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Iovance Biotherapeutics Completes Biologics License Application (BLA) Submission for Lifileucel in Advanced Melanoma

Globenewswire | March 27, 2023 | Read time : 02:00 min

Iovance Biotherapeutics Completes

Iovance Biotherapeutics, Inc. a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, announced it has completed its rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for lifileucel. Lifileucel is a tumor infiltrating lymphocyte (TIL) therapy intended as a treatment for patients with advanced melanoma who progressed on or after prior anti-PD-1/L1 therapy and targeted therapy, where applicable. There are no FDA approved therapies in this treatment setting.

Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, “Completing our BLA submission for lifileucel is a critical step forward in our journey to deliver the first individualized, one-time cell therapy for a solid tumor. I would like to acknowledge the patients and physicians who participated in the C-144-01 clinical trial and the FDA review team for their commitment and support, as well as our internal team for their tremendous effort in completing the first BLA submission for Iovance. Our preparations for commercialization remain on track to support a launch later this year. We look forward to continued collaboration with the FDA as they review this new class of treatment for advanced melanoma patients with limited options.”

The BLA submission for lifileucel is supported by positive clinical data from the C-144-01 clinical trial in patients with advanced post-anti-PD1 melanoma. Following a successful pre-BLA meeting with the FDA, Iovance is pursuing accelerated approval in this indication. Iovance also reached agreement with the FDA regarding the registrational trial design for the Phase 3 TILVANCE-301 trial of lifileucel in combination with pembrolizumab in frontline advanced melanoma. TILVANCE-301 is intended to support full approval of lifileucel in post-anti-PD-1 advanced melanoma and is also designed to support registration for lifileucel in combination with pembrolizumab as therapy for advanced melanoma in the frontline setting. Startup activities for TILVANCE-301 are ongoing and the trial is expected to be well underway at the time of potential accelerated approval for lifileucel in advanced post-anti-PD-1 melanoma.

Marc Hurlbert, Ph.D., CEO of the Melanoma Research Alliance (MRA), said, “MRA congratulates Iovance for completing the BLA submission and moving closer toward making TIL therapy an option for people with advanced melanoma who have progressed following prior treatments. We hope for an FDA approval as quickly as possible for patients with significant unmet need who have no approved treatment options.”

Following receipt of the complete rolling BLA submission for lifileucel, the FDA has 60 days to determine the acceptability of the BLA for review. The rolling BLA allowed Iovance to submit portions of the BLA to the FDA on an ongoing basis, enabling the FDA to begin review as early as possible as documents were received. The rolling BLA submission and eligibility for priority review are benefits available under the FDA’s guidance on expedited programs for serious conditions, which allow for an expedited six-month review from the time of BLA acceptance. In addition, the FDA previously granted a regenerative medicine advanced therapy (RMAT) designation for lifileucel in advanced melanoma.

About Iovance Biotherapeutics, Inc.

Iovance Biotherapeutics aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. Our lead late-stage TIL product candidate, lifileucel for metastatic melanoma, has the potential to become the first approved one-time cell therapy for a solid tumor cancer. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer.

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Poseida Therapeutics Announces FDA Clearance of Investigational New Drug Application for P-CD19CD20-ALLO1, an Allogeneic Dual CAR-T Cell Therapy

