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Clinical trial news definitely picked up this final week of January. Here’s a look. Clinical trial news definitely picked up this final week of January. Here’s a look. COVID-19-Related Todos Medical announced a data lock for the interim data analysis of its Tollovir Phase II trial in hospitalized COVID-19 patients. The results were on January 27, 2022. Tollovir is a 3CL protease inhibitor and anti-cytokine therapeutic candidate. Arcturus Therapeutics updated data from clinical development programs for ARCT-154 and ARCT-165, investigational, next-generation, self-amplifying mRNA vaccine candidates. Full cohort data from the ARCT-154 arm of the ongoing Phase I/II booster trial showed a 30-fold increase in neutralizing antibody geometric mean concentrations against SARS-CoV-2 at Day 15 post-boost and maintained a 28-fold increase at Day 29. ARCT-165 and ARCT-154 also demonstrated robust neutralizing antibody responses post-boost against several variants of concern and variants of interest. Arcturus also said that, when administered in low doses at least five months after initial vaccination with Comirnaty, the Pfizer-BioNTech vaccine, the candidates provided robust increases of 54- and 47-fold, respectively, in neutralizing antibody responses against the Omicron variant. Sinovac Vaccine A Phase IV study out of Brazil and the University of Oxford evaluated the Chinese-made Sinovac COVID-19 vaccine plus booster shots from either Sinovac, AstraZeneca -Oxford, Pfizer - BioNTech or Johnson & Johnson . The study found that patients who received Sinovac first then received a different booster had significantly higher immune responses to variants. Mixed vaccines induced antibodies at “8-22-fold higher” levels than when older adults were given another shot of the Sinovac vaccine. And giving the boosters from other vaccines increased immunity against both Omicron and Delta variants. Moderna launched the Phase II trial of its Omicron-specific booster vaccine candidate, dosing the first patient. The study will focus on adults 18 years and older and have two cohorts, people who previously received the two-dose Moderna series at least six months ago and people who received the two-dose primary series plus a 50-microgram booster dose of the original vaccine at least three months ago. The company expects to enroll about 300 people in each cohort. Galecto published full results of a trial of its inhaled galectin-3 inhibitor, GB0139, in hospitalized patients with COVID-19 who required oxygen but not mechanical ventilation. It was compared to patients who received only standard of care. The drug hit the primary endpoint of safety with no treatment-related adverse events. The mean age of patients was 65 years, with a mean BMI of 32, multiple comorbidities and breathlessness, but they were all able to effectively inhale the drug and hit pharmacologically relevant plasma levels. Galectin-3 plays a central role in COVID-19-related acute lung injury, cytokine storm, T-cell exhaustion and organ micro-thrombosis. The drug also appeared to rapidly decrease markers of inflammation associated with cytokine storm, micro-thrombosis and short or long-term fibrosis. BerGenBio and Oslo University Hospital will initiate a study of BerGenBio’s bemcentinib in hospitalized COVID-19 patients. The EU-SolidAct trial — European DisCoVeRy for Solidarity: An Adaptive Pandemic and Emerging Infection Platform Trial — is part of EU-RESPONSE, a pan-European research project to identify drugs against COVID-19. Bemcentinib, an AXL inhibitor, will be evaluated in up to 500 hospitalized COVID-19 patients. TFF Pharmaceuticals completed enrollment in its Phase I trial of a dry powder formulation of niclosamide for COVID-19. Niclosamide is a potential antiviral treatment. Non-COVID-19-Related TScan Therapeutics received the green light from the FDA to run a Phase I trial of TSC-100 for patients with hematologic malignancies undergoing allogeneic hematopoietic cell transplantation (HCT). TSC-100 targets the minor histocompatibility antigen HA-1. Applied Pharmaceutical Science got the go-ahead from the FDA to initiate a Phase I trial of APS03118 for multiple cancers. The drug is a next-generation selective