Zacks Small Cap Analysis – ARWR: PALISADE Section 3 Outcomes Printed in NEJM… – Cyber Tech

By David Bautz, PhD

NASDAQ:ARWR

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Section 3 PALISADE Outcomes Printed in The New England Journal of Drugs

On September 2, 2024, Arrowhead Prescribed drugs, Inc. (NASDAQ:ARWR) introduced outcomes for the Section 3 PALISADE trial of plozasiran in sufferers with familial chylomicronemia syndrome (FCS). The outcomes had been offered in a late-breaking oral presentation on the European Society of Cardiology (ESC) Congress 2024 and in addition revealed in The New England Journal of Drugs (Watts et al., 2024). The corporate held an investor name on September 3, 2024 and the slides for it may be discovered right here. Plozasiran was additionally not too long ago granted Breakthrough Remedy designation by the U.S. FDA. It beforehand obtained Orphan Drug Designation and Quick Monitor Designation by the FDA together with Orphan Drug Designation by the European Medicines Company.

The PALISADE trial enrolled 75 sufferers with persistent chylomicronemia, with or and not using a genetic prognosis of FCS, who had been randomized to subcutaneous plozasiran at 25 mg (n=26), 50 mg (n=24), or placebo (n=25) each three months for 12 months. Inclusion standards included a historical past of a number of triglyceride (TG) measurements above 1000 mg/dL (11.3 mmol/L) together with a minimum of one of many following: prior genetic testing prognosis for FCS; recurrent episodes of acute pancreatitis; recurrent hospitalizations for extreme belly ache with out different explainable trigger; historical past of childhood pancreatitis; or household historical past of HTG-induced acute pancreatitis. The next desk exhibits the baseline traits for the sufferers, together with the median baseline TG stage of 2044 mg/dL throughout all cohorts.

The next graphs present the median discount in triglycerides and stage of APOC3. On the ten-month time level, the median discount in fasting triglycerides was -80% within the 25 mg cohort, -78% within the 50 mg cohort, and -17% for the placebo cohort (P<0.001). The discount in APOC3 was -93% within the 25 mg cohort, -96% within the 50 mg cohort, and -1% within the placebo group (P<0.001).

An vital secondary efficacy endpoint in contrast the incidence of positively adjudicated acute pancreatitis (AP) in a pre-specified pooled evaluation of the 25 mg and 50 mg plozasiran teams versus the pooled placebo group. Of the 38 suspected circumstances of acute pancreatitis that had been referred for adjudication, 9 episodes in seven sufferers had been positively adjudicated. The outcomes confirmed an 83% discount within the danger of creating acute pancreatitis for plozasiran-treated sufferers in comparison with these handled with placebo. A complete of two circumstances occurred in two of fifty sufferers (4%) receiving plozasiran versus seven circumstances that occurred in 5 of 25 sufferers (20%) receiving placebo (OR 0.17; P=0.03).

The next desk supplies the abstract of opposed occasions through the trial. There was a better fee of great or extreme opposed occasions within the placebo group, no modifications had been seen in platelet counts, and there have been no deaths reported within the examine.

Comparability with Olezarsen

Ionis Prescribed drugs is creating olezarsen, an antisense oligonucleotide that targets APOC3. Ionis not too long ago reported that the NDA for olezarsen for the remedy of FCS was accepted for Precedence Evaluate with a PDUFA date of December 19, 2024. The outcomes from the Section 3 BALANCE trial of olezarsen in FCS had been not too long ago revealed in The New England Journal of Drugs (Stroes et al., 2024). Whereas troublesome to carry out cross-trial comparisons, the next chart supplies a few of the parameters and outcomes for the Section 3 trials for olezarsen and plozasiran.

A number of factors that we predict are worthwhile for buyers to contemplate embody:

•   Olezarsen is dosed as soon as each 4 weeks whereas plozasiran is dosed as soon as each three months

•   All the topics within the olezarsen examine had genetically confirmed FCS, whereas between one-half to two-thirds of topics within the plozasiran trial had generically confirmed FCS

•   The triglyceride and APOC3 reductions had been better for plozasiran than for olezarsen (with the caveat of median vs. imply values reported), with much less variability over time for plozasiran than for olezarsen.

•   Plozasiran confirmed a statistically vital lower within the danger of creating acute pancreatitis (AP), nonetheless the olezarsen trial was not powered for statistical significance however nonetheless confirmed a positive fee ratio.

Total, we imagine the plozasiran outcomes are extremely aggressive and may place the product to do effectively within the FCS market.

Conclusion

The outcomes from the Section 3 PALISADE trial of plozasiran in FCS are very encouraging and we imagine the drug compares fairly favorably to olezarsen, which has a PDUFA date of December 19, 2024 for FCS. We sit up for the corporate submitting the NDA for plozasiran and extra info on plans for its potential industrial launch subsequent 12 months. With no modifications to our mannequin our valuation stays at $70 per share.

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