“Close your eyes and relax”: the role of hypnosis in reducing anxiety, and its implications for the prevention of cardiovascular diseases
Ellipses Pharma: Maximizing KOL Input To Increase The Success Rate Of Oncology R&D
Key opinion leader (KOL) input is associated with a higher success rate in drug development. However, many oncology programs fail to effectively use this critical resource, resulting in poor decision making, unnecessary delays in development, and conflicts that bias asset selection and clinical trial design. Ellipses Pharma is taking a different approach.
Ellipses, a developer of innovative cancer medicines, has built a cloud-based platform to crowdsource scientific and clinical insight from a large number of global oncology KOLs—200 and climbing—in its scientific affairs group (SAG). Because the KOLs provide responses anonymously, and the data are blinded when reviewed internally, the platform guards against groupthink, enables open expression of views, and mitigates the risk that Ellipses will value some opinions more highly than others.
The large number of KOLs offering expert critique and input increases confidence in the feedback and allows Ellipses to capture heterodox views—outliers that challenge assumptions and lead to better decisions. KOLs are located in leading centers of excellence around the world and provide expert reviews of the pre-clinical and clinical data for each asset alongside insights into local practices, patient populations, and real-world evidence of the competitive landscape.
How Ellipses uses the platformEllipses uses the platform in its due-diligence process before it in-licenses new assets (Fig. 1). By gathering feedback from around 50 to 70 experts simultaneously on each asset, Ellipses' investment team utilizes the scientific consensus to de-risk the development of its drugs by better understanding data on potential acquisition targets, the indications a drug candidate could address, and how important a molecule could be for patients.
Fig. 1Capabilities of the Ellipses platform. The secure anonymized online asset-review process for ellipses key opinion leaders (KOLs) promotes freedom to express opinions and an unbiased appraisal of the clinical data. SAG, scientific affairs group.
The feedback can reveal new and tailored opportunities for drug candidates, for example by showing that Ellipses' new RET receptor tyrosine kinase inhibitor (EP0031) has applications in cancers that mutate and escape existing treatments, as well as in tumors that are RET positive when first detected. Such input enables Ellipses to understand and maximize the value of assets by allowing the design and execution of more efficient clinical trials with enhanced success rates.
After acquiring an asset, Ellipses gathers continual input from the KOLs to enable the nimble adaptation of drug development in response to new internal and external data. The experts comment on clinical trial design, patient stratification, biomarker selection, and how to increase the chances of success. The input has enabled Ellipses to open studies and enroll participants faster than industry norms.
Ellipses' constant engagement with KOLs facilitates flexibility. Recently, Ellipses responded to feedback from its KOLs after requesting a review of the development plan for one of its drugs in the clinic. KOLs provided real-world data, garnered in recent months in their clinics and presented at a leading cancer conference, to argue against focusing on development of a triplet combination. Noting the toxicity profile of the currently used combination, the KOLs made the case for prioritizing a doublet that would likely be better for patients, be more attractive to investigators and physicians, and provide more relevant data to inform the design of any successful triplet combination needed in the future. Ellipses responded by running preclinical doublet studies and pivoting to the new approach.
Validating the KOL-enabled modelKOLs have embraced the model. The platform is easy to use, with a professor at Newcastle University and University Hospitals, UK, praising the ability to "evaluate in stages" and complete work in their "own time, when more convenient." KOLs value participation. A professor at Cambridge University, UK, described their role as "fulfilling academically and altruistically," while a peer at University Hospitals Leuven, Belgium, said it would be "a missed opportunity not to work with Ellipses." A professor of investigational cancer therapeutics at the University of Texas, USA explained "Being part of the SAG allows me to make an impact in an even earlier stage of an asset, when the investment decisions are made."
Ellipses has early evidence that its KOL-enabled approach is working. Seven of the nine assets chosen and developed thus far with the input of the experts remain in active development, putting the company above the historical one-in-eight success rate seen in oncology drug development. If Ellipses can increase the success rate to one in seven or one in six, it will mean more approved therapies for patients, lower drug development costs, and ultimately better value for payers.
Work is underway to further improve the system, with Ellipses aiming to increase the membership of its SAG to 300 oncology KOLs split between Europe, the US, the Asia-Pacific (APAC) region, and the rest of the world while making its platform even easier to use. The improvements will position Ellipses to continue to use KOL input to inform a disruptive approach to drug development that is designed to de-risk oncology drug selection and quickly advance multiple high-quality assets.
