Category Archives: Biotech Companies Gene Therapy

XyloCor and SmartCella enter into license agreement for use of the Extroducer to administer novel gene therapy XC001 to the heart

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  • The Extroducer® Infusion Catheter System® enables local delivery of XC001 to the heart without the need for surgery.
  • XC001 has achieved positive Phase 1/2 results in the EXACT Trial validating its transformative potential for treatment of refractory angina in patients who have exhausted available treatment options and have a debilitating quality-of-life.

XyloCor Therapeutics, Inc. (“XyloCor”), a clinical-stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, and SmartWise, a unit of SmartCella Holding AB (“SmartCella”), have entered into a licensing agreement under which XyloCor has rights to the Extroducer® Infusion Catheter System ®, a first-in-class endovascular device designed to deliver advanced therapies directly into the heart [and hard-to-reach tissues] .  XyloCor plans to deploy the Extroducer to support catheter-based endocardial delivery of its lead gene therapy candidate, XC001 (encoberminogene rezmadenovec), in future clinical studies and commercial use.  

XyloCor and SmartCella enter into license agreement for use of the Extroducer to administer novel gene therapy XC001 to the heart

“This agreement with SmartCella will enable XyloCor to build upon its robust foundation of efficacy and safety data for XC001 by offering the potential for improved safety and ease of delivery without surgery via this novel catheter,” said Al Gianchetti, President and CEO of XyloCor. “Teaming up with SmartCella will help in our effort to optimize patient safety and tolerability while maintaining accurate delivery of XC001 to target areas in the heart for patients with refractory angina. It also opens up the potential to develop XC001 earlier in the coronary artery disease progression for even larger patient groups.”

XC001 is designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple isoforms of vascular endothelial growth factor (VEGF). With the use of the Extroducer catheter, XyloCor can offer patients a better delivery option for local administration of XC001 directly to the heart, that is less invasive and eliminates potential risks associated with surgical administration.

“We welcome the Extroducer delivery of XC001 as it offers a more efficient method for gene therapy administration for patients with refractory angina,” said Timothy D. Henry MD, Interventional Cardiologist and Director of the Lindner Center, The Christ Hospital, Cincinnati, Ohio. “Preclinical models provide strong evidence that this approach will maintain, or even improve the efficacy when compared to surgical delivery and it should lower the risk of complications that may arise from surgical administration. I am looking forward to initiating the Phase 2b trial of XC001 in patients with refractory angina using this innovative administration approach.”

The recently published EXACT Phase 1/2 trial assessed the use of one-time gene therapy with XC001 as a new therapeutic approach in refractory angina – a debilitating and chronic condition that impacts over one million people in the United States and is growing in prevalence. In the EXACT trial, 42 patients with class II-IV angina were treated with XC001 directly administered to the heart following minimally invasive surgical access. The results demonstrated that treatment with XC001 can be safely administered and achieve durable clinical improvements of exercise duration, and angina frequency, due to a decrease in ischemic burden, as measured by Positron Emission Tomography (PET) imaging. Notably, six months after treatment 43% of patients had no chest pain with ordinary activities and 58% reported no angina episodes at 12-month clinical follow up. XC001 was well tolerated in the patient population and there were no serious adverse events related to the drug.  The Phase 2b trial will be a randomized double-blinded study assessing the safety and efficacy of XC001 administered via the Extroducer® Delivery Catheter in coronary artery disease patients with refractory angina.

“The collaboration underscores the transformative potential of the Extroducer in delivering XC001 therapy for patients with refractory angina. A great example of a powerful combination of delivery system and drug therapy representing a substantial advancement in treatment options. The collaboration with such a distinguished partner as XyloCor marks a significant milestone for our global expansion efforts and will also enable us to further explore and harness the future capabilities of the Extroducer, ultimately expanding the benefits to a greater number of patients in need,” said Niklas Prager, CEO of SmartCella.

Terms of the agreement include a global license to XyloCor for use of the Extroducer for the administration of XC001 and provide for SmartCella to supply catheters to XyloCor in clinical trials and commercial use in exchange for an upfront payment, clinical, regulatory and commercial milestones and a royalty on sales. Total deal value amounts to approximately USD 130 million and mid-single digit royalties.

