Clinical Catch-Up: December 30, 2019 – January 2, 2020

Research

Even with the holiday week, there was still some clinical trial news coming out. Here’s a look.

Hookipa Pharma dosed the first patient in its Phase I/II clinical trial of HB-201 for Human Papillomavirus 16-positive cancers. HB-201 is a TheraT platform-based vector from the arenavirus family that expresses a non-oncogenic but highly antigenic E6/E7 fusion protein from HPV16. It is an open-label dose-escalation and dose-expansion trial in 100 patients with treatment-refractory HPV16+ cancers.

X4 Pharmaceuticals initiated a Phase Ib clinical trial of mavorixafor (X4P-001) in combination with ibrutinib (Imbruvica) for Waldenstrom’s macroglobulinemia (WM). WM is a rare form of non-Hodgkin’s lymphoma. Mavorixafor is a potential first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4. Imbruvica is Janssen, a Johnson & Johnson company’s Bruton’s tyrosine kinase inhibitor.

WAVE Life Sciences reported disappointing data from its ongoing Phase Ib/IIa PRECISION-HD2 trial in Huntington’s disease (HD). The trial was evaluating WVE-120102 in HD. Analysis showed that all patients receiving multiple intrathecal doses of the therapeutic compared to placebo, a statistically significant reduction of mutant huntingtin (mHTT) protein was seen in cerebrospinal fluid (CSF) of 12.4%. Further analysis of a dose response across treatment groups suggested a statistically significant response in mHTT reduction at the highest doses. Although the reduction in mHTT with the drug compared to placebo was statistically significant, there was no change in total huntingtin protein (tHTT) compared to placebo.

WVE-120102 is an allele-selective molecule that preferentially lowers mHTT protein by targeting a point mutation called single nucleotide polymorphism (SNP) rs362331 (SNP2), which maintains the level of health HTT protein relatively intact. The wtHTT protein plays an important role in neuronal function and is potentially neuroprotective in adult brains.

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Principia Biopharma announced updated anticipated timing for final results from its Phase III trial of PRN1008 in pemphigus, in addition to complete response rates from its Phase II Part B open-label trial. PRN1008 is an oral, small molecule, reversible covalent inhibitor of Bruton tyrosine kinase (BTK). It is present in the signaling pathways of most types of white blood cells except for T-cells and plasma cells. The company believes its accelerated enrollment timelines will produce final data in the second half of 2021 compared to the first half of 2022. Also, in the Phase II Part B trial, the complete response rate was 40%.

Jazz Pharmaceuticals enrolled the first patient in the Phase II/III trial of JZP-458. The compound is a recombinant Erwinia asparaginase molecule that uses a novel Pseudomonas fluorescens expression platform. The trial is evaluating the compound as a potential treatment for pediatric and adult acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) patients who are hypersensitive to E. coli-derived asparaginases.

Lineage Cell Therapeutics announced additional patient data from its ongoing Phase I/IIa clinical trial of OpRegen. OpRegen is the company’s retinal pigment epithelium (RPE) transplant therapy for dry age-related macular degeneration. The first Cohort 4 patients treated using both a subretinal delivery system and the company’s new thaw-and-inject formulation of OpRegen continues to show improvements in vision, having gained 25 readable characters six months after administration.

BioXcel initiated its SERENITY program, two Phase III trials of BXCL501 for acute treatment of agitation in patients with schizophrenia and bipolar disorder. Topline data are expected in mid-2020. BXCL501 is a potential first-in-class, proprietary sublingual thin film of dexmedetomidine, a selective alpha-2a receptor agonist for acute agitation.

Clover Biopharmaceuticals dosed the first patient in its Phase III trial of SCB-808, a proposed biosimilar to Enbrel (etanercept) in a prefilled syringe formulation for rheumatic diseases, including ankylosing spondylitis and rheumatoid arthritis. The Phase III, multi-center trial is in China.

Eli Lilly and Company opened the LIBRETTO-531 clinical trial of selpercatinib (LOXO-292) for treatment-naïve RET-mutant medullary thyroid cancer (MTC). The trial will enroll 400 patients with advanced or metastatic RET-mutant MTC who have received no previous systemic therapy for metastatic disease. LOXO-292 is a highly selective and potent, oral new drug that inhibits native RET signaling as well as anticipated acquired resistance mechanisms.

Sol-Gel Technologies announced topline results from two Phase III trials for Twyneo. Twyneo is the company’s combination of microencapsulated tretinoin 0.1% and microencapsulated benzoyl peroxide 3% cream. The trials showed statistically significant improvement on all co-primary endpoints in acne vulgaris.

Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, announced results from a Phase II trial of AKCEA-APO(a)-LRX (TQJ230) in patients with established cardiovascular disease and elevated lipoprotein (a) levels. The results were published in The New England Journal of Medicine (NEJM). The drug is an antisense therapy developed using Ionis’ advanced LICA technology platform designed to inhibit the production of apoliprotein(a). The data demonstrated the drug could get 98% of patients below 125 nmol/L, the established threshold for a Lp(a)-driven CVD in patients already on statins.

Novan reported topline results from its Phase III B-SIMPLE trial for SB206 for molluscum contagiosum. Although the trial did not hit its primary endpoint in B-SIMPLE1 and B-SIMPLE2, the company says that multiple sensitivity analyses support continuation. As a result, the topline results are for efficacy data only. SB206 is a topical nitric oxide-releasing therapy.

The primary endpoint was the proportion of patients with complete clearance of all treatable molluscum lesions at the 12-week point. The company emphasizes that the data was “near statistically significant” in B-SIMPLE2 and was statistically significant in the secondary endpoint and several pre-specified sensitivity analyses. The secondary endpoint was complete clearance at Week 8. They also say that B-SIMPLE1, which also did not show statistical significance in the primary endpoint, was supportive and consistent with the B-SIMPLE2 results.

Incyte Corporation’s pivotal Phase III GRAVITAS-301 trial of itacitinib in combination with corticosteroids in treatment-naïve acute graft-versus-host disease (GVHD) didn’t hit the primary endpoint. Itacitinib is a novel and selective JAK1 inhibitor. The primary endpoint was improved overall response rate (ORR) at Day 28 compared to placebo plus corticosteroids. The ORR was 74% compared to 66.4%, respectively. Although there was improvement, it did not achieve statistical significance. There was also no difference seen in non-relapse mortality (NRM) at Month 6, which was the trial’s key secondary endpoint.

DURECT Corporation announced results from its Phase IIa trial of DUR-928 in mild to moderate plaque psoriasis. The drug did not demonstrate a benefit over placebo. The company indicates it does not plan to continue development of topical DUR-928 in psoriasis. DUR-928 appears to modulate the activity of nuclear receptors playing an important regulatory role in cellular functions, such as lipid homeostasis, inflammation, and cell survival. It is a sulfated oxysterol that epigenetically modifies gene activity without changing the DNA sequence itself.

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