TScan Therapeutics To Host Virtual KOL Event To Discuss Clinical Updates From The ALLOHA™ Phase 1 Trial And Heme Development Strategy
WALTHAM, Mass., Dec. 02, 2024 (GLOBE NEWSWIRE) -- TScan Therapeutics, Inc. (Nasdaq: TCRX), a clinical-stage biotechnology company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer, today announced the Company will host a virtual key opinion leader (KOL) event to discuss data from the ALLOHA™ Phase 1 heme trial presented at the ASH Annual Meeting and the clinical development strategy for the heme program. Additionally, the Company will provide an update on its PLEXI-T Phase 1 solid tumor trial. The virtual event will take place on Tuesday, December 10, at 8:00 a.M. ET.
The event will provide an in-depth review of the oral presentation describing the preliminary results from TScan's ongoing ALLOHA Phase 1 heme trial of TSC-100 and TSC-101 in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (HCT) with reduced intensity conditioning. The Company will also provide updates with regards to a potential registrational path for the program following its initial meeting with the U.S. Food and Drug Administration (FDA), as well as future plans to expand the program.
Featured speakers include:
Registration for the live event can be found here. A replay will be made available on the "Events and Presentations" section of the Company's investor relations website at ir.Tscan.Com.
About TScan Therapeutics, Inc.
TScan is a clinical-stage biotechnology company focused on the development of T cell receptor (TCR)-engineered T cell (TCR-T) therapies for the treatment of patients with cancer. The Company's lead TCR-T therapy candidates are in development for the treatment of patients with hematologic malignancies to prevent relapse following allogeneic hematopoietic cell transplantation (the ALLOHATM Phase 1 heme trial). The Company has developed and continues to expand its ImmunoBank, the Company's repository of therapeutic TCRs that recognize diverse targets and are associated with multiple HLA types, to provide customized multiplex TCR-T therapies for patients with a variety of cancers and is enrolling patients into its ongoing PLEXI-T Phase 1 solid tumor trial.
Contacts
Heather SavelleTScan Therapeutics, Inc.VP, Investor Relations857-399-9840hsavelle@tscan.Com
Maghan MeyersArgot Partners212-600-1902TScan@argotpartners.Com
Sensorion Reports Data In The Audiogene Phase 1/2 Gene Therapy Clinical Trial
SENS-501 gene therapy product and surgical procedure are well tolerated by the first two treated patients and encouraging behavioural changes are observed in both toddlers
Sensorion plans on hosting a KOL event in early 2025 to further comment and discuss the first cohort safety data, qualitative and quantitative efficacy measurements, and next steps for Audiogene Phase 1/2 clinical trial, including planned interactions with the U.S. Food and Drug Administration.
MONTPELLIER, France, December 18, 2024--(BUSINESS WIRE)--Regulatory News:
Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company specializing in the development of novel therapies to restore, treat and prevent hearing loss disorders, announced today latest data updates confirming the safety of SENS-501 in the two first patients injected in the Company's Audiogene study. This is a Phase 1/2 clinical trial evaluating SENS-501 in patients aged 6 to 31 months and naïve of cochlear implant, who are suffering from a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene.
The Audiogene clinical trial assesses, as primary endpoint in the first part of the dose escalation study, the safety of an intra-cochlear injection of SENS-501 in infants and toddlers aged 6 to 31 months at the time of gene therapy treatment. Sensorion's objective in targeting the first years of life, when brain plasticity is optimal, is to maximize the chances of these young children with pre-linguistic hearing loss to acquire normal speech and language, thus, potentially transforming these toddlers' lives. For the first two toddlers treated in the first cohort, SENS-501 gene therapy product and surgical procedure were well tolerated: the intra-cochlear administration of the gene therapy product was uneventful, and no serious adverse events were reported. In addition, encouraging behavioural improvements were observed in both toddlers. Sensorion plans on hosting a KOL event in early 2025 to further comment and discuss the first cohort safety data, qualitative and quantitative efficacy measurements, and next steps for Audiogene Phase 1/2 clinical trial, including planned interactions with the U.S. Food and Drug Administration. Further details regarding the event shall be disclosed in due course.
The Company continues to anticipate the completion of the first cohort enrollment by the end of the year and the recruitment of the second cohort by the end of H1 2025.
