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Clovis Oncology CLVS Stock News

Clovis Oncology (NASDAQ:CLVS)

On Monday, Clovis Oncology stock closed higher by 9% after it announced that the FDA had approved its drug. The drug in question is known as rucaparib – to be marketed as Rubraca. This was for early approval of rucaparib, which is used to treat patients with advanced ovarian cancer. The stock had a little trouble maintaining all of its gains throughout the day. That’s because it gave back a lot of of what it had gained initially. The stock was initially trading higher by as much as 22%, but gave most of the gains back to only close higher by 9%.

CLVS Big Surprise

The FDA approval of rucaparib came in a lot sooner than what was expected. That’s because the FDA granted approval using a program known as Accelerated Approval. This is where a drug can garner earlier approval if it treats a serious or life-threatening disease. This is based on clinical data that meets typical approval standards and helps those with no other treatment options. First off, the FDA establishes what is known as a surrogate endpoint that is used to determine clinical benefit. In other words, such an endpoint would be used to determine if the drug is efficacious enough to warrant early approval. The FDA wasn’t supposed to decide on approval for rucaparib until February. That’s why this early approval came as such a huge surprise.

Clinical Trial

The phase 3 trial showed some substantial efficacy, which is why the drug has been approved early. In addition, the drug targets a patient population with a huge unmet medical need. There were two single-arm trials done to prove efficacy of rucaparib. The two trials recruited up to a total of 160 patients with advanced ovarian cancer. The ovarian cancer patients recruited into the trial had to meet another requirement. That requirement is that they had to have the BRCA mutation in order to enroll into the clinical trial. That wasn’t the only requirement though, because patients also had to have been treated with two prior chemotherapy treatments. The final result of the trial was that 54% of patients experienced complete or partial shrinking of their tumor over a median of 9.2 months.

Looking Forward

This FDA accelerated approval for rucaparib is good because now CLVS can go after additional indications. Other indications being targeted include prostate cancer, breast cancer, and gastroesophageal cancer. This early approval for ovarian cancer bodes well for the company and its stock. The stock has been beaten down pretty badly in the past for pipeline setbacks, but now it seems like things are finally starting to shape up for it. Ovarian cancer is a large unmet medical need because not many treatment options exist for it. At least 22,000 women are diagnosed with ovarian cancer each year. Out of all the patient population, at least 15% to 20% have the BRCA mutation needed to take the drug rucaparib. The fact that its a rare form of cancer means that the whole market of the BRCA  mutation will belong to CLVS. That means that the future is very bright indeed for the company.

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Ophthotech Corporation OPHT Stock News

Ophthotech Corporation (NASDAQ:OPHT)

On Monday, Ophthotech stock tumbled lower by 86% after it announced that its phase 3 trial in patients with wet age-related macular degeneration (Wet AMD) failed to meet the primary endpoint of the study. The stock was slaughtered immediately, causing a lot of panic selling, but for good reason. The key thing to keep in mind is that there were two different phase 3 trials treating these patients with Wet AMD. The results were quite disappointing, as they did not show statistical significance whatsoever. Investors were really looking forward to good results, because a prior phase 2 trial showed that the drug did achieve statistical significance.

OPHT Phase 3 Results

The two phase 3 trials were known as OPH1002 and OPH1003 respectively. Neither one of them achieved statistical significance, and that’s why the stock was crushed on Monday. Both trials were testing the company’s drug, Fovista, in combination with Novartis’ Lucentis. This combination was being tested against Lucentis alone. The reason for that was to determine if adding Fovista to Lucentis would boost efficacy. Unfortunately, this was not the case. Adding Fovista to Lucentis was no better than Lucentis alone. How, exactly, were the results calculated to determine if Fovista together with Lucentis was better than Lucentis alone? It was done by patients having to undergo a standardized eye chart. This eye chart would be where patients could determine how many letters they were able to read up to with the vision they had after receiving treatment. The patients in the Fovista/Lucentis combination therapy achieved a mean gain of 10.24 letters. That is really good, however, compared to Lucentis alone it’s not a lot. Patients that took Lucentis alone achieved a mean gain of 10.01 letters. Comparing the two numbers, it is obvious that it is not statistically significant. Thus, the main reason why the stock crashed down to $5.29 per share at the bell.

