke by more than leading NRT’s. This is a great step forward for smoking cessation and tobacco harm reduction and will be the first innovation in NRT for decades.”

ENHALE™ features an intuitive, easy-to-use, certified medical device with replaceable single-dose nicotine cartridges. The product is unique in utilizing low-temperature vaporisation technology, which heats the nicotine formulation to much lower temperatures when compared to vape products. Operating at lower temperatures ensures that ENHALE™ can safely and effectively deliver nicotine without any thermal degradation or metals which are of particular concern in vaping products.

About Ventus Medical
Ventus Medical is dedicated to the development of inhalation technology and is a certified medical device manufacturer based in the UK. The founding team of Ventus Medical were responsible for the first and only approved medical vape product (e-VOKE), which was licensed in 2013 by the MHRA.

David Lawson
Ventus Medical LTD
+44 333 344 0201
david.lawson@ventusmedical.com

Apr 22
Love0

Centessa Pharmaceuticals Announces Open IND for ORX750; Proof-of-Concept Data in Sleep-Deprived Healthy Volunteers Planned for 2H 2024

By Advent Life Sciences, Press Release
Press Release.

 

Centessa Pharmaceuticals Announces Open IND for ORX750; Proof-of-Concept Data in Sleep-Deprived Healthy Volunteers Planned for 2H 2024
April 22, 2024

BOSTON and LONDON, April 22, 2024 (GLOBE NEWSWIRE) — Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company that aims to discover and develop medicines that are transformational for patients, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug application (IND) to initiate a Phase 1 first-in-human, clinical trial of ORX750 for the treatment of narcolepsy. ORX750 is an investigational, orally administered, highly potent and selective orexin receptor 2 (OX2R) agonist designed to directly target the underlying pathophysiology of orexin neuron loss in narcolepsy type 1 (NT1), with potential applicability to narcolepsy type 2 (NT2), idiopathic hypersomnia (IH), and other sleep-wake disorders with normal orexin levels.

The Phase 1 study will evaluate the safety, tolerability and pharmacokinetics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of ORX750 in healthy adult subjects. In parallel to the SAD, a cross-over pharmacodynamic (PD) assessment will be performed utilizing the Maintenance of Wakefulness Test (MWT) and Karolinska Sleepiness Scale (KSS) in acutely sleep-deprived healthy adult subjects which is intended to provide proof-of-concept data to enable dose selection for NT1, NT2 and IH indications. The study has a maximum exposure limit specified by the FDA which the Company believes significantly exceeds the predicted efficacious doses of ORX750 in indications associated with or without orexin loss; therefore, the Company does not expect this limit to affect any of the planned clinical development activities for ORX750. The Company expects to commence dosing of the Phase 1 study in healthy volunteers imminently, and proof-of-concept data are anticipated in the second half of 2024.

The Phase 1 study design of ORX750 includes SAD combined with PoC cohorts to assess PD effects of ORX750 by measuring sleep latency with the MWT and subjective sleepiness with the KSS in acutely sleep-deprived healthy subjects.

SAD combined with PoC cohorts to assess PD effects of ORX750 by measuring sleep latency with the MWT and KSS in acute sleep-deprived healthy subjects.

“This is a significant milestone for the development of our potential best-in-class OX2R agonist, ORX750, for the treatment of narcolepsy and other sleep-wake disorders,” said Saurabh Saha MD PhD, Chief Executive Officer of Centessa. “We are excited to begin executing what we believe is an elegant, adaptive Phase 1 study aimed at generating early proof-of-concept data for ORX750 in acutely sleep-deprived healthy volunteers in the second half of this year. We expect this study to enable dose selection for planned studies evaluating ORX750 in patients with NT1 and in patient populations with normal orexin levels, including NT2 and IH.”

About ORX750
ORX750 is an investigational, orally administered, highly potent and selective orexin receptor 2 (OX2R) agonist designed to directly target the underlying pathophysiology of orexin neuron loss in narcolepsy type 1 (NT1). ORX750 has been shown to potently activate the OX2R with an in vitro EC50 of 0.11 nM and 9,800-fold selectivity over the human orexin receptor (hOX1R). ORX750 is Centessa’s first orexin product candidate being developed for the treatment of narcolepsy with potential expansion into narcolepsy type 2 (NT2), idiopathic hypersomnia (IH) and other sleep-wake disorders.

