March 1, 2013 (Vol. 33, No. 5)

J.P. Morgan Healthcare Conference Attendees Share Their Reasons to Anticipate Good Times

While many large-cap biotech companies that presented during this year’s “J.P. Morgan Healthcare” conference are retrenching and repurchasing stock, the small and medium cap biotechs are looking forward optimistically. Innovative firms are anticipating late-stage clinical trial results and, generally, good financial years.

BioTime’s agreement to acquire Geron’s embryonic stem cell program assets was one of the most-discussed conference highlights. “The concept of consolidating the intellectual property is strong enough to build a one-stop shop for the more than 600 patents and applications in this field,” said Michael West, Ph.D., CEO of BioTime.

The deal forms a new company, BioTime Acquisition Corporation (BAC), a wholly owned subsidiary of BioTime. “We know this is the first time we’ve had critical mass,” said Tom O’Karma, president and CEO of BAC, and former president and CEO of Geron.

As Dr. West stated, “We feel strongly about the power of pluripotency. With Geron’s assets, we can create a product model for cell therapy, so vials containing pure cells can be frozen and shipped like a pill. You can only do that if you have a pluripotent cell line.”

The company initially plans to address multiple sclerosis spinal conditions, enabling a single cell to treat many forms of MS spine and spinal injuries. “This has been demonstrated to be safe and feasible in humans, and we are ready to move it to other diseases,” O’Karma remarked.

At Vertex, “We’re sharpening our focus for the future to create transformative medicine for specialty markets—not drugs that are merely wanted, but drugs that are needed to transform lives,” said Jeff Leiden, M.D., Ph.D., chairman, president, and CEO. Dr. Leiden called innovation “the secret sauce to push the frontiers of personalized medicine.”

The company is interested in collaborative options for high-value targets that will help sustain growth. VX-509 for autoimmune diseases, for example, may be a platform for multiple disease therapeutics. Kalydeco™, currently a monotherapy for cystic fibrosis, also has the potential for label expansion. It was approved in the U.S. and Canada and will launch in Europe later in 2013.

The company just received breakthrough therapy designations from the FDA recently for VX-809 combined with ivacaftor for cystic fibrosis and for ivacaftor alone in people above age six who have the G551D mutation. Late-stage projects are under way for cystic fibrosis, hepatitis C, and autoimmune disease and, Dr. Leiden added, “early-stage research has an interesting molecule for genetic and specialty diseases.”

Savara Pharmaceuticals also is focused on cystic fibrosis. In November, the FDA granted orphan drug status to AeroVanc™, a vancomycin hydrochloride inhalation powder, to treat pulmonary methicillin-resistant Staphylococcus aureus infections in cystic fibrosis patients. “By delivering the drug directly to the infection site, we’re delivering 1/20 of the standard intravenous dose but gaining 100 times more concentration at the infection site. Therefore, we also have 1/20 of the toxicity,” said Rob Neville, CEO.

The therapeutic is delivered with a small device that fits inside a pocket. Hospitals currently are using IV vancomycin off-label by means of a large nebulizer, Neville pointed out. Savara filed an IND in December targeting MRSA and anticipates trials to begin in the first quarter of 2013. “We hope to show reduction in the number of MRSA infections in patients with CF,” he said.

A magnified photo of vancomycin hydrochloride powder particles used in Savara’s AeroVanc for the treatment of MRSA lung infection in cystic fibrosis patients.


Cerulean Pharma’s approach to cancer treatment uses antibody drug conjugate-style nanotherapeutics to slip through the leaky vasculature inherent in tumor cells. “Because our drug is covalently bonded, it can be gradually released directly to the tumor,” Oliver Fetzer, Ph.D., president and CEO, said. Molecular targets often fail to elicit a durable response because of tumor heterogeneity, he pointed out. “Our approach combines a targeted delivery system and highly potent drug that is released from within tumor cells.”

Lead compound CRLX101 is in four Phase II trials, including a Phase IIb trial for non-small-cell lung cancer. Overall survival is the endpoint. Mono- and combination therapies are in development. “Patients are living longer than we expected, but we don’t yet have all the data,” Dr. Fetzer says.

He predicted that another compound, CRLX301, will be released to the clinic in 2014. Preclinical work shows complete tumor eradication within two weeks of dosing and 100% survival, he claimed.

Immune Pharmaceuticals is developing nanoMAbs. “We conjugate nanoparticles, rather than toxic payloads, to antibodies. We deliver two drugs in one nanoparticle, enabling direct delivery and minimizing side effects,” said Daniel Teper, Pharm.D., CEO.