PRNewswire | July 10, 2023

Poseida Therapeutics, Inc. a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for P-CD19CD20-ALLO1, the Company's first allogeneic dual CAR-T cell product candidate, which targets both CD19 and CD20 antigens for the treatment of relapsed or refractory B-cell malignancies and is being developed in partnership with Roche. "We are pleased to receive IND clearance for P-CD19CD20-ALLO1, our third fully allogeneic CAR-T cell product candidate and the second therapy in our partnership with Roche for hematological malignancies that will enter the clinic. We believe this represents the FDA's first known IND clearance of an allogeneic dual CAR-T therapy targeting CD19 and CD20," said Kristin Yarema, Ph.D., President, Cell Therapy at Poseida. "The dual-targeting approach leverages our proprietary non-viral piggyBac® DNA Delivery System, which enables expression of two fully functional CAR molecules into T cells from healthy donors for the treatment of B-cell malignancies that may have heterogeneous antigen expression. We believe that targeting both CD19 and CD20 has the potential to overcome the limitations of currently available CD19-directed CAR-T products where antigen escape has been observed as an important resistance mechanism. It has been estimated that up to 40% of cases where B-cell malignancies relapse or are refractory to CD19 targeting autologous CAR-T therapy may involve antigen escape. We look forward to dosing the first patients in this study." P-CD19CD20-ALLO1 will be evaluated in a Phase 1 multi-center, open-label, dose-escalation study that will enroll up to 70 adult patients with relapsed or refractory B-cell malignancies. The study will evaluate the safety, tolerability, and preliminary efficacy of P-CD19CD20-ALLO1. After enrollment, patients will receive a chemotherapy-based lymphodepletion regimen followed by administration of P-CD19CD20-ALLO1 allogeneic CAR-T cells. With the P-CD19CD20-ALLO1 IND now cleared, the Company is actively focused on opening clinical sites. About P-CD19CD20-ALLO1 P-CD19CD20-ALLO1 is an allogeneic CAR-T cell therapy product candidate being developed for relapsed or refractory B-cell malignancies in partnership with Roche. P-CD19CD20-ALLO1 expresses two fully functional CAR molecules to target cells that express either CD19 or CD20. The dual targeting approach employed in P-CD19CD20-ALLO1 aims to overcome the antigen escape limitations of CD19-only targeted CAR-T therapies by simultaneously targeting both CD19 and CD20. In addition to the dual targeting, P-CD19CD20-ALLO1 uses a novel CD19 binder that showed greater potency in in vivo preclinical models when compared to the canonical FMC63 Single-chain variable fragment (scFv) binder. P-CD19CD20-ALLO1 is an off-the-shelf CAR-T therapy for which patients do not have to undergo apheresis and wait for the cells to be manufactured, which can potentially overcome the limitation of autologous CAR-T therapies associated with significant manufacturing times. P-CD19CD20-ALLO1 will be studied in multiple B-cell malignancies About Poseida Therapeutics, Inc. Poseida Therapeutics is a clinical-stage biopharmaceutical company advancing differentiated cell and gene therapies with the capacity to cure certain cancers and rare diseases. The Company's pipeline includes allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors as well as in vivo gene therapy product candidates that address patient populations with high unmet medical need. The Company's approach to cell and gene therapies is based on its proprietary genetic editing platforms, including its non-viral piggyBac® DNA Delivery System, Cas-CLOVER™ Site-Specific Gene Editing System and nanoparticle and hybrid gene delivery technologies.

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Medical, Diagnostics

Aspen Neuroscience to Partner with Rune Labs and Emerald Innovations to Incorporate both Active and Passive Digital Health Monitoring

