Biotech Stocks: Why Expensive U.S. Drug Trials are Pushing Pharma Research Overseas
PALM BEACH, Florida:
American pharmaceutical and biotech companies who need to conduct clinical trials are looking more and more to conduct them overseas in the European Union, because the EU offers lower cost alternatives to U.S. trials... which not only saves money, buy also can save time to market.
Many U.S. based companies have taken notice. A recent article with an industry insider said A typical drug is unlikely to reach pharmacy shelves for a decade after scientists first develop it, and getting it through all of the regulatory hurdles can cost billions of dollars. That's a serious challenge for smaller pharmaceutical companies... (companies have) found a shortcut by conducting clinical trials in (the E.U.), where it's been able to move much faster than its ongoing American experiments. Like any small biotech company, we have to make the most of limited resources... The big challenge there ultimately comes to time and money, and of course those two are inextricably related. The more time you spend, the more money you're burning; we look for every opportunity to shorten the pathway. Active biotech and pharma companies in the markets this week include Moleculin Biotech, Inc. (NASDAQMBRX), Zynerba Pharmaceuticals, Inc. (NASDAQ ZYNE), Esperion Therapeutics, Inc. (NASDAQ ESPR), Athenex, Inc. (NASDAQ ATNX), TherapeuticsMD, Inc. (NASDAQ TXMD).
The insider went on to provide some other reasons, in addition to lower costs, that the E.U. is offering better options. It continued In the U.S., there are so many clinical trials happening that... participants... (have) become a hot commodity. Not so (overseas)... It took us nearly a year in the US to fill the first patient cohort, (the insider) said. We filled the first patient cohort... (overseas) in the first month and a half. The article also addressed the high degree of efficacy of safety standards in the E.U., saying ... as far as safety standards and rigor go, the trials are the same... (citing) the Clinical Trial Site Standards Harmonization Action Collaborative, an international agreement that set universal safety standards for clinical research no matter where it occurs... It's fairly common for small biotechs to look for opportunities outside the U.S. that may help accelerate the process...
Moleculin Biotech, Inc. (NASDAQMBRX) BREAKING NEWS Moleculin Biotech, a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, today announced additional positive interim safety and efficacy data from its ongoing open label, single arm Phase 1/2 study of Annamycin in Poland. After receiving a single starting dose of 120 mg/m2 in the first cohort of the dose escalation phase of the trial, 2 of 3 patients treated responded sufficiently to qualify for a potentially curative bone marrow transplant. The results for all 3 patients were reviewed by the Safety Review Committee, which determined that no drug-related adverse events were observed that would prevent advancing the trial to the next higher dose level of 150 mg/m2. To date in the European trial, one patient experienced grade 2 mucositis (which resolved to grade 1 within 2 days) and no other adverse events related to Annamycin have been reported. No additional patient data have been developed in the Company's parallel US clinical trial, which is currently recruiting its second cohort to be given a dose level of 120 mg/m2 (the US trial started at a lower initial dose of 100 mg/m2).
The progress in Europe, specifically Poland, continues to be encouraging, commented Walter Klemp, Moleculin's Chairman and CEO. In just a few months, we have completed the first cohort and we've seen a partial response from 2 out of 3 patients sufficient to qualify them for a potentially curative bone marrow transplant. It's important to remember that these are relapsed or refractory patients for whom the standard of care failed. So, to see this kind of response at the starting dose level in the dose-ranging phase, we believe is quite remarkable. And, although this data is still preliminary and may not be indicative of the ultimate outcome of the trial, the improvement in activity at 120 mg/m2 in Poland as compared with the 100 mg/m2 cohort in the US is consistent with our expectations for Annamycin.
Mr. Klemp continued Recognizing that cardiotoxicity is a significant limitation of existing therapies, we are pleased that we continue to see no evidence of cardiotoxicity in any of the patients treated thus far. Specifically, there was no observed reduction in left ventricle ejection fraction, which is the standard metric for acute cardiotoxicity, nor any change in biomarkers that would indicate the potential for long-term cardiovascular impairment. This is an important step in the ongoing clinical study of Annamycin and toward our goal of ultimately demonstrating the drug's safety and effectiveness to support regulatory approval in both the US and European Union.
