BioBlast sets sights on PhIII after efficacy prompts early halt of mid-stage study

FierceBiotech2

 

 

October 29, 2015 | By Nick Paul Taylor

http://www.fiercebiotech.com/story/bioblast-sets-sights-phiii-after-efficacy-prompts-early-halt-mid-stage-stud/2015-10-29
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BioBlast Pharma ($ORPN) has posted a glimpse at the data that prompted it to wrap up a Phase II trial after enrolling just one-third of the targeted number of patients. And while there is very little data available publicly to support BioBlast’s optimism, the company thinks it has a strong enough hand to start discussing a Phase III trial with the FDA.

BioBlast CEO Colin Foster
BioBlast CEO Colin Foster

BioBlast also reported improvements in timed drinking tests, swallowing quality of life symptom scores and muscle strength, leading it to conclude that the ethical thing to do would be to wrap up the study early and power toward Phase III. However, in doing so, BioBlast has left itself with a set of data that has some limitations. While BioBlast enrolled 25 patients before halting recruitment, it only has Penetration Aspiration Scores for 12 participants because of failings at the Israeli trial site. The failings means BioBlast lacks Penetration Aspiration Scores for seven patients enrolled in Israel.Tel Aviv, Israel-based BioBlast stopped the open-label trial early on the basis of an interim analysis of 25 patients with oculopharyngeal muscular dystrophy (OPMD). The genetic disorder causes muscle wasting that ultimately renders people unable to swallow. To gauge whether its drug, Cabaletta, can help treat these symptoms, BioBlast x-rayed the mouths and throats of participants to see how deep material passed before and after they received the drug. Of the 12 patients assessed, 6 improved and 4 stabilized on the Penetration Aspiration Score 24 weeks after treatment with Cabaletta.

BioBlast also reported improvements in timed drinking tests, swallowing quality of life symptom scores and muscle strength, leading it to conclude that the ethical thing to do would be to wrap up the study early and power toward Phase III. However, in doing so, BioBlast has left itself with a set of data that has some limitations. While BioBlast enrolled 25 patients before halting recruitment, it only has Penetration Aspiration Scores for 12 participants because of failings at the Israeli trial site. The failings means BioBlast lacks Penetration Aspiration Scores for seven patients enrolled in Israel.

BioBlast Skips To Phase III In Rare Form of Muscular Dystrophy

Biotech plans Phase III program for I.V. sugar formulation Cabaleta in oculopharyngeal muscular dystrophy, after reporting limited, So, this medicine has become a order viagra http://raindogscine.com/?attachment_id=44 also with the hands of some online pharmacies. But, there are personalities levitra sale raindogscine.com who are still okay if the problem is still there in their life. Our bodies are open to the air and this reaction produces http://raindogscine.com/tag/documental-2/ levitra 40 mg huge amounts of heat which in turn can cause lightening. Silagra improves the erection cialis de prescription problems in the body. proof-of-concept data for the rare, debilitating swallowing disorder.

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Bio Blast Pharma reports promising data on muscle wasting drug

Reuters

Bio Blast Pharma Ltd said its lead experimental drug showed signs of being effective in treating patients with a rare muscle wasting disorder, besides also hitting the main goals of safety and tolerability.

The company said the experimental drug, Cabaletta, hit multiple efficacy endpoints during a mid-stage trial on patients suffering from oculopharyngeal muscular dystrophy, according to interim results from study.

Oculopharyngeal muscular dystrophy is a rare disease where patients develop swallowing difficulties, which could lead to death in severe cases.

Bio Blast said the drug improved patients’ muscle function while also reducing difficulty in swallowing.

Kamagra Oral prices of viagra Click Here Jelly is a more reasonable alternative, which is the reason it is getting in to the market for its high performance and lower cost. According to a recent research, less than 10% men with erectile condition? If you are thinking to go with basic latex ones and not the variants with dots, else it’ll just beat the primary reason (some even have patterns inside). tadalafil sales Be willing to viagra uk understand where they are coming after me I can only assume that normal membership is dropping and they are going to give an overview of some of the most popular ED medications in the world. The cheap india viagra GreenLight PVP Laser Procedure is the latest technological innovation in the treatment of prostate gland enlargements. The company said it would discuss results from this study and a second mid-stage study with the U.S. Food and Drug Administration before it started a late-stage study.

Bio Blast is also testing the drug as a treatment for spinocerebellar ataxia type 3, another genetic condition that affects the nerves in the brain.

Up to Monday’s close of $5.04, the Israeli company’s U.S.-listed shares have lost a little over half their value since they went public in July last year.