prnewswire | August 28, 2023

Aspen Neuroscience today announced that it is partnering with Emerald Innovations, a pioneer in the creation of "invisible" off-body sensors for measuring health analytics, and Rune Labs, a software and data analytics company for precision neurology, to incorporate digital health (DHT) modalities in the company's Trial-Ready Screening Cohort Study. The Trial Ready Screening Cohort study was launched in 2022 to screen, enroll and begin manufacturing cells for potential patient candidates for the future Phase 1/2a clinical trial for ANPD001, a personalized (autologous) cell therapy to treat Parkinson's disease (PD) by replacing lost dopamine neurons. Aspen Neuroscience is advancing the collection of objective measures of motor function via passive in-home monitoring, working with Emerald Innovations' contactless biosensors, and active monitoring, working with Rune Labs' clinical development platform, StriveStudy. The use of DHT empowers trial investigators to include objective, long-term (pre-treatment) motor and sleep symptom capture, to provide data on each patient's rate of disease progression. "Parkinson's disease is very personal in nature, and we are working to develop a personal cell therapy for PD," said Damien McDevitt, PhD, Aspen president and CEO. "Everyone with Parkinson's has a unique experience, with varied symptoms. We look forward to working closely with the innovative and collaborative teams at Rune Labs and Emerald Innovations to capture a holistic and precise pattern of motor symptom activity over time for people in our screening study." "Parkinson's is a heterogeneous condition, adding to the challenge of assessing treatment benefit without a personalized view of each trial participant's symptom activity," said Brian Pepin, CEO of Rune Labs. "Through StriveStudy, our biopharma partners are able to collect large quantities of continuous multimodal data to characterize participant's disease activity across a range of clinical features. We are excited to collaborate with Aspen Neuroscience to help them characterize patient symptoms as they bring their personalized cell therapy into clinical testing." "We are delighted to collaborate with Aspen Neuroscience," said Dr. Dina Katabi, MIT professor and the co-founder and president of Emerald Innovations, Inc. "A significant hurdle in Parkinson's disease clinical trials is the reliable monitoring of disease progression and medication response without the need to enroll hundreds or even thousands of patients. Empirical evidence has demonstrated that readings from the Emerald sensor offer a dependable marker for tracking disease progression. The Emerald sensor gathers this data without body contact, relying solely on the analysis of radio signals in the environment as patients go about their normal lives." About DHT in the Trial Ready Cohort Screening Study Aspen will partner with Rune Labs to deeply characterize each patient's symptoms in the Trial Ready Cohort, including the collection of multimodal data, thereby enabling robust disease characterization over time. This will include objective measures of tremor and dyskinesia levels captured on FDA-cleared StrivePD on the Apple Watch, medication monitoring, as well as Apple HealthKit, including global activity (e.g., daily steps). Aspen and Emerald will collaborate to assess passive "invisible" in-home monitoring of participants enrolled in the Trial Ready Cohort. This will include gait speed measured across walking intervals, sleep onset, the amount of time between entering different sleep stages; sleep duration, sleep efficiency and WASO: the total time spent in the WAKE stage after sleep onset. About Parkinson's Disease Parkinson's disease is the world's second most common neurodegenerative disease, affecting more than 10 million people worldwide, including more than 1 million in the U.S., where an additional 90,000+ people are diagnosed every year. Patients can experience non-motor symptoms such as mental and behavioral changes, sleep problems, depression, memory difficulties and fatigue; and motor symptoms caused by the loss of dopamine neurons in the brain, including unintended or uncontrollable movements (dyskinesia), shaking (tremor), stiffness, and difficulty with balance and coordination, which worsen over time. By the time of diagnosis, it is common for people to have lost the majority of cells in the brain that make dopamine. To date, there is no disease-modifying therapy that can stop, replace or prevent the loss of dopamine neurons or slow the progression of PD. About Aspen Neuroscience Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on personalized (autologous) regenerative medicine. The company is developing patient-derived iPSCs to create personalized cell therapies that address diseases with high unmet medical needs, beginning with autologous neuron replacement for Parkinson's disease and extending across the brain and affected organs. A leading iPSC platform company, Aspen combines stem cell biology with the latest artificial intelligence and genomic approaches to investigate patient-specific, restorative treatments. The company has developed a best-in-class platform to create and optimize iPSC-derived cell therapies, which includes in-house bioinformatics, manufacturing and QC. About Emerald Innovations, Inc. Emerald Innovations is a health analytics company that specializes in digital health solutions for passive, contactless in-home monitoring. Emerald seamlessly captures continuous health data from patients as they go about their normal lives. The Emerald biosensors fall under a cutting-edge category known as "Invisibles." Installed in the homes of study participants, these sensors continuously analyze surrounding radio signals. Using artificial intelligence, they extract patients' physiological data while prioritizing their privacy. Emerald simplifies clinical trials for its clients by obtaining statistically significant biomarkers, making them readily available to clinicians and data analysts, and consistently providing updates on the progress of clinical trials. Emerald's clientele encompasses big pharma, Fortune 500 pharmaceutical firms, innovative biotech companies, and leading disease research foundations. About Rune Labs Rune Labs is a software and data analytics company for precision neurology, supporting care delivery and therapy development. StrivePD is the company's care delivery ecosystem for Parkinson's disease, enabling patients and clinicians to better manage Parkinson's by providing access to curated dashboards summarizing a range of patient data sources, and by connecting patients to clinical trials. For therapeutics development, biopharma and medical device companies leverage Rune's technology, network of engaged clinicians and patients, and large longitudinal real-world datasets to expedite development programs. The company has received financial backing from leading investors such as Eclipse Ventures, DigiTx, TruVenturo and Moment Ventures. For more information, please visit runelabs.io and strive.group.