RET inhibitor. Tango Therapeutics ’ IND for a Phase I/II trial of TNG908 for cancers with MTAP deletions, including non-small cell lung cancer, mesothelioma, pancreatic cancer and cholangiocarcinoma, was cleared by the FDA. TNG908 is a synthetic lethal small molecule inhibitor of protein arginine methyltransferase 5 (PRMT5). SQZ Biotech expects to initiate its Phase I/II COMMANDER-001 trial of SQZ-eAPC-HPV in HPV16+ solid tumors after the FDA approved its IND. The therapy was developed by delivering five different mRNAs into a patient’s monocytes, B cells, T cells and NK cells. Lyell received the go-ahead from the FDA to launch a Phase I trial of LYL132 in solid tumors expressing New York esophageal squamous cell carcinoma 1 (NY-ESO-1). LYL132 is a T-cell receptor (TCR) therapy that incorporates Epi-R, the company’s epigenetic reprogramming technology. Genentech published one-year results from four pivotal Phase III trials of faricimab in wet or neovascular, age-related macular degeneration (AMD) and diabetic macular edema (DME). All four studies hit their primary endpoints, demonstrating that patients receiving faricimab up to every four months achieved non-inferior vision gains compared to aflibercept, given every two months. Faricimab is a bispecific antibody designed for the eye that targets and inhibits two pathways linked to several vision-threatening retinal conditions by neutralizing Ang-2 and VEGF-A. Lyra Therapeutics initiated the Phase III ENLIGHTEN trial of LYR-210 in adult, surgically naïve chronic rhinosinusitis patients. LYR-210 utilizes the company’s proprietary XTreo platform to enable six months of local, intra-nasal, anti-inflammatory therapy for a single treatment. Connect Biopharma announced data from the Phase IIb trial of CBP-20-1 administered subcutaneously to adults with moderate-to-severe atopic dermatitis. CBP-201 is an antibody designed to target interleuekin-4 receptor alpha. MediWound announced positive topline results from its Phase II trial of EscharEx for debridement of venous leg ulcers. It hit the primary endpoint, showing that patients receiving the treatment had a statistically significant higher incidence of complete debridement compared to the gel vehicle. Erytech Pharma presented results of two trials of eryaspase in combination with mFOLFIRINOX as first-line treatment for advanced pancreatic cancer. Erypase (GRASPA) consists of L-asparaginase encapsulated inside donor-derived RBCs and targets the cancer cells’ altered asparagine and glutamine metabolism. Mustang Bio announced the FDA had placed a clinical hold on its IND for the planned Phase II study of MB-207 for X-linked severe combined immunodeficiency (XSCID), also known as “bubble boy syndrome.” The hold is in place pending CMC clearance. MB-207 is a gene therapy. Mind Medicine reported the FDA had approved its IND for a Phase IIb trial of MM-120 for generalized anxiety disorder (GAD). It was previously on hold. MM-120 is a pharmacologically optimized form of LSD. Imara expects to launch a Phase II trial of tovinontrine for heart failure with preserved ejection fraction (JFpEF). Tovinontrine is a highly selective and potent small molecule inhibitor of phosphodiesterase-9 (PDE9). Nanoscope received IND clearance from the FDA for a Phase II trial of MCO-010 ambient-light activatable optogenetic monotherapy in Stargardt patients. Stargardt is an inherited retinal disease. The therapy, MCO-010, is a gene therapy that reprograms healthy retinal cells to make them photosensitive. SwanBio Therapeutics received the green light from the FDA for a Phase I/II trial of SBT101 for disease-causing ABCD1 mutation in adrenomyeloneuropathy. SBT101 is an AAV-based gene therapy. Cerevance dosed the first patient in its Phase I trial of CVN766. CVN766 is a potent and highly selective small molecule antagonist of the orexin 1 receptor (Ox1R), which is expressed in the areas of the brain important for regulating fear, anxiety, reward and emotion. The company is developing the drug for panic disorder and schizophrenia. Checkpoint Therapeutics announced positive topline results from its Phase III trial of cosibelimab in metastatic cutaneous squamous cell carcinoma (cSCC). The drug is an anti-PD-L1 checkpoint inhibitor. The