Géraldine Honnet, M.D., Chief Medical Officer of Sensorion, said: "I'm very pleased with the progress made in the Audiogene Phase 1/2 clinical trial evaluating SENS-501, a potentially game changing hearing loss therapeutic being developed in collaboration with the Institut Pasteur. Today's data results confirm an excellent safety profile for the first toddlers treated with SENS-501 and I am happy to report early signs of encouraging behavioural changes in both patients. We look forward to treating the third patient of the first cohort imminently, thus achieving an important development milestone for Sensorion. We will host a KOL call in early 2025 and I am looking forward to presenting further safety and efficacy data for patients included in our first cohort.
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I am confident that Sensorion's differentiated clinical approach will set new standards in the field of gene therapy for otoferlin deficiency as the study has indeed been designed to assess whether SENS-501 can demonstrate not only hearing restoration in a very homogeneous population of infants and toddlers but also enable language acquisition and development, and the resulting significant improvements in quality of life. I would like to reiterate my gratitude to the parents of the treated patients for their trust, and to the participating investigators for their ongoing commitment to the Audiogene study and conviction in the potential of SENS-501 to create a new treatment paradigm for this debilitating form of deafness."
Professor Natalie Loundon, M.D., Director of the Center for Research in Pediatric Audiology, Pediatric Otolaryngologist and Head and Neck Surgeon, Necker Enfants Malades, AP-HP, in Paris, France, Coordinating Investigator of the Audiogene clinical study, commented: "The preliminary results provide satisfactory data on the safety of SENS-501 for patients. Gene therapy represents real hope for a therapeutic treatment and improvement hearing, speech acquisition and quality of life for children born deaf due to DFNB9 mutations. I look forward to the next data update on Audiogene's first cohort, where patients will have received the lowest dose investigated for the restoration of hearing in DFNB9 patients and I'm excited to continue our work on the Audiogene clinical study."
About the Audiogene Trial
Audiogene aims to evaluate the safety, tolerability and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in infants and toddlers aged 6 to 31 months at the time of gene therapy treatment. By targeting the first years of life, when brain plasticity is optimal, the chances of these young children with pre-linguistic hearing loss acquiring normal speech and language are maximized. The study comprises two cohorts of two doses followed by an expansion cohort at the selected dose. While safety will be the primary endpoint of the first part of the dose escalation study, auditory brainstem response (ABR) will be the primary efficacy endpoint of the second part of the expansion. Audiogene will also evaluate the clinical safety, performance and ease-of-use of the delivery system developed by Sensorion.
About SENS-501
SENS-501 (OTOF-GT) is an innovative gene therapy program developed to treat a specific form of congenital deafness linked to mutations in the OTOF (otoferlin) gene. This gene plays a key role in the transmission of auditory signals between the hair cells of the inner ear and the auditory nerve. When this gene is defective, affected individuals are born with severe to profound hearing loss.
The aim of SENS-501 (OTOF-GT) is to restore hearing by introducing a functional copy of the OTOF gene directly into hair cells via viral vector technology (AAV). By replacing the defective gene, this therapy aims to restore the normal process of converting sound into electrical signals, enabling patients to regain their hearing ability. Currently in clinical research phase, this gene therapy program represents significant hope for families affected by this rare form of genetic deafness. SENS-501 (OTOF-GT) embodies a commitment to scientific innovation in the field of hearing, with the potential to dramatically improve the quality of life of patients suffering from genetic deafness.
This gene therapy for patients suffering from otoferlin deficiency has been developed in the framework of RHU AUDINNOVE, a consortium composed of Sensorion with the Necker Enfants Malades Hospital, the Institut Pasteur, and the Fondation pour l'Audition. The project is partially financed by the French National Research Agency, through the "investing for the future" program (ref: ANR-18-RHUS-0007).
About Sensorion
Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates.
It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses.
Sensorion's portfolio also comprises clinical-stage small molecule programs for the treatment and prevention of hearing loss disorders. Sensorion's clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) progressing in a planned Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner Cochlear Limited, has completed in a study of SENS-401 in patients scheduled for cochlear implantation. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022.
www.Sensorion.Com
Label: SENSORIONISIN: FR0012596468Mnemonic: ALSEN
Disclaimer
This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2023 full year report published on March 14, 2024, and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.
View source version on businesswire.Com: https://www.Businesswire.Com/news/home/20241217687410/en/
Contacts
Investor RelationsNoémie Djokovic, Investor Relations and Communication Associateir.Contact@sensorion-pharma.Com
Press RelationsUlysse CommunicationBruno Arabian / 00 33(0)6 87 88 47 26barabian@ulysse-communication.ComNicolas Entz / 00 33 (0)6 33 67 31 54nentz@ulysse-communication.Com
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