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MedTech, Industry Outlook

Vaxxinity Demonstrates Target Engagement of Toxic Alpha-Synuclein in Parkinson’s Patients

Globenewswire | July 18, 2023

Vaxxinity, Inc. a U.S. company pioneering the development of a new class of medicines, announced new data from a Phase 1 clinical trial demonstrating that antibodies derived from its investigational immunotherapeutic for Parkinson’s disease (PD), UB-312, slows seeding of alpha-synuclein (aSyn) in cerebrospinal fluid (CSF) of patients with PD as demonstrated using multiple target engagement assays. These data signify that UB-312 has established clear target engagement in PD patient CSF, and provides further validation of Vaxxinity’s platform technology in neurodegenerative disease. “This is a major milestone for Vaxxinity in our quest to help Parkinson’s patients. Our candidate has shown target engagement of the toxic species of alpha-synuclein in patients, demonstrating not only proof of our technology platform, but also proof of the mechanism of our vaccine-derived antibodies specifically engaging with the toxic target in vivo,” said Mei Mei Hu, CEO of Vaxxinity. “Showing target engagement has always been a key challenge to overcome in neurodegeneration, and is of critical importance when demonstrated – a milestone worth celebrating. It is beyond our expectation to see this in our Phase 1 trial. We are endlessly grateful to the patients who participated, and to The Michael J. Fox Foundation and our collaborators for their work on these cutting-edge assays that supported this breakthrough.” UB-312 is designed to target aggregated forms of aSyn, the toxic species that underlies Parkinson’s disease and other synucleinopathies. Last month, Vaxxinity announced clinical data from Part B of its Phase 1 clinical trial of UB-312 demonstrating that UB-312 was well-tolerated and induced anti-aSyn antibody responses in participants with early PD, and that antibodies were detectable in the CSF. As part of this trial, The Michael J. Fox Foundation (MJFF) funded a 2-year collaborative project between Vaxxinity, the Mayo Clinic, and UTHealth Houston to analyze CSF collected from patients, and to conduct exploratory research to characterize the anti-aSyn antibodies produced after UB-312 administration and assess target engagement. Analyses from this and related research yielded insights about the pharmacodynamic effects of anti-aSyn antibodies generated by UB-312 in the Phase 1 trial. UB-312-derived antibodies show preferential binding to aggregated aSyn isolated from patients with PD and Multiple System Atrophy (MSA), as measured by dot blot. Preclinical data published in Alzheimer’s Research & Therapy in 2020 showed similar characteristics of UB-312-derived antibodies. UB-312-derived antibodies successfully demonstrate inhibition of aggregation of aSyn in both a seed amplification assay (SAA) and a protein misfolding cyclic amplification assay (PMCA). These techniques can potentially be used to identify people with PD, and also to measure the treatment response and pharmacodynamic properties of UB-312-derived antibodies from subjects in clinical trials. Importantly, aSyn aggregation was slowed down in CSF samples from PD patients who received UB-312, as compared to those who received placebo, in the Phase 1 trial. Vaxxinity plans to continue analyses of the clinical data as part of the collaborative project with MJFF, in addition to completing other target engagement assays and additional antibody characterization studies for binding kinetics and specificity. Mark Frasier, Ph.D., Chief Scientific Officer of MJFF, commented, “Integration of critical biomarker insight into therapeutic development programs is essential for building confidence in the treatment approach, and for designing informative trials. We’re pleased to support efforts of this kind that can have major impact for people with Parkinson’s disease.” About Parkinson’s Disease Parkinson’s disease (PD) affects approximately one million people in the United States and more than 10 million people worldwide. PD is a chronic and progressive neurodegenerative disorder that affects predominately dopamine-producing (“dopaminergic”) neurons in the substantia nigra area of the brain. While today’s approved products are aimed at providing symptomatic relief, they often produce significant side effects and lose their beneficial effects over time. There are no currently approved disease-modifying therapeutics for PD. Alpha-synuclein (aSyn) is a protein highly expressed in neurons, mostly at presynaptic terminals, suggesting a role in synaptic vesicle trafficking, synaptic functions and in regulation of neurotransmitter release at the synapse. Mutations in the gene encoding aSyn are known to cause or increase the risk of developing PD or dementia with Lewy bodies (DLB) and have been shown to alter the secondary structure of aSyn, resulting in misfolded and aggregated forms of the protein (i.e., pathological forms). While mutations in the aSyn gene are rare, aggregates of aSyn in the form of Lewy bodies (LB) and Lewy neurites are common neuropathological hallmarks of both familial and sporadic PD, suggesting a key role of aSyn in PD neuropathogenesis. Immunotherapy approaches targeting aSyn have been shown to ameliorate aSyn pathology as well as functional deficits in mouse models of PD and are now being investigated in the clinic. About UB-312 UB-312 is a vaccine candidate targeting pathological forms of alpha-synuclein (aSyn) for the disease-modifying treatment and prevention of Parkinson’s disease (PD) and other synucleinopathies. Preclinical data indicated that UB-312 elicits antibodies that preferentially recognize pathological forms of aSyn, and improve motor performance in mouse models of synucleinopathies. Clinical data from the Phase 1 trial indicate that UB-312 elicits antibodies that target aggregated aSyn, and that these antibodies slow the aggregation of alpha-synuclein in the cerebrospinal fluid of patients with PD. The European Medical Agency has granted UB-312 orphan designation for multiple system atrophy. About Vaxxinity Vaxxinity, Inc. is a purpose-driven biotechnology company committed to democratizing healthcare across the globe. The company is pioneering a new class of medicines aimed at disrupting the existing treatment paradigm for chronic disease, increasingly dominated by monoclonal antibodies, which suffer from prohibitive costs and cumbersome administration. The company’s proprietary technology platform has enabled the innovation of novel synthetic peptide immunotherapy candidates designed to bring the efficiency of vaccines to the treatment of chronic diseases, including Alzheimer’s disease, Parkinson’s disease, migraine, and hypercholesterolemia. The technology is also implemented as part of a COVID-19 vaccine program. Vaxxinity has optimized its pipeline to achieve a potentially historic, global impact on human health.