One Failure Too Many

With this failure in the Wet AMD arena, that is one failure too many. That is because now OPHT only has one other drug left in the pipeline. That drug candidate is known as Zimura, and it’s being developed for patients with dry age-related macular degeneration (dry AMD). This is still a large patient population of at least 8 million patients, but losing the ability to get Fovista to market is a big missed opportunity. The hope is that the last drug in the pipeline can make it to the finish line. There is, however, one problem. That problem is that both Fovista and Zimura are based on the aptamer technology. The only hope is that the drug may work in the dry AMD population.

Moving Forward

The way to trade the OPHT stock now is to wait a few days for a possible bounce. It might be a good stab once the stock has found a bottom. If one is attempting to try a risky stock such as this, that would be the best time to buy. The Zimura indication might actually work out, but the downside is that it will take years for that to happen. A phase 2/3 trial could take years to run, and even then, there is no guarantee that the drug will be successful in the Dry AMD patient population. Ophthotech’s failure was a huge gain for Regeneron Pharmaceuticals, which already has a large foothold in the Wet AMD market. The Regeneron stock was higher in morning trading on the failed OPHT news, and eventually ended higher by 3.83 for the day. It seems that Regeneron will keep its place as king in the Wet AMD market, and unfortunately, OPHT will continue to feel the pain of this trial failure.

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MEI Pharma MEIP Stock News


On Monday, MEI pharma released results of its phase 2 clinical trial in elderly patients with Acute Myeloid Leukemia – AML. These results were presented at a prestigious medical conference known as the American Society of Hematology or ASH. The results themselves were outstanding, but the stock didn’t budge at all. Matter of fact, the MEIP stock only closed higher by 1.27%. The problem is that the data was sufficient enough to carry the stock higher by at least 20% or more. Typically, when a biotech in phase 2 releases positive clinical results, the stock should rise by at least 20% or more. On the other hand a trial that fails can fall by as much as 50% or more, on average. There is no denying that the stock should have traded higher, but this can be attributed to a few reasons. One reason being that the company will now have to run a large phase 3 trial in the same elderly population. That means the company will need to eventually raise additional capital. In addition, the success rate for AML drugs in phase 3 clinical development is really low. These two reasons are probably why the stock traded close to flat on Monday. It is  highly possible that the stock could trade higher in the coming weeks if big positions were to pour in.

Phase 2 Results

This phase 2 study used the drug Pracinostat to treat these elderly patients with AML. The study was open-label in nature and recruited a total of 50 patients. The open-label nature of the trial let researchers and management know whether the patients received the drug or not. Patients were given 60mg of Pracinostat orally three times a week for a total of three weeks. Then patients were given a chance to rest away from treatment, and then followed up with a subcutaneous injection or intravenous infusion of azacitidine for the first 7 days of each 28-day cycle. The treatment was safe but there were some grade 3 to grade 4 adverse events. The thing to note is that the adverse events weren’t anything in which the patient could die from. The study results were nothing short of amazing. The median overall survival was 19.1 months, which is much better than azacitidine alone. The one year survival rate was 62% and the complete response rate was 42%. One of the investigators of the trial, Dr. Garcia-Manero, had this to say:

“The results from this study of Pracinostat and azacitidine in elderly patients deemded unfit for intensive therapy are particularly encouraging. Despite recent advances in the treatment of AML, options for these elderly unfit patients remains unlimited. The combination of Pracinostat and azacitidine appears to show a long-term survival benefit in this population, including an unprecedented two-year survival rate of 41% in this study”

These results are highly notable, especially in a population that can’t take many other forms of treatment because of how frail their bodies are. Remember, these are patients that are at a median age of 75. Having to take chemotherapy or other invasive forms of treatments is not ideal at all.

Looking Forward

The phase 2 results are highly encouraging, so much so that MEIP is already recruiting for a phase 3 trial of newly diagnosed elderly patients that are 75 and older. The stock didn’t react appropriately as expected, but the truth is that the long-term story remains intact. Over time new investors will find out the true value of the company, especially once the phase 3 trial enrollment nears completion. For now, investors will have to continue to believe that the data observed to date warrants continuing to hold their investment. Sometimes biotechnology stocks don’t move up until a few days or a few weeks after results. This is because some results may take time to interpret the data. MEIP should be in good standing considering that it has a good pipeline of cancer drugs. In addition, to date, the company has shown that its drug continues to work in this hard-to-treat patient population.