About Centessa Pharmaceuticals
Centessa Pharmaceuticals plc is a clinical-stage pharmaceutical company that aims to discover and develop transformational medicines for patients. Our most advanced programs include a hemophilia program, an orexin agonist program for the treatment of narcolepsy and other sleep-wake disorders and an immuno-oncology program focused on our LockBody® technology platform. We operate with the conviction that each of our programs has the potential to change the current treatment paradigm and establish a new standard of care. For more information, visit www.centessa.com, which does not form part of this release.

Forward Looking Statements
This press release contains forward-looking statements. These statements may be identified by words such as “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue,” “ongoing,” “aim,” “seek,” and variations of these words or similar expressions that are intended to identify forward-looking statements. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements, including statements related to the Company’s ability to discover and develop transformational medicines for patients; the timing of commencement of new studies or clinical trials of ORX750 and other potential orexin agonist candidates; research and clinical development plans and the timing thereof; the Company’s ability to differentiate ORX750 and other potential orexin agonist candidates from other treatment options; the development and therapeutic potential of ORX750 and other potential orexin agonist candidates; predicted efficacious doses of ORX750; the Company’s ability to successfully conduct its clinical development of ORX750 below the maximum exposure limit set by the FDA or, in the event the Company plans to exceed the maximum exposure limit, the Company’s ability to successfully have the maximum exposure limit removed; and other regulatory matters, including the timing and likelihood of success of obtaining authorizations to initiate or continue clinical trials. Any forward-looking statements in this press release are based on our current expectations, estimates and projections only as of the date of this release and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks related to the safety and tolerability profile of our product candidates; our ability to protect and maintain our intellectual property position; business (including commercial viability), regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about the Company; risks inherent in developing product candidates and technologies; future results from our ongoing and planned clinical trials; our ability to obtain adequate financing, including through our financing facility with Oberland, to fund our planned clinical trials and other expenses; trends in the industry; the legal and regulatory framework for the industry, including the receipt and maintenance of clearances to conduct or continue clinical testing; future expenditures risks related to our asset-centric corporate model; the risk that any one or more of our product candidates will not be successfully developed and/or commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; geo-political risks such as the Russia-Ukraine war and conflicts in the Middle East. These and other risks concerning our programs and operations are described in additional detail in our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and our other reports, which are on file with the U.S. Securities and Exchange Commission (SEC). We explicitly disclaim any obligation to update any forward-looking statements except to the extent required by law.

Contact: 
Kristen K. Sheppard, Esq.
SVP of Investor Relations
investors@centessa.com

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/b64204a9-7c64-47fd-b834-598ca0e45978

Apr 15
Love0

AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations

• AVB-101 is an investigational gene therapy designed to deliver a functional copy of the GRN gene directly to the brain, thus restoring progranulin levels and potentially stopping disease progression

• Milestone marks first-in-human intrathalamic gene therapy delivery for any adult neurodegenerative disease

• ASPIRE-FTD trial will enroll patients at sites in Europe and the United States

LONDON — APRIL 15, 2024 — AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). Clinical trial sites are currently open in Poland, Spain, and the Netherlands with additional sites expected to open in multiple countries, including the United States.

“AviadoBio is committed to bringing forward an innovative gene therapy treatment for FTD-GRN and this moment marks an important milestone for the FTD community and our company,” said Lisa Deschamps, CEO. “We are thankful for the dedication of the ASPIRE-FTD clinical investigators studying AVB-101 and immensely grateful for the families who participate in clinical trials for new treatment options that may change the future for generations of families living with FTD-GRN.”

FTD is a devastating form of early-onset dementia that typically leads to death within 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 It is a leading cause of dementia in people under the age of 65 and is often misdiagnosed.⁴People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain.

“There is a critical need for new treatments for people living with FTD-GRN. The lack of effective disease-modifying treatments makes this diagnosis particularly difficult for patients and their families who don’t currently have any available treatment options for this devastating and progressive disease,” said David Cooper, M.D., Chief Medical Officer. “Treating the first patient in ASPIRE-FTD marks an important step forward in understanding AVB-101’s potential, which has already shown promise in preclinical studies.”