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Industry Outlook

Poseida Therapeutics Announces FDA Clearance of Investigational New Drug Application for P-CD19CD20-ALLO1, an Allogeneic Dual CAR-T Cell Therapy

PRNewswire | July 10, 2023

Poseida Therapeutics, Inc. a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for P-CD19CD20-ALLO1, the Company's first allogeneic dual CAR-T cell product candidate, which targets both CD19 and CD20 antigens for the treatment of relapsed or refractory B-cell malignancies and is being developed in partnership with Roche. "We are pleased to receive IND clearance for P-CD19CD20-ALLO1, our third fully allogeneic CAR-T cell product candidate and the second therapy in our partnership with Roche for hematological malignancies that will enter the clinic. We believe this represents the FDA's first known IND clearance of an allogeneic dual CAR-T therapy targeting CD19 and CD20," said Kristin Yarema, Ph.D., President, Cell Therapy at Poseida. "The dual-targeting approach leverages our proprietary non-viral piggyBac® DNA Delivery System, which enables expression of two fully functional CAR molecules into T cells from healthy donors for the treatment of B-cell malignancies that may have heterogeneous antigen expression. We believe that targeting both CD19 and CD20 has the potential to overcome the limitations of currently available CD19-directed CAR-T products where antigen escape has been observed as an important resistance mechanism. It has been estimated that up to 40% of cases where B-cell malignancies relapse or are refractory to CD19 targeting autologous CAR-T therapy may involve antigen escape. We look forward to dosing the first patients in this study." P-CD19CD20-ALLO1 will be evaluated in a Phase 1 multi-center, open-label, dose-escalation study that will enroll up to 70 adult patients with relapsed or refractory B-cell malignancies. The study will evaluate the safety, tolerability, and preliminary efficacy of P-CD19CD20-ALLO1. After enrollment, patients will receive a chemotherapy-based lymphodepletion regimen followed by administration of P-CD19CD20-ALLO1 allogeneic CAR-T cells. With the P-CD19CD20-ALLO1 IND now cleared, the Company is actively focused on opening clinical sites. About P-CD19CD20-ALLO1 P-CD19CD20-ALLO1 is an allogeneic CAR-T cell therapy product candidate being developed for relapsed or refractory B-cell malignancies in partnership with Roche. P-CD19CD20-ALLO1 expresses two fully functional CAR molecules to target cells that express either CD19 or CD20. The dual targeting approach employed in P-CD19CD20-ALLO1 aims to overcome the antigen escape limitations of CD19-only targeted CAR-T therapies by simultaneously targeting both CD19 and CD20. In addition to the dual targeting, P-CD19CD20-ALLO1 uses a novel CD19 binder that showed greater potency in in vivo preclinical models when compared to the canonical FMC63 Single-chain variable fragment (scFv) binder. P-CD19CD20-ALLO1 is an off-the-shelf CAR-T therapy for which patients do not have to undergo apheresis and wait for the cells to be manufactured, which can potentially overcome the limitation of autologous CAR-T therapies associated with significant manufacturing times. P-CD19CD20-ALLO1 will be studied in multiple B-cell malignancies About Poseida Therapeutics, Inc. Poseida Therapeutics is a clinical-stage biopharmaceutical company advancing differentiated cell and gene therapies with the capacity to cure certain cancers and rare diseases. The Company's pipeline includes allogeneic CAR-T cell therapy product candidates for both solid and liquid tumors as well as in vivo gene therapy product candidates that address patient populations with high unmet medical need. The Company's approach to cell and gene therapies is based on its proprietary genetic editing platforms, including its non-viral piggyBac® DNA Delivery System, Cas-CLOVER™ Site-Specific Gene Editing System and nanoparticle and hybrid gene delivery technologies.