trial hit the primary endpoint, demonstrating a confirmed objective response rate (ORR) of 47.4%. Sierra Oncology announced positive topline data from the Phase III MOMENTUM trial of momelotinib in myelofibrosis patients who are symptomatic and anemic and previously treated with an approved JAK inhibitor. The trial hit all primary and key secondary endpoints. Momelotinib is a potent, selective and orally bioavailable JAK1, JAK2 and ACVR1/ALK2 inhibitor. Vaccinex reported positive interim response data in its Phase Ib trial of its pepinemab with Merck ’s Keytruda (pembrolizumab). Pepinemab is a humanized IgG4 monoclonal antibody. In the study, two patients with recurrent or metastatic head and neck squamous cell carcinoma showed complete responses. Gilead Sciences reported the FDA placed a partial clinical hold on studies assessing the combination of magrolimab plus azacytidine over unexpected serious adverse events. Magrolimab is a potential, first-in-class monoclonal antibody against CD47 and a macrophage checkpoint inhibitor. Azacitidine is a chemotherapy agent marketed under the brand names Vidaza and Onureg, both manufactured by Bristol Myers Squibb . 4D Pharma announced additional clinical data from Part A of its Phase I/II study of MRx-4DP0004 for asthma. The drug is an orally delivered single strain Live Biotherapeutic. The study hit the primary endpoint and the safety pro comparable to placebo. Biogen and its partner Eisai Company released additional details about the Phase IV post-marketing study of Aduhelm (aducanumab) for Alzheimer’s disease. As part of the Phase IV trial, the companies plan to enroll 18% of U.S. participants from Black/African American and Latinx populations. The primary endpoint of the study will be measured via the Clinical Dementia Rating-Sum of Boxes (CDR-SB) at 18 months after the beginning of treatment. This is a validated measure of both cognition and function broadly used in clinical studies of early symptomatic Alzheimer’s and is consistent with the two Phase III EMERGE and ENGAGE trials. They also plan to increase enrollment from 1,300, which they had previously announced, to 1,500 patients with Mild Cognitive Impairment due to Alzheimer’s and mild Alzheimer’s disease, with confirmation of amyloid beta pathology. They expect to initiate patient screening in May 2022. GeNeuro announced the completion of its ProTEct-MS Phase II trial of temelimab in patients whose multiple sclerosis progresses without relapsed as they have previously received chronic anti-CD20-Ab therapy with rituximab. They expect to report final results by the end of March. Cognito Therapeutics and Providence Health Plan announced plans to run a Phase III trial of its optogenetic, disease-modifying treatment for patients with mild-to-moderate Alzheimer’s disease. Moderna launched a Phase I clinical trial at George Washington University organized by IAVI to evaluate an mRNA vaccine to deliver HIV immunogens to patients. Funded by the Bill & Melinda Gates Foundation, it will evaluate 56 healthy, HIV-negative adults. Excision BioTherapeutics initiated a Phase I study of EBT-101 to remove sections of the HIV genome from the human body. EBT-101 is a CRISPR-based technology. Taysha Gene Therapies reported a patient death in otherwise positive interim data from their gene therapy for Sandhoff and Tay-Sachs diseases. TSHA-101 delivers both the HEXA and HEXB genes that make up the beta-hexosaminidase A enzyme. The patient death appears likely related to an initial COVID-19 infection, which the patient recovered from, but then contracted a secondary hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection and died. It does not appear to be associated with the therapy.

