BioPharm America 2014: Partnerships to Solve the World’s Unmet Needs

Event Recap by Karen Ring, Big3Bio Event Correspondent

How important is it that the major players in the life sciences — pharma, biotech, startups, academics, etc. – increase partnering to tackle unmet patient needs and develop innovative treatments for serious diseases?

bpa1BioPharm America 2014, held in Boston from September 22-24, brought together the leaders from these organizations to meet and discuss just how essential partnerships are for the progress and success of the life sciences industry. The 7th annual edition of the event had an impressive turnout with 770 delegates, 504 companies, 2,343 one-on-one meetings, 787 licensing opportunities posted, and 72 company presentations. The conference was organized by EBD Group and hosted by MassBio.

A chronicle of the three-day program:

Welcoming the Masses

Bob Coughlin, CEO of MassBio, opened the event by highlighting this year’s success in the life sciences industry –16 companies going IPO in 2014 (See Bruce Booth’s article on 2014 Biotech IPOs). However, even with these successes, he emphasized that there is much work to do to ensure the success of Massachusetts and US biotech companies.

Coughlin suggested that companies connect with new non-traditional funding sources to fill the funding gap. He acknowledged that while there was an impressive amount of early stage funding that was poured into Massachusetts companies, there still needs to be more.

“Continued discussion, continued partnerships, and collaborations can make a future where patients are helped,” he said. “We need to solve the worlds unmet medical needs (through avenues of partnerships and collaborations).”

That became the underlying theme of the conference.

John Carroll Announces Fierce15: Go Big or Go Home

John Carroll, editor in chief of FierceBiotech, gave a special live announcement of the “Fierce 15”, a list of 15 innovative and up-and-coming biotech companies chosen by the publication. Carroll described the Fierce 15 as a way to “celebrate what’s great about this industry. [These are] groups of people who can take ridiculous risks, who try to develop game changing therapies, [and create] something that the world hasn’t seen.”

John Carroll on the right with some of the 2014 Fierce 15 winners

Carroll explained that this year’s group was different than previous years owing to changes in financial trends (IPO window still open, less overall VC funding, successful VCs starting new funds). As a result, this year’s group is dominated by early stage development companies led by experienced research and management teams.

Carroll highlighted that many of the Fierce 15 are clustered around the “Big 3” biotech hubs (six companies from the Boston area, two from San Francisco, and two from San Diego); these companies are “going where the money is” with most of these companies in the 15 lying within a 50 mile radius of one of these hubs. Carroll predicted future trends by mentioning that many top-level academic scientists are leading research teams at these companies and more are looking into crossing the divide into biotech. When announcing the 15, Carroll said these companies will “change the way diseases are treated. It’s go big or go home.”

This year’s Fierce 15: Adapt Immune, Aduro Biotech, Calico, Cidara Therapeutics, Cleave BioSciences, Dimension Therapeutics, Editas Medicine, Juno Therapeutics, Molecular Partners, Navitor Pharmaceuticals, Seres Health, Spark Therapeutics, Spero Biopharma, Sutro Biopharma, Voyager Therapeutics, and Mapp Biopharmaceuticals named as the “Fierce 15 Extra”. For more details and company profiles check out FierceBiotech’s 2014 Fierce 15.

Gary Nabel’s Keynote Address on Rare Diseases

Gary Nabel, Chief Scientific Officer at Sanofi, argued in his keynote speech that rare diseases are a perfect model for determining how to translate academic innovation into industry. Nabel told the story of Gaucher’s disease and Genzyme’s enzyme replacement therapy drug called Ceredase. Nabel was frank: “Nothing in biotechnology is simple…there is a need for other innovative therapies in rare diseases.”

He went on to explain that while Ceredase was very successful, not all patients responded positively to the drug. Genzyme addressed this issue by developing another option for Gaucher’s patients: Cerdelga. This is a first-line oral therapeutic that attacks Gaucher’s disease by a different mechanism and was approved recently by the FDA in August 2014.

Nabel then shared his two cents on the rare disease pharma model. Nabel explained that the traditional approach to translational medicine (starting with a target, developing a drug, then treating disease) needs to be switched to a more dynamic model of identifying a disease with an unmet need, determine a target, figure out the mechanism, and then develop a therapeutic.

“The rare disease model can teach us how to be successful in trying this approach. The power of genetics is great. If we can get big data on human polymorphisms and disease phenotypes, connect the genetics to the cellular and molecular pathways in humans, explore traditional and non-traditional approaches, and understand the disease well enough, then drug development risk can be reduced. If there is an outstanding medical need, the probability of creating transformative medicine increases, transformative medicines will be approved and reimbursed, and [we] will impact patient lives!”

On partnering, Nabel talked about the value chain of innovation on a spectrum of early (academics, small biotech, government research), middle (biotech), and late (large biotech, pharma) development. The challenge he said is to know where and when to get engaged and to determine what move would have the highest probability of success.

Nabel concluded by emphasizing the need for more company co-creation. Early-stage innovative ideas should be identified earlier by VCs and pharma, and companies formed around these ideas. He ended on an upbeat note: “This is an exciting time for the industry, and I can’t wait to see what innovative medicines come in the next couple of years!”

Key Highlights from Panels

The remainder of BioPharm America continued with partnering meetings, company presentations, and panels that discussed important topics in the industry.  Key highlights from a few of the panels:

  • In Getting Real Panel: Biopharma innovation and its impact on patients, Chairman and Chief Executive Officer of Alkermes, Richard Pops, noted: “Biotech should take on large chronic diseases because they are affecting the large global economic future of the population.” He suggested repurposing non-substitutable orphan disease drugs for chronic disease treatment to take advantage of an easier regulatory pathway
  • In State of the Industry: A focus on the financial landscape in the life sciences, Bruce Booth, a prominent partner at Atlas Ventures, concisely summarized where the life science industry stands:
  1. There is an unprecedented market demand for biotech innovation.
  2. We are in the longest and largest IPO window for therapeutic biotech companies.
  3. Mergers and acquisitions are doing well.
  4. Disconnect between supply and demand (not enough supply and increasing demand).
  5. The number of startup biotechs is relatively flat over the past 5 years.

Conclusions/possible solutions: We need to change our investment thesis by increasing VC funding for startups, building and recruiting more talent to executive boards, managing the liquidity process, and have more
VCs exit from board positions.

  • Quote of note in “All innovation roads lead to Massachusetts: Pharma’s approach to the Boston biotech cluster”: “Boston is experiencing a massive influx of people in the biomedical industry. This is generating a hotbed of innovation and cutting edge research and attracting startups with new game-changing technologies,” said Leigh Zawel, site head at CTI Boston (Pfizer). “In order to keep these companies in MA, Pharma and VCs have to continue to invest in these small companies and keep the cluster together.”

All of the panels provided in-depth discussions of existing and emerging partnerships that will revolutionize the life sciences industry and patient care.

The overall mood of the conference was positive, yet there was also a sense of urgency to address important issues such as tackling unmet patient needs, the future of biotech funding, recruiting top talent to boardrooms, and translating early stage innovation from academia to industry.  With all the excitement surrounding the success of the life science industry in 2014, next year’s conference will be sure to be an exciting and transformative experience. BioPharm America 2015 will be held on September 15-17 in Boston, MA.