Read More

Medical, Diagnostics

Aspen Neuroscience to Partner with Rune Labs and Emerald Innovations to Incorporate both Active and Passive Digital Health Monitoring

prnewswire | August 28, 2023

Aspen Neuroscience today announced that it is partnering with Emerald Innovations, a pioneer in the creation of "invisible" off-body sensors for measuring health analytics, and Rune Labs, a software and data analytics company for precision neurology, to incorporate digital health (DHT) modalities in the company's Trial-Ready Screening Cohort Study. The Trial Ready Screening Cohort study was launched in 2022 to screen, enroll and begin manufacturing cells for potential patient candidates for the future Phase 1/2a clinical trial for ANPD001, a personalized (autologous) cell therapy to treat Parkinson's disease (PD) by replacing lost dopamine neurons. Aspen Neuroscience is advancing the collection of objective measures of motor function via passive in-home monitoring, working with Emerald Innovations' contactless biosensors, and active monitoring, working with Rune Labs' clinical development platform, StriveStudy. The use of DHT empowers trial investigators to include objective, long-term (pre-treatment) motor and sleep symptom capture, to provide data on each patient's rate of disease progression. "Parkinson's disease is very personal in nature, and we are working to develop a personal cell therapy for PD," said Damien McDevitt, PhD, Aspen president and CEO. "Everyone with Parkinson's has a unique experience, with varied symptoms. We look forward to working closely with the innovative and collaborative teams at Rune Labs and Emerald Innovations to capture a holistic and precise pattern of motor symptom activity over time for people in our screening study." "Parkinson's is a heterogeneous condition, adding to the challenge of assessing treatment benefit without a personalized view of each trial participant's symptom activity," said Brian Pepin, CEO of Rune Labs. "Through StriveStudy, our biopharma partners are able to collect large quantities of continuous multimodal data to characterize participant's disease activity across a range of clinical features. We are excited to collaborate with Aspen Neuroscience to help them characterize patient symptoms as they bring their personalized cell therapy into clinical testing." "We are delighted to collaborate with Aspen Neuroscience," said Dr. Dina Katabi, MIT professor and the co-founder and president of Emerald Innovations, Inc. "A significant hurdle in Parkinson's disease clinical trials is the reliable monitoring of disease progression and medication response without the need to enroll hundreds or even thousands of patients. Empirical evidence has demonstrated that readings from the Emerald sensor offer a dependable marker for tracking disease progression. The Emerald sensor gathers this data without body contact, relying solely on the analysis of radio signals in the environment as patients go about their normal lives." About DHT in the Trial Ready Cohort Screening Study Aspen will partner with Rune Labs to deeply characterize each patient's symptoms in the Trial Ready Cohort, including the collection of multimodal data, thereby enabling robust disease characterization over time. This will include objective measures of tremor and dyskinesia levels captured on FDA-cleared StrivePD on the Apple Watch, medication monitoring, as well as Apple HealthKit, including global activity (e.g., daily steps). Aspen and Emerald will collaborate to assess passive "invisible" in-home monitoring of participants enrolled in the Trial Ready Cohort. This will include gait speed measured across walking intervals, sleep onset, the amount of time between entering different sleep stages; sleep duration, sleep efficiency and WASO: the total time spent in the WAKE stage after sleep onset. About Parkinson's Disease Parkinson's disease is the world's second most common neurodegenerative disease, affecting more than 10 million people worldwide, including more than 1 million in the U.S., where an additional 90,000+ people are diagnosed every year. Patients can experience non-motor symptoms such as mental and behavioral changes, sleep problems, depression, memory difficulties and fatigue; and motor symptoms caused by the loss of dopamine neurons in the brain, including unintended or uncontrollable movements (dyskinesia), shaking (tremor), stiffness, and difficulty with balance and coordination, which worsen over time. By the time of diagnosis, it is common for people to have lost the majority of cells in the brain that make dopamine. To date, there is no disease-modifying therapy that can stop, replace or prevent the loss of dopamine neurons or slow the progression of PD. About Aspen Neuroscience Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on personalized (autologous) regenerative medicine. The company is developing patient-derived iPSCs to create personalized cell therapies that address diseases with high unmet medical needs, beginning with autologous neuron replacement for Parkinson's disease and extending across the brain and affected organs. A leading iPSC platform company, Aspen combines stem cell biology with the latest artificial intelligence and genomic approaches to investigate patient-specific, restorative treatments. The company has developed a best-in-class platform to create and optimize iPSC-derived cell therapies, which includes in-house bioinformatics, manufacturing and QC. About Emerald Innovations, Inc. Emerald Innovations is a health analytics company that specializes in digital health solutions for passive, contactless in-home monitoring. Emerald seamlessly captures continuous health data from patients as they go about their normal lives. The Emerald biosensors fall under a cutting-edge category known as "Invisibles." Installed in the homes of study participants, these sensors continuously analyze surrounding radio signals. Using artificial intelligence, they extract patients' physiological data while prioritizing their privacy. Emerald simplifies clinical trials for its clients by obtaining statistically significant biomarkers, making them readily available to clinicians and data analysts, and consistently providing updates on the progress of clinical trials. Emerald's clientele encompasses big pharma, Fortune 500 pharmaceutical firms, innovative biotech companies, and leading disease research foundations. About Rune Labs Rune Labs is a software and data analytics company for precision neurology, supporting care delivery and therapy development. StrivePD is the company's care delivery ecosystem for Parkinson's disease, enabling patients and clinicians to better manage Parkinson's by providing access to curated dashboards summarizing a range of patient data sources, and by connecting patients to clinical trials. For therapeutics development, biopharma and medical device companies leverage Rune's technology, network of engaged clinicians and patients, and large longitudinal real-world datasets to expedite development programs. The company has received financial backing from leading investors such as Eclipse Ventures, DigiTx, TruVenturo and Moment Ventures. For more information, please visit runelabs.io and strive.group.

