I just became aware of this after someone I follow posted their ER yesterday on Stocktwits. First attraction was cash value. Sitting on about $5 million over MV. So now, I dug deeper on their only candidate. And my conclusion is that Macrilen approval appears a done deal for the following reasons:
1. Nov 6, 2014, the FDA issued a CRL stating that Macrilen NDA could not be approved in form it was filed. The FDA was concerned with "the lack of complete and verifiable source data for determining whether patients were accurately diagnosed with AGHD." After meeting with the FDA regarding the approval deficiencies in the CRL, Company got a panel of US and EU endocrinology experts to advise it regarding options for Macrilen. Now NOTE THIS: "The panel advised the Company to continue to seek approval for the compound because of their confidence in its efficacy and because there currently is no FDA-approved diagnostic test for AGHD." So, Company formulated another proposal to the FDA.
2. March 6, 2015, Company met with the FDA agreed on the general design of a confirmatory study as well as evaluation criteria. On April 13, 2015, Company announced a new Phase 3 study that will demonstrate the efficacy of Macrilen and satisfy the registration requirements of the European Medicines Agency and the FDA.
3. Oct 26, 2016, Company announced completion of patients enrollment for the Phase 3 and intention to file a NDA for Macrilen with the FDA during the first half of 2017, NOTE "if the results of the trial warrant doing so."
4. Feb 13, 2017, Company announced completion of the Phase 3. More importantly, it announced that using the insulin tolerance test (ITT) as a comparator, it concluded that Macrilen "demonstrated performance supportive of achieving registration with" the FDA. FURTHERMORE: "The FDA has agreed to consider the Company’s conclusions during a Type A meeting." NOW READ THIS: the new Phase 3 confirmed that "Macrilen stimulates the pituitary gland to produce growth hormone more effectively than the ITT; in approximately 80% of all patients, measured growth hormone levels following the administration of Macrilen were equal to or higher than the growth hormone levels produced by the ITT."
5. March 29, 2017, Company met with the FDA regarding the new Phase 3. At the end of the meeting, the FDA stated that the clinical studies addressed the prior deficiencies mentioned in the November 2014 complete response letter. Further, the FDA "indicated that the summary data we submitted prior to the meeting appear to support the propositions we advanced." Mr. Dodd concluded: "Although there can be no assurance of approval of any NDA, we believe that we are now one important step closer to the commercialization of Macrilen"
Mar 30, 2017
NOW REGARDING MARKET ADOPTION AND COMMERCIALIZATIOn FOLLOWING LIKELY APPROVAL, PLEASE SEE PR OF OCTOBER 26, 2016, stating "the Company believes that Macrilen™, if it is approved, is likely to be rapidly adopted by physicians as the preferred means of evaluating AGHD for the following reasons:
it is safer than the ITT because it does not require the patient to become hypoglycemic and thus avoids the symptoms and potential complications of hypoglycemia;
Macrilen™ is administered orally, while the ITT requires an intravenous infusion of insulin; the evaluation of AGHD using Macrilen™ is significantly less time consuming and labor intensive than the ITT and, therefore, it is less expensive to conduct; and
the evaluation can be conducted in the physician’s office rather than in a hospital setting.
As a result, the Company believes that Macrilen™, if it is approved, may be used for 40% to 50% of AGHD evaluations in the U.S. during the first year after its introduction and that the percentage could grow to as high as 85% within the first three years of commercialization."
CATION I BOT SOME YESTERDAY BUT WILL LOAD MORE TODAY