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 safely and effectively cross the blood-brain barrier, delivering targeted treatment directly to the brain to restore progranulin levels in the frontal and temporal cortex where it is needed most, while at the same time minimizing the dose required and thereby limiting any potential systemic exposure.

The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital is the first and only center in Europe currently performing MRI-guided infusions of gene therapies. “Our team is excited to participate in this important scientific research and to perform the first intrathalamic administration of AVB-101 in the ASPIRE-FTD global trial,” said Prof. Mirosław Ząbek, M.D., Ph.D., the chairperson of the department of neurosurgery at INC.

“As a neurologist and founder of the Neuro-Care Clinic, I’ve seen the difficult struggles of my patients with dementia and their families,” said Gabriela Klodowska, M.D., Ph.D. “It means a lot to our patients with FTD to bring this type of study offering a potential one-time treatment for patients with a GRN mutation to Poland and to be the first to enroll patients in this global study.”

In November 2023, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to AVB-101. 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. This designation is intended to bring new medicines to patients earlier. It has also received orphan drug designation by both the FDA and European Commission.

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

  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. Moore KM et al. Lancet Neurol. 19(2):145–156
  7. Kansal K et al. Dement Geriatr Cogn Disord. 2016;41:109–122
  8. Galvin JE et al. Neurology. 89(20):2049–2056
  9. Young JJ et al. Ther Adv Psychopharmacol. 2018;8(1):33–48
  10. Kuang, L., et. al. Frontotemporal dementia non-sense mutation of progranulin rescued by aminoglycosides. Human Molecular Genetics 2020;29(4):624-634

About ASPIRE-FTD

ASPIRE-FTD is an open-label, multi-center, Phase 1/2 dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, patients will receive a single administration of AVB-101 delivered as a one-time infusion into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.

About AVB-101

An investigational 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 infusion directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center.

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 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 people.⁵ FTD typically strikes younger than Alzheimer’s disease and the majority of FTD cases occur between 45 and 68 years of age.⁶,⁷ Given the early onset, FTD can have a substantially greater impact on work, family, and finances than Alzheimer’s disease.⁸ 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.⁹ Approximately 11,000 people in the U.S. and EU5 are living with FTD-GRN with approximately 2,200 new cases per year.¹,¹⁰ 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

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with 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), SV Health Investor’s Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), and LifeArc.

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

Mar 13
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Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

By Eloxx Pharmaceuticals, Press Release, Private Companies
Press Release.

 

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

  • Almirall obtains exclusive global rights to develop and commercialize ZKN-013 for the treatment of rare dermatological and other diseases associated with nonsense mutations
  • ZKN-013 is a phase I ready oral therapy designed to overcome nonsense mutations that cause a premature stop codon resulting in nonfunctional protein production for example in recessive Dystrophic Epidermolysis Bullosa (RDEB), Junctional Epidermolysis Bullosa (JEB) and familial adenomatous polyposis (FAP)

BARCELONA, Spain and WATERTOWN, Mass., March 13, 2024 (GLOBE NEWSWIRE) — Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013. Under the agreement, Almirall obtains global rights to develop and commercialize ZKN-013, including for the use in orphan dermatological diseases. ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases.

The drug candidate is expected to shortly enter into Phase I development in healthy volunteers.

As part of this agreement, Eloxx will receive an upfront of $3 million, and additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million, as well as tiered royalties based on any potential future global sales.

“We are very excited about this agreement with Almirall to develop and distribute ZKN-013, our lead TURBO-ZM™ based molecule, as we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM™ platform.”

“This license agreement is aligned with Almirall’s R&D strategy to develop novel treatments to help people with dermatological conditions, including rare diseases,” said Karl Ziegelbauer, Executive Vice President R&D and Chief Scientific Officer of Almirall. “We look forward to progressing the development of ZKN-013 to find a potentially impactful solution for patients suffering from rare and devastating diseases caused by nonsense mutations.”

RDEB /JEB are rare skin diseases characterized by defects in the Collagen7 gene which is essential for the correct formation of the skin structure and barrier function. ZKN-013 has demonstrated robust functional preclinical activity in RDEB/JEB patient cells and in APCmin (multiple intestinal neoplasia) mice. The studies demonstrated that ZKN-013 induces the production of functional, full-length COL7 in RDEB patient cells.