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MedTech, Industry Outlook

Vaxxinity Demonstrates Target Engagement of Toxic Alpha-Synuclein in Parkinson’s Patients

Globenewswire | July 18, 2023

Vaxxinity, Inc. a U.S. company pioneering the development of a new class of medicines, announced new data from a Phase 1 clinical trial demonstrating that antibodies derived from its investigational immunotherapeutic for Parkinson’s disease (PD), UB-312, slows seeding of alpha-synuclein (aSyn) in cerebrospinal fluid (CSF) of patients with PD as demonstrated using multiple target engagement assays. These data signify that UB-312 has established clear target engagement in PD patient CSF, and provides further validation of Vaxxinity’s platform technology in neurodegenerative disease. “This is a major milestone for Vaxxinity in our quest to help Parkinson’s patients. Our candidate has shown target engagement of the toxic species of alpha-synuclein in patients, demonstrating not only proof of our technology platform, but also proof of the mechanism of our vaccine-derived antibodies specifically engaging with the toxic target in vivo,” said Mei Mei Hu, CEO of Vaxxinity. “Showing target engagement has always been a key challenge to overcome in neurodegeneration, and is of critical importance when demonstrated – a milestone worth celebrating. It is beyond our expectation to see this in our Phase 1 trial. We are endlessly grateful to the patients who participated, and to The Michael J. Fox Foundation and our collaborators for their work on these cutting-edge assays that supported this breakthrough.” UB-312 is designed to target aggregated forms of aSyn, the toxic species that underlies Parkinson’s disease and other synucleinopathies. Last month, Vaxxinity announced clinical data from Part B of its Phase 1 clinical trial of UB-312 demonstrating that UB-312 was well-tolerated and induced anti-aSyn antibody responses in participants with early PD, and that antibodies were detectable in the CSF. As part of this trial, The Michael J. Fox Foundation (MJFF) funded a 2-year collaborative project between Vaxxinity, the Mayo Clinic, and UTHealth Houston to analyze CSF collected from patients, and to conduct exploratory research to characterize the anti-aSyn antibodies produced after UB-312 administration and assess target engagement. Analyses from this and related research yielded insights about the pharmacodynamic effects of anti-aSyn antibodies generated by UB-312 in the Phase 1 trial. UB-312-derived antibodies show preferential binding to aggregated aSyn isolated from patients with PD and Multiple System Atrophy (MSA), as measured by dot blot. Preclinical data published in Alzheimer’s Research & Therapy in 2020 showed similar characteristics of UB-312-derived antibodies. UB-312-derived antibodies successfully demonstrate inhibition of aggregation of aSyn in both a seed amplification assay (SAA) and a protein misfolding cyclic amplification assay (PMCA). These techniques can potentially be used to identify people with PD, and also to measure the treatment response and pharmacodynamic properties of UB-312-derived antibodies from subjects in clinical trials. Importantly, aSyn aggregation was slowed down in CSF samples from PD patients who received UB-312, as compared to those who received placebo, in the Phase 1 trial. Vaxxinity plans to continue analyses of the clinical data as part of the collaborative project with MJFF, in addition to completing other target engagement assays and additional