Clinical Catch-Up: October 7-11

Clinical Trial Data

It was a busy week for clinical trial announcements. Here’s a look.

ReveraGen BioPharma presented topline data from an 18-month treatment of Duchenne muscular dystrophy (DMD) patients with vamorolone, a first-in-class steroidal anti-inflammatory. It is being developed as an alternate to corticosteroids such as prednisone or deflazacort. The trial data demonstrated that 48 DMD patients receiving a broad range of doses of vamorolone for six months showed dose-related improvement in multiple gross motor outcomes. After finishing the six-month period, almost all patients and their doctors decided to continue treatment with the drug. Of the group, 45 boys participated in a two-year long-term extension trial with dose escalation to 2.0 or 6.0 mg/kg/day of vamorolone.

Citius Pharmaceuticals announced it had hit the data cutoff for the most recent patient treated and completed eight weeks of observation in its Phase III pivotal trial for Mino-Lok. The primary endpoint is time to catheter failure event between randomization and the test of cure (TOC). Mino-Lok is an antibiotic lock solution to treat patients with CLABSIs/CRBSIs, or catheter-related or central line-associated bloodstream infection.

Zogenix presented positive topline results from its pivotal Phase II RETRO trial, a global retrospective study of SET, a fixed combination treatment of two pyrimidine nucleosides for TK2 deficiency. TK2 is a rare, debilitating and often fatal genetic disorder that mostly affects infants and children. There are currently no approved therapies. The trial is utilizing MT1621, the company’s deoxynucleoside substrate enhancement therapy.

NGM Biopharmaceuticals announced positive preliminary data from a prespecified interim analysis of the aldafermin 1 mg 24-week Cohort 4, the final cohort of its Phase II trial. The trial is evaluating aldafermin in biopsy-confirmed non-alcoholic steatohepatitis (NASH). The interim analysis showed statistically significant change in the absolute liver fat content of -7.9% compared to -2.0% in the placebo arm.

ADMA Biologics presented data from compassionate use of Asceniv to treat Respiratory Syncytial Virus (RSV) infection in two immunocompromised children. The children were admitted to the Mayo Clinic with T-cell lymphoblastic lymphoma. Both children appeared to recover from their RSV infections and were discharged from the hospital. The data suggests the drug may be useful in treating severe RSV infections.

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ProQR Therapeutics presented topline data from the Phase I/II trial of sepofarsen in LCA10 patients. LCA10 is Leber’s congenital amaurosis 10 caused by the p.Cys998X mutation in the CEP290 gene. Sepofarsen is an RNA-based oligonucleotide. Data is being presented at the American Academy of Ophthalmology’s annual meeting.

NovartisAveXis announced new interim data from the Phase I/II STRONG trial of intrathecal (IT) administration of AVXS-101 in older patients with spinal muscular atrophy (SMA) Type 2. The interim data showed a mean increase of 5.9 points from baseline in HFMSE scores, almost double the clinical meaningful threshold, at a mean duration of follow-up time of 9.3 months. This is a mean increase of 4.2 points from baseline in HFMSE scores that the company presented in May 2019.

Oculis SA dosed the first patient in its Phase IIb trial of OCS-01 for inflammation and pain following cataract surgery. OCS-01 is a topical formulation of dexamethasone based on the company’s Solubilizing NanoParticle (SNP) technology platform. It may potentially become the first once daily topical steroid for this indication. The trial is being run in 20 specialized ocular surgery centers in the U.S.

Helixmith indicated its VM202 met the primary and secondary endpoints in its Phase III extension trial for diabetic peripheral neuropathy (DPN). The trial was an extension of the original 500-subject DPN 3-1 study. The study showed the drug to be safe and well-tolerated and had clinically meaningful and statistically significant decrease in pain compared to placebo at months 6, 9, and 12. VM202 (donaperminogene seltosplasmid) is a first-in-class, proprietary, non-viral, potentially regenerative plasmid DNA gene therapy.

Minoryx Therapeutics completed enrollment in its FRAMES Phase II trial of leriglitazone in Friedreich’s Ataxia. FRAMES is a multicenter, randomized, double-blind, placebo-controlled trial to test the efficacy and safety of leriglitazone in this patient population. It has recruited 39 patients in four European countries. Leriglitazone is a novel, brain penetrant, orally bioavailable and selective PPARγ agonist.