ZKN-013 is also being developed for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and progression to colon cancer. FAP is a rare GI disease with patients progressing to colon cancer caused by mutations in the APC gene.

ZKN-013 is Eloxx’s lead TURBO-ZM based molecule. Eloxx’s TURBO-ZM platform uses chemistry technology to develop novel Ribosome Modulating Agents to target the human ribosome to develop new potential therapeutics. Ribosomes form the translation machinery that generates functional proteins from genetic sequences; modulating the ribosome subunits provides a therapeutic approach to address a number of different diseases.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations.

For more information, please visit www.eloxxpharma.com.

About Almirall  

Almirall is a global pharmaceutical company dedicated to medical dermatology. We closely collaborate with leading scientists, healthcare professionals, and patients to deliver our purpose: to transform the patients’ world by helping them realize their hopes and dreams for a healthy life. We are at the forefront of science to deliver ground-breaking, differentiated medical dermatology innovations that address patients´ needs.

Almirall, founded in 1944 and headquartered in Barcelona, is publicly traded on the Spanish Stock Exchange (ticker: ALM). Almirall (total revenue in 2023: €898.8 MM, 1900 employees globally) has a direct presence in 21 countries and affiliates in over 70 others.

For more information, please visit almirall.com

Corporate Communications contact Almirall: Investors’ Relations contact
Corporate communications team Pablo Divasson del Fraile
corporate.communication@almirall.com investors@almirall.com
Phone: (+34) 659 614 173 Phone: (+34) 93 291 30 87
Eloxx Contacts:
Investors:
John Woolford
john.woolford@westwicke.com
443.213.0506
Media:
Laureen Cassidy
laureen@outcomescg.com

Eloxx Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement and the expected timeline for clinical development and efficacy of ZKN-013 are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability, or our licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our, and our licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financials-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Almirall Legal warning
This document includes only summary information and is not intended to be exhaustive. The facts, figures and opinions contained in this document, in addition to the historical ones, are “forward-looking statements”. These statements are based on the information currently available and the best estimates and assumptions that the Company considers reasonable. These statements involve risks and uncertainties beyond the control of the Company. Therefore, actual results may differ materially from those declared by such forward-looking statements. The Company expressly waives any obligation to revise or update any forward-looking statements, goals or estimates contained in this document to reflect any changes in the assumptions, events or circumstances on which such forward-looking statements are based, unless required by the applicable law.

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Feb 28
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Pulsed Field Ablation Innovator Argá Medtech Announces €54 Million Oversubscribed Series B Capital Raise

By Argá Medtech, Press Release, Private Companies
Press Release.

 

Pulsed Field Ablation Innovator Argá Medtech Announces €54 Million Oversubscribed Series B Capital Raise

– Funding will advance US and EU clinical studies of innovative pulsed field ablation system for treating atrial fibrillation  – 

Lausanne, Switzerland and San Diego, CA, USA — February 27, 2024 — Argá Medtech, a private company developing Coherent Sine-Burst Electroporation™ (CSE™), a next-generation cardiac ablation system for treating cardiac arrhythmias, including atrial fibrillation (AF), today announced the closure of a €54M oversubscribed Series B funding. The round was led by the existing investors, Advent Life Sciences (UK) and Earlybird Health (Germany), as well as new investor Gilde Healthcare (the Netherlands) and an undisclosed strategic investor. The financing enables Argá Medtech to advance the development of its innovative CSE™ Pulsed Field Ablation (PFA) system for treating AF through the execution of an IDE study in the US and a CE Mark study in the EU. With the funding, the company will also expand its US offices in San Diego, CA, in anticipation of its US clinical activities.

“We are pleased to secure the support of such marquee investors who believe that Argá Medtech will revolutionize the atrial fibrillation ablation field,” said David Neale, CEO of Argá Medtech. “This financing enables us to advance toward our goal of validating the CSE™ PFA system in Europe and the US as we work to deliver a safe, fast, and effective treatment to millions of people affected by cardiac rhythm disorders and atrial fibrillation. We are proud of our accomplishments to date, including conducting a 48-patient first-in-human study in Europe, which demonstrated the high performance of our platform in treatingatrial fibrillation.”

Argá Medtech’s CSE™ ablation system enables electrophysiologists to treat any region in the heart safely and efficiently using a single, multi-configurable catheter while titrating lesion depths according to the location within the heart.

