Neuroplast BV

16 patients have been enrolled in a multicenter international, randomized, placebo-controlled, double-blinded Phase II clinical trial Goal: to determine the effect size of Neuro-Cells ® , a stem-cell treatment for Traumatic Spinal Cord Injury (TSCI) Confirmed excellent safety and tolerability of Neuro-Cells® Preclinical evidence showcases Neuro-Cells ® potential in other indications, including traumatic brain injury Primary outcomes expected in February 2024, full study completion projected in August 2024 GELEEN, The Netherlands, Sept. 7, 2023 /PRNewswire/ -- Neuroplast, a Dutch clinical-stage biotech, focusing on cell-based treatments for neurodegenerative diseases, has successfully completed patient inclusion of its Phase II randomized placebo-controlled double-blinded clinical trial to evaluate the effect size of Neuro-Cells ® for Traumatic Spinal Cord Injury (TSCI). Preliminary data indicate an excellent safety profile due to the complete absence of product related adverse events. Furthermore, p atient feedback confirms excellent tolerability. The trial is conducted in collaboration with Hospital Nacional de Parapléjicos in Toledo, Spain, and Rigshospitalet in Copenhagen, Denmark. The Neuro-Cells® technology platform uses the patient's own bone marrow to create a stem cell treatment that modulates inflammation and improves regeneration potential in the central nervous system. Neuro-Cells® are administered intrathecally to patients in the sub-acute stage. This autologous treatment aims to preserve and restore function, mobility, and therefore independence. Double-blinded, randomized, placebo-controlled multi-center study The trial was conducted by Principal Investigators Antonio Oliviero, MD, PhD and Prof. Jörg Mey from Hospital Parapléjicos in Toledo, Spain, as well as Professor Fin Biering-Sørensen and MD, PhD Claus Andersen from Rigshospitalet in Copenhagen, Denmark. The study is a randomized, double-blinded and placebo-controlled trial, with an early and late intervention cross-over design. The intervention group received Neuro-Cells® in the sub-acute phase after sustaining trauma, with six months follow-up to their primary endpoints. The placebo group received a placebo at first but have been or will be treated with Neuro-Cells® after the initial six-month follow-up period. The multi-faceted follow-up for both groups includes standardized and validated outcome measures on motor and sensory function and multiple blood and cerebrospinal fluid measurements. Next to the absence of product related adverse events, the patients describe the intervention as easy and feasible as it does not require significant changes in daily living or medication intake. Antonio Oliviero, MD, PhD, Principal Investigator at Hospital Nacional de Parapléjicos de Toledo, Spain, states: "In our collaboration with Neuroplast to establish the role of cell transplantation in the recovery of individuals with spinal cord injury, it is great to hear that patients are pleased with the treatment that they experience as easy, feasible and safe." Fin Biering-Sørensen, MD, PhD, Principal investigator at Rigshospitalet, Denmark, adds : "For decades we have been seeking a cure for spinal cord injury. This project may be one step on that road, which is very exciting, due to the fact that so far, we have only been able to treat the symptoms and complications caused by the spinal cord injury." In total, the trial involves 16 patients that were included six to ten weeks after sustaining trauma to the spinal cord. The trial is conducted under official approval from the Spanish and Danish medical ethical committees and competent authorities. Neuroplast CEO Johannes de Munter concludes: "This milestone takes us one step closer to offering an effective treatment for patients suffering from traumatic spinal cord injury. We are especially glad to see further confirmation of the excellent tolerability and safety of our Neuro-Cells® product." Potential relevance to other neurological conditions Preclinical evidence suggests that the Neuro-Cells® treatment may have broader applications in addressing various neurodegenerative diseases. Neuroplast has obtained orphan disease designations for traumatic spinal cord injury and frontotemporal dementia. Ongoing research efforts are actively exploring additional potential applications. Neuroplast is open to discussing investment opportunities aimed at advancing the clinical pathways for addressing a broader range of conditions affecting the central nervous system, including traumatic brain injury. About Traumatic Spinal Cord Injury (TSCI) Acute TSCI causes incurable impairment to the spinal cord, affecting approximately 12,000 people across Europe and 17,000 across the USA annually. The damage or trauma interrupts communication of the brain with the body regions below the site of injury. Spinal