Based: Redwood City, CA
CEO: Charles (Chuck) Bramlage
The Scoop: Pearl Therapeutics delivered compelling results from a Phase IIb trial of its lead therapy PT003, for chronic obstructive pulmonary disease (COPD) in May, showing the inhaled LAMA/LABA combo therapy beat existing products Foradil and Spiriva in a key measurement of efficacy. The data helped propel the feisty upstart in the race with Big Pharma outfits to bring potential blockbuster treatments for the lung disorder to market. And the biotech sent a clear message to its competition that its sights are set on an eventual market debut for PT003 when it brought on Chuck Bramlage, who worked on multiple respiratory product launches for GlaxoSmithKline ($GSK), to be its CEO.
What Makes It Fierce: Through last year, Pearl had proven that it could rally investors to support its development of promising formulation technology taken from Nektar Therapeutics ($NKTR) for the respiratory market; its total venture haul reached $102.5 million after raising $69 million in a Series C financing last October. Now the firm can back up its success on the financing front with exciting safety and efficacy data on its top drug.
The company's formulation technology enables multiple drugs to be delivered in a single inhaled product. With PT003, the company uses its technology--which includes porous particles that are designed to efficiently carry drugs to the lungs--to pack glycopyrrolate, a long-acting muscarinic antagonist (LAMA), and formoterol fumarate, a long-acting beta-2 agonist (LABA) into one product. The latter drug ingredient is also used in an inhaled treatment for COPD marketed as Foradil.
Yet Pearl's story is about much more than a platform technology.
"It's not the formulation technology that sells, right, it's that we've got the drugs to go with it," Bramlage said. "The formulation [technology] got us here, but the real win is that fact that you're able to put these drugs in a combined product and give it to patients, and they take one product, and they get a lot better."
In its Phase IIb trial results presented at the annual American Thoracic Society meeting in May, the company revealed that PT003 bested comparator therapies Foradil and Spiriva in a lung function measure (called FEV1 AUC0-12) after one week of treatment as a primary endpoint. On this score, Pearl's drug provided a 47% improvement over Foradil and 49% over Spiriva.
To build on that data and lay the groundwork for a planned Phase III next year, the company announced the initiation of four additional Phase II trials for its PT003 program in June. The firm has already wrapped up one of those four studies, providing more safety and efficacy data on the drug and helping the firm zero in on a dose to test in Phase III. An important goal of the mid-stage studies is to find an optimal dose for PT003, which Pearl is developing to be taken twice a day, Bramlage said. The CEO also shared some early good news from the studies. Swift enrollment in them has put the firm about 6 months ahead of the previously scheduled completion date of June 2012, and the expected completion time is now in late December.
The sooner the data are available the better as Pearl races GSK, which is in late-stage development of a LAMA/LABA product for COPD, and other experimental products to market. The combo products are expected to be an improvement for patients with the lung disease, which actually describes multiple ailments including emphysema, chronic bronchitis and some asthma cases.
"We would love to be first [to market] and we're working as fast as we can on these trials to get there," Bramlage said. There's an estimated $2 billion to $4 billion annual market for combo treatments like PT003. If Pearl doesn't cross the finish line first, he added, "I see value if we're third or forth. It's just going to be a smaller share [of the market]."
The data are also relevant to Pearl's hunt for one or more partners to aid in commercializing the product. Bramlage is open to striking separate deals that would cover U.S. rights to PT003 and territories in the rest of the world, but his preference is to garner one global pact that would give his firm the right to co-promote the treatment in the U.S. After all, the CEO said he wants to build a sales unit at Pearl like he did earlier in his career at GSK, where he played key roles in the commercial releases of respiratory drugs such as Advair and Flovent. Bramlage said he also takes Advair for his asthma.
The company has come a long way since former Nektar employees and biotech vets Sarvajna Dwivedi, Pearl's executive VP of R&D, and Adrian Smith formed the start-up to attack respiratory diseases with Nektar's drug-delivery technology about 5 years ago. Now we'll see how the firm fares in its pursuit of a commercial partner for PT003 and late-stage development of the treatment.
Venture backers: 5AM Ventures, Clarus Ventures, New Leaf Venture Partners and Vatera Healthcare Partners.