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Nivalis Therapeutics NVLS Stock News

Nivalis Therapeutics (NASDAQ:NVLS)

On Monday Nivalis Therapeutics stock fell by more than 52% after the company announced that it had failed its phase 2 trial in patients with Cystic Fibrosis — CF. As soon as the results were released the stock tumbled as investors sold their shares to the open market. The phase 2 trial used the company’s drug known as cavosonstat to treat these patients with CF that had the F508del-CFTR mutation. The trial tested the safety and efficacy of two doses of cavosonstat of 200 mg and 400 mg. One thing to note is that these patients that were given the NVLS drug had already been taking treatment with Orkambi.

Trial Bust

Patients were already on Orkambi, because that is what is being used as the current therapy for these patients. Orkambi was approved by the FDA back in July of 2015. It was developed by a pharmaceutical company by the name of Vertex Pharmaceuticals. The hope was that the NVLS drug cavosonstat would be able to achieve the primary endpoint of the study in order to improve clinical outcome. The problem is that the trial failed on the primary endpoint of the study. The primary endpoint was to determine if the drug could change the FEV1 measurement from baseline compared to the placebo compound. Not even the secondary endpoint of reducing sweath chloride at week 12 was successful. Unfortunately, the trial didn’t achieve any endpoint. The CEO of the company Jon Congleton had this to say about the results:

“While we are disappointed in the outcome of this trial, we plan to continue to investigate the therapeutic potential of cavosonstat and our S-nitrosoglutathione reductase (GSNOR) inhibitor portfolio to  determine  next steps”

Competition Wins

There is no denying that the phase 2 trial was being watched closely by NVLS management and investors. There was, however, another key player that was really watching to see how the trial went. That key player being Vertex Pharmaceuticals, which is the one that produced Orkambi as standard of care treatment for CF patients. The reason why Vertex was looking at this trial was to see if cavosonstat would steal its thunder. In other words, if cavosonstat was positive and made it to market it could compete toe to toe with Orkambi. That’s not the case anymore and now Vertex Pharmaceuticals can breathe a sigh of relief. This means that Vertex still maintains its position as the leader in the Cystic Fibrosis space. Although, this means that NVLS will not be a direct competitor. If NVLS can’t find a way to come back from this, then the company doesn’t have that much of a bright future in its path.

Looking Forward

It’s tragic that the trial did not do much compared to placebo in helping these patients with CF. The truth is that the drug cavosonstat targeting CF was the lead candidate of the pipeline. This is a bad blow for the company which was heavily relying on a positive clinical outcome in this phase 2 study. The NVLS stock will continue to suffer because of this setback. It is not clear whether or not the company will continue to develop the cavosonstat drug in the CF indication. The company says it wants to evaluate the next steps for this program but the truth is that it doesn’t have that much of a future left after missing the primary endpoint. The good news is that there is another drug candidate in the pipeline that is being developed to treat asthma. The bad news is that it is still in earlier stage clinical trials. It will take many years before it makes to a phase 2 trial. That means investors will be cautious of investing in this stock as it doesn’t have much of a pipeline left.

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Arrowhead Pharmaceuticals ARWR Stock News

Arrowhead Pharmaceuticals (NASDAQ:ARWR)

On Monday, Arrowhead Pharmaceuticals stock closed down by 1.58%, even though it announced that it had completely finished the deal with Amgen. The deal between Arrowhead and Amgen had finally closed today. Investors weren’t too enthusiastic, as the stock closed lower instead of heading higher. This news is definitely good news, and the stock should have reacted appropriately to this type of news. Typically, when a biotechnology company posts a final deal with a big pharma company, its stock soars higher. In this case it was totally different, but there is a good reason why. Not too long ago, Arrowhead received a clinical hold from the FDA for its Heparc-2004 study dealing with Hepatitis B. This caused a sudden drop in the share price, so the news posted today, while good, was not enough to overcome the clinical hold for the trial.

ARWR Deal With Amgen

The deal between ARWR and Amgen was established back at the end of September of this year. The deal established was that Arrowhead, pending certain milestones over time, could obtain up to $674 million in cash.  The way the deal worked out was that Amgen received an upfront payment of $55.6 million, and the potential to earn the other $617 million. The reason for Amgen doing this deal was to obtain the early pre-clincial work in the cardiovascular space. ARWR had a pre-clinical program in this space, and that was something which Amgen was greatly interested in. The upfront payment that ARWR got is huge. It can use this cash to develop other drug candidates in the pipeline. This does two different things. For one, it advances other drugs into the clinic to increase the stock price and shareholder value. Secondly, it allows additional partnership opportunities to unfold. This will greatly continue to increase the value of the company itself. The initial drug candidate is known as ARC-LPA and showed a 98% knockdown in mice with a single injection. Amgen must have been impressed enough to make the decision to create this partnership.