cord injuries are mainly caused by accidents and - in most of the cases - result in life-long loss of control of motor functions and sensations. After the primary injury to the spinal cord, a cascade of events leads to progressive loss of tissue which may further deteriorate the patient's prognosis. Current treatment approaches for TSCI are only symptomatic, leaving the underlying pathophysiology unchanged. TSCI has a serious impact on the quality of life of patients, with severe implications on mobility and loss of independence. In addition, TSCI creates a lifetime financial burden for patients, payors, healthcare systems and societies at large. About Neuro-Cells® Neuro-Cells® is a transformative platform treatment under GMP. It contains a non-substantially manipulated bone marrow-derived integrated mixture of stem cells including hematopoietic and mesenchymal stem cells, manufactured from a patient's own bone marrow (donor and receiver are the same person). Inflammatory inducing components and pathogens are removed during this process. About Neuroplast Neuroplast is a Dutch stem cell technology company focusing on fast-track development programs using autologous cell products for treatment of neurodegenerative diseases, with the aim of giving back perspective to people who suffer from those conditions. The company was founded in August 2014 by physician Johannes de Munter and neurologist Erik Wolters. Current funders are Lumana Invest, Brightlands Venture Partners, LIOF and the Netherlands Enterprise Agency. Neuroplast is located in The Netherlands. For more information, please visit About Hospital de Parapléjicos, Toledo, Spain Hospital Nacional de Parapléjicos is the reference public hospital in Spain for the treatment of spinal cord injury, recognized by the Ministry of Health of the Government of Spain. About Rigshospitalet, Copenhagen, Spain Rigshospitalet is one of the two Danish national centers offering highly specialized treatment, rehabilitation and care of individuals with spinal cord lesions. About Lumana Invest Investment company Lumana was established by entrepreneurs and unique due to not having a predetermined investment horizon. The Lumana founders showcase strong commitment to their portfolio companies by actively supporting management in strategic decision making. About Brightlands Venture Partners Brightlands Venture Partners (BVP) is the fund manager of Chemelot Ventures and is a so-called ecosystem investor. BVP invests in companies benefiting from and contributing to the Brightlands campuses in the south of The Netherlands. Other funds under management are BVP Fund IV, Brightlands Agrifood Fund and Limburg Ventures. The funds of BVP focus on sustainability and health; together the funds have made 50 investments. About LIOF LIOF is the regional development agency for Limburg and supports innovative entrepreneurs with advice, network and financing. Together with entrepreneurs and partners, LIOF is working towards a smarter, more sustainable and healthier Limburg by focusing on the transitions of energy, circularity, health and digitalization. About The Netherlands Enterprise Agency The Netherlands Enterprise Agency operates under the auspices of the Dutch Ministry of Economic Affairs and Climate Policy. It facilitates entrepreneurship, improves collaborations, strengthens positions and helps realize national and international ambitions with funding, networking, know-how and compliance with laws and regulations. Forward looking statements All statements other than statements of historical facts, including the statements about the clinical and therapeutic potential and future clinical milestones of Neuro-CellsÒ, the indications we intend to pursue and our possible clinical or other business strategies, and the timing of these events, are forward-looking statements. Forward-looking statements can be identified by terms such as "believes", "expects", "plans", "potential", "would" or similar expressions and the negative of those terms. These forward-looking statements are based on our management's current beliefs and assumptions about future events and on information currently available to management. Neuroplast B.V. does not make any representation or warranty, express or implied, as to the improper use of this article, accuracy, completeness or updated status of above-mentioned statements. Therefore, in no case whatsoever will Neuroplast B.V. be legally liable or liable to anyone for any decision made or action taken in conjunction with the information and/or statements in this press release or for any related damages. In case of any further questions, please contact: Neuroplast Johannes de Munter, CEO T: +31 (0)85 076 1000 E: [email protected] LifeSpring LifeSciences Communication, Amsterdam Leon Melens T: +31 6 538 16 427 E: [email protected] SOURCE Neuroplast

