Sarepta Versus The FDA, And Other News: The Good, Bad And Ugly Of Biopharma – Seeking Alpha

Sarepta Therapeutics (SRPT) fell victim to the FDA's whims as the stock tumbled more than 3 percent following the issues raised by the FDA against its drug candidate Vyondys 53 (golodirsen). The drug was earlier rejected by the FDA last year. However, given what we can only call "popular protests," later the agency reversed its own decision in December and gave it a green signal. Now that the drug has been approved, the FDA decided to make its initial Complete Response Letter public, sending investors into a tizzy about its motives.

The latest move by the FDA is concerned with the impact of the drug on a wide range of physical activities. The document released by the FDA talks about low levels of effectiveness, kidney toxicity and infections as some of the reasons behind the original rejection handed by the FDA. While some of these reasons were mentioned when the drug was rejected, new documents shed more light on those issues.

Though it is commendable that the FDA has provided more details for explaining its course of action, the FDAs wobbly posture on the future of the drug is a worrying cause for investors. It also shows that despite granting approval to the drug, the agency still is skeptical about the performance of the treatment. If so, why not maintain its stand and not grant approval in the first place?

The agencys reluctance in making things clear is likely to cause damage to the company and its stock performance. The FDA could have chosen to make all the facts clear at the time of rejection, allowing the company to properly address those issues as happened in the issue related to kidney toxicity. The FDA concern regarding the impact of the drug on the kidney was properly tackled by Sarepta, as the company recommended monthly monitoring of the patients.

The now-public CRL is much more scathing in its tone than was earlier anticipated. Apart from raising issues regarding the efficacy and safety profile of the drug, the CRL is also critical of the companys then failure to start a mandatory confirmatory clinical trial for a previously approved exon 51 skipping DMD drug, Exondys 51 or eteplirsen.

The December approval for the drug was mainly granted on the basis of surrogate endpoint data from a Phase II study, and the positive decision had taken many by surprise. It would have been more of a prudent move by the FDA to clear all the concerns regarding the drug before granting it approval than raising issues after granting the green signal.

The company has tried to assuage the market concerns by claiming that the concerns mentioned in the CRL are not unique to Sarepta. However, the FDA actions are likely to have a somewhat negative impact on the drug, which may otherwise help the company in leading the Duchenne treatments market. After Exondys 51, Vyondys 53 is the companys second treatment for addressing Duchenne muscular dystrophy. The ailment affects the production of a protein called dystrophin that is required for ensuring muscle health.

Sarepta stock had a tumultuous past year, where it offered a slightly negative 12-month return. The stock showed strong movements, making it highly volatile.

Evoke Pharma (EVOK) stock jumped up as the company reported its new collaboration with Eversana. The pact involves the commercialization and distribution of Gimoti nasal spray in the United States. This new agreement comes after EVOK terminated its similar deals with Novos Growth Partners. The now defunct agreement was signed a year back.

Eversana is a life science commercial services provider and will be entitled to receive certain reimbursements from EVOK pertaining to the costs. It will also receive a percentage of profit once Gimoti revenue surpasses a certain level. In return, Eversana will provide a revolving credit facility of up to $5 million to EVOK.

Gimoti is designed to treat symptoms of acute and recurrent diabetic gastroparesis in adult women. Its target goal date under the Prescription Drug User Fee Act (PDUFA) has been set for June 19, 2020. The new deal requires to be ratified by the FDA. Eversana has various resources, including experienced personnel, to help EVOK in marketing and commercializing its drug candidate. The companies plan to launch Gimoti in the market within this year, subject to FDA approval.

The terms of the agreement will remain applicable for five years post FDA approval. After the expiry of this lockdown period, EVOK will regain the entire product sales and related commercialization tasks. The drug candidate enjoys patent protection in the United States till 2030. It has the potential to become the only non-oral and out-patient treatment for female patients suffering from diabetic gastroparesis in the United States.

Applied Genetic Technologies Corp. (AGTC) reported encouraging six month data from its Phase studies of its AAV gene therapy in patients suffering from color blindness caused by mutations in the ACHM CNGA3 genes or ACHM GNGB3. The trial was carried out on a cohort of 22 patients. The market reacted negatively to the news and the stock tumbled more than 30 percent.

Applied Genetic did not provide any specific results. The company communiqu only stated that the trial observed encouraging signs of biologic activity. It also stated that such activity came with a favorable safety profile. Applied Genetic plans to release data from additional dose group time points and age groups during the second half of this year. The company is currently in the process of dosing three high dose groups in pediatric patients and in two groups of adult patients. The results from these trials will be used for measuring the readiness to move to pivotal trials.

Achromatopsia is an inherited retinal disease and is distinguished by the lack of cone photoreceptor function. The ailment causes extreme light sensitivity leading to day blindness, complete loss of color discrimination and reduced visual acuity.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

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.

Read the original here:
Sarepta Versus The FDA, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha