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Exclusive Life Science Feature all rights to its novel ribosome-targeting antibiotics (RX-04) program. "At the time the Sanofi deal was completed, we believed it was the best way to advance the RX-04 platform, and Sanofi contributed meaningfully to its advancement," says Szela. "However, with the clinical and development resources of our lead investor Vatera, and with a new management team in place, the company now has the resources to advance the program independently and has worked with Sanofi to obtain full rights. We were thrilled they agreed to return the rights since we believe we are in a strong position to move RX-04 into the clinic." Szela says the company remains committed to developing its pipeline of novel antibiotics. It is conducting a Phase 3 clinical trial of delafloxacin for acute bacterial skin and skin structure infections (ABSSSI), the first of two planned Phase 3 trials for the drug in ABSSSI, the second one for an oral formulation later in 2013 or in early 2014. It plans to launch human trials for the lead molecule in the RX-04 program in cations. Once there, they must design the preclinical and early clinical studies as carefully as possible to address safety and effectiveness in the targeted areas. Rabin of ACT emphasizes getting the right experts to plan those studies. "Many of our scientists are trained as developmental biologists and have the ability to elucidate the conditions that occur during 'organogenesis' for any type of tissue — that is, to figure out the natural signals that occur as an embryonic stem cell becomes a differentiated cell as part of an organ in the body." That solves the challenge of producing stem cells, but none of the company scientists had worked with animal models, so ACT turned to academic collaborations to explore how the cells might treat human diseases. Now, Rabin says, the company has some of the leading scientists in its targeted disease areas to guide its clinical development programs. It has achieved U.S. and European orphan status for its Stargardt's disease program, shortening the "The cost of building our own GMP space has proved to be far less expensive than the CMO route and probably far speedier in terms of the time to get to clinical trials." Gary Rabin, president and CEO, Advanced Cell Technology 2014 for life-threatening, gram-negative pathogens. "We are very lucky to have a group of investors who believe in the scientific and technological underpinnings of the company, and they are very supportive of our efforts, so we are well-funded to complete the development and commercialization of our products," Szela says. "Drug development is a difficult endeavor, and the main hurdles we face in the R&D; of our products are achieving the clinical and regulatory milestones necessary to commercialize them." For ACT, Rabin says, working with human embryonic stem cells gives it some uniqueness, which has attracted private investors and helped it weather the Bush-era moratorium on government funding. "The federal-funding issue has proved to be both a positive and a negative. While we would like to see greater NIH investment in our programs, as well as access to our human ES cells by other researchers in regenerative medicine, the moratorium actually gave us a gigantic advantage because we used private money and thus became a forerunner in both clinical development and in securing very broad patent positions in HES — as a consequence of getting there first." STAY THE COURSE! — WHERE'S THE MAP? By choosing the novel-drug development path, micro-innovators assume the mighty challenge of actually going down it. The first real step is proof-of-concept (PoC), but as we've seen, companies may spend years getting to the point of proving the particular concept that will drive development of their products' initial indi- 32 LifeScienceLeader.com January 2014 timeline and offering possible compassionate use, and its dry AMD program may be eligible for accelerated approval and priority review at the FDA, after Phase 2 studies. "Some of the common issues in development include figuring out which 'box' your product fits in for the FDA or other regulatory agencies — particularly for cell therapies, how to be regulated as a drug versus device (and especially avoid being regulated as both) — and developing a strategy for educating regulators about your product so that your safety and efficacy studies are approved in a way that maximizes the chance for success," Rabin says. "Another possible common feature relates to drug pricing and reimbursement. There are set CPT and HCPCS drug and product codes, and those existing codes may not always be ideal (or even work) when it comes to completely different approaches to treating a disease where existing therapies exist and can be equally problematic where there is no existing treatment for a particular disease." Melinta is expediting its development programs under the U.S. Generating Antibiotic Incentives Now (GAIN) Innovation Act, passed in July 2012 to encourage drug development aimed at resistant pathogens. Delafloxacin was one of the first compounds to receive Qualified Infectious Disease Product (QIDP) designations under the Act, for ABSSSI and community-acquired bacterial pneumonia (CABP). Companies with QIDPs gain incentives such as an additional five years of market exclusivity, priority review, and eligibility for fast-track review status.