"Shaping" new vaccines

On March 8th 2011, Liquidia Technologies announced that it has received $10 million from the Bill and Melinda Gates Foundation, in order to develop new vaccine candidates utilizing its proprietary PRINT (Particle Replication In Non-Wetting Templates) technology. For those that have been following the activities of the Gates Foundation, it will immediately be evident that this type of activity, i.e. akin to a VC firm, is quite different to the normal grant process that the Foundation was, and still is, employing when financing a project. One can refer to the Collaboration for AIDS Vaccine Discovery (CAVD), a network of research teams that the Foundation set up and funded with $355 million in order to work towards development of an HIV vaccine, to understand the scope and basic functions of this charity organization.

Liquidia Technologies was founded in 2004 and is currently being backed up (besides the Gates Foundation) by New Enterprise Associates, Canaan Partners, Pappas Ventures, Firelake Capital Management, the Wakefield Group as well as private investors. This is a strong group of investors that, in theory, could assist Liquidia in its plans for rapid growth. Canaan Partners are well known and active investors in companies such as VaxInnate (recently in the news for receiving $117.9 million over three years-potentially rising to $196.6 million over five years- by the HHS, for development of influenza vaccine candidates) and Chimerix, a company that develops orally available antiviral agents, using its Phospholipid Intramembrane Microfluidization (PIM) technology.

What is really exciting about Liquidia's PRINT technology is the ability to control and modify at will the shape, size, chemical composition and, in general, most parameters that play a role in the immunomodulatory properties of a vaccine candidate. The company proposes formulating a vaccine agent or a drug, on a PRINT-produced dissolvable particle. Making use of the ability of the immune system to recognize and distinguish between 3D shapes of proteins and pathogenic/self structural components, such a particle could possess the shape of a bacterial component that triggers a specific immune response, or the structural conformation of a cytoplasm-localized protein, that would facilitate transport of the active ingredient through the cell membrane.

From an investor's perspective, Liquidia constitutes an attractive case. PRINT technology should generally increase the efficacy of a vaccine and could be employed to generate an adjuvant effect; these features will drive costs of vaccine production down and discard the need for use of non-approved adjuvant agents (the FDA is known for its reluctance to approve novel adjuvants). PRINT particles can also be combined with existing vaccines, and this opens up a wealth of opportunities for Liquidia. The company has recently announced the initiation of its first clinical trial, testing its lead seasonal flu vaccine candidate, LIQ001. A quick look at the clinicaltrials.gov website reveals that LIQ001 is being tested as a combination with sanofi's Fluzone; the trial has finished recruitment and is expected to complete in late 2011 with results available in early 2012. As this trial constitutes the company's first attempt of progressing the PRINT-based technology from proof-of-concept into the clinic, we are waiting impatiently to see the results. Liquidia has also signed an agreement with the PATH Malaria Vaccine Initiative (MVI) for the use of PRINT technology in the development of a new generation of malaria vaccines; we believe this is a strategic step, crucial for the company's further development and a collaboration that should yield large amounts of clinical trial data, necessary for further evaluation of PRINT.

Looking into the future, a company such as Liquidia, constitutes an attractive target for acquisition/partnership from a larger biotech or pharma company. Despite the company's recent investment influx and collaborative agreements, it still remains very interesting to see how PRINT technology will navigate the regulatory path and to pressure-test its limitations and advantages.