Penny stock forges ahead with an NDA for Duchenne treatment, seeking priority review – Endpoints News

Af­ter set­backs and de­lays, the Swiss biotech San­thera is look­ing to have its treat­ment for Duchenne mus­cu­lar dy­s­tro­phy get past the FDA.
San­thera, along with Rever­a­Gen Bio­Phar­ma, an­nounced it has com­plet­ed the rolling sub­mis­sion for an NDA to US reg­u­la­tors and is seek­ing a pri­or­i­ty re­view for va­morolone to treat Duchenne. San­thera states that the “core” of its NDA is the pos­i­tive da­ta from a Phase IIb tri­al that show the safe­ty and ef­fec­tive­ness of the drug.
The re­sults, which were post­ed last year, ex­am­ined dosage lev­els of 2 mg/kg per day and 6 mg/kg per day, look­ing at how 121 pa­tients’ time to stand from ly­ing flat was short­ened af­ter 24 weeks of treat­ment. In the high dose arm, pa­tients re­duced their time to stand from 6 sec­onds to 4.6 sec­onds, while the place­bo group went from 5.4 to 5.5 sec­onds. That equat­ed to a p-val­ue of p=0.002. San­thera didn’t re­veal the time dif­fer­ence for the low­er dose but re­port­ed a p-val­ue of p=0.02.
So far, the news of San­thera’s march to­ward the NDA has slight­ly perked up the ears of in­vestors, but the stock price $SANN.SW is still well down in the pen­ny stock range as it faces a price falloff of over 50% since last Oc­to­ber.
“Com­ple­tion of the va­morolone NDA sub­mis­sion is a ma­jor step to­wards our goal of bring­ing this in­ves­ti­ga­tion­al ther­a­py to pa­tients liv­ing with DMD, and rep­re­sents a vi­tal mile­stone for San­thera,” San­thera CEO Dario Ek­lund said in a state­ment.
With San­thera ap­ply­ing for pri­or­i­ty re­view, it ex­pects the process to take 60 days if the re­view is grant­ed and is an­tic­i­pat­ing an ap­proval date for some time in the mid­dle of next year. San­thera al­so sub­mit­ted its pitch to the EMA in Sep­tem­ber, seek­ing ap­proval for va­morolone in the EU, Nor­way, Liecht­en­stein and Ice­land.
San­thera has had a wild cou­ple of years, as it was forced to halt a Phase III study in 2020 for its now-de­funct idebenone pro­gram af­ter it failed an in­ter­im analy­sis. The com­pa­ny had been gear­ing up for a po­ten­tial com­mer­cial launch through­out Eu­rope and had to with­draw its mar­ket­ing au­tho­riza­tion ap­pli­ca­tion from the EMA. This led to most of its staff be­ing laid off in the sub­se­quent re­struc­tur­ing.
Af­ter the Phase IIb wins for va­morolone in Duchenne, San­thera was ex­tend­ed a life­line as it opened up some liq­uid­i­ty to get in­to the next year. How­ev­er, San­thera was hop­ing that the FDA would con­sid­er the fil­ing com­plete and ac­cept­ed for re­view by the end of Au­gust or ear­ly Sep­tem­ber.
Digital medicine has become a major component in an ever-evolving healthcare environment. You’re likely already familiar with digital technologies such as telehealth platforms and electronic health records. Plus, there are hundreds of thousands of wellness apps available to anyone on smartphone app stores.
But there’s a reason digital medicine and even more specifically, digital therapeutics exist in a subcategory of their own. A digital therapeutic is typically described as an evidenced-based software solution, which are held to the same standards of regulatory oversight as more traditional medical devices. Digital Therapeutics can positively impact the trajectory of a patient’s disease and are approved to deliver specific clinical outcomes. In other words, Digital Therapeutics are viewed as clinically validated software solutions used by clinicians to treat, manage, and prevent a broad spectrum of diseases and disorders. This differs significantly from consumer health apps that track health or aid in the care process in some way.
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The vaccine R&D revolution has gone global. How will the lessons from Covid-19 reshape the way we study, test, produce and distribute vaccines around the world? Join me on Wednesday for a panel discussion. You can sign up at no cost here.
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On Friday afternoon, the FDA’s oncology drug advisory committee unanimously voted 16-0 against the approval of Y-mAbs’ experimental drug for CNS metastases in pediatric neuroblastoma patients. The key issue the FDA pointed to was that the external control group for Y-mAbs’ pivotal study was very different from the treatment group at baseline, an issue it highlighted in briefing docs released yesterday.
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One of the key programs that inspired Sanofi’s $2.5 billion buyout of Synthorx in late 2019 is now the subject of a $1.6 billion writedown.
Sanofi disclosed in its Q3 earnings that it’s closing down Phase II platform trials for SAR444245, an IL-2 candidate that it had hoped would serve as a next-gen foundation of the oncology franchise. An early look at the data, it said, suggests efficacy that’s “lower than projected.”
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Alnylam Pharmaceuticals, the pharma market’s leader in RNAi therapies, reported its third quarter earnings Thursday, updating shareholders on its pipeline progress and initial sales figures for the recently approved vutrisiran (marketed as Amvuttra).
But tucked away near the bottom of its report, the company revealed it would scrap plans to launch a Phase III trial for vutrisiran in the rare Stargardt disease, appearing to blame President Joe Biden’s Inflation Reduction Act in the process.
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Following the US’ lead, the EMA is cutting back on the use of JAK inhibitors for some patients over a suite of safety concerns.
The EMA’s safety committee issued new guidance on Friday, recommending that a group of JAK inhibitors used for chronic inflammatory disorders are only used in certain at-risk patients if no alternatives are available.
That includes patients 65 years and older, those at risk of major heart problems, those who smoke or who have extensively in the past and those at risk of cancer. Patients at risk of blood clots in the lungs and deep veins (VTE) should take caution, the committee says, while recommending lower doses for some patients at risk of VTE, cancer or major heart problems.
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Ahead of a key meeting of the FDA’s Oncologic Drugs Advisory Committee tomorrow, the FDA is raising serious concerns regarding the efficacy of Y-mAbs Therapeutics’ potential pediatric neuroblastoma treatment.
Part of the problem for FDA is that the entire application for 131I-omburtamab is based on a small, single-arm trial from New York’s Memorial Sloan Kettering Cancer Center that found the three-year overall survival rate after CNS/LM relapse in the efficacy population of 94 patients was 54%.
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Gilead was following German biotech Myr and its hepatitis delta virus candidate for “quite some time” before finally pulling the trigger on a $1.7 billion buyout deal. With an EU authorization already under its belt, an OK in the US was all but a done deal — or so it thought.
The company reported an FDA rejection during its Q3 earnings call on Thursday, leaving CMO Merdad Parsey “disappointed” and spoiling the $360 million milestone that Myr would have reaped on an FDA approval. So what happened?
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RNA editing biotechs pitch impermanence. Instead of making lasting changes to the DNA, RNA editors rewrite the intermediate message before it is translated into proteins. And many RNA editing biotechs, such as Shape Therapeutics, Wave Life Sciences, Korro Bio, and appropriately named ADARx Pharmaceuticals, take advantage of machinery already found in the body — ADAR editing.
ADAR editing converts the RNA code from A to I, which is interpreted as a G. While found in humans, it’s a method used much more actively by octopi and squid to drastically change the code of their nervous system proteins.
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