FDA Clinical Hold

As noted before, the FDA had placed a clinical hold on Arrowhead’s hepatitis B trial program. This action was completed a few weeks ago on November 9, and the stock has suffered because of it since. The clinical hold is on Heparc-2004 which is treating 12 patients with the Hepatitis B virus. The trial has been placed on hold and is not recruiting patients for the moment. The reason for the hold was a problem observed in the nonclinical toxicology study being done with non-human primates. More specifically, the FDA wants answers to questions surrounding the company’s EX1 delivery technology. That technology is being used to deliver targets to the liver intravenously (through a vein).

Looking Forward

The good news about the clinical hold is that it deals with a higher dose in a non-human clinical study. That means that ARWR should easily answer the FDA’s questions to get the clinical hold lifted quickly. In the meantime the stock will continue to suffer for it, so investors should be cautious in the short term. Long term, though, this clinical hold should eventually be dealt with, and that will be a huge positive. The deal with Amgen is what should be driving the company forward, because it received a lot of cash upfront. As noted before it can do many different things with this cash, therefore it should help create shareholder value over time. The stock currently boasts a $300M market cap, which is pretty good considering the pipeline it has, although it doesn’t account for the potential $617 million extra in cash it could obtain. The thing is that as investors realize the true value of ARWR, the stock should start to climb again.

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Dynavax Technologies DVAX Stock News

Dynavax Technologies (NASDAQ:DVAX)

On Monday, Dynavax Technologies stock closed the day lower by 64.55% after the company announced that the FDA has rejected Hepislav-B, its lead vaccine product. This product is being used to treat patients with the Hepatitis B virus. Not many treatment options are available to those with the Hepatitis B virus, therefore a vaccine of this caliber is more severely wanted. The problem is that DVAX has had many problems in the past in bringing this vaccine to market. This has led many investors to doubt that the company is even capable of bringing this product to market on its own. This is evidenced by the fact that this is the second time in three years that the FDA has flat out rejected the vaccine with a Complete Response Letter — CRL.

Rejection Continues

As soon as DVAX announced the rejection by the FDA, the stock traded lower for the day by as much as 72%, falling to $3.20 per share. This is the lowest trading level the stock has seen since 2008. The good news, if it can be construed as good news, is that the stock was able to close lower by 65.55% instead. That’s still a steep drop, but for good reason. DVAX had the same issue back in 2013 when the FDA cited that Hepislav-B couldn’t be approved in its current form because of safety issues. The same items have been indicated in this CRL – such issues as clarification with adverse events observed in clinical trials, cardiac events, and other safety issues. DVAX CEO Eddie Gray has given investors a positive outlook citing that approval of Hepislav-B is still doable. Unfortunately, that doesn’t bring a whole lot of confidence to those investors who have been waiting for the approval of this vaccine for many years now. The problem is that the drug has proven to be effective, but the safety of patients taking the vaccine has always been a concern for the FDA. Yet again, safety played an important role in the FDA deciding to reject the vaccine again.

Safety Issues

The FDA has cited that, while Hepislav-B is very efficacious, it can’t approve the vaccine without addressing a lot of the safety issues that it has. Adverse events are something that DVAX must address before going back to the FDA to seek approval for its product. The CEO claims that the safety issues can easily be addressed and that the vaccine can easily approved by the FDA pending certain clarification issues. There is just one major hurdle in the way, and that is that the company is not at all confident in bringing this product to market. The CEO stated in the press release that it will have to meet with the FDA to figure out how to better address the safety issues. In addition, the CEO stated that it will not be able to go the FDA route alone. This means that it will have to now bring a financial or pharmaceutical partner on board to help carry the Hepislav-B vaccine to the finish line. The problem with this is that there can be no assurance that such a partner will be able to achieve this goal.