Dr. Robert Shepard, Moleculin's Chief Medical Officer for Annamycin added With the Polish trial now progressing to 150 mg/m2, we expect to see even better results. And, although 150 mg/m2 was the maximum tolerable dose established by the prior developer of Annamycin due to the incidence of mucositis in patients above that dose level, now that the cryotherapy protocol is well understood to mitigate the potential for dose-limiting mucositis, there is a good opportunity for dose levels to progress even beyond 150 mg/m2, so the potential to help patients is very exciting.
The insider went on to provide some other reasons, in addition to lower costs, that the E.U. is offering better options. It continued In the U.S., there are so many clinical trials happening that... participants... (have) become a hot commodity. Not so (overseas)... It took us nearly a year in the US to fill the first patient cohort, (the insider) said. We filled the first patient cohort... (overseas) in the first month and a half. The article also addressed the high degree of efficacy of safety standards in the E.U., saying ... as far as safety standards and rigor go, the trials are the same... (citing) the Clinical Trial Site Standards Harmonization Action Collaborative, an international agreement that set universal safety standards for clinical research no matter where it occurs... It's fairly common for small biotechs to look for opportunities outside the U.S. that may help accelerate the process...
Moleculin Biotech, Inc. (NASDAQMBRX) BREAKING NEWS Moleculin Biotech, a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors, today announced additional positive interim safety and efficacy data from its ongoing open label, single arm Phase 1/2 study of Annamycin in Poland. After receiving a single starting dose of 120 mg/m2 in the first cohort of the dose escalation phase of the trial, 2 of 3 patients treated responded sufficiently to qualify for a potentially curative bone marrow transplant. The results for all 3 patients were reviewed by the Safety Review Committee, which determined that no drug-related adverse events were observed that would prevent advancing the trial to the next higher dose level of 150 mg/m2. To date in the European trial, one patient experienced grade 2 mucositis (which resolved to grade 1 within 2 days) and no other adverse events related to Annamycin have been reported. No additional patient data have been developed in the Company's parallel US clinical trial, which is currently recruiting its second cohort to be given a dose level of 120 mg/m2 (the US trial started at a lower initial dose of 100 mg/m2).
The progress in Europe, specifically Poland, continues to be encouraging, commented Walter Klemp, Moleculin's Chairman and CEO. In just a few months, we have completed the first cohort and we've seen a partial response from 2 out of 3 patients sufficient to qualify them for a potentially curative bone marrow transplant. It's important to remember that these are relapsed or refractory patients for whom the standard of care failed. So, to see this kind of response at the starting dose level in the dose-ranging phase, we believe is quite remarkable. And, although this data is still preliminary and may not be indicative of the ultimate outcome of the trial, the improvement in activity at 120 mg/m2 in Poland as compared with the 100 mg/m2 cohort in the US is consistent with our expectations for Annamycin.
Mr. Klemp continued Recognizing that cardiotoxicity is a significant limitation of existing therapies, we are pleased that we continue to see no evidence of cardiotoxicity in any of the patients treated thus far. Specifically, there was no observed reduction in left ventricle ejection fraction, which is the standard metric for acute cardiotoxicity, nor any change in biomarkers that would indicate the potential for long-term cardiovascular impairment. This is an important step in the ongoing clinical study of Annamycin and toward our goal of ultimately demonstrating the drug's safety and effectiveness to support regulatory approval in both the US and European Union.
Dr. Robert Shepard, Moleculin's Chief Medical Officer for Annamycin added With the Polish trial now progressing to 150 mg/m2, we expect to see even better results. And, although 150 mg/m2 was the maximum tolerable dose established by the prior developer of Annamycin due to the incidence of mucositis in patients above that dose level, now that the cryotherapy protocol is well understood to mitigate the potential for dose-limiting mucositis, there is a good opportunity for dose levels to progress even beyond 150 mg/m2, so the potential to help patients is very exciting.