The company’s lead compound, however, is bertilimumab for ulcerative colitis (in Phase II), for Crohn’s disease and severe asthma (in Phase I), and for ophthalmology and bullous penphigoid diseases (in preclinical stages). “Physicians need new alternatives to treat disease earlier, and more tightly targeted drugs. Bertilimumab, a fully human mAb targeting eotaxin-1, has a direct correlation with severity of disease, because eotaxin modulates the disease and also is a biomarker,” he said.

Gut Microbiome

Second Genome is intent on cracking the code of the gut microbiome, which plays a role in metabolic and autoimmune processes. “There’s a large amount of bacterial diversity, but much of it is functionally conserved,” said Peter DiLaura, CEO. “The link between the microbiome and disease is strongest in inflammatory and metabolic diseases, and in hospital-acquired infections.”

The company is developing microbiome modulators to promote the growth of beneficial bacteria. “We’ve built a platform to look at microbial interactions and are conducting preclinical research on obesity and type 2 diabetes. Last year, we got blank stares when we discussed the gut microbiome. This year, we’re in back-to-back meetings,” he said. “Second Genome has collaborations with Merck and Danisco, and an early-stage pipeline is emerging.”

Elcelyx Therapeutics is using its gut sensory modulators (GSMs) to target diabetes and obesity. This first-in-class therapeutic platform facilitates glucose regulation and increases the sense of satiety, said Alain Baron, M.D., president and CEO. These GSMs activate enteroendocrine cells in the lining of the lower intestine to increase the release of the hormone GLP-1 that regulates glucose, Dr. Baron explains.

Lovida™, an over-the-counter weight loss product will likely be released first, followed by NewMet™, a reformulation of metformin, for patients with type 2 diabetes. NewMet makes it possible to lower the dose about 75% without reducing efficacy.

“Metformin has no known mechanism of action, but we believe we’ve cracked it, making the molecule safer and more convenient,” Dr. Baron said. Phase II studies are near completion and Phase IIb studies will begin in March. “So far, we maintain the full effect of metformin without side effects, and offer once-per-day oral dosage. It can be combined with DPP inhibitors for single-dose administration.”


Amicus Therapeutics uses pharmaceutical chaperones and oral monotherapy to stabilize patients’ own natural enzymes to combat rare and orphan diseases, “especially lysomal conditions,” said John F. Crowley, chairman and CEO. That approach enables co-administration, thus increasing tissue uptake and reducing the immunogenicity of enzyme replacement therapy.

“We have Phase II data in Fabry and Pompe diseases, and are in late preclinical development targeting Parkinson’s disease. We are entering the biologics space, partnering with GlaxoSmithKline, Laureate Biopharma, and JCR Pharmaceuticals.”

In the case of Fabry disease, “We have more than 220 patient-years of data with no adverse events, and 57 of 59 patients in a 12-month trial wanted to continue to use migalastat HCl monotherapy as their only therapy for Fabry.” Crowley says Amicus characterized and built constructs for each of the more than 500 known mutations that lead to Fabry’s, “so we can predict with nearly 100 percent certainty who will respond to this medicine.”

Antifungals and Antibacterials

NovaDigm Therapeutics plans to begin Phase II studies targeting fungal Candida infections by mid-2013. “In Phase I trials, we’ve seen very rapid antibody responses to our antigen (NDV-3), because almost everyone has been pre-exposed to Candida,” said M. Timothy Cooke, Ph.D., CEO. “When we give a single dose, it looks like a booster dose.”

Typically, the response is evident at day 7 and peaks at day 14. NVD-3 also appears to protect against S. aureus, he noted. The HYR1 antigen may be combined with NDV-3 for a bivalent vaccine, he added.

“For the five percent of women with recurrent vaginal yeast infections, the only other option is the chronic use of antifungals. We intend to vaccinate women and reduce the number of recurrences, their severity, or both,” Dr. Cooke says.

Hospital-acquired infections offer another large target. “Candida is the third most common hospital-acquired infection, behind pseudomonas and S. aureus, and may become invasive,” Dr. Cooke points out. He said NovaDigm envisions labeling by type of medical intervention—high risk surgery or catheter use, for example.

Cubist Pharmaceuticals is developing therapeutics for the acute care environment. “We’re channel-focused, not therapy-based. Therefore, we have a different business model from most biotechs that embraces multiple therapeutic areas,” Michael W. Bonney, CEO, said. Therapeutically, Cubist focuses on antibacterials and, recently, nonopiate pain management.

Bonney said the company is profitable and revenue-generating. His goal is to double revenue by the end of 2017. To do that, “Cubist is expanding its acute care focus, developing a compelling pipeline of well-differentiated late-stage compounds, and building a European presence.”

Four new late-stage trials are expected to be in the clinic by 2017, taking advantage of breaking trends including antibiotic-resistant infections, outcomes management, and financial pressures. Cubist reported 23% growth in 2012.

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