Clinical Catch-Up: 89bio, Tonix, Arcutis and More

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As the summer winds down, many biopharma companies are starting up new trials and reporting data from existing ones. Here’s a look at last week’s top clinical trial news.

89bio presented data from its Phase II ENTRIGUE proof-of-concept trial of pegozafermin in severe hypertriglyceridemia (SHTG) at the European Society of Cardiology (ESC) Congress 2022.

The data, consistent with previous studies, demonstrated that the drug significantly decreased triglyceride levels, reduced atherogenic lipoproteins, and improved liver fat and glycemic control markers in select patients. It was generally well-tolerated with a favorable safety profile. The drug is a specifically engineered glycoPEGylated analog of fibroblast growth factor 21 (FGF21) being developed for the treatment of non-alcoholic steatohepatitis (NASH) and SHTG.

Tonix Pharmaceuticals enrolled the first patient in the Phase II PREVAIL trial of TNX-102 SL for a subset of patients with Long COVID syndrome whose symptoms overlap with fibromyalgia.

Long Covid, or Post-Acute Sequelae of COVID-19 (PASC), has a long list of symptoms, including multi-site pain, fatigue and sleep disturbance, all of which are associated with central sensitization syndromes like fibromyalgia. TNX-102 SL is a patented sublingual formulation of cyclobenzaprine hydrochloride that allows rapid transmucosal absorption and decreased production of a long half-life active metabolite, norcyclobenzaprine. It is being developed for Long Covid, fibromyalgia, PTSD, alcohol use disorder and agitation in Alzheimer’s disease.

Belite Bio initiated enrollment for the U.S. Phase III trial of LBS-008 in patients with Stargardt Disease (STGD1), a progressive blinding disease.

It is the most common inherited retinal dystrophy that causes blurring and/or loss of central vision in adults and children. LBS-008 is an oral, small molecule retinol binding protein 4 (RBP4) antagonist that selectively decreases the delivery of vitamin A (Retinol) to the eye, resulting in a decrease of toxic vitamin A byproducts implicated in the onset and progression of the disease.

Immatics treated the first patient in its Phase Ib expansion cohort C of IMA203CD8 for hard-to-treat solid tumors.

The drug is a second-generation TCR-T monotherapy where a proprietary CD8aß co-receptor is added to PRAME-specific IMA203 T cells. The CD8 co-receptor plays a key role during T cell antigen recognition and activation, allowing the engagement of CD8 and CD4 T cells in their attack against cancer cells. PRAME refers to a melanoma antigen preferentially expressed in tumors.

Arcutis Biotherapeutics completed enrollment of its INTEGUMENT-2 pivotal Phase III trial of roflumilast cream 0.15% in adults and children with atopic dermatitis (AD).

The cream is a once daily topical formulation of roflumilast, a potent and selective phosphodiesterase type 4 inhibitor (PDE4). The company also wrapped enrollment in an identically designed pivotal Phase III trial, INTEGUMENT-1, earlier in August. Topline data from both trials is expected by the end of this year.

Apellis Pharmaceuticals announced topline data at 24 months, demonstrating increased effects over time with intravitreal pegcetacoplan.

The results were from the Phase III DERBY and OAKS trials of the drug, a targeted C3 therapy, in geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Both monthly and every-other-month pegcetacoplan demonstrated clinically meaningful reduction in GA lesion growth from baseline compared to sham. Between months 18-24, the treatment effect accelerated compared to the previous six months.

Ranok Therapeutics initiated patient dosing in the U.S. for the Phase I/II CHAMP-1 trial of RNK05047 for advanced solid tumors or diffuse large B-cell lymphoma.

The drug is the first based on the company’s CHAMP technology and the first BRD4 protein degrader to enter clinical testing. BRD4 is a master regulator of oncogenes found in multiple cancer types. The CHAMP, or Chaperone-mediated Protein Degrader/Degrader, platform leverages the cellular chaperone network, which regulates the folding and stability of proteins.

Anthos Therapeutics initiated its second Phase III trial of abelacimab for patients at risk of thrombosis and are diagnosed with gastrointestinal/genitourinary (GI/GU) cancers.

These cancers come with a higher risk of bleeding than other cancers. Abelacimab is a novel dual-acting human monoclonal antibody targeting both Factor XI and Factor XIa. In the MAGNOLIA trial, monthly treatment with abelacimab is being compared to once daily injection of dalteparin, the current standard of care anticoagulant for patients with GI/GU cancers.

TG Therapeutics published the results from the ULTIMATE I and II Phase III trials of ublituximab in patients with relapsing forms of multiple sclerosis.

The results were published in The New England Journal of Medicine. The drug is an anti-CD20 monoclonal antibody. The studies found that the treatment, compared to teriflunomide, resulted in significantly lower annualized relapse rates, a decrease in the total number of MRI-detectable lesions, and improved rates of patients achieving no evidence of disease activity (NEDA).

Pfizer reported its Phase III trial for its respiratory syncytial virus (RSV) vaccine demonstrated efficacy in 85.7% of adults over 60 years of age with a more severe form of the infection.

The RENOIR trial studied the company’s prefusion F vaccine candidate (RSVpreF). Approximately 37,000 patients participated globally.

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