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one-time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About Extroducer® Infusion Catheter System

The Extroducer® Infusion Catheter System is a first-in-class endovascular delivery device which enables direct-to-tissue drug delivery. The Extroducer® addresses a significant unmet need in the field of novel therapies, enabling targeted delivery of a wide range of modalities for solid tumor treatment, genetic disorders and tissue repair, to name but a few. Using standard equipment and routine interventional radiology approaches, the Extroducer provides access to hard-to-reach tissues by safely penetrating the vessel wall and delivering payload directly to the target location. Smartwise received U.S. Food and Drug Administration (FDA) clearance under 510(k) for the Extroducer® delivery catheter in June 2022.

About XyloCor

XyloCor Therapeutics, Inc. is a private, clinical-stage biopharmaceutical company developing potential best-in-class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co-founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

About SmartCella

SmartCella, founded in 2014, is an innovative biotechnology company based in Stockholm, Sweden. SmartCella’s vision is to combine first-in-class delivery platforms with cutting-edge cell and mRNA therapies to unleash the full potential of targeted therapies. The company has three main business units, Smartwise, SmartCella Solutions and ProCella. For more information, visit www.smartcella.com.

EyeDNA Therapeutics Announces Positive 24-month Data from Ongoing Phase I/II Trial of HORA-PDE6b Gene Therapy in Patients with Retinitis Pigmentosa Caused by Bi-allelic Mutations in PDE6b

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Clinically meaningful benefit in visual functions and good safety profile of HORA-PDE6b is confirmed at 24-month follow-up

Phase I/II results will be discussed with US and European health authorities to define optimal path to making HORA-PDE6b available to patients with PDE6b Retinitis Pigmentosa

Paris, France, May 7, 2024 – eyeDNA Therapeutics (‘eyeDNA’), a newly created subsidiary of Coave Therapeutics (‘Coave’), a genetic medicine company focused on developing life-changing therapies, today announces positive 24-month follow-up results from its Phase I/II study (NCT03328130) evaluating the safety and efficacy of HORA-PDE6b, its investigational gene therapy for retinitis pigmentosa (RP) caused by bi-allelic mutations in the PDE6b gene (PDE6b RP). These data were reported during an oral presentation* on May 6, at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting in Seattle, WA, US.

The positive 24-month follow-up data presented confirm results from the previous interim analysis of the trial conducted at the 12-month follow-up point and support preparation for a registrational trial for HORA-PDE6b in PDE6b RP patients. Further discussions with health authorities in the US and Europe are planned to define the optimal path to making HORA-PDE6b available to PDE6b RP patients.

To date, HORA-PDE6b has been administered in 17 evaluable patients aged 18 years and older presenting an advanced form of PDE6b RP using two ascending doses in four consecutive cohorts. The treatment was administered in the more affected eye while the other eye served as an untreated control.

In a subgroup of clinical interest of six patients receiving the high dose, with less advanced disease (Best Corrected Visual Acuity (BCVA) score ≤75 ETDRS letters [ ≤ 20/32 Snellen equivalent], Goldmann Visual Field (GVF) ≥ 10 degrees), positive efficacy results were reported at 24 months on the BCVA and the GVF outcomes. The BCVA mean change from baseline increased by +0.09 LogMar in the untreated eye, while acuity in the treated eye was stabilized (+0.02 LogMar). From baseline, the mean reduction of the GVF was over 300 deg² superior in the untreated eyes compared to the treated eye.

Interestingly, long-term data available from patients from the low dose group (n=7) followed up over a five-year period found that the BCVA of the untreated eyes consistently declined (increase of 0.05 to 0.08 LogMAR/year from the second year), which is in line with the natural history of the disease. Meanwhile, the mean change from baseline of BCVA of the treated eyes in the same group is stabilized (between +0.03 and +0.06 LogMAR over the same follow-up period). The difference of BCVA mean change from baseline between the treated eyes and the untreated eyes at five years is 0.25 LogMAR (12 Letters).

Furthermore, the full-field stimulation test (FFST) in blue light assessing rod function continues to show an improvement of the light perception threshold in favor of the treated eyes, which is considered clinically meaningful (improvement of almost six decibels). The interesting positive trend on the retinal anatomical evaluation by Optical Coherence Tomography (OCT; Ellipsoid Zone horizontal length) observed at 12-month follow-up on the subgroup of clinical interest is maintained after 24 months.

Following the 24-month study period, both doses were well tolerated (n=17). Five ocular Serious Adverse Events (SAEs) occurred including two resolved SAEs possibly related to HORA-PDE6b (one chorioretinitis and one reduced visual acuity). Patients did not receive preventive oral corticosteroids.

An additional cohort of four to six younger patients aged 13-17 years old with a GVF at baseline ≥ 20 degrees in each meridian and at an earlier disease stage is ongoing with three patients enrolled.