The company’s CSE™ PFA platform combines its proprietary CSE™ PFAgenerator with a multi-configurable catheter to provide unmatched flexibility in AF treatment. Argá Medtech’s CSE™ system uses a sinusoidal/sine wave, while other PFA platforms are typically powered by square wave energy sources. This approach offers several advantages, such as allowing physicians to configure the energy delivery and titrate the depth to the location in the heart. Additionally, the waveform is delivered through a versatile, all-in-one catheter that can be shaped to create circular, linear, or focal ablation lesions, eliminating the need to perform catheter exchanges to achieve the desired lesion set. Thus, the procedure is simplified, reducing the risk of introducing air bubbles as one catheter is removed and another is reinserted, as well as reducing costs.

AF is the most common heart arrhythmia, affecting about 38 million patients worldwide1, rendering them five times more likely to have a stroke.2 While ablation using thermal energy has proven effective in alleviating AF symptoms, higher risks are associated with its use.3In contrast, PFA energy delivery offers a tissue-selective, precise ablation of the intended heart tissue while preserving nearby tissues and minimizing the risks of thermal injury, such as those to the esophagus or the phrenic nerve.

About Argá Medtech SA

Argá Medtech SA is developing a Coherent Sine-Burst Electroporation™ (CSE™), Pulsed Field Ablation (PFA) system to treat patients with cardiac arrhythmias. Argá Medtech is setting the standard for the next-generation PFA systems with its versatile catheter design able to create circular, linear, and focal lesions, coupled with a unique CSE™ PFA waveform that allows energy titration based on tissue depth to achieve durable lesions. The company was co-founded in June 2020 by veteran medical device executive David Neale, CEO, and serial entrepreneur Randy Werneth, CTO, and has offices in Lausanne, Switzerland and San Diego, CA, USA. With over 50 years of combined experience in creating and commercializing cardiac and arrhythmia management products, the leadership team is well connected in electrophysiology and understands the current challenges faced by physicians and patients.

To learn more, visit https://argamedtech.com.

References: 

1. Lippi, Giuseppe et al. “Global epidemiology of atrial fibrillation: An increasing epidemic and public health challenge.” International journal of stroke: official journal of the International Stroke Society vol. 16,2 (2021): 217-221. doi:10.1177/1747493019897870. https://pubmed.ncbi.nlm.nih.gov/31955707/

2. World Stroke Organization. Stroke and Atrial Fibrillation. Accessed February 16, 2024.  https://www.world-stroke.org/world-stroke-day-campaign/prevent-stroke/stroke-and-atrial-fibrillation

3. Aldaas OM, Malladi C, Aldaas AM, Han FT, Hoffmayer KS, Krummen D, Ho G, Raissi F, Birgersdotter-Green U, Feld GK, Hsu JC. Safety and acute efficacy of catheter ablation for atrial fibrillation with pulsed field ablation vs thermal energy ablation: A meta-analysis of single proportions. Heart Rhythm O2. 2023 Sep 12;4(10):599-608. doi: 10.1016/j.hroo.2023.09.003. PMID: 37936671; PMCID: PMC10626185. https://pubmed.ncbi.nlm.nih.gov/37936671/

Media Contact: 

Finn Partners

Glenn Silver

glenn.silver@finnpartners.com

Feb 27
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Curve Therapeutics raises £40.5 million to turbocharge discovery platform and advance breakthrough pipeline

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

 

Curve Therapeutics raises £40.5 million to turbocharge discovery platform and advance breakthrough pipeline

  • Series A financing led by Pfizer Ventures with new investors Columbus Venture Partners and British Patient Capital which join founding investor Advent Life Sciences and seed investor Epidarex Capital
  • Goal to advance proprietary discovery platform and progress pipeline of assets addressing challenging cancer targets into the clinic

Southampton, UK, 27 February 2023 – Curve Therapeutics (“Curve” or the “Company”), a private biotechnology company pioneering a revolutionary intracellular screening platform addressing complex and challenging disease targets, today announces the close of its successful £40.5 million Series A financing. Pfizer Ventures led the round with participation from Columbus Venture Partners and British Patient Capital, which join founding investor Advent Life Sciences and co-lead from the seed round, Epidarex Capital.