Fast-track status is granted for frontotemporal dementia, next to the existing designation for traumatic spinal cord injury An orphan medicinal product designation or orphan disease designation (ODD) grants the holder a fast-track development pathway to market authorization with market exclusivity This second ODD for Frontotemporal Dementia highlights the broader applicability of Neuro-Cells® for both chronic and trauma-induced neurodegenerative diseases; many of which share underlying disease mechanisms. Stem cell biotech Neuroplast already holds an ODD for Traumatic Spinal Cord Injury for which a Phase II clinical trial is ongoing Further applications of Neuro-Cells® are being explored, with targets on both the chronic- and the trauma-induced spectrum. These include ALS, Alzheimer's, Parkinson's Disease and Traumatic Brain Injury GELEEN, Netherlands, Sept. 8, 2022 /PRNewswire/ -- The European Union has granted stem cell biotech Neuroplast an orphan medicinal product designation for the applicability of its stem cell technology platform to frontotemporal dementia (FTD), following a positive opinion from The European Medicines Agency (EMA). With the existing orphan disease designation (ODD) for traumatic spinal cord injury (TSCI), Neuro-Cells® is now approved for a fast-track development pathway with market exclusivity for both a trauma-induced and a chronic degenerative central nervous system disorder. This marks an important milestone in the development roadmap of Neuroplast's Neuro-Cells® platform, as a stepping stone to other chronic neurodegenerative diseases such as Alzheimer's, ALS and Parkinson's Disease. The potential width in therapeutic applicability of the Neuroplast technology gives perspective to millions of people suffering from neurodegenerative diseases that currently have no outlook on effective treatment. One technology addresses underlying mechanisms of multiple acute and chronic neurological disorders Several conditions of the central nervous system, even when they seem unrelated at first and may have distinctive causes, have similar underlying disease mechanisms in common. These include unprogrammed cell death boosted by inflammation. Neuro-Cells®, an autologous, bone-marrow derived Advanced Therapy Medicinal Product, addresses that disease mechanism by moderating inflammation of damaged cells in the central nervous system, to limit further impairment. The treatment objective in acute disorders is to limit impact of sudden injury, where the treatment objective in chronic disorders is to limit progression of the disease. Neuroplast is already running a fast-track development pathway for traumatic spinal cord injury (TSCI), with a Phase II clinical trial in progress. This designation for frontotemporal dementia illustrates the broader applicability of the same technology for acute as well as chronic neurodegenerative disorders, paving the way to explore further applicability to conditions such as ALS, Alzheimer's disease, traumatic brain injury, subarachnoid stroke and Parkinson's Disease. Orphan disease designation for FTD awarded based on pre-clinical evidence Orphan disease designations are restricted to products for rare conditions for which there are no satisfactory methods of treatment authorized. It allows for a faster market authorization pathway and ten-year market exclusivity. Frontotemporal dementia (FTD) is a degenerative condition in the brain that affect approximately 3.8 people in 10,000 persons in the EU. Typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play. For this approval, the European Union followed the positive opinion from the EMA after the EMA followed positive recommendations from the Committee for Orphan Medicinal Products (COMP). COMP partly based their conclusions on the availability of pre-clinical evidence in mice, that showed decrease in neuroinflammation markers and rescue of cognitive and social behavioral deficits. Examples include reduction of anxiety, depressive-like behavior and abnormal social behavior. Neuroplast CEO Johannes de Munter states: "This designation for frontotemporal dementia is an important milestone in expanding the Neuro-Cells® development to a wider range of therapeutic areas. Using the same technology platform for traumatic spinal cord injury and frontotemporal dementia, illustrates an unusual range of acute and chronic neurological disorders that could potentially benefit from this." Neuroplast is open to discuss investor opportunities to effectuate the clinical pathways to a wider scope of neurological conditions. About Frontotemporal dementia Frontotemporal dementia (FTD) is a degenerative condition in the brain that is characterized by behavioral and language impairments. Depending on the variant, patients experience changes in personality, emotion, speech or motor functions. Patients may first become indifferent or careless and have difficulty