R&D expenses increase as Ophthotech advances Fovista clinical trials

Burgeoning patient enrollment for clinical trials of Fovista, Ophthotech’s leading product candidate, largely shaped the company’s balance sheet for the first quarter of 2015.

Perspective from Andre Witkin, MD

During a company-sponsored conference call to report first quarter financial results and ongoing enrollment in the trials, CEO David R. Guyer, MD, discussed efforts to advance Fovista, an anti-PDGF agent, in the treatment of wet age-related macular degeneration.

Company resources are also being dedicated to Zimura, an anti-complement agent, in the treatment of wet AMD.

“The first quarter of this year has resulted in solid progress for Ophthotech,” Guyer said. “We are excited with the progress of the Fovista phase 3 pivotal program and reaching our first major recruitment milestone.”

Michael G. Atieh, executive vice president and chief financial and business officer, reported that in the first quarter of 2015, research and development expenses totaled $24.6 million, up from $14.4 million in the first quarter of 2014.

“Over half of the increase in R&D expense in the quarter relates to our Fovista phase 3 clinical program, where enrollment continues to increase, and the Fovista expansion studies,” Atieh said. “Costs related to Fovista include clinical trial costs and costs to manufacture Fovista for the clinical studies. Increased compensation expenses including share-based comp associated with additional research and development staffing, along with an increase in manufacturing spending related to the Zimura clinical program, accounted for the remaining changes in expenses. … As we continue into 2015, our R&D expenses will increase significantly and may fluctuate from quarter to quarter. Manufacturing-related expenses will also increase as we prepare for the start of the Zimura dry AMD study.”

Enrollment for the first phase 3 pivotal trial of Fovista used in combination with Lucentis (ranibizumab, Genentech) is complete. Enrollment for a second phase 3 trial of Fovista in combination with Lucentis is expected to be completed by the end of the third quarter. A third phase 3 trial of Fovista in combination with Eylea (aflibercept, Regeneron) or Avastin (bevacizumab, Genentech) is currently recruiting patients, Guyer said.

Enrollment for the three phase 3 trials is expected to total about 1,866 patients, he said.

A phase 2/3 clinical trial of Zimura in combination with anti-VEGF therapy for patients with polypoidal choroidal vasculopathy, a variant of wet AMD, is planned for launch later this year.

The company is also conducting three expansion studies for Fovista. The first expansion study is designed to investigate the potential of Fovista in combination with anti-VEGF therapy to reduce subretinal fibrosis in patients with wet AMD. The second expansion study is designed to look at the potential of Fovista to reduce the treatment burden associated with anti-VEGF monotherapy in patients with wet AMD. The third expansion study is designed to evaluate the benefits of initiating Fovista combination therapy in patients who respond poorly to anti-VEGF monotherapy, president Samir Patel, MD, said.

Six-month data presented at the Association for Research in Vision and Ophthalmology meeting showed that 10 of 27 patients who underwent pretreatment with Fovista gained an average of 16.5 letters of visual acuity, while those who were not pretreated gained 4.41 letters, Patel said.

“We believe these findings are consistent with our trial rationale for the planned treatment resistance study employing Fovista combination therapy,” he said.

In other financial news, the company’s net income for the first quarter totaled $7.6 million, compared with a net loss of $20.7 million in the first quarter of 2014, Atieh said.

The increase in net income was partly attributed to $41.7 million in collaboration revenue related to a second $50 million enrollment milestone received from Novartis Pharma. A $30 million enrollment-based milestone from Novartis is expected to be earned upon completion of the final phase 3 trial for Fovista, Atieh said. The enrollment-based milestones are being paid under a licensing and commercialization agreement that Novartis and Ophthotech entered in May 2014. – by Matt Hasson

Perspective

Andre Witkin, MD

Ophthotech has developed an aptamer called Fovista, which targets platelet-derived growth factor (PDGF). PDGF plays a role in supporting blood vessel pericytes in choroidal neovascular (CNV) membranes. Anti-VEGF agents reduce CNV permeability and growth; however, the addition of an anti-PDGF agent may stimulate regression of CNV. In the phase 2 trials, eyes with neovascular age-related macular degeneration that received Fovista in combination with Lucentis (ranibizumab, Genentech) achieved better visual acuity results than with Lucentis alone. Despite the exclusion of a significant number of patients based upon strict enrollment criteria, the combination of these two medications remains the first approach that has demonstrated better visual acuity results than anti-VEGF medication alone. With the upcoming completion of the phase 3 trials using Fovista in combination with anti-VEGF therapy, we hope to better understand the amount of visual acuity benefit achievable with this new class of medication and may be able to determine which patients might gain the most benefit using this combination therapy approach.

Andre Witkin, MD

Assistant professor, Tufts Medical Center,

Boston

Disclosures: Witkin reports no relevant financial disclosures.