valuation time, page-21

Ok here's highlights of the data released following the 3rd Cohort & timeline of events following ---- make up your own mind.

--- 4 weeks following data EMA grants Orphan Designation

--- 7 weeks further FDA grants Orphan Designation




>>> Convincing evidence that PAT-SM6 is active in patients with end-stage disease who are resistant to other marketed therapies

>>> Two multi-resistant patients experienced stable disease post treatment with PAT-SM6
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>>> PAT-SM6 seems to prolong the time to the next therapy and this is deemed clinically significant

>>> No serious adverse events reported
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>>> Final (4th) cohort of patients currently being recruited


All of the six patients treated in these cohorts tolerated PAT-SM6 very well. There were no drug-related serious adverse events and no dose-limiting toxicities. On the basis of these safety data, the independent Drug Safety Monitoring Board (DSMB) gave approval for the 4th and final cohort to commence. Patients in this final cohort will receive a minimum of four doses of PAT-SM6, each dose being at 6mg/kg/dose. This is more in line with dosing levels of other antibodies currently on the market.

Of these six patients treated with PAT-SM6, two have shown stable disease. The first of these (from the 2nd cohort) was a 73 year-old man who had previously received six lines of therapy including a stem cell transplant. At the time of entry into the trial he was resistant to all-available therapies and had rapidly advancing disease. At day +36 after treatment with four doses of PAT-SM6, he showed evidence of stable disease according to the International Myeloma Working Group criteria.


“Our trial with PAT-SM6 is producing some very exciting data and it is particularly encouraging to see two patients, with end-stage multiple myeloma, respond so positively to treatment with this novel antibody,” commented Patrys’ CEO, Dr. Marie Roskrow. “All of the patients being recruited into this trial are resistant to the currently marketed drugs and PAT-SM6 is inducing strong and sustained immunological responses.”

“As we move into the final cohort of this trial, Patrys is beginning to attract the attention of potential new investors and commercial partners. We are delighted with the progress of the trial and are most grateful to Dr. Rasche and his colleagues for their continuing clinical support,” Dr. Roskrow said."




4th cohort should be complete & updated in days / weeks.

Still further to go through trials of course but just as a guide to FDA in relation to approval stats after Orphan Designation -

In 2012 of the 48 new drugs approved by the FDA 15 of those where Orphan Drugs & that has increased through this year with FDA giving marketing approval to 21 new ODD's in the first 9 months of 2013. <<<< This is what draws big pharma in.