By Malorye A. Branca, Editor-in-Chief, PharmaWeek
New technologies
ranging from software programs to whole drug platforms headlined
the partnering session of this year's Molecular Medicine
Tri-Conference in
San Francisco, February 21. The annual meeting gives young
companies a chance to explain, briefly, their value to would-be
investors and then have an opportunity to meet with them. More
than 40 companies participated this year. (To see a list of these
companies, visit this
link.)
"Don't ever
let anyone say there is no capital for you," advised
biotechnology guru G. Steven Burrill, who opened the meeting.
"And when they are passing it around, reach for it." The
terms have gotten tougher, but for young companies, such capital
is the difference between an idea and a product. Burrill also
warned that "Biotechs need to think globally from day
one." That doesn't just mean thinking of new overseas markets
but also of new overseas competitors. "The Chinese
will quickly be major global players," he said.
In terms of
investments, Burrill said his own top interests now are areas
including nutriceuticals, personalized medicine,
"boomer"-related conditions such as memory loss, and
pandemic diseases. Companies representing a much wider range of
technologies, however, were showcased at the meeting.
While small
molecules are still the most popular target for drug developers, a
number of companies are trying to spearhead completely new drug
platforms.
ProNAi, for
example, is using DNAi to develop a totally new class of drugs.
The company's nucleic-acid-based interfering technology
"solves a non-trivial problem," said Neal Goodwin, chief
scientific officer at the company. "We do not use toxic
nucleic acids." Targeting of DNA and RNA have both been
problematic, but ProNAi claims it has a "bold new nucleic
acid therapeutic approach" that has "great
bioavailability" and that the company will soon have
large-scale manufacturing capacity. That is critical because the
company aims to launch a clinical trial of its lead candidate in
2006.
Another company
with a new approach to drug making is Catalyst Biosciences, which
is developing Alterases — engineered proteases. "Proteases
have a very focused activity, but we believe we can expand this
and bring catalysis to address unmet needs," said David
O'Reilly, the company president. While small molecules act in a
stoichiometric (or one-to-one) manner, proteases act in a
catalytic fashion, so one molecule can affect hundreds or even
thousands of targets.
The explosion of
research on protease inhibitors over the last decade or so has
been a boon to groups such as Catalyst, which is interested in now
using actual proteases as drugs. Several proteases are also
already on the market. "The benefits of these products are
very apparent," said O'Reilly. Another side benefit of these
compounds is that they create "direct, detectable biomarkers
of unique activity," he said.
Helios
Bioscience, meanwhile, is using pathways informatics to help
companies select, classify, and validate drug targets. Building
dynamic models is still "very challenging," said Jean
Baptiste Dumas, Helios's CEO. However, the company is getting
excellent results comparable to those of other methods. In a
recent study using its Immuno-dyn model, the company identified 27
molecules with the desired immune properties out of 350
candidates. Data were already available on 18 of those compounds,
and the company was able to confirm that 70% of these compounds
were indeed known to have those immune properties. Four of the
molecules were novel targets that could be further investigated.
All of these
companies are at relatively early stages in their development, but
they are also reaching for major milestones, such as first
clinical trials and partnerships, that will help them get the cash
and momentum they need to stay in the game.