Looking Forward

Things were not shaping up from September for DVAX anyway, because that is when the FDA cancelled a schedule advisory panel meeting. This was where the advisory committee was to review Hepislav-B for both efficacy and safety. Unfortunately, that never happened, since the FDA has completely cancelled the meeting. The ironic thing is that most believed that the cancelling of the meeting was highly bullish. In other words, the FDA would approve the vaccine. As can be seen, that’s not how things played out, and now DVAX has about $109.9 million in cash left as of the end of the third-quarter. That gives DVAX enough cash for at least three more quarters, but, obviously, it will have to seek other options before then. It will have to either partner with a pharmaceutical company or find a new form of financing to continue operations. Considering that DVAX has yet to find a partner after so many years, this doesn’t bring a lot of confidence to investors. All is now dependent upon management’s actions over the course of this year. If there are positive signs that the company can turn things around, it will be great for investors; if not, then DVAX will fall into an bottomless pit with no end in sight.

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ACADIA Pharmaceuticals ACAD Stock News

Acadia Pharmaceuticals (NASDAQ:ACAD)

On Monday, Acadia Pharmaceuticals announced that it had initiated a phase 2 trial in patients with Alzheimer’s Disease Agitation. Once again ACAD will use its drug Pimavanserin to see if the drug will work in this targeted indication. The company has already received FDA approval for Pimavanserin in Parkinson’s Disease Psychosis. Now the drug is being tested in another indication and it is difficult to gauge whether or not it will be successful in that disease. Still, considering that ACAD already has FDA approval for Parkinson’s Disease Psychosis, any kind of failure in this new indication won’t really hinder the stock in any severe way.

Phase 2 Trial Design

The most important aspect of a biotechnology company is for the technology or drug to be superior to the current standard of care treatments. Despite that, there are other things that management must do in order to ensure that a trial is successful. In this case, ACAD announced the phase 2 trial initiation for patients with Alzheimer’s Disease Agitation. This phase 2 trial will be known as “SERENE”. It will recruit a total of 430 patients who have Alzheimer’s Agitation or aggression. Patients in the trial will be split up into three different groups. One group of patients will take one daily dose of 34 mg pimavanserin, one group will take 20 mg pimavanserin, and the final group will take the placebo compound instead. One thing to keep in mind is that each dose will be given once a day over a 12-week period. That sounds like a short study, and while treatment time will be short it will take a long time to recruit that many patients into the trial. That means that it could take between 1 to 2 years before results from this trial are announced. That means that it won’t be a catalyst for quite some time with respect to this study. The primary endpoint of the trial will be looking for a reduction of an Alzheimer’s agitation score. This score will be measured using a system called “Cohen-Mansfield Agitation Inventory”.

Severe Need

This indication that ACAD is targeting next is very important, because like Parkinson’s Disease Psychosis, there are no other treatment options. That means that the company has the opportunity to tap into another market that has no current therapies for. Of course it will also be good for patients because nothing exists to help them with respect to their disease. The Executive Vice President of ACAD, Serge Stankovic had this to say:

“AD Agitation is a common condition and a major cause of distress for Alzheimer’s patients, their families and caregivers”

As noted before, there is no current therapy to treat this disease. As mentioned above Alzheimer’s Disease Agitation not only affects the patient, but also has a devastating affect on families and caregivers as well. If Pimavanserin can be successful in this indication it would bring a lot of relief for many families out there.

Moving Forward

ACAD stock is almost appropriately priced at $23.31 per share. That means that it has a market cap of $2.79 billion, and considering that it has already received approval for Pimavanserin in Parkinson’s Disease Psychosis it makes plenty of sense. As mentioned before the phase 2 trial won’t complete until a few years from now so any catalyst from the new indication won’t happen for quite some time. The good news is that there might be a short-term bump in the stock as investors cheer the new indication targeting a large market. It is still highly bullish that the trial has started and a good reason for the stock to trade up because of this. Also it doesn’t hurt that Q3 earnings are on the way and any positive indication for Nuplazid — previously Pimavanserin — sales going up will be highly bullish for the stock.

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Inovio Pharmaceuticals INO Stock News

Inovio Pharmaceuticals (NASDAQ:INO)

On Monday, Inovio Pharmaceuticals closed the day down by 17% after the company announced that the FDA had placed its trial initiation on hold. This refers to the phase 3 clinical trial that was supposed to begin shortly treating women with cervical dysplasia or CIN 2/3 lesions. The vaccine to be used in the clinical trial is known as VGX-3100. A clinical hold is a trial that is either halted while ongoing or delayed if the trial has not yet begun.

