Clinical Catch-Up: May 10-14

Doctor and Patient

Although a quiet week for COVID-19-related clinical trial announcements, there were plenty of others. Read on for more information.

COVID-19-Related

Biophytis completed recruitment of 155 patients to its COVA Phase II/III trial of Sarconeos in COVID-19 patients. This will allow the independent Data Monitoring Committee to run its second interim analysis. Sarconeos is an oral, small molecule being developed for sarcopenia in a Phase II trial, but also for severe respiratory aspects of COVID-19.

Non-COVID-19-Related

Mustang Bio received the go-ahead from the FDA for a Phase I/II trial of MB-106 for relapsed or refractory CD20+ B-cell non-Hodgkin lymphoma and chronic lymphocytic leukemia. MB-106 is the company’s CD20-targeted CAR-T therapy.

Daiichi Sankyo Company and AstraZeneca presented preliminary response and disease control of datopotamab deruxtecan (Dato-DXd) in metastatic triple negative breast cancer (TNBC) with disease progression after standard treatment. Data-DXd is a TROP2 directed DXd antibody drug conjugate (ADC).

Axcella initiated its Phase IIb EMMPACT trial of AXA1125 in nonalcoholic steatohepatitis (NASH). AXA1125 is an oral drug that is a composition of six amino acids and derivatives that target multiple metabolic pathways linked to fatty liver disease.

G1 Therapeutics initiated its PRESERVE 4 Phase II trial of Cosela (Trilaciclib) administered prior to docetaxel in metastatic non-small cell lung cancer (NSCLC) in the 2nd and 3rd-line setting who have previously received a checkpoint inhibitor and chemotherapy. Cosela appears to help protect bone marrow cells from damage caused by chemotherapy by inhibiting cyclin-dependent kinase 4/6.

BioSight completed enrollment in its ongoing Phase IIb trial of aspacytarabine (BST-236) as a single-agent first-line acute myeloid leukemia (AML) therapy for patients who cannot receive standard intensive chemotherapy. Aspacytarabine is a novel anti-metabolite composed of cytarabine covalently bound to asparagine, acting as a pro-drug of cytarabine.

UNITY Biotechnology announced initial safety and tolerability data from its Phase I trial of UBX1325 for diabetic macular edema and age-related macular degeneration. The drug is a small molecule inhibitor of Bcl-xL.

Larimar Therapeutics announced topline data from its Phase I trial of CTI-1601 for Friedreich’s Ataxia (FA). The drug resulted in dose-dependent increases in frataxin levels from baseline compared to placebo controls. The drug is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with FA.

Orchard Therapeutics published safety and efficacy data from three Phase I/II trials of its OTL-101 gene therapy for adenosine deaminase severe combined immunodeficiency (ADA-SCID). The data showed sustained ADA gene expression, metabolic correction, and functional immune reconstitution in 48 out of 50 patients.

Sequana Medical announced positive topline results from the RED DESERT study with repeated doses alfapump DSR (Direct Sodium Removal) in diuretic-resistant heart failure patients. The alfapump is a fully implantable wireless device that automatically pumps fluid from the abdominal cavity into the bladder.

Clene and its subsidiary Clene Nanomedicine presented updated blinded interim efficacy data from its ongoing Phase II RESCUE-ALS trial of CNM-Au8 for early symptomatic amyotrophic lateral sclerosis (ALS) patients. The blinded data suggest that CNM-Au8 may have potential for neuro-repair in these patients. The therapy is a bioenergetic nanocatalyst, a stable, aqueous suspension of catalytically active gold (Au) nanocrystals.

Amylyx Pharmaceuticals presented the trial design of its Phase III PHOENIX study of AMX0035 (sodium phenylbutyrate (PB)-taurusodiol (TURSO)) for ALS. The trial will evaluate the safety and efficacy of the drug.

QPex Biopharma dosed the first patient in its Phase I trial of ORAvance in drug-resistant gram-negative bacterial infections. ORAvance is an ultra-broad-spectrum oral beta-lactamase inhibitor for use in combination with beta-lactam antibiotics.

Checkpoint Therapeutics completed enrollment for the metastatic cutaneous squamous cell carcinoma cohort in its registration-enabling trial of its anti-PD-L1 checkpoint inhibitor, cosibelimab. Top-line data is expected in the fourth quarter of this year and if successful, the company plans to submit a Biologics License Application (BLA) to the FDA for the drug in the first half of 2022, with a similar application in Europe shortly afterwards.

Ocuphire Pharma completed enrollment in the VEGA-1 Phase II trial of Nyxol and low-dose pilocarpine in presbyopia (farsightedness). Nyxol is a 0.75% phentolamine ophthalmic solution, a once-daily preservative-free eye drop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist.

Progenity presented preliminary results from its Drug Delivery System (DDS) clinical trial (as well as a preclinical study of PGN-600, liquid tofacitinib delivered by DDS). The DDS allows for delivery of high concentrations of proprietary drug formulations by a novel, orally ingestible capsule to the precise site of disease along the GI tract. The results support the use of the DDS for the company’s lead ulcerative colitis combination product candidate and for other uses targeting GI diseases.

