Pacanalotamab

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

It was a busy week for clinical trial updates. Here’s a look. 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 RESOLUTE SM 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.