Curve’s powerful Microcycle® platform enables the direct discovery of biologically active molecules against targets that have been difficult to address using conventional drug discovery methods. Curve has built a discovery pipeline of assets including a first-in-class dual-inhibitor of HIF-1 and HIF-2 that addresses survival mechanisms in more than half of solid tumours, and a first-in-class inhibitor of ATIC dimerization that targets an important vulnerability in multiple cancers. The financing will enable the Company to progress development of these assets rapidly towards clinical development and to expand the discovery platform beyond challenging and complex intracellular protein targets.

Simon Kerry, PhD, MBA, Chief Executive Officer of Curve Therapeutics, said: “This financing will enable us to expand our team, progress our lead assets into the clinic and to expand our drug discovery platform. We welcome our new investors alongside our existing strong syndicate and look forward to working together to take Curve to its next stage of growth.”

Professor Ali Tavassoli, Chief Scientific Officer of Curve Therapeutics, added: “Curve’s Microcycle platform is a powerful tool for drug discovery, enabling an unparalleled advantage in the discovery of functional hits and leads. We look forward to maximising the potential of our platform to further develop a rich pipeline of programmes with the potential to treat unmet clinical needs in a diverse range of diseases, including cancer.”

Dr. Marie-Claire Peakman, Partner at Pfizer Ventures, commented: “We are delighted to have led this financing and to work with great co-investors and leadership team.  Curve’s platform provides an exciting new approach designed to tackle tough, high priority targets which have been difficult to progress in the past. We are eager for the Company to progress the platform with the goal of identifying new therapeutics for challenging diseases.” 

Curve’s platform allows functional screening and enrichment of highly diverse, gene-encoded, Microcycle libraries within the cytoplasm of mammalian cells to identify library members that have a desired biological activity against a therapeutic target. Importantly, the compact size of Microcycles enables their transformation to non-peptide small molecules for lead optimisation and development, an unparalleled advantage compared to other cyclic peptide technologies.

Curve occupies state of the art laboratories at the Southampton Science Park, a move prompted by the expansion of the Company’s research team which included the recent appointments of Dr Monika Ermann as Vice President of Drug Discovery, and Dr Sally Price as Vice President of Biology. With four regional Universities and pioneering medical institutions such as the Centre for Cancer Immunology in Southampton, Hampshire is an attractive location for life science companies, and just an hour from key biotech centres in Oxford and London.

For more information, please contact:

Curve Therapeutics
Simon Kerry
Chief Executive Officer
info@curvetx.com

Optimum Strategic Communications
Mary Clark, Vici Rabbetts, Joshua Evans
+44 (0) 208 078 4357
curve@optimumcomms.com

About Curve Therapeutics

Curve Therapeutics is a private biotechnology company pioneering a revolutionary intracellular screening platform to enable the discovery of innovative therapeutics that address complex and challenging disease targets with the potential to transform the lives of patients. Curve originated from world-leading Microcycle® research conducted by Professor Tavassoli’s group in the Department of Chemistry at the University of Southampton, UK. Curve is backed by blue chip investors including Advent Life Sciences, Epidarex Capital, Pfizer Ventures, Columbus Venture Partners and British Patient Capital. Curve has a US$1.7bn global research collaboration with MSD the trade name of Merck & Co., Inc., Kenilworth, NJ USA, to discover and validate modulators of up to five therapeutic targets using its Microcycle® technology, initially for oncology and neurology indications. For more information visit: www.curvetx.com

About Curve’s Microcycle® platform

Curve has developed an IP-protected, mammalian cell platform technology for functional screening and enrichment of diverse hexameric cyclic peptide Microcycle® libraries to identify those library members that have the desired biological activity against a therapeutic target. Curve’s platform allows direct screening for biologically active library members inside mammalian cells and facilitates small molecule hit-to-lead programmes. A key advantage of the technology is that both the library and the target are present in all of their native conformations within a cell. Uniquely, the compact size and rigid structure of Microcycles® enables the design of non-peptide small molecule leads. The platform can be used for a wide range of therapeutically relevant targets, including protein-protein and protein-DNA interactions and has been used by Curve to develop a pipeline of cancer programmes against targets including a dual HIF-1/HIF-2 inhibitor and an inhibitor of ATIC homodimerization.

Feb 21
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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