understanding sentences. While the condition progresses, patients may become language impaired, lack initiative and lose executive functions. The typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play. FTD affects approximately 3.8 people in 10,000 persons in the EU, for whom there are no effective treatments available. Patients typically receive antipsychotics to limit behavioral symptoms. About Neuro-Cells® Neuro-Cells® is a transformative treatment under GMP. It contains non-substantially manipulated bone marrow-derived hematopoietic and mesenchymal stem cells, manufactured from a patient's own bone marrow (donor and receiver are the same person). Inflammatory inducing components and pathogens are removed during this process. About Neuroplast Neuroplast is a Dutch stem cell technology company focusing on fast-track development programs using autologous cell products for treatment of neurodegenerative diseases, with the aim of giving back perspective to people who suffer from those conditions. The company was founded in August 2014 by physician Johannes de Munter and neurologist Erik Wolters. Current funders are Lumana Invest, Brightlands Venture Partners, LIOF and the Netherlands Enterprise Agency. Neuroplast is located at Brightlands Chemelot Campus in The Netherlands. For more information, please visite About Lumana Invest Investment company Lumana was established by entrepreneurs and unique due to not having a predetermined investment horizon. The Lumana founders showcase strong commitment to their portfolio companies by actively supporting management in strategic decision making. About Brightlands Venture Partners Brightlands Venture Partners (BVP) is the fund manager of Chemelot Ventures and is a so-called ecosystem investor. BVP invests in companies benefiting from and contributing to the Brightlands campuses in the south of The Netherlands. Other funds under management are BVP Fund IV, Brightlands Agrifood Fund and Limburg Ventures. The funds of BVP focus on sustainability and health; together the funds have made over 40 investments. About LIOF LIOF is the regional development agency for Limburg and supports innovative entrepreneurs with advice, network and financing. Together with entrepreneurs and partners, LIOF is working towards a smarter, more sustainable and healthier Limburg by focusing on the transitions of energy, circularity, health and digitalization. About The Netherlands Enterprise Agency The Netherlands Enterprise Agency operates under the auspices of the Dutch Ministry of Economic Affairs and Climate Policy. It facilitates entrepreneurship, improves collaborations, strengthens positions and helps realize national and international ambitions with funding, networking, know-how and compliance with laws and regulations. Forward looking statements All statements other than statements of historical facts, including the statements about the clinical and therapeutic potential and future clinical milestones of Neuro-CellsÒ, the indications we intend to pursue and our possible clinical or other business strategies, and the timing of these events, are forward-looking statements. Forward-looking statements can be identified by terms such as "believes", "expects", "plans", "potential", "would" or similar expressions and the negative of those terms. These forward-looking statements are based on our management's current beliefs and assumptions about future events and on information currently available to management. Neuroplast B.V. does not make any representation or warranty, express or implied, as to the improper use of this article, accuracy, completeness or updated status of above-mentioned statements. Therefore, in no case whatsoever will Neuroplast B.V. be legally liable or liable to anyone for any decision made or action taken in conjunction with the information and/or statements in this press release or for any related damages. In case of any further questions, please contact: Neuroplast Johannes de Munter, CEO T: +31 (0)85 076 1000 E: [email protected] LifeSpring LifeSciences Communication, Amsterdam Leon Melens T: +31 6 538 16 427 E: [email protected] Logo: SOURCE Neuroplast

