Drug Development: What’s Hot? What’s Not?
Acquisitions, technology, patient engagement, real world evidence, and artificial intelligence are hot trends transforming pharma. A new option for drug-delivery captured investor attention at BIO 2017. An entirely new take on cardiovascular care could mean billions for Novartis. And yet, what’s not hot remains a global threat to health and is using crowd sourcing to try to find answers.
Hot Topics at the Annual Drug Information Association Meeting
- Real world evidence and data continue to be a focus but no consensus on either has yet emerged.
- Artificial intelligence and wearables as a data source were both hot topics this year while neither were on the agenda last year.
- Excitement and interest in forthcoming Chinese regulatory reforms that are expected to make clinical trials and review of clinical trial applications much faster.
- Regulators from developed and developing countries discussed how regulators can better cooperate.
- Involving patients in clinical trials and clinical trial design as well as drug affordability, pricing, and reimbursement.
Parexel Acquired by Private Equity Firm
On June 20, Biopharma company Parexel announced it had been acquired by Pamplona Capital for $5 billion. In May, Parexel said it planned to would eliminate 1200 jobs and shift positions to lower-cost locations such as India. Regarding the sale, Parexel CEO Josef von Rickenbach said: “We believe the more flexible corporate structure afforded by this transaction will better position us to advance PAREXEL’s strategy in light of these realities and to shape the Company to best capitalize on our exciting market opportunities.”
Deal Making at the Biotechnology Innovation Organization (BIO) Business Forum
Cardiometabolic and cancer companies drew the most interest at the BIO 2017. Flagship Pioneering announced a mega $120 million Series B for start-up Rubius Therapeutics. Rubius reengineers red blood cells into ‘drug-carriers.’ Because red blood cells circulate for up to 120 days, there is potential for a longer treatment effect. It’s expected that RED cell therapeutics will be used for many different therapeutic applications including: enzyme replacement, cancer immunotherapy, blood cancers, and triggering apoptosis in tumor cells. Rubius has performed preliminary work on RCT’s potential in autoimmune, metabolic, rare, and infectious diseases. There was quite a bit of interest in Rubius from institutional investors. The $120 Million to Rubius is the largest so far, this year.
Novartis shares rose 3% in the wake of phase III data showing that the active ingredient (canakinumab or ACZ885) in its anti-inflammatory drug Ilaris reduced major adverse cardiovascular events in patients with prior heart attack and inflammatory atherosclerosis when added to standard therapy. It is the first proof that targeting inflammation reduces cardiovascular risk.
The inflammation hypothesis holds that plaque repairs blood vessels damaged by inflammation and oxidative stress.
If it is approved, some investors think it could bring in $2 Billion annually. Although there will likely be payer push-back. The list price for Ilaris is $200,000/year. Data must still be presented to cardiologist. This will likely happen at the European Society of Cardiology Congress in August.
Melinta Therapeutics won FDA approval for its antibiotic delafloxin. To be sold as Baxdela, the antibiotic is approved for serious skin infections. While this is a win, an ongoing supply of new antibiotics is needed to combat antibiotic resistance. There are a few, but not enough players in antibiotic R & D.
It can take decades and millions of dollars to develop an antibiotic only to have them become ineffective as bacteria fight back. The disincentives are real. Despite that, one scientist took it upon himself to go old-school with a twist in a personal quest to find new options. Doctor Adam Roberts took up where researchers left off decades ago, looking for answers in the dirtiest and most germ infested places. He extended his reach by crowdsourcing the search for naturally occurring antibiotics. Using a Facebook page, he sells sample kits which people use to send samples from the most unsanitary places they can find. His painstaking work caught the attention of the Liverpool School of Tropical Medicine where he is expanding his quest. His goal is to develop a thousand new drugs, only releasing new ones when the old ones are no longer effective.
Of his vision, he says “I think that is achievable,” he says. “But it’s going to take a massive amount of work.”
Incentives to encourage the development of new antimicrobials is only one part of the solution to resistance. We cannot end the development of new super-bugs, we can only slow it down. The use and overuse of antibiotics worldwide must change. Antibiotic resistant bacteria can develop anywhere and impact people in any country. Just this past fall, a strain of E. coli, resistant to an antibiotic seldom used in the United States was found in Chinese pigs. A patient in Pennsylvania was found to have that strain of E. coli only a half year later.
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