The following 5 under-the-radar healthcare stocks had important announcements this week that may not have made the front page news.
Raptor Pharmaceuticals (RPTP) has had a hectic week. On Monday, the stock surprisingly tumbled 28% after releasing what seemed to be strong results for its pivotal Phase 3 clinical trial. On Tuesday, the stock jumped 13% to claw back some of those losses. The company announced that its Phase 3 clinical trial of Delayed Release or DR Cysteamine, known as study drug RP103, for the treatment of nephropathic cystinosis, met the primary endpoint of non-inferiority compared with Cystagon, immediate-release cysteamine bitartrate. The comparison was based on white blood cell ("WBC") cystine levels, the established efficacy surrogate biomarker and sole primary endpoint in the clinical trial. The company also reported that there were no unexpected serious safety concerns experienced by patients in the trial attributable to RP103.
Of 41 patients who completed the Phase 3 protocol, 38 were included in the evaluable data set, 3 not being fully compliant with the protocol. On average, the peak WBC cystine level measured in patients treated with Cystagon was 0.54 +/- 0.05 nmol 1/2 cystine/mg protein, compared with an average peak value of 0.62 +/- 0.05 nmol 1/2 cystine/mg protein for patients treated with RP103. The mean difference was 0.08 nmol 1/2 cystine/mg protein, with a 95.8% confidence interval of 0.00-0.16 (one sided p=0.021). As stipulated in the Statistical Analysis Plan, the non-inferiority endpoint of the clinical trial would be achieved when the upper end of the confidence interval around the mean difference of WBC cystine levels did not exceed an absolute value of 0.3. The upper end of the confidence interval in the Phase 3 clinical trial was determined to be 0.16, thus achieving the non-inferiority endpoint.
Additionally, the endpoint was achieved at a lower average daily dose of RP103, compared with Cystagon. In the course of the study, seven serious adverse events ("SAEs") requiring a visit to the emergency room or hospital, were reported for seven individual patients. Of these seven SAEs, six were determined by the Principal Investigator to be unrelated to either RP103 or Cystagon. One SAE, gastric intolerance, was graded as "possibly related" to RP103 and was subsequently resolved. Further analyses of non-serious adverse events are underway by the company's statistical contractor.
Furiex Pharmaceuticals (FURX) rose 2.2% on Tuesday after it confirmed that Takeda Pharmaceutical resubmitted two new drug applications (NDAs) for alogliptin to the FDA. One application is for marketing approval of alogliptin, and the second is for the fixed-dose combination therapy alogliptin/pioglitazone. Under Furiex's agreement with Takeda, these two resubmissions do not trigger a milestone payment to Furiex. If U.S. approval is granted, Furiex would be eligible to receive a $25 million milestone payment as well as potential royalties and sales-based milestones. Furiex presently receives royalty payments from Takeda for the sale of alogliptin, tradename Nesina, in Japan.
Nephros (NEPH.OB) has had a volatile week, jumping 30% on Monday and falling 14% on Tuesday. After the close on Tuesday, the company provided a corporate update which included its plans for a new 510(k) application. Nephros anticipates submitting a new 510(k) application to market its leading-edge hemodiafiltration (HDF) system for end-stage renal disease (ESRD) in the U.S. in the third quarter of 2011 which would be subject to the FDA's standard 90-day review period. The application will detail Nephros's OLpur MD220 diafilter and Nephros's OLpur H2H Hemodiafiltration module. Nephros's OLpur MD220 is a dialyzer designed expressly for HDF therapy that employs Nephros's proprietary Mid-Dilution diafiltration technology. Nephros's OLpur H2H Hemodiafiltration module enables the most common types of standard dialysis machines to perform HDF therapy. Nephros believes that, if approved, its technology would be the first approved on-line HDF therapy available in the U.S.
Ligand Pharmaceuticals (LGND) jumped 8% on Tuesday following a positive clinical trial update. The company announced that it received positive data from ENABLE-1, the first of two Phase III studies examining Promacta (eltrombopag) in patients with hepatitis C-related thrombocytopenia, and that full data will be released at an upcoming scientific conference.
Chelsea Therapeutics International (CHTP) rallied 5% on Tuesday after announcing positive preliminary data from a Phase II study. The company announced positive top-line results of an investigator-led Phase II clinical study of droxidopa in combination with carbidopa in adults with attention deficit hyperactivity disorder (ADHD). The single-center, 12-week study enrolled 20 patients diagnosed with adult ADHD. The top-line results showed that droxidopa dramatically improved the patients' mean score on the adult ADHD Investigator Symptom Rating Scale (AISRS).
Upon enrollment, patients in the study had a mean AISRS score of 34. After three weeks of open-label droxidopa monotherapy (titration from 200mg-600mg TID), the mean AISRS score decreased by approximately 47% to 19 (p<0.0001). The reduction in AISRS score was maintained with the addition of carbidopa (25mg or 50mg) for another three weeks. Droxidopa was well tolerated with no serious adverse events observed. The most common adverse events observed throughout both the open-label and double-blind portions of the study were headache (n=5; 25%) and drowsiness (n=5; 25%), both of which were mild.
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.