Major Hold

In the case of Inovio, the phase 3 trial had not yet started. It was supposed to start soon, but now it looks like the phase 3 trial will be delayed much longer until Inovio can get the FDA to lift the clinical hold. The good news to come out of this is that no other trial is affected, only the phase 3 trial that was supposed to start. Many investors were disappointed by this news, as evidenced by the stock falling 17% for the day.

The company may have received notice beforehand, but it didn’t get every exact detail necessary to take the necessary course of action right away. In other words, Inovio is awaiting a letter from the FDA which is set to come within the next 30 days. In the initial communication between Inovio and the FDA, the FDA has requested additional information on its CELLECTRA 5PSP immunotherapy delivery device.

Months Of Delay

Investors of Inovio were already anxious for the phase 3 trial to get started, but now it looks like they will have to wait a lot longer. Why is this the case? The reason is because Inovio stated in its press release that data to satisfy the FDA won’t be ready until the end of this year. This means that the data wont’ be ready until probably late December, and that is if the company remains on track. If Inovio needs more time, or if the FDA has additional questions from comments then it is possible that the delay could be stretched out even longer.

If that were to happen then the stock could fall into a further decline. It is never a good thing when there is a clinical hold on a trial, and biotechnology companies’ stock tends to tumble as a result. That seems to be the case so long as the issue remains unresolved. That means Inovio will have to work quickly with the FDA to rectify the situation in a timely manner. There is, however, one major problem. Even if everything was to go smoothly, Inovio anticipates that the phase 3 trial would start in the first quarter of 2017.  Even then that doesn’t mean that it will be in the beginning of 2017. It could be pulled back as much as by the end of the first quarter of 2017. Again, that is if there is no other delays in the dialogue between Inovio and the FDA.

Moving Forward

The good news is that nothing else in the pipeline was affected, therefore all the other clinical trials are running as normal. In addition, the clinical hold had nothing to do with the vaccine itself. The company’s vaccine is safe, and the hold wasn’t even because of the safety of the CELLECTRA device used to inject the vaccine. The clinical hold was because the FDA was worried about the shelf life of the delivery device product. Something so trivial had caused the FDA to put a hold on the trial. The truth is that this matter should be resolved fairly easily, the bad news is that it will delay the phase 3 trial initiation by 5 to 6 months. As long as Inovio is able to resolve this issue with the FDA then the long-term future of the company should remain on track.

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PTC Therapeutics PTCT Stock News

PTC Therapeutics (NASDAQ:PTCT)

On Monday, PTC Therapeutics fell by 40% to $7.97 per share after the company had announced that the FDA refused its appeal for a refuse-to-file letter. PTC Therapeutics has not been able to get the FDA to back its Duchenne Muscular Dystrophy — DMD — drug. What happened was that PTC had filed an appeal to the agency to reconsider its refuse-to-file letter date back in February of this year. This further signifies that the FDA is not interested in approving PTC’s drug Translarna. Another deadly blow to PTC Therapeutics, which has not been having the best year at all. News of this appeal denial by the FDA led the PTC stock lower by 40%.

Major Problem

A major problem or hurdle for PTC is that, while its commitment to get its drug approved to treat DMD patients is sound, it is not highly justified at all. The reason being is that it had run two phase 3 clinical trials that had both failed. Thinking about this more clearly, it doesn’t make too much sense on why PTC is still pursuing this indication at all. Both phase 3 trial were completed and both had failed the primary endpoints of the studies. PTC has a claim of its own on why it is still considering to pursue the DMD indication. It has noted that despite failing both phase 3 trials, there might be a subset of patients that would greatly benefit from treatment with Translarna.

Not Backing Down

PTC had sent its appeal to the FDA and had the request denied. Instead of taking this as a hint that maybe the efficacy of its drug is not as strong as intended, the company is pushing forward. It has stated that it will now send the appeal to a higher level in the agency. The problem is that sending an appeal to the next higher level of the FDA may not work. In that case, PTC may have to end up submitting multiple appeals to each higher level. Even if it does this there can be no guarantee that it will have a positive outcome. After all, if the lower branch of the FDA doesn’t feel that the drug provides enough efficacy, then why would the higher level of the FDA feel differently? It might be a good idea for PTC to move on towards other drugs in the pipeline.