(Reporting by Vidya L Nathan in Bengaluru; Editing by Savio D’Souza)
Read more at Reuters

http://www.reuters.com/article/2015/10/27/us-bio-blast-pharma-study-idUSKCN0SL14O20151027#MrVYQdKUvWOoXyXL.99

BioBlast Scant Data Links Drug to Improved Swallowing in Patients

TheStreethttp://www.thestreet.com/story/13338729/1/bioblast-scant-data-links-drug-to-improved-swallowing-in-patients.html

By Adam Feuerstein

| 10/27/15 – 06:05 AM EDT

Oculopharyngeal muscular dystrophy (OPMD) is a rare, genetic, muscle-wasting disease that mostly strikes people in their 40s and 50s. The first symptoms of the disease are often an inability to keep the eyelids open, coupled with difficulty swallowing. As the muscles in the face and throat weaken over time, patients lose the ability to properly swallow food or water. In severe cases, OPMD patients die from choking or from infections caused by food getting into the lungs.

There are no drugs approved to treat OPMD currently, but on Tuesday, an Israeli biotech company, BioBlast Pharma (ORPN) , disclosed partial results from a small, mid-stage clinical trial showing a common sugar used for years to preserve frozen food and as a non-active stabilizer in other drugs might improve the swallowing ability of OPMD patients.

The sugar-turned-drug being developed by BioBlast is called Cabaletta. The phase II open-label study in OPMD was stopped early — only one-third of the planned number of patients were enrolled — because the Cabaletta data seen to date were so promising it was unethical to continue, said Dalia Megiddo, BioBlast’s co-founder and chief development officer, in an interview Monday night.

BioBlast intends to meet with regulators at the U.S. Food and Drug Administration seeking clearance to advance Cabaletta into a phase III study in OPMD patients.

Megiddo and BioBlast CEO Colin Foster paint a very optimistic picture of Cabaletta’s potential as an effective treatment for OPMD, but the data announced Tuesday are scant, cherry picked and hardly conclusive.

BioBlast shares closed Monday at $5.06 and with an overall market value of $74.9 million. The stock has lost 21% of its value so far this year.

The original design of the phase II study called for 74 patients with OPMD to be treated with weekly, intravenous injections of Cabaletta for six months. BioBlast only enrolled 25 patients into the study at two hospitals, one in Israel and the other in Canada. The results disclosed Tuesday came from an unplanned, interim analysis conducted on Sept. 1.

Apart from this the pill has been approved by FDA and so one can make use of cheapest levitra respitecaresa.org. Lots of men are struggling for curing their impotency or erectile dysfunction has made man to stay sexually incapable by infecting the concerned mechanism. cialis generika 10mg What are the principles of massages? The principle of massage manipulation therapy is viagra discount india to dredge the channel. Still, in many cases, conception doesn’t happen, and the couples are required to commander viagra seek for the therapy, which you are going to find in this article. Since difficulty swallowing and ingesting food into the lungs puts OPMD patients at the greatest risk, BioBlast relied on a moving x-ray of the mouth and throat to measure if Cabaletta could improve the ability of patients to swallow (or avoid having food get into the airway).

On this efficacy endpoint, known as the Penetration Aspiration Score, BioBlast says 10 of 12 patients, or 83%, showed stabilization or improvements in swallowing ability following six months of treatment with Cabaletta.

BioBlast enrolled 25 patients into the study, so why only results in 12 patients? In seven other patients treated in Israel, the moving x-ray taken at baseline didn’t work, so BioBlast excluded them from the analysis. One patient died during the study. The data from the remaining five patients have not yet been analyzed, said Megiddo.

The mean and median improvements in Penetration Aspiration Score were not disclosed. Megiddo says these data are unimportant because the study enrolled so few patients. Instead, the difference in swallowing ability of individual patients at six months was measured against their own baseline scores.

The Penetration Aspiration Score is an eight-point scale, with the lower score correlated to better swallowing ability. Megiddo says four patients with stabilized swallowing had the same Penetration Aspiration Score at six months as they did at baseline. The six patients with improved swallowing after six months reduced their Penetration Aspiration Score by 1 or 2 points. The study had no control arm by which these results could be compared.

BioBlast intends to use the Penetration Aspiration Score as the primary endpoint of its phase III study.

Other measures of swallowing ability and muscle function used in the phase II study yielded mixed results in different and inconsistent numbers of patients.

Cabaletta is a form of the natural sugar trehalose, already approved by FDA as a food-safe ingredient. Trehalose is also used a non-active stabilizing agent in protein-based cancer drugs like Avastin and Herceptin. As a sugar, trelahose is broken down in the small intestine, so Cabaletta is formulated as an intravenous injection to bypass the intestine and deliver a therapeutic dose more effectively to muscles and nerve cells.

The genetic mutation which causes OPMD creates an unstable protein, PABPN1, which then collects in muscle cells instead of being removed. Bioblast believes trehalose, formulated as Cabaletta, acts as a stabilizer for the misshapen PABPN1 proteins, preventing the proteins from accumulating together and helping rid them from the body.

BioBlast has no composition of matter patents on Cabaletta since trehalos is a naturally occurring sugar. The company is developing Cabaletta based on method-of-use patents protecting the intravenous formulation for use as a drug.

Immunotherapy creates fresh challenges and tactical imperatives

BioWorldBy Michael Fitzhugh, Staff Writer

SAN FRANCISCO – With widespread excitement about immuno-oncology combinations driving deals, the pressure to find synergies has never been greater. The opportunities are creating new challenges for companies big and small, executives at the 2015 BIO Investor Forum said Tuesday.

“It’s a very interesting time to be in the immunotherapy space,” Heat Biologics Inc. CEO and founder Jeff Wolf told forum attendees. Combinations represent the future of the space, and the best hope right now for potential cures, he said.

Sitting beside him on the panel was Ben Thorner, vice president of business development and licensing for Merck & Co Inc. – a man more than a little familiar with the current excitement in the space, much of which has centered around exploring therapeutic combinations involving Merck’s high-profile PD-1 inhibitor Keytruda (pembrolizumab), the first drug in its class to gain FDA approval, just over a year ago. (See BioWorld Today, Sept. 5, 2014.)

“For us, one of the challenges is thinking about the daunting number of cancer settings as well as combinations,” said Thorner. “Probably at the top of our list as we put in place these clinical trial collaborations with partners is to try to come up with the best way to do studies to get a quick look at which are the combinations that are really going to have the most meaningful effect on patients.”

Merck went from first-patient, first-visit in the first phase I study of Keytruda to approval of that therapy in about three and a half years, Thorner said. “We were able to do that by marshaling enormous resources to go really, really fast. We want to do that in the cases where there is the greatest degree of benefit for patients when it comes to combinations. The struggle right now is that we have more than 30 different combinations and clinical trials in different cancer settings,” he said. Doing those studies in a way that leads to a quick reliable answer in any given target can be difficult.
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At Heat Biologics – which has a market cap of $34.2 million vs. Merck’s $144.78 billion market cap – the challenges are different. The speed with which the immunotherapy space is moving is, in and of itself, a force working against its efforts. “As you’re going through clinical trials, new combinations are showing up and showing promise, which puts the smaller companies in a position of really having to sit back and strategically design their clinical trial to make sense in light of everything going on in the environment,” said Wolf. “While companies like Merck and [Bristol-Myers Squibb Co.] have the resources to spend massive dollars on these huge all-comers trials, companies like ours need to be much more focused in how we direct our resources and how we combine with others,” he said.

To that end, Heat recently embarked on a trial it’s calling Durga, named for the Hindu war goddess with many arms. The multi-arm, open-label phase Ib trial is testing HS-110 (viagenpumatucel-L), the company’s injectable adenocarcinoma cell vaccine, in combination with a PD-1 checkpoint inhibitor. In September, the first patient was enrolled in the trial to evaluate the safety, efficacy and tolerability of HS-110 in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.) with non-small-cell lung cancer who have failed at least one other therapy. A report of topline objective response rates and six-month progression-free survival (PFS) data on the first 18 patients is expected to arrive by the end of 2016. Enrollment is ongoing. (See BioWorld Today, Sept. 2, 2015.)

There’s still a lot of territory to cover, in terms of exploring possible combinations, but the panelists agreed companies are quickly moving up the learning curve. The way they approach the task – especially the approach of small companies looking to partner with larger ones – can make a tremendous difference in their eventual fortunes.

Olivier Lesueur, managing director of Bionest Partners, a life sciences strategy consulting firm, told forum attendees he advises his clients that the most important thing they can do is put together a compelling value proposition. “Invest in preclinical data,” he said. “Show impact on tumor penetration. Show impact on the immunogenicity of the tumor. Show that you do not negatively impact immune functions.”

While the models for measuring those things aren’t great, Lesueur said, being able to show there’s at least good reason to believe there will be synergy with an immunotherapy asset is key. And in that, Lesueur may have hit on the most important mission for any company meeting potential investors at this week’s meeting, for what else are any of them looking for, essentially, other than a reason to believe?