Taysha said the data for TSHA-101 is the “first-ever to support the bicistronic vector approach in humans delivering both HEXA and HEXB genes in the endogenous ratio.” Shares of Taysha Gene Therapies are down more than 10% after a patient’s death marred positive interim data for the company’s experimental gene therapy treatment for Sandhoff and Tay-Sachs diseases, which are two forms of GM2 gangliosidosis. The first patient treated with Texas-based Taysha’s TSHA-101 was diagnosed with Sandhoff disease, a rare inherited disorder that progressively destroys brain and spinal cord nerve cells. After administering TSHA-101, an investigational gene therapy that delivers both the HEXA and HEXB genes that make up the β-hexosaminidase A enzyme, the patient demonstrated clinical improvement after three months. The patient was cleared to travel home. However, Taysha said the unvaccinated patient was exposed to a family member who was symptomatic of an upper respiratory infection, which was likely COVID-19. The Taysha patient was hospitalized with pneumonia. While there, the patient contracted a secondary hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection and ultimately succumbed. Trial investigators have made the initial assessment that the patient’s death was not likely due to the gene therapy. However, according to the company, a review from the independent data safety monitoring board is being conducted and results are anticipated in the near term. Prior to the patient’s death, Taysha said their disease had realized normalization of Hex A enzyme activity by the end of the first month following treatment with the gene therapy. The levels were “58-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 3,” the company said. According to available data, the patient achieves Hex A enzyme activity of 190% and 288% of normal at month 1 and month 3, respectively. The second patient to receive the gene therapy is a Tay-Sachs patient. Another rare genetic disorder, Tay-Sachs is caused by the absence of an enzyme that helps break down fatty substances known as gangliosides. Without the ability to break them down, gangliosides build up to toxic levels in the brain and spinal cord, leading to early death. After dosing with TSHA-101, the Tay-Sachs patient achieved Hex A enzyme activity of 25% of normal at month 1. That data represents a 5-fold improvement above the presumed asymptomatic level of 5% of normal identified by natural history. Despite the early death that is possibly related to COVID-19, Taysha said the data for TSHA-101 is the “first-ever to support the bicistronic vector approach in humans delivering both HEXA and HEXB genes in the endogenous ratio.” Although the cause of the patient’s death is still under review, the company said preliminary data suggest that TSHA-101 was well-tolerated with no significant drug-related events. TSHA-101 is the only bicistronic vector currently in clinical development for GM2 gangliosidosis and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration. It has also been granted Orphan Drug designation from the European Commission. “TSHA-101 is the first bicistronic vector in clinical development, representing an important first for the field of gene therapy,” RA Session II, president, founder and chief executive officer of Taysha, said in a statement. “TSHA-101 demonstrated expression of both HEXA and HEXB genes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis. We expect to provide continued updates on the program, with additional clinical data anticipated by the end of 2022.”

It was a busy week for clinical trial updates. Here’s a look. COVID-19-Related China’s Clover Biopharmaceuticals terminated its partnership with GlaxoSmithKline to develop a COVID-19 vaccine using GSK’s adjuvant. Clover was testing two vaccine candidates, one with GSK’s adjuvant and the other with an adjuvant from Dynavax, and originally said the one with the GSK product would move to mid-to-late stage clinical trial in December. But they decided to instead initiate Phase II and III trials using the Dynavax adjuvant after taking into account “scale-up manufacturing considerations.” Covaxx announced the Taiwan Ministry of Health and Welfare granted conditional approval to begin a Phase II trial of UB-612, its COVID-19 vaccine candidate. It is a multitope protein/peptide-based vaccine. Abpro Corporation announced results from a Phase I trial of its ABP 300, a human neutralizing antibody against COVID-19 derived from recovered patients. It was conducted in 42 healthy subjects. After it was finished in December 2020, several Phase II/III registrational studies were initiated recently. CalciMedica presented new clinical data from its trial of Auxora in severe COVID-19 pneumonia. In the study, 17 patients with severe COVID-19 