at press time
Genaera's AMD Treatment Creates a Stir
Company Gets Lots of Attention from Early Results.

Anyone even remotely familiar with the pharmaceutical industry will easily recognize the names Genentech, Pfizer, Novartis and Alcon. But until recently, hardly anyone could tell you much about a company named Genaera.

Those who did know about Genaera could tell you that the drug development company, based in Plymouth Meeting, Pa., began in 1987 as Magainin Pharmaceuticals and has gone through more than $180 million in the past 16 years without one FDA approval to show for its efforts.

Initially, the company focused its research on a class of microbial peptides called magainins, which had shown an ability to defend against invading bacteria. More recently, the company has been best known for its research efforts with squalamine, a patented, synthesized aminosterol with antiangiogenic properties, originally found in its natural state in the livers of certain types of sharks.

Because antiangiogenic agents inhibit the growth of new blood vessels -- blood vessels that could nourish tumors -- Magainin, and then Genaera, essentially saw squalamine as a potential treatment for many types of cancers. The company conducted several Phase I/II clinical trials employing squalamine as a cancer-fighting agent.

And though Genaera's chief operating officer Kenneth Holroyd, M.D., says that the company is still pursuing squalamine as a treatment for prostate and lung cancer, Genaera is now primarily focusing on squalamine as a potential treatment for wet AMD. This puts little Genaera squarely in a race against the deep pockets of Genentech/Novartis, Alcon and Pfizer/Eyetech to develop a new and effective next-generation drug to combat wet AMD.

In the trial, 40 patients were given four weekly doses of squalamine, administered intravenously, with no further maintenance therapy. The 4-month results reported were highly positive, with all patients having improved or preserved vision and 10 patients improving by 3 or more lines on an EDTRS chart. Patients' angiograms are being sent to reading centers for a confirmatory study.

"These data are very preliminary. They need to do a lot more," says Jerry Chader, Ph.D., M.D., chief scientific officer for The Foundation Fighting Blindness. "But the data look good."

"We are planning to begin a Phase II controlled trial in the second quarter of 2004," says Dr. Holroyd. "We'd like to have a partner, but we can do the Phase II study alone."

Genaera has emphasized that squalamine is delivered by patient-friendly intravenous injection, which can treat both eyes at once, while other wet AMD treatments under development are administered directly into or adjacent to the eyeball.

Though experts such as William Li, M.D., president and medical director of The Angiogenesis Foundation, point out that intravenous squalamine has been well-tolerated in cancer trials, questions have been raised by some researchers about intravenous delivery of an antiangiogenic drug for an eye disease. Could it cause more undesirable systemic side effects than an antiangiogenic agent delivered directly to the eye, especially in an elderly patient population?

"These questions are under investigation," explained Dr. Peyman. "However, if a drug is given systemically for a limited time and is effective, it would be desirable and acceptable to the patients."

"This (systemic side effects) is a concern for all of the compounds, not just ours," says Dr. Holroyd. "This question will be answered over time, with additional trials."

Skeptics have pointed to the fact that the Biotechnology Value Fund (BVF) provided Genaera with several million dollars of new funding in May in return for about a 10.5% equity stake in the company, but sharply reduced its holdings of Genaera shortly after the positive trial results had been released.

While Magainin/Genaera's track record of the past 16 years invites skeptics, Dr. Holroyd is optimistic.

"We're not the only company that's been in this type of position," he notes. "Eyetech is a small company that got a partnership with Pfizer. Regeneron is another small biotech that just did a $500 million partnership deal with Aventis. It took Scios 21 years to get its first drug approved, and they just got bought out by Johnson & Johnson for $2.4 billion." 

AAO: End O.D. Surgery in VA Hospitals
Legislation Is Sought After Negotiations Fail.

The American Academy of Ophthalmology (AAO) is backing legislation that would prohibit optometrists from performing eye surgery in Veterans Administration (VA) hospitals.

An Academy-backed bill, the Veterans Eye Treatment Safety (VETS) Act, was introduced in November by Rep. John Sullivan (R-Okla.) after the VA refused to revoke the surgery privileges of an optometrist who had performed glaucoma- and diabetes-related laser surgery at a VA facility in Wichita, Kan.

In a statement, the AAO said it sought a legislative remedy "following extensive negotiations with VA officials that did not lead to patient safety assurances deemed necessary to ensure high quality care."

Rhonda Edens, public affairs officer for the Robert J. Dole VA Medical Center in Wichita, said Michelle Dent, O.D., had been licensed in Oklahoma to perform laser surgery for the treatment of glaucoma- and diabetes-related eye diseases. Oklahoma is the only state that allows optometrists to perform these types of laser surgeries.

Edens said Dr. Dent had performed a total of three such laser surgeries during her 3 years working at the VA facility and that all of the treatments were successful.

According to Edens, Dr. Dent voluntarily stopped performing these surgeries in March after she was told objections had been raised.

"Currently, Dr. Dent isn't doing laser surgery and we're waiting for a decision from VA headquarters in Washington, D.C.," Edens told Ophthalmology Management.

The Wichita VA facility has two ophthalmologists on staff. It's not known whether an ophthalmologist was present when Dr. Dent performed any or all of these laser procedures.

A recent AAO-funded survey on veterans' health care found that 95% of 1,000 veterans surveyed thought it was very important to have a licensed medical doctor specializing in eye care performing eye surgery, while 88.7% preferred to have an ophthalmologist, rather than an optometrist, perform eye surgery on them in a VA facility.

Allergan and Inspire Will Co-Promote Drug
Elestat Seeks Share of Growing Ocular Allergy Marketplace.

In a new agreement between two companies that have been partnering on drug development, Allergan and Inspire Pharmaceuticals will co-promote epinