Today’s Daily Dose brings you news about disappointing results from Soleno’s Prader-Willi syndrome trial; the Court ruling in a patent infringement lawsuit filed by Opiant/Emergent against Teva; encouraging additional data from Sarepata’s limb-girdle muscular dystrophy Type 2E trial; and Enochian’s path forward for ENOB-HV-01, a potential cure for HIV.
1. Soleno’s DCCR Fails To Control Appetite
Shares of Soleno Therapeutics Inc. (SLNO) plunged more than 40 percent in extended trading on Monday, following disappointing results from the DESTINY PWS trial.
DESTINY PWS is a phase III study evaluating Diazoxide Choline Controlled-Release for Prader-Willi syndrome or PWS in short. PWS is a rare disease, which is characterized by short stature, cognitive disabilities, and an insatiable appetite (hyperphagia) in children that often contributes to obesity and type 2 diabetes. This complex genetic neurobehavioral/metabolic disorder is caused due to the loss or lack of expression of a set of genes on chromosome 15.
The DESTINY PWS study, which involved 127 PWS patients at 29 sites in the U.S. and UK, did not meet its primary endpoint of change from baseline in hyperphagia, the hallmark of PWS.
However, the Company noted that there were significant improvements in a prespecified subgroup with severe hyperphagia and significant positive changes in two of three key secondary endpoints.
An open-label, safety extension study of Diazoxide Choline Controlled-Release (DCCR) tablets in PWS patients completing the DESTINY PWS trial, dubbed C602, is ongoing. An interim analysis of this study has shown a further reduction in hyperphagia of 48% after 6 months of DCCR treatment.
To date, there has been no approved drug for PWS that is effective in controlling the appetite of the patients (increased appetite).
Pfizer’s (PFE) recombinant human growth hormone Genotropin (somatropin) and Sandoz/Novartis’ Omnitrope, a biosimilar for Genotropin, are the FDA-approved drugs for the treatment of growth failure in children with PWS.
SLNO closed Monday’s trading at $3.24, down 0.61%. In after-hours, the stock was down 48.77% at $1.66.
2. Opiant, Emergent Hit As Court Rules In Teva’s Favor
Shares of Opiant Pharmaceuticals Inc. (OPNT) and its commercial partner Emergent BioSolutions Inc. (EBS) took a hit on Monday after a Court ruled in favor of Teva in a patent infringement suit related to NARCAN Nasal Spray.
Developed by Opiant and licensed to Adapt Pharma, which is now under the aegis of Emergent BioSolutions, NARCAN Nasal Spray is approved for the emergency treatment of known or suspected opioid overdose.
Between 2016 and 2018, TEVA filed applications with the FDA seeking regulatory approval to market a generic version of NARCAN before the expiration of the ‘253, ‘747, ‘177, ‘965, ‘644, and ‘226 patents. Perrigo is another company that is seeking to market generic versions of NARCAN. Opiant filed a suit against Teva in October 2016, and against Perrigo in 2018, alleging the proposed generic versions of NARCAN infringes on the patent rights.
Teva received final approval for its generic version of NARCAN Nasal Spray last April.
NARCAN accounts for a major chunk of Emergent BioSolutions’ product sales. In 2019, NARCAN accounted for $280.4 million out of the total product sales of $903.5 million.
Emergent intends to appeal the decision to the Court of Appeals for the Federal Circuit.
In February of this year, Emergent and Perrigo entered into a settlement agreement to resolve the ongoing litigation related to NARCAN. The Perrigo settlement agreement is subject to review by the U.S. Department of Justice and the Federal Trade Commission, and entry of an order dismissing the litigation by the U.S. District Court for the District of New Jersey.
Now that the Court has ruled in favor of Teva, it remains to be seen what impact it’s going to have on the settlement that has been reached with Perrigo.
EBS closed Monday’s trading at $68.69, down 20.96%. OPNT plunged 22.74% to close Monday’s trading at $9.04.
3. Sarepta On A Strong Footing
Sarepta Therapeutics Inc. (SRPT), which announced positive and robust expression and biomarker data and positive 9-month functional results from the low-dose cohort (Cohort I) of SRP-9003, its investigational gene therapy to treat limb-girdle muscular dystrophy Type 2E, trial, last year, announced encouraging preliminary results from Cohort 2 on Monday.
Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.
Results reported on Monday included safety and expression results from three clinical trial participants in the high-dose cohort (Cohort 2) measured at 60 days and one-year functional data from three clinical trial participants in the low-dose cohort (Cohort 1).
According to the Company, preliminary results from Cohort 2 showed a strong dose-dependent increase in transduction and expression transduction and expression when compared with Cohort 1.
The three participants showed robust mean expression of 72.3% of transduced beta-SG, properly localized to the muscle sarcolemma, as measured by immunohistochemistry (IHC). These results exceeded the pre-defined measure of success for the study of 50% positive fibers which was previously achieved in Cohort 1. The beta-sarcoglycan gene is involved in autosomal recessive limb-girdle muscular dystrophy.
Mean fiber intensity, as measured by IHC, was 73.1% compared to normal control.
All participants showed robust quantification of beta-SG, as measured by Western blot, with mean beta-SG of 62.1% of normal control.
All participants showed a reduction in serum creatine kinase (CK) levels from pre-treatment baseline measure to the last measure at 90 days, with a mean CK reduction of 89.1% from baseline. CK is an enzyme biomarker strongly associated with muscle damage.
The one-year functional data from three clinical trial participants in the low-dose cohort (Cohort 1) showed that there were continuous improvements from baseline across all functional measures, including the North Star Assessment for Limb-Girdle Muscular Dystrophies, time-to-rise, four-stair climb, 100-meter walk test, and 10-meter walk test.
SRPT touched a new 52-week high of $159.75 in intraday trading on Monday, before closing at $159.68, up 7.90%.
4. Enochian On Track With Potential HIV Cure
Shares of Enochian Biosciences Inc. (ENOB) soared as much as 222% in early trading on Monday, following successful completion of an Initial Targeted Engagement for Regulatory Advice (INTERACT) meeting with the FDA related to ENOB-HV-01, a potential cure for HIV.
The Company’s ENOB-HV-01 is a novel approach to autologous stem cell transplantation, with the potential to cure HIV by increasing engraftment of gene-modified cells that are resistant to HIV infection.
A pre-IND submission for the Company’s gene-modified cell therapy platform ENOB-HV-01 is expected to be made in 2021.
ENOB touching a high of $13.43 in early trade on Monday, before closing at $6.06, up 47.09%.
5. Stocks That Moved On No News
TCR2 Therapeutics Inc. (TCRR) closed Monday’s trading at $16.85, up 31.23%.
NextCure Inc. (NXTC) closed Monday’s trading at $30.38, up 19.32%.
Genfit SA (GNFT) closed Monday’s trading at $6.44, up 19.26%.
Protara Therapeutics Inc. (TARA) closed Monday’s trading at $36.26, down 26.57%.
InflaRx N.V. (IFRX) closed Monday’s trading at $6.58, down 12.15%.
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