pneumonia were randomized to receive Auxora, a potent and selective small-molecule CRAC channel inhibitor, plus standard of care, and nine patients received only standard of care. The data demonstrated that levels of D-dimer, a key marker of clotting and inflammation, decreased in patients receiving Auxora and SOC but increased in patients receiving SOC alone. Moscow’s Gamaleya Institute published the efficacy results of its Sputnik V vaccine in The Lancet from a Phase III trial. The vaccine demonstrated efficacy of 91.6% against the original viral strain. Although designed for two doses, primary analysis of AstraZeneca and the University of Oxford’s Phase III data for its COVID-19 vaccine found it to have 76% efficacy after the first dose. That protection was maintained to a second dose, with an interval of 12 weeks or more, the efficacy increased to 82%. This is welcome news as drug companies and governments work to manufacture and distribute more vaccines to get as many people vaccinated as possible. However, when the second dose was provided less than six weeks after the initial dose, the efficacy was 54.9%. Vaxart announced preliminary data from its Phase I trial of VXA-COV2-1, its oral COVID-19 vaccine candidate. The data suggests it was generally well-tolerated with immunogenicity against several strains of the virus. However, neutralizing antibodies were not seen in volunteers after a single dose. Biophytis SA plans to begin patient recruitment for most clinical centers in Brazil and the U.S. for Part 2 of its COVA Study of Sarconeos (BIO101) for acute respiratory failure associated with COVID-19. Sarconeos is a small molecule, oral treatment for sarcopenia and respiratory manifestations of COVID-19, as well as in Duchenne muscular dystrophy (DMD). The University of Oxford in the UK, with the National Immunisation Schedule Evaluation Consortium, is planning a trial to evaluate the use of first and second doses of different COVID-19 vaccines. The first dose is viewed as the “prime” vaccination with the follow-up a “booster” shot. The study will begin with a first dose of the AstraZeneca-Oxford vaccine followed by either the Pfizer-BioNTech vaccine or another dose of the AstraZeneca-Oxford vaccine. It will also investigate a first dose of the Pfizer-BioNTech vaccine followed by either the AstraZeneca-Oxford shot or another dose of the Pfizer-BioNTech vaccine. In addition, there will be two different dosing schedules, four weeks and 12 weeks. Agenus announced positive preliminary data from its Phase I trial of iNKT cell therapy in moderate to severe COVID-19 through its subsidiary, AgenTus Therapeutics. Preliminary data showed that of the four patients dosed to date, three were extubated and released after treatment, and two of them were extubated within 24 hours of dosing. Non-COVID-19-Related Merck released first-time data from the Phase III KEYNOTE-598 trial of Keytruda in combination with Bristol Myers Squibb’s Yervoy (ipilimumab) compared to Keytruda alone as first-line therapy for metastatic non-small cell lung cancer (NSCLC) without EGFR or ALK genomic tumor aberrations and whose tumors express PD-L1. Adding Yervoy to the therapy did not improve overall survival (OS) or progression-free survival (PFS) but did add toxicity to Keytruda monotherapy. Fractyl Laboratories published data from the investigator-initiated INSPIRE trial demonstrating that a majority of patients with type 2 diabetes treated with a combination of ReVita DMR and a glucagon-like peptide-1 receptor agonist were able to discontinue and remain off insulin therapy for 18 months after treatment. All treated patients demonstrated significant improvements in measures of glucose regulation and metabolic health. Revita DMR (duodenal mucosal resurfacing) is a first-in-class intervention designed to reverse insulin resistance and metabolic disease progression. Vaccitech dosed the first patient in HBV002, its Phase Ib/IIa clinical trial of VTP-300 with and without a low-dose anti-PD-1 checkpoint inhibitor in chronic hepatitis B. VTP-300 uses Vaccitech’s ChAdOx1-MVA prime-boost combination to elicit an immune response against HBV. Spark Therapeutics, a Roche company, dosed the first patient in the Phase I/II RESOLUTESM trial