Competitor Appearance

One of the many reasons that PTC might be pushing forward is that it is pointing towards Sarepta’s case. Sarepta Therapeutics just received approval for its DMD drug Eteplirsen about a month ago. While PTC does have a point that Sarepta didn’t back enough clinical efficacy to warrant approval, it doesn’t have the backing by the FDA at all. At least when Sarepta submitted its application for its NDA, the FDA took at a look into it. Even then, it was awfully close as the FDA raised concerns over Eteplirsen as well. Sarepta may have been lucky or may have had the proper papework. In any case, it at least got to have an advisory committee review and FDA review of its drug. This is where PTC lacks because it has to file a massive amount of appeals in hopes that it can get the FDA to give it a chance for possible approval.

Moving Forward

It doesn’t seem like things are going well for PTC Therapeutics on the U.S. front with the FDA. That means the stock will continue to fall, especially after this most recent denial for appeal. The good news is that the company has an application submitted in Europe which may prove to be helpful. Europe is more so trying to work with PCT about getting Translarna approved. The company has submitted additional supplemental information to the European Agency, and a decision for this country will come down sometime before the end of the year. If PTC receives a positive recommendation from Europe, then there is a possibility that the stock might be able to recover.

Tesaro TSRO Stock News


On Monday, Tesaro Stock closed higher by 18.79% after the company had announced over the weekend that its drug was successful in a clinical trial in patients with ovarian cancer. The results were outstanding, and a big reason why the stock closed higher for the day. What makes the results even more impressive is the fact that they were presented at a European Medical Conference. Original data was released earlier in the year, in June, but detailed results of the trial was released over the weekend. Even though a majority of these results were known, it didn’t stop the stock from big gains for the day.

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TSRO Trial Results

The phase 3 clinical trial was known as ENGOT-OV16/NOVA, and it recruited a total of 533 recurrent Ovarian Cancer patients. When it is said that patients have recurrent Ovarian Cancer, that means that no matter how many treatments they take, the cancer comes back after being eradicated from the body. Tesaro’s drug, niraparib, was combined with platinum-based chemotherapy. That combination was tested against the platinum-based chemotherapy alone.

The primary endpoint of the clinical trial was to assess the progression-free survival — PFS — of those patients taking niraparib compared to the chemotherapy. The trial was set up with two cohorts because TSRO was testing two sets of Ovarian Cancer patients. That means one set had the BRCA mutation version of the cancer, and the other set was the non-BRCA mutation cancer version. The trial was successful in both populations, which makes the results even that much more significant.

In the Ovarian cancer patients with the BRCA mutation, the median PFS for niraparib was 21 months compared to 5.5 months in the placebo side. Obviously the drug was clearly built for the BRCA Ovarian cancer patients. While the non-BRCA mutation didn’t succeed with as large as a gap in PFS like the BRCA group it was still successful. The non-BRCA group patients taking niraparbib achieved a median PFS of 9.3 months compared to placebo obtaining 3.9 months. Having both trials successful in Ovarian cancer is no easy feat at all.

Ovarian Cancer

There are around 200,000 cases of Ovarian Cancer per year in the United States. It is a difficult type of cancer to treat because there aren’t many treatment options other than platinum-based chemotherapy. It is also a type of cancer that is hard to detect because symptoms don’t become known until the cancer has spread to the pelvis and belly of the patient. The problem with that is that once it has spread to other parts of the body, it becomes a lot more difficult to treat. Surgery and chemotherapy are both current treatments but carry heavy risks. Especially chemotherapy which carried a lot of side effects. TSRO drug niraparib was safe for the most, but had some grade 3 and 4 adverse events reported in the trial. The investigator has noted that most of these were resolved by adjusting the dosage level of the drug.

Moving Forward

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Tesaro has a lot going for its drug. With the results helping the stock post some gains, the truth is that there is another catalyst yet to come. That is TSRO stating that it will be able to complete its rolling FDA submission of its NDA for niraparib by the 4th quarter of this year. Considering that the drug was shown to be highly superior to current treatment options along with the fact that there weren’t very many safety issues, then it should be able to easily obtain FDA approval. The market opportunity for targeting Ovarian Cancer could be up to $1.4 billion. This drug has shown that it can help these patients with recurrent ovarian cancer over standard of care, and that will be key for its marketing success.

[Image Courtesy of Wikipedia]

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