Your Daily Pharma Scoop: Reata Provides Update, Assembly Biosciences Data, Bellicum's BPX-501

Summary

• Reata provided an update on the CARDINAL study of bardoxolone.
• Assembly Biosciences reports results for ABI-H0731.
• FDA lifts clinical hold on Bellicum’s BPX-501 studies.

Analysis focus: Reata Pharmaceuticals

Today we will discuss Reata Pharmaceuticals (NASDAQ:RETA), which provided an update on the phase 2 portion of the CARDINAL study of bardoxolone methyl (bardoxolone) in patients with chronic kidney disease (CKD) due to Alport syndrome.

Bardoxolone is an oral, once-daily activator of Nrf2. A transcription factor, Nrf2 induces molecular pathways that help in reducing inflammation. It restores mitochondrial function, reduces oxidative stress and inhibits pro-inflammatory signaling. The investigational candidate has already been granted an Orphan Drug designation by the FDA for the treatment of Alport syndrome and pulmonary arterial hypertension.

The phase 2 CARDINAL study enrolled 30 patients, who received bardoxolone orally, once-daily for two years. The company said that 90% of the patients remain on the study. These patients will be included in the Week 52 withdrawl analysis.

Reata reported that efficacy results show that significant increases in kidney function are maintained at week 36. The efficacy was measured by estimated glomerular filtration rate (eGFR). At week 36, the mean improvement from baseline in eGFR was 11.3 mL/min/1.73 m2 (n=27; p<0.0000001). This is not significantly different than the change observed at Week 12.

Alport syndrome is a rare disease, affecting an estimated 30,000 to 60,000 people in the U.S. alone, according to data from the Alport Syndrome Foundation.

Reata has a separate phase 3 study with bardoxolone. The phase 3 CATALYST study is evaluating bardoxolone as a treatment of connective tissue disease associated pulmonary arterial hypertension.

Reata shares are down nearly 26% this year. The company ended 2017 with $129.78 million in cash on its balance sheet. The company’s current cash position represents nearly 25% of market capitalization. With two late-stage programs, we believe RETA is worth a look based on current valuation.

Stocks in News: Analysis of ASMB

Assembly Biosciences (ASMB) announced promising results from phase 1a and 1b studies of lead candidate ABI-H0731. The company is currently developing the treatment for chronic hepatitis B virus (HBV) infection.

Analysis: Assembly Biosciences reported results from the completed phase 1a study in healthy volunteers. The results showed ABI-H0731 to be safe and well-tolerated with no serious adverse events or dose-limiting toxicities. The company said that data from ongoing phase 1b study in treatment-naïve HBV patients showed viral load declines of as much as 99% in both e-antigen-positive and e-antigen-negative participants in a dose-dependent manner.

In other news

Bellicum Pharmaceuticals (BLCM) announced that the FDA has lifted clinical hold on studies of BPX-501 in the U.S. The company is currently working with U.S. clinical sites to resume patient recruited based on protocols it amended in consultation with the FDA, including guidance on monitoring and management of neurologic adverse events.

Bristol-Myers Squibb (BMY) announced that it has entered into a research collaboration with Harvard Stem Cell Institute's Harvard Fibrosis Network. As per the terms of the collaboration agreement, Harvard academic researchers will collaborate with BMY scientists on four projects over three years.

Akers Biosciences (AKER) announced that it has engaged two new U.S. distributors for its point-of-care PIFA Heparin/PF4 Rapid Assay.

Argenx (ARGX) announced the receipt of third preclinical milestone from its collaboration with LEO Pharma, following the approval of the clinical trial application (CTA) filing for ARGX-112.

Aeglea BioTherapeutics (AGLE) announced preliminary results from a phase 1/2 clinical evaluating AEB1102 AEB1102 (pegzilarginase) in two patients with Arginase 1 Deficiency showed a treatment effect as measured by standardized assessment tools. The data reported showed improvement in two patients in the Six-Minute Walk Test (6MWT) of 31.4% and 23.4%, respectively. Both were significantly above the Minimal Clinically Important Difference (MCID) of 9% at week 8.

Pfizer (PFE) announced that the first patient has been dosed in a phase 1b clinical study evaluating Duchenne muscular dystrophy (DMD) candidate PF-06939926.

Johnson & Johnson (JNJ) announced that additional analyses from the CANVAS studies showed the renal protective benefits in type 2 diabetics treated with INVOKANA. The data reported INVOKANA showed reduced urinary albumin to creatinine ratio, a biomarker for chronic kidney disease, by 17% from baseline in people with T2D with preserved estimated glomerular filtration rate (eGFR), a measure of kidney function. The reduction was 23% in those with reduced eGFR.

Analyst’s Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.