“The highly encouraging safety and efficacy data observed in patients two years after treatment with HORA-PDE6b continue to support our view that this novel gene therapy could provide an important clinical benefit for PDE6b RP patients. These data will support our discussions with regulators to determine the optimal route for getting HORA-PDE6b to patients,” said Rodolphe Clerval, Chief Executive Officer. “At the same time, we continue to evaluate HORA-PDE6 in an expansion cohort of younger patients with less advanced disease, for whom treatment with HORA-PDE6b could have an even greater therapeutic impact.”

“PDE6b retinitis pigmentosa is a progressive and irreversible inherited degenerative disease that leads to significant visual impairment and blindness. These two-year safety and promising efficacy results are of great medical interest and could represent a significant step towards providing an effective treatment for patients with this devastating disease,” commented Dr. Jean-Baptiste Ducloyer, MD, Nantes University Department of Ophthalmology.

*Abstract 2134:

JB Ducloyer, et al. 12-month Safety and Efficacy Evaluation of HORA-PDE6b, a Gene Therapy Targeting Patients with Retinitis Pigmentosa Due to Biallelic PDE6B Gene Mutation

***

About eyeDNA Therapeutics and HORA-PDE6b

eyeDNA Therapeutics, a wholly owned subsidiary of Coave Therapeutics, is a clinical-stage gene therapy company, focused on developing life-changing therapeutics for inherited retinal disorders. Our lead program HORA-PDE6b, an AAV5-based gene replacement therapy, is being evaluated in a Phase I/II trial for the treatment of retinitis pigmentosa (RP) caused by bi-allelic mutations of the PDE6b gene (PDE6b RP) (NCT03328130).

eyeDNA and Théa Open Innovation (‘TOI’) are partners for the development and commercialization of HORA-PDE6b. eyeDNA is responsible for the global development of HORA-PDE6b and retains commercial rights to the product in the US, Japan, South Korea, China and other territories outside Europe. In Europe and certain other countries, HORA-PDE6b is being co-developed by Coave and TOI under a license and development agreement with exclusive rights granted to TOI to commercialize HORA-PDE6b in these territories.

About Coave Therapeutics

At Coave Therapeutics, we are leading the transition of genetic medicine from rare to prevalent conditions, starting with neurodegenerative and eye diseases. Our proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform introduces chemical modifications onto AAV capsids or Lipid Nanoparticles (LNPs) to overcome the limitations of current vectors on efficacy, safety, and manufacturability.

With low doses and optimized routes of administration, our conjugated vectors have demonstrated markedly improved transduction and biodistribution in the central nervous system and the eye across different species. Our diverse pipeline of novel genetic medicines can potentially transform the lives of people afflicted by rare and prevalent neurodegenerative and ocular diseases – including genetically and non-genetically defined indications.

Coave recently created its subsidiary eyeDNA Therapeutics to focus on the development – up to the marketing authorization application – of its unique gene therapy HORA-PDE6b for the treatment of inherited retinal diseases caused by mutations in the human PDE6b gene.

Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit https://coavetx.com/ and follow us on LinkedIn.

CONTACTS
eyeDNA Therapeutics and Coave Therapeutics
Rodolphe Clerval, CEO
contact@coavetx.com

MEDiSTRAVA
Sylvie Berrebi, Leila Adlam, Mark Swallow
coavetx@medistrava.com

Laverock Therapeutics announces appointment of Ali Elsley as Finance Director and expansion of team

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STEVENAGE, United Kingdom, March 26, 2024 (GLOBE NEWSWIRE) — Laverock Therapeutics, a gene-silencing company with a uniquely powerful technology for the development of programmable advanced therapies to tackle major diseases, today announces the appointment of Ali Elsley as Finance Director.

Ali has extensive experience in the biotechnology and pharma sector working with both private and public companies. She joins from Acacia Pharma Group plc, an early commercial-stage listed pharma company, where she was Group Financial Controller and Company Secretary and focused on equity fundraising, M&A activity and all aspects of financial operations. Prior to this, she spent 10 years as an auditor with PwC LLP in Cambridge, with a focus on early-stage biotech and pharma companies, together with advisory IPO work. She is a fellow of the Institute of Chartered Accountants in England and Wales and holds a degree in Archaeology and Anthropology from the University of Cambridge.

In addition to Ali’s appointment, Laverock has significantly grown its team to 24 people, representing growth of 50% over the past year, and expanded its R&D facilities in the Stevenage Bioscience Catalyst as it continues to build its capabilities following its £13.5m expanded seed round announced in September last year, the second highest seed investment by a UK company in 2023.

