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Beacon Therapeutics

Dec 06
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Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP)

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

 

Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP)

  • To date, laru-zova has been well-tolerated by all participants in the Phase 2 DAWN study.
  • Data show promising early improvements in low luminance visual acuity (LLVA), a critical measure of visual function.
  • Data build confidence in laru-zova as a potential treatment for patients with XLRP.
  • Pivotal Phase 2/3 VISTA trial for laru-zova in XLRP is currently enrolling.

London, UK, Cambridge, MA, 6 December 2024 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a purpose to save and restore the vision of patients with blinding retinal diseases, today announced the presentation of 3-month interim safety and efficacy results of the Phase 2 DAWN trial in patients with XLRP at the FLORetina-ICOOR Meeting 2024 in Florence, Italy.

Key presentation highlights:

  • The three-month data show that laru-zova has been well-tolerated by all participants.
  • No study agent-related treatment emergent adverse events (TEAEs) were reported, including no ocular inflammatory adverse events.
  • Data also show promising early improvements in LLVA, a critical measure of visual function.
  • The benefit-risk profile supports on-going clinical development for the treatment of patients with XLRP caused by RPGR mutations.

DAWN is a non-randomized, open-label study of laru-zova in participants with XLRP who have previously been treated with a full-length AAV-vector based gene therapy targeting the RPGR protein. The purpose of DAWN is to assess two different doses of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously received gene therapy for XLRP.

XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP primarily affects young males with an estimated prevalence of 1 in 25,000 males in US, Europe and Australia having XLRP with RPGR mutations. Laru-zova expresses the full length RPGR protein and is therefore expected to address the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss, representing a potential best-in-class treatment for progressive vision loss in patients with XLRP.

Lance Baldo, MD, chief executive officer of Beacon Therapeutics, stated, “We are encouraged by the early results from the Phase 2 DAWN study. The strong safety profile observed to date is complemented by promising early improvements in low luminance visual acuity – a meaningful and functional measure of vision in patients with XLRP. These data not only support the ongoing pivotal VISTA study, but also strengthen our commitment to this opportunity to bring hope to patients and families affected by this devastating disease.”

Beacon Therapeutics is also enrolling patients for its pivotal Phase 2/3 VISTA trial of laru-zova as it develops this potential treatment for patients with XLRP.

Presentations –
• Subretinal Gene Therapy laru-zova (laru-zova (AGTC-501)) for X-Linked Retinitis Pigmentosa (XLRP) Phase 2 Multicenter Study (DAWN): Preliminary Results

• RPGR gene therapy and the Beacon clinical trials: Beacon Therapeutics Subretinal Gene Therapy laru-zova (laru-zova (AGTC-501)) for X-Linked Retinitis Pigmentosa (XLRP)

Presenting Author – Professor Paulo Eduardo Stanga, Founder and Chief Medical Officer, The Retina Clinic London

Contact:
info@beacontx.com

Media:
beacontherapeutics@edelman.com

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) and two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD). Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

Lead development candidate laru-zova (laru-zova (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, primarily in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Laru-zova expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon 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.

Jul 25
Love0

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