of SPK-3006 for late-onset Pompe disease, a rare, inherited lysosomal storage disorder. SPK-3006 is an investigational liver-directed AAV gene therapy. It is designed to deliver a single IV infusion of a modified enzyme, secretable GAA, which is produced by the liver. Circularity Healthcare executed a contract with Professional Educational Research Institute (PERI) in 2020 to begin a new clinical trial of D’Oxyva to prevent amputations and treat human diabetic foot ulcers. D’Oxyva is a painless, noninvasive transdermal delivery system used to improve blood perfusion, tissue oxygenation and wound healing. Applied Therapeutics announced the FDA had lifted the hold and the AT-007 ACTION-Galactosemia Kids pediatric clinical trial will resume immediately. The trial was previously divided into two separate clinical studies, a dose escalation and biomarker study followed by a separate long-term clinical outcomes study. They have now been combined into a single two-part study. AT-007 is a CNS penetrant Aldose Reductase inhibitor to treat Galactosemia. Mustang Bio announced that the FDA had removed the clinical hold on the MB-107 pivotal Phase II trial on January 28. The agency removed the hold on the Investigational New Drug (IND) application after reviewing a comprehensive CMC package submitted by the company in late December 2020. The company plans to initiate the pivotal Phase II trial in newly diagnosed X-linked severe combined immunodeficiency (XSCID) patients. It expects to enroll the first patient in the second quarter of 2021. XSCID is also known as bubble boy syndrome. Immunovant voluntarily paused its ongoing Phase IIb clinical trial of IMVT-1401 in Thyroid Eye Disease (TED). Immunovant reports that they became aware of a physiological signal made up of elevated cholesterol and LDL levels in patients receiving the drug in the ASCEND GO-2 trial. In previous trial of the drug in Myasthenia Gravis (MG) and in healthy patients, cholesterol levels were not measured. “Out of an abundance of caution,” Immunovant decided to voluntarily pause dosing in both TED and in a trial in Warm Autoimmune Hemolytic Anemia in order to inform everyone involved, in addition to modifying the monitoring program. ASCEND GO-2 is evaluating the drug in TED at different doses, each given weekly for 12 weeks. They found on preliminary, unblinded data from 40 patients through week 12, that mean LDL at week 12 had increased by about 65% in the 680-mg dose group and by about 40% in the 340-mg dose group, but not in the control group. Average HDL and triglyceride levels increased to a much lower degree. No serious cardiovascular events had been reported in IMVT-1401 clinical trials. Bristol Myers Squibb announced positive data from POETYK PSO-2, the second Phase III trial of deucravacitinib for moderate to severe plaque psoriasis. The drug was being evaluated against placebo and Amgen’s Otezla (apremilast). The trial hit both co-primary endpoints compared to placebo, with significantly more patients achieving Psoriasis Area and Severity Index (PASI 75), which is at least a 75% improvement of baseline PASI, and the static Physician’s Global Assessment (sPGA) score of clear or almost clear (sPGA 0/1) after 16 weeks, compared to placebo. The trial also hit multiple key secondary endpoints, including demonstrating that 6 mg deucravacitinib once a day was superior to Otezla in proportion of patients reaching PASI 75 and sPGA 0/1 at Week 16. Deucravacitinib is a once-daily, oral, selective tyrosine kinase 2 (TYK2) inhibitor. The drug is designed to inhibit interleukin (IL)-12, IL-23 and Type 1 interferon (IFN) pathways. It binds to the regulatory domain of TYK2, which is structurally distinct from the Janus kinase (JAK) 1, 2 and 3 kinases. Prothena Corporation announced that after discussions with the FDA and further analysis of the data, it is advancing birtamimab into the confirmatory Phase III AFFIRM-AL trial in patients with AL amyloidosis. Birtamimab is a humanized immunoglobulin G1 designed to directly neutralize soluble toxic aggregates and promote clearance of amyloid via phagocytosis. NeurMedix received FDA approval to initiate a pivotal Phase III trial of NE3107 in Alzheimer’s disease. The drug is a 17alpha-ethynyl-androst-5-ene-3,7,17-triol, a