David Venables, CEO of Laverock, said: “Ali has an exceptional track record working with innovative biotechnology and pharma companies. I very much look forward to working closely with her and the rest of our fast-growing team as we continue to apply our uniquely powerful gene-silencing technology to the development of a new generation of programmable advanced therapies to tackle major diseases.”

Ali Elsley, FD of Laverock, said: “Laverock’s technology has the potential to create a step-change in advanced therapies and I have been hugely impressed with the pace of progress since joining the company. The management team and Board are exceptionally strong, and I believe we have a very solid foundation from which to deliver on the promise.”

For further information please contact:

Laverock Therapeutics:

E: contact@laverocktx.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 Laverock Therapeutics

Laverock Therapeutics is a gene-silencing company with a uniquely powerful technology which harnesses the cell’s natural gene regulatory mechanisms to develop a new generation of programmable advanced therapies to tackle major diseases. Laverock’s truly innovative technology re-directs naturally-occurring micro RNAs (miRNAs) to conditionally silence genes in a way which is programmable, tunable, stable and specific. This opens a path to more effective, safer and accessible advanced therapies. Laverock is applying its technology through its own pipeline – targeting regenerative medicine and oncology – and through partnerships.

Laverock has a highly experienced leadership team with proven track records in biotechnology, pharma and academia, and an exceptionally strong Board. Laverock completed a £13.5m seed round in September 2023. Its high-calibre investors include Calculus Capital, Eli Lilly and Company, Mercia Ventures, Maven Capital Partners, Eos, UK Innovation & Science Seed Fund and Tekfen Ventures.

For more information, please visit www.laverocktx.com and follow us on LinkedIn.

Alpha Cognition – developing novel treatments for neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis

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Alpha Cognition is a clinical stage, biopharmaceutical company dedicated to developing novel treatments for under-served neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis (ALS).

Alpha Cognition - developing novel treatments for neurodegenerative diseases such as Alzheimer’s Dementia and Amyotrophic Lateral Sclerosis

ALPHA-1062, a patented new chemical entity that has demonstrated safety and improved ALPHA-1062, is a patented new chemical entity being developed as a new generation acetylcholinesterase inhibitor for the treatment of Alzheimer’s disease, with expected minimal gastrointestinal side effects. ALPHA-1062’s active metabolite is differentiated from donepezil and rivastigmine in that it binds neuronal nicotinic receptors, most notably the alpha-7 subtype, which is known to have a positive effect on cognition. ALPHA-1062 is also being developed in combination with memantine to treat moderate to severe Alzheimer’s dementia and as an intranasal formulation for traumatic brain injury.

ALPHA-0602 (Progranulin) is expressed in several cell types in the central nervous system and in peripheral tissues, promotes cell survival, regulates certain inflammatory processes, and plays a significant role in regulating lysosomal function and microglial responses to disease. Its intended use for the treatment of neurodegenerative diseases has been patented by the Company and Alpha-0602 has been granted an Orphan Drug Designation for the treatment of Amyotrophic Lateral Sclerosis by the FDA.

ALPHA-0702 and ALPHA-0802 are Granulin Epithelin Motifs, or GEMs, derived from full length progranulin which have therapeutic potential across multiple neurodegenerative diseases. Progranulin is comprised of 7½ Granulin Epithelin Motifs (GEMs). GEMs are important in regulating cell growth, survival, repair, and inflammation. Individual granulin domain peptides are approximately 60 amino acids in length (~7kDa). GEMs are metabolically stable and resistant to proteolysis (long half-life). Evidence suggests that not all GEMs are equally potent at promoting cell survival signaling or reducing proteinopathy, and may compete (e.g., binding to effectors) with active GEMs and effectively decrease their potency. Specific GEM combinations have been shown to be more effective than full length progranulin in promoting Cathepsin D maturation. GEMs have been shown to be important in regulating cell growth, survival, repair, and inflammation. Alpha-0702 and ALPHA-0802 are designed to deliver this with potentially lower toxicity, and greater therapeutic effect.

More info : http://www.alphacognition.com/

Keywords : Alpha Cognition , neurodegenerative diseases , dementia , Alzheimer’s Dementia , Alzheimer Disease , Amyotrophic Lateral Sclerosis , acetylcholinesterase inhibitor , orphan drug , CNS , neuroscience , traumatic brain injury , Granulin Epithelin Motifs , Gene Therapy

Cytena – single cells on demand

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Cytena – single cells on demand

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