MorphoSys and Incyte dosed the first patient in their Phase III frontMIND trial of tafasitamab and lenalidomide in addition to rituximab, cyclophosphamide, doxorubicin, vincristine and prednisone (R-CHOP) compared to R-CHOP alone as first-line treatment for high-intermediate and high-risk patients with untreated diffuse large B-cell lymphoma (CLBCL). Tafasitamab is a humanized, monoclonal antibody against the B-cell specific antigen CD19 and to induce immune cell activation.

Allay Therapeutics announced data from a Phase Ib/IIa trial of its first candidate, ATX-101, for pain after total knee replacement. ATX-101 is a small dissolvable drug that incorporates the local analgesic bupivacaine.

Merck announced positive results from the pivotal neoadjuvant/adjuvant Phase III KEYNOTE-522 trial of Keytruda (pembrolizumab) in combination with chemotherapy as pre-operative (neoadjuvant) treatment and then continuing as a single agent (adjuvant) treatment after surgery. It met the dual primary endpoint of event-free survival (EFS).

Amgen and AstraZeneca published detailed results from the NAVIGATOR Phase III trial of Tezepelumab in severe asthma. Tezepelumab is being jointly developed. It is a potential first-in-class human monoclonal antibody that focuses on the airway epithelium by targeting and blocking thymic stromal lymphopoietin (TSLP).

Exscientia noted that Sumitomo Dainippon Pharma Co. will initiate a Phase I trial of DSP-0038 in the U.S. for Alzheimer’s disease psychosis. DSP-0038 is the third molecule created using Exscientia’s AI technologies to enter clinical trials. They designed DSP-0038 to be a single small molecule with high potency as an antagonist for the 5-HT2A receptor and agonist for the 5-HT1A receptor, while selectively avoiding similar receptors and unwanted targets.

uniQure presented 26-week data from the Phase III HOPE-B gene therapy trial of etranacogene dezaparvovec in hemophilia B patients with pre-existing neutralizing antibodies to AAV5. The therapy is made up of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua).

Beyond Air presented data from both LungFit PRO programs, acute viral pneumonia (including COVID-19) and bronchiolitis, demonstrated a favorable safety profile and encouraging efficacy trends using high concentrated inhaled nitric oxide (NO) for acute viral lung infections in hospitalized patients.

Sio Gene Therapies presented new biomarker data from its trial of AXO-AAV-GM1, for GM1 gangliosidosis. The product is an AAV9-based gene therapy. The data provides direct evidence of biodistribution and biochemical effect in the CNS at the lowest dose.

AzurRx BioPharma announced positive interim data from the first 18 out of 20 patients in its Phase II trial of MS1819 in combination with current standard of care, porcine-derived pancreatic enzyme replacement therapy (PERT), for the treatment of severe exocrine pancreatic insufficiency (EPI) in cystic fibrosis patients. There were clinically meaningful improvements in the primary efficacy endpoint, the Coefficient of Fat Absorption (CFA), with an average gain of 5.9 points from baseline. MS1819 is a recombinant lipase enzyme.

Aspira Women’s Health launched a prospective Clinical Study for Ovarian Cancer Risk Detection. It will enroll over 600 prospective women with adnexal masses, as well as more than 2000 women at high risk for ovarian cancer. Ovarian cancer risk will be evaluated by both CA125 and Aspira’s OVASight proprietary algorithm. The objective is to increase total trial enrollment to validate the serial monitoring aspects of the OVASight algorithm in women who present with an adnexal mass.

Biogen reported that its gene therapy, cotoretigene toliparvovec, failed to hit the primary endpoint of the Phase II/III XIRIUS trial for X-linked retinitis pigmentosa (XLRP). XLRP is a rare, inherited disease of the retina associated with progressive loss of vision as the cells in the retina that sense light slowly deteriorate. The gene therapy is an AAV8 vector-based gene therapy administered by subretinal injection. It delivers a full-length functioning retinitis pigmentosa GTPase regulator (RPGR) protein in XLRP patients whose disease is caused by mutations in the RPGR gene.

Fate Therapeutics announced encouraging interim Phase I data from its ongoing Phase I dose-escalation study of FT516 as monotherapy in relapsed/refractory acute myeloid leukemia (AML). FT516 is the company’s off-the-shelf, iPSC-derived natural killer (NK) cell program.

Genenta Science presented new clinical data from its Phase I/IIa trial of Temferon in glioblastoma multiforme (GBM). The presentation focused on patients who have undergone a follow-up surgical procedure for their cancer. Temferon is based on ex-vivo gene transfer into autologous hematopoietic stem/progenitor cells (HSPCs) to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages (Tie2 Expression Monocytes – TEMs).

Tessa Therapeutics announced early clinical data from its ongoing Phase I dose-escalation study of TT11X in patients with CD30+ lymphomas. TT11X is a CD30-CAR EBVST therapy being co-developed by Tessa and Baylor College Medicine. The data highlighted six patients treated with the therapy and demonstrated a favorable safety profile and encouraging clinical activity even at lower doses.

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