AVROBIO dosed the first patient in its AVR-RD-04 gene therapy program for cystinosis, a lysosomal storage disease. This is part of its ongoing Phase I/II clinical trial sponsored by the University of California San Diego. The gene therapy is derived from the patient’s own hematopoietic stem cells, which are then genetically engineered to produce functional cystinosis, a protein.

Galera Therapeutics announced final data from its two-year tumor outcomes follow-up of patients with locally advanced squamous cell head and neck cancer treated with GC4419 (avasopasem manganese). The drug is a highly selective and potent small molecule dismutase mimetic designed to decrease the incidence and severity of radiation-induced severe oral mucositis. The data showed the drug, with standard radiotherapy, maintained the treatment of head and neck cancer while decreasing the radiation-induced oral mucositis.

Salarius Pharmaceuticals added Memorial Sloan Kettering Cancer Center and Nationwide Children’s Hospital in Columbus, Ohio to its clinical trial sites for its ongoing Phase I/II trial of seclidemstat for Ewing sarcoma. It brings the total number of active clinical trial sites to eight. The trial is an open-label dose escalation and dose expansion study to determine the maximum tolerated dose and define the safety profile of seclidemstat.

Rafael Pharmaceuticals enrolled 100 patients in its Phase III trial of CPI-613 (devimistat) in combination with modified FOLFIRINOX (mFFX) for first-line therapy for metastatic adenocarcinoma of the pancreas. It is active and still open for patient enrollment. Devimistat is a first-in-class compound that targets enzymes involved in cancer cell metabolism.

ImaginAb published data from its Phase I trial of its CD8 tracer 89Zr-Df-IAB22M2C in visualizing the immune system. The data was published in The Journal of Nuclear Medicine. The data showed the tracer was well tolerated with no immediate or delayed side effects. It appeared to successfully target CD8+ T-cell-rich tissues.

Alder BioPharmaceuticals dosed its first patient in a Phase I trial of ALD1910 as a prevention for migraine. ALD1910 is a monoclonal antibody that inhibits pituitary adenylate cyclase-activating polypeptide (PACAP). The trial will enroll about 100 healthy men and women between the ages of 18 and 55 to assess the safety, tolerability and pharmacokinetics of the drug at various doses. Initial results are expected in the second half of 2020.

City of Hope opened a first-in-human clinical trial of IL13Ralpha2 antigen, a CAR-T cell, with Bristol-Myers Squibb’s Opdivo and Yervoy in brain tumors. The trial will deliver CAR-T cells locally to the brain via direct injection to the tumor site and by way of infusion into the ventricular system. Mustang Bio is the exclusive licensee of City of Hope patents covering the IL13Ralpha2-specific CAR-T therapy.

Gilead Sciences and Galapagos NV released Week 52 data from the Phase III FINCH 1 and FINCH 3 trials of filgotinib, a JAK1 inhibitor, for moderately-to-severely active rheumatoid arthritis (RA). The data was consistent with the data from the Week 12 and 24 analyses that was presented earlier. The marketing authorization for RA is being evaluated by the European Medicines Agency, and an application has been submitted to the Japanese Ministry of Health, Labor and Welfare. Gilead expects to file an NDA with the FDA by the end of this year.

Evevensys presented data from part 1 of its Phase I/II trial of non-infectious uveitis (NIU) at the Ophthalmology Innovation Summit’s 11th Annual OIS@AAO conference. The company’s technology is a non-viral gene therapy ocular drug delivery platform that utilizes a two-part Electrotransfection System, including an Ocular Device and Electrical Pulse Generator, that delivers DNA plasmids encoding therapeutics proteins into the ciliary muscle. The study showed no serious adverse events.

Menlo Therapeutics presented successful Phase II results of serlopitant for pruritis in patients with psoriasis. The data showed a statistically significant decrease in itching in patients treated with serlopitant compared to placebo. It also showed consistent benefit that favored serlopitant when studying response rates across patient demographic categories.

AMAG Pharmaceuticals published data from its two pivotal, double-blind, placebo-controlled Phase III trials and its voluntary open-label one-year extension study of Vyleesi (bremelanotide injection) in hypoactive sexual desire disorder (HSDD). The data was published in the online issue of Obstetrics & Gynecology and will appear in the November print issue.

Geron Corporation dosed the first patient in the IMerge Phase III trial of imetelstat in lower risk myelodysplastic syndromes. IMerge is a two-part Phase II/III trial of the drug in transfusion dependent patients with lower risk MDS who are relapsed after or refractory to erythropoiesis-stimulating agents. The data suggested meaningful and durable transfusion independence and potential disease-modifying activity.

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