It was another busy week for clinical trial news. Here’s a look. COVID-19-Related Boehringer Ingelheim enrolled the first patient in a new clinical trial in COVID-19 patients with respiratory failure. Weill Cornell Medicine and New York-Presbyterian Hospital are leading the study in whether these patients are more susceptible to developing progressive Interstitial Lung Disease (scarring of the lungs). Synairgen reported it had hit its recruitment target of 610 participants for its global Phase III SPRINTER trial of SNG001 for hospitalized COVID-19 patients. The study is being run in 17 countries. The drug is an inhaled formulation of interferon beta, a broad-spectrum antiviral protein. A topline readout is expected in early 2022, and if successful, the company plans to an Emergency Use Authorization (EUA) in the U.S. GlaxoSmithKline and Vir Biotechnology announced headline data from the COMET-TAIL Phase III trial of IM sotrovimab for mild-to-moderate COVID-19 in high-risk, non-hospitalized adults and adolescents. It was non-inferior to IV administration. Sotrovimab is a SARS-CoV-2 neutralizing monoclonal antibody. Non-COVID-19-Related Bristol Myers Squibb’s Phase III CheckMate -816 trial hit the primary endpoint in an interim analysis. The study was evaluating the company’s checkpoint inhibitor Opdivo (nivolumab) and chemotherapy in patients with resectable stage IB to IIIA non-small cell lung cancer (NSCLC), which is a neoadjuvant setting—a first step to shrink a tumor before the main treatment, typically surgery. The combination demonstrated a statistically significant and clinically meaningful improvement in event-free survival (EFS) compared to chemotherapy alone when dosed before surgery. GlaxoSmithKline announced positive results in its Phase III trials of daprodustat. The drug, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor met the primary and secondary endpoints in the ASCEND-ND and ASCEND-D trials for anemia due to chronic kidney disease. Novartis announced results from a pooled post-hoc analysis of Phase III ORION-9, -10, and -11 trials. The studies were looking at the impact of body mass index (BMI) levels on the safety and efficacy of twice-yearly Leqvio (inclisiran). The drug was well-tolerated and demonstrated effective and sustained reduction of about 50% in low-density lipoprotein cholesterol (LDL-C) when used with other lipid-lowering therapies. The company also presented positive Phase III results from the JUNIPERA study in support of Cosentyx (secukinmab) in pediatric patients with juvenile psoriatic arthritis (JPsA) and enthesitis-related arthritis (ERA). The drug significantly delayed time to flare versus placebo. The company also published data from HER-MES, a Phase IV study that compares Aimovig (erenumab) with an oral prophylactic migraine treatment (topiramate). Aimovig has a superior efficacy versus topiramate with patients having a significantly higher probability of having a clinically meaning improvement in migraine frequency. Prelude Therapeutics reported the FDA had accepted its Investigational New Drug (IND) application for PRT2527. The company hopes to start a Phase I trial of the drug by the end of the year. PRT2527 is a potent and selective CDK9 inhibitor being developed for patients with selected solid tumors, including sarcoma, prostate cancer, breast cancer, and other cancers with genomic alterations that lead to MYC dependence. TFF Pharmaceuticals completed dosing in the Phase Ib study of Voriconazole Inhalation Powder for treatment and prevention of Invasive Pulmonary Aspergillosis. The drug is a next-generation, direct-to-lung, inhaled dry powder version of voriconazole. Selecta Biosciences announced topline results from a joint Selecta and AskBio Phase I trial of its ImmTOR platform to mitigate the formation of neutralizing antibodies against an adeno-associated viral serotype 8 serotype capsid used in gene therapies. At day 30, in patients receiving a single 0.3 mg/kg dose of ImmTOR, the company observed a median anti-AAV8 neutralizing antibody titer of 1:5, a 250-fold lower level than in subjects dosed with AAV8 capsid alone. AstraZeneca presented three new sub-analyses from the DAPA-CKD Phase III trial of Farxiga (dapagliflozin). The drug is a sodium-glucose cotransporter 2 (SGLT2) inhibitor. The data supports the consistency of the drug’s cardiorenal and mortality benefits in treating chronic kidney disease with and without type 2 diabetes. Crinetics Pharmaceuticals presented new data from ACROBAT Advance, the ongoing open label extension (OLE) trial of paltusotine in patients with acromegaly. ACROBAT Edge and Evolve are two separate Phase II studies. Treatment with paltusotine resulted in median serum insulin-like growth factor-1 (IGF-1) levels that were lower than those observed in the untreated period in the parent studies and were stably maintained at levels achieved on prior SRL therapy for up to 51 weeks. Affimed and NKGen Biotech initiated patient recruitment in the Phase I/IIa trial of the innate cell engager (ICE) AFM2 (Affimed’s tetravalent, bispecific EGFR and CD16A-binding ICE) in combination with SNK01, NKGen’s ex vivo expanded and activated autologous NK cell therapy. iOnctura fully enrolled the metastatic uveal melanoma expansion cohort of the DIONE-01 trial of IOA-244. IOA-244 is a selective PI3Kδ inhibitor. AbbVie presented integrated data from two Phase II trials, KEEPsAKE 1 and 2, of Skyrizi (risankizumab) in adults with active psoriatic arthritis. The data demonstrated that 24-weeks of treatment with the drug caused greater