antibody characterization studies for binding kinetics and specificity. Mark Frasier, Ph.D., Chief Scientific Officer of MJFF, commented, “Integration of critical biomarker insight into therapeutic development programs is essential for building confidence in the treatment approach, and for designing informative trials. We’re pleased to support efforts of this kind that can have major impact for people with Parkinson’s disease.” About Parkinson’s Disease Parkinson’s disease (PD) affects approximately one million people in the United States and more than 10 million people worldwide. PD is a chronic and progressive neurodegenerative disorder that affects predominately dopamine-producing (“dopaminergic”) neurons in the substantia nigra area of the brain. While today’s approved products are aimed at providing symptomatic relief, they often produce significant side effects and lose their beneficial effects over time. There are no currently approved disease-modifying therapeutics for PD. Alpha-synuclein (aSyn) is a protein highly expressed in neurons, mostly at presynaptic terminals, suggesting a role in synaptic vesicle trafficking, synaptic functions and in regulation of neurotransmitter release at the synapse. Mutations in the gene encoding aSyn are known to cause or increase the risk of developing PD or dementia with Lewy bodies (DLB) and have been shown to alter the secondary structure of aSyn, resulting in misfolded and aggregated forms of the protein (i.e., pathological forms). While mutations in the aSyn gene are rare, aggregates of aSyn in the form of Lewy bodies (LB) and Lewy neurites are common neuropathological hallmarks of both familial and sporadic PD, suggesting a key role of aSyn in PD neuropathogenesis. Immunotherapy approaches targeting aSyn have been shown to ameliorate aSyn pathology as well as functional deficits in mouse models of PD and are now being investigated in the clinic. About UB-312 UB-312 is a vaccine candidate targeting pathological forms of alpha-synuclein (aSyn) for the disease-modifying treatment and prevention of Parkinson’s disease (PD) and other synucleinopathies. Preclinical data indicated that UB-312 elicits antibodies that preferentially recognize pathological forms of aSyn, and improve motor performance in mouse models of synucleinopathies. Clinical data from the Phase 1 trial indicate that UB-312 elicits antibodies that target aggregated aSyn, and that these antibodies slow the aggregation of alpha-synuclein in the cerebrospinal fluid of patients with PD. The European Medical Agency has granted UB-312 orphan designation for multiple system atrophy. About Vaxxinity Vaxxinity, Inc. is a purpose-driven biotechnology company committed to democratizing healthcare across the globe. The company is pioneering a new class of medicines aimed at disrupting the existing treatment paradigm for chronic disease, increasingly dominated by monoclonal antibodies, which suffer from prohibitive costs and cumbersome administration. The company’s proprietary technology platform has enabled the innovation of novel synthetic peptide immunotherapy candidates designed to bring the efficiency of vaccines to the treatment of chronic diseases, including Alzheimer’s disease, Parkinson’s disease, migraine, and hypercholesterolemia. The technology is also implemented as part of a COVID-19 vaccine program. Vaxxinity has optimized its pipeline to achieve a potentially historic, global impact on human health.

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