small molecule, orally-administered, anti-inflammatory, insulin-sensitizing agent. The trial will be run at about 30 U.S. clinical trial sites in subjects with mild to moderate Alzheimer’s. electroCore announced full enrollment for its TR-VENUS study of non-invasive vagal nerve stimulation (nVNS) for acute treatment of stroke. The study will be conducted at nine major medical centers across Turkey. gammaCore Sapphire is the company’s non-invasive, hand-held medical therapy applied at the neck. Amgen released news of five cancer programs that have either been halted or abandoned. One of the programs was the company’s BCMA BiTE (bispecific T cell engager) program for pavurutamab (AMG 701), which Amgen was developing to replace AMG 420. The company stated, “Enrollment in the Phase I study has been paused while we discuss protocol modifications to optimize safety monitoring and mitigation with the FDA. Currently enrolled patients who are demonstrating benefit may continue to receive investigational product and the Company expects to resume patient enrollment in H1 2021.” BiTE is a class of artificial bispecific monoclonal antibodies. They direct the body’s immune system, specifically the cytotoxic activity of T cells, against cancer cells. They are fusion proteins made up of two single-chain variable fragments of different antibodies. Also paused was AMG 673, a CD33-targeting BiTE for acute myelogenous leukemia (AML). They are delaying that program while they determine if AMG 330 would be a better choice. Three other programs were halted altogether. One is AMG 596, abandoned because of reprioritization of their R&D programs. The Phase I trial of AMG 397, an oral MCL-1 inhibitor, was halted to shift to intravenous MCL-1 inhibitor AMG 176, which is being evaluated in a Phase I study for hematologic malignancies. And then Imlygic (talimogene laherparepvec)’s Phase III in combination with Merck’s checkpoint inhibitor Keytruda (pembrolizumab) for melanoma—it was halted over futility. Sio Gene Therapies dosed the first patient with infantile Tay-Sachs disease in its Phase I/II trial of AXO-AAV-GM2. The therapy is a gene therapy that aims to restore HexA function by inserting a functional copy of the HEXA and HEXB gene by way of two co-administered AAVrh8 vectors. Intrommune Therapeutics got the go-ahead from the FDA for its INT301, an oral mucosal immunotherapy for the treatment of peanut allergies. The product is a specially formulated toothpaste to stabilize and deliver allergenic proteins to immunologically active areas of the oral cavity. Reistone Biopharma Co. announced that SHR0302 hit primary and key secondary endpoints in its Phase II AMBER2 clinical trial for moderate to severe ulcerative colitis. SHR0302 is a selective Janus kinase type 1 (JAK1) inhibitor. AstraZeneca reported that its Phase III KESTREL trial of Imfinzi (durvalumab) failed to meet the primary endpoint of improving overall survival (OS) compared to the EXTREME treatment regimen, which was chemotherapy plus cetuximab, a standard of care. The trial is in first-line treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) whose tumors expressed high levels of PD-L1. Imfinzi is a human monoclonal antibody, a checkpoint inhibitor, that binds to PD-L1, blocking the interaction of PD-L1 with PD-1 and CD80. Sanofi’s venglustat failed to hit the target in its Phase II trial in Parkinson’s disease. Venglustat is an oral CNS-penetrating glucosylceramide synthase inhibitor. The focus was on Parkinson’s with a mutation in the GBA gene, which is seen in up to 10% of patients and associated with early onset and worse outcomes. PTC Therapeutics announced that its Phase II trial of Translarna in Duchenne muscular dystrophy (DMD) failed to significantly improve dystrophin expression. The study evaluated 20 ambulatory DMD patients ages 2 through 8 who received the drug. Spero Therapeutics reported that the FDA placed a clinical hold on its Phase IIa trial of SPR720 in patients with nontuberculous mycobacterial pulmonary disease (NTM-PD). The hold is a precautionary measure after some animal toxicology results. SPR720 is a novel class of antibacterial agent that targets enzymes essential for bacterial DNA replication. Most Read Today