improvements in PsA signs and symptoms compared to placebo. Eli Lilly reported sub-analysis of the EMPEROR-Preserved Phase III trial of Jardiance (empagliflozin) for adults with heart failure with left ventricular ejection fraction (LVEF) over 40%. The drug decreased the risk for the composite primary endpoint of cardiovascular death or hospitalization for heart failure and slowed kidney function decline regardless of chronic kidney disease status at baseline. Rain Therapeutics plans to initiate a Phase II MANTRA-3 trial of milademetan as a monotherapy for Merckel cell carcinoma patients refractory to immune checkpoint inhibition. The drug is an oral mouse double minute 2 (MDM2) inhibitor. The trial is expected to start mid-2022. SCYNEXIS completed its Phase I trial of a liposomal IV formulation of ibrexafungerp. The data support the safety of the liposomal IV formulation. Ibrexafungerp is an antifungal agent, a novel class of structurally distinct glucan synthase inhibitors, triterpenoids. Neuroplast announced positive Phase I results in patients suffering from Traumatic Spinal Cord Injury (TSCI). The company’s Neuro-Cells appeared safe and well tolerated. The treatment uses the patient’s own stem cells to prevent further loss of function after sustaining TSCI. Noxopharm initiated patient enrollment in its DARRT-2 Phase II trial. DARTT is an experimental immunotherapy based on Veyonda combining with a well-tolerated, low dosage of external beam radiotherapy to trigger an immune response. Jasper Therapeutics and AVROBIO are collaborating on using Jasper’s JSP191, an anti-CD117 monoclonal antibody as a targeted conditioning agent for patients with Fabry disease and/or Gaucher disease type 1 who are receiving one of AvroBio’s ex vivo lentiviral gene therapies. JSP191 blocks stem cell factor receptor signaling. Immutep announced final OS data from its Phase IIb AIPAC trial. The study evaluated eftilagimod alpha in combination with paclitaxel in metastatic breast cancer. The data supported Phase III development, with an OS benefit trend with median survival benefit of +2.9 months. Oncorus presented Phase I data on ONCR-177 in advanced injectable tumors. ONCR-177 is an oncolytic herpes-simplex virus-1 expressing five immunomodulatory transgenes. OncoC4 presented preliminary clinical data from the ongoing Phase Ia PRESERVE-001 trial of ONC-392 in cancer patients. ONC-392 is a nextgen anti-CTLA-4 monoclonal antibody. Aravive announced positive new data from the Phase Ib portion of its open-label Phase Ib/II trial of batiraxcept (AVB-500) in combination with cabozantinib in clear cell renal cell carcinoma. Batiraxcept is a therapeutic recombinant fusion protein that neutralizes GAS6 activity by binding to GAS6, selectively inhibiting the GAS6-AXL signaling pathway, which is upregulated in multiple cancers including ovarian, renal and pancreatic. CureVac announced promising data from the completed dose-escalation portion of the Phase I trial of CV8102. CV8102 is a single-stranded non-coding RNA optimized to maximize activation of cellular receptors that normally detect viral pathogens entering cells but is designed to recruit and activate antigen-presenting cells at the site of injection to present tumor antigens released from tumor cells to T cells in the draining lymph node. 89bio presented a sub-analysis of its Phase Ib/IIa proof-of-concept study of BIO89-100 in non-alcoholic steatohepatitis (NASH), looking at the correlation of liver fat and spleen volume. BIO89-100 is a potential best-in-class fibroblast growth factor 21 (FGF21) analog. Athira Pharma presented an overview and updates from two ongoing Phase III trials, ACT-AD and LIFT-AD of ATH-1017 in mild-to-moderate Alzheimer’s disease. ATH-1017 is a small molecular designed to enhance the activity of Hepatocyte Growth Factor (HGF) and its receptor, MET. AC Immune announced that Genentech presented the full topline data from the Phase III LAURIET trial of semorinemab in mild-to-moderate Alzheimer’s disease. The drug is an investigational anti-Tau monoclonal antibody. As previously announced, the trial hit one of two co-primary endpoints, demonstrating statistically significant 43.6% reduction in rate of cognitive decline, but missed the Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL) or the secondary efficacy endpoints. Yumanity Therapeutics announced that YTX-7739 hit the primary endpoints in its Phase Ib trial in Parkinson’s disease. The drug is a potential first-in-class disease-modifying mechanism of action that inhibits stearoyl-CoA desaturase (SCD). It was generally well tolerated and demonstrated favorable PK/PD and safety pro Apellis Pharmaceuticals presented data from the Phase III DERBY and OAKS trials of pegcetacoplan in geographic atrophy (GA) secondary to age-related macular degeneration. The drug is a targeted C3 therapy. It hit the primary endpoint in the OAKS study of significantly reducing GA lesion growth compared to pooled sham at 12 months. The DERBY study narrowly missed the primary endpoint, demonstrating a decrease in GA lesions growth with monthly and every-other-month treatment compared to pooled sham at 12 months. Lindis Blood Care published results from the clinical pilot study of Catuvab for the removal of tumor cells from surgical blood. The results in 16 cancer patients undergoing abdominal tumor resection demonstrated efficacy and feasibility with no residual malignant cells detected in the final erythrocyte concentrates after the procedure. Catuvab is a medical device used to remove EpCAM-positive tumor cells from surgical blood and is designed to enable the use of machine autotransfusion technology (MAT). Nouscom announced promising interim safety, immunogenicity, PK/PD and clinical activity data from the Phase I trial of NOUS-209. The drug is an off-the-shelf cancer vaccine based on shared neoantigens, administered in combination with Merck’s Keytruda (pembrolizumab) for Microsatellite Instable High (MSI-H) gastric, colorectal and gastro-esophageal junction solid tumors. Finch Therapeutics reported positive topline data from PRISM-EXT, an open-label extension of its PRISM3 Phase II trial of CP101 for prevention of recurrent C. difficile infection. There were no treatment-related serious adverse events, and the drug showed an overall safety pro with what was seen in PRISM3. Freeline Therapeutics announced new data from its ongoing Phase I/II MARVEL-1 dose-finding trial of FLT190 for Fabry disease. The data of the trial has seen expression of alpha-galactosidase A reaching near-normal levels. HOOKIPA Pharma advanced HB-201 to Phase II. The drug is being evaluated in combination with Merck’s Keytruda (pembrolizumab) as a first or second-line treatment for HPV16+ squamous cell head and neck cancers (NHSCC). HB-201 is a single-vector compound with an arenavirus backbone that expresses an E7E6 fusion protein derived from HPV16. Rafael Pharmaceuticals enrolled the first patient in the APOLLO 613 Phase I/II trial of CPI-613 (devimistat) in combination with hydroxychloroquine in patients with clear cell sarcoma at City of Hope in Duarte, Calif. Two other sites, Cleveland Clinic Children’s and University of Michigan’s Rogel Cancer Center have also been opened. Adaptimmune Therapeutics reported updated clinical and translational data from its SPEARHEAD-1 Phase III trial of afamitresgene autoleucel in advanced synovial sarcoma or myxoid/round cell liposarcoma. Based on the reported overall response rate, it is expected to meet its primary endpoint in the final analysis planned later this year. Merck initiated the VICTOR Phase III trial of Verquvo (vericiguat) in patients with chronic heart failure and reduced ejection fraction of 40% or less who have not had a recent worsening heart failure event. The drug is already approved in the U.S. to reduce the risk of CV death and heart failure hospitalization following a hospitalization for heart failure or need for outpatient IV diuretics in adults with symptomatic chronic heart failure and ejection fraction less than 45%. Eisai and Biogen presented results of sensitivity analyses evaluating the consistency of lecanemab efficacy across multiple statistical models in patients with Mild Cognitive Impairment (MCI) due to Alzheimer’s disease and mild AD. Lecanemab is an anti-amyloid beta protofibril antibody. Biogen also reported that its Aduhelm (aducanumab) significantly lowered plasma p-tau181, a biomarker of tau in Alzheimer’s disease. This was from two Phase III trials. They say change in plasma p-tau181 was significantly correlated with change in amyloid beta plaque, and it reduced cognitive and functional decline on all primary and secondary Alzheimer’s measurements. Horizon Therapeutics enrolled the first patient in a Phase IIb trial of hZN-825 for diffuse cutaneous systemic sclerosis, a subset of systemic sclerosis (scleroderma). The drug is a lysophosphatidic acid receptor 1 (LPAR1) antagonist. Ikena Oncology presented data from their IK-175 AHR antagonist program. The ongoing trial is recruiting patients in both monotherapy and combination cohorts with nivolumab. It is being evaluated in multiple tumor types, including urothelial carcinoma, a type of bladder cancer. Vaccitech reported results from ongoing Phase I and Phase Ib/IIa trials of VTP-300 for chronic hepatitis B infection. The drug is a heterologous prime-boost vector combination of ChAdOx1-HBV + MVA-HBV. It induced robust T cells against targeted HBV antigens. Cue Biopharma presented updated interim data from its ongoing trial of CUE-101 as a monotherapy for recurrent/metastatic HPV+ head and neck cancer. The drug is a Fc-fusion biologic that incorporates peptide-major histocompatibility complex molecules with engineered IL2.