BioCryst Berotralstat PDUFA, And Other News: The Good, Bad And Ugly Of Biopharma

Summary

• BioCryst's Berotralstat PDUFA has been set for December 3, 2020.
• Sarepta progresses with muscular dystrophy study.
• Aimmune's Palforzia produces positive data.
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BioCryst Pharmaceuticals Goes Ahead with Expanded Access Program for Berotralstat

BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX) announced launching its Expanded Access Program for berotralstat. The program will allow physicians to request the drug for hereditary angioedema patients who do not have access to the product through a clinical trial. BioCryst has filed an application with the FDA for berotralstat. The PDUFA date has been set at December 3, 2020.

As per the FDA, expanded access program is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. Berotralstat is indicated for patients suffering from hereditary angioedema, which is an inherited disorder. The ailment causes recurring episodes of severe swelling. Such swelling can be found in the limbs, intestinal tract, airway and face. The drug candidate is designed to be taken orally once a day.

The company recently released long-term data for berotralstat, highlighting its safety and tolerability. The data collected from the APeX-2 trial demonstrated that 150 mg dosage of the drug reduced patients' monthly use of Standard of Care medication by 53.6 percent. It also reduced the number of HAE incidents needing acute SoC treatment by 49.2 percent in comparison to placebo. The data also showed that the percentage of HAE incidents needing re-treatment with multiple doses of on-demand therapy was lower for patients administered berotralstat than the patients who were on placebo.

Patients who were administered the drug candidate during the trial also demonstrated quick and steady decline in the frequency of their HAE incidents during the course of 48 weeks. Thirty patients who completed 48 weeks of the regimen had mean attack rate of 1.4 attacks per month after a month of treatment. This metric further declined to 1.0 attacks per month at month 12. The baseline stat for this metric was at 2.9 incidents per month.

Swift and enduring results were also shown by patients who switched from placebo to 150 mg drug after week 24. There was significant decrease in their HAE attack rate. The mean attack rate declined to 0.5 attacks per 28 days at month seven mark and then to 0.4 attacks per 28 days at month 12. The company also carried out an integrated 48-week analysis across both the APeX-2 and APeX-S trials. The analysis showed that the drug candidate was found to be safe and generally well tolerated in 342 patients. The most frequent adverse drug reactions were mild-to-moderate in their intensity. Some patients complained of gastrointestinal events. However, even these events were brief in duration and were self-limited.

Apart from berotralstat, BioCryst also has several other drug candidates in its product development portfolio. Its BCX9930 is an oral Factor D inhibitor, being developed for treating complement-mediated diseases. Its other drug candidate galidesivir is emerging as a potential treatment for COVID-19, Marburg virus disease and Yellow Fever. The company is also working on BCX9250, an ALK-2 inhibitor for treating fibrodysplasia ossificans progressiva.

Sarepta Therapeutics Moves Ahead with Muscular Dystrophy Study

Sarepta Therapeutics (SRPT) announced positive results from a study of its lead drug candidate SRP-9003. The data comprised safety and expression results from its three clinical trial participants in the high-dose cohort examined at 60 days. It also included one year functional data from three clinical trial participants in the low dose group.

The study consists of two cohorts, based on their dosage of the drug candidate. The low dose cohort or Cohort 1 participants were given a one-time infusion of SRP-9003 dosed at 5x1013 vg/kg while the patients in the high dose cohort or Cohort 2 were administered a one-time infusion of 2x1014 vg/kg. Doug Ingram, President and CEO, Sarepta said, "SRP-9003 employs the same vector, AAVrh74, and same promoter, MHCK7, as SRP-9001, our therapy in development to treat Duchenne muscular dystrophy. And Cohort 2 received a similar dose as our ongoing SRP-9001 studies for Duchenne." The company is currently working on selecting the dose for its pivotal trial of the drug candidate.

Preliminary results from cohort 2 showed that there was strong dose-dependent increase in transduction and expression transduction and expression in high dose cohort in comparison to low dose cohort. The mean expression of transduced beta-SG, properly localized to the muscle sarcolemma, was at 72.3 percent for Cohort 2. Mean fiber intensity for the cohort was measured at 73.1 percent compared to normal control. All the members of the cohort showed decline in their serum creatine kinase levels from baseline. Cohort 2 showed one serious adverse event of dehydration resulting from vomiting 3 days after infusion. Rest of the adverse events were generally mild to moderate in their severity.

At one-year milestone, the patients in cohort 1 kept showing improvements in all functional measures such as time to rise, 100-meter walk test etc. over the baseline. The group did not show any new drug related safety signal.

Aimmune Posts Results from Palforzia

Aimmune Therapeutics (AIMT) reported positive data from its follow-on study of PALISADE trial. The company said that the data showed 80 percent of patients treated by the drug candidate to be successfully desensitized to peanut proteins after two years of daily dosing. Palforzia was approved by the FDA in January. The drug seeks to reduce sensitivity reaction to peanuts by exposing patients to peanuts over a period of time.

The data has been collated from ARC004, an open-label, rollover study of the pivotal Phase 3 PALISADE trial. Daniel Adelman, M.D., Chief Medical Officer of Aimmune said, "Additionally, new insights about the daily burden of living with peanut allergy will be presented to highlight the need for an approved treatment for peanut allergy. To that end, we take great pride in our leadership and dedication to improving the lives of those affected by peanut allergy." The follow up data also provided insights about safety and efficacy profile of Palforzia.

At year 2, patients given daily dose exhibited ongoing immunomodulation. They also showed elevated desensitization which further increased over time. The patients on the therapy also experienced lower rates of adverse events. 80 percent of the patients who participated in a food challenge showed desensitivity to 2,000 mg of peanut protein.

PALFORZIA is an oral immunotherapy indicated for the mitigation of allergic reactions, including anaphylaxis, that may occur with accidental exposure to peanut. The drug has been approved by the FDA for treating patients with a confirmed diagnosis of peanut allergy. Initial Dose Escalation may be administered to patients aged 4 through 17 years while Up-Dosing and Maintenance may be continued in patients 4 years of age and older. The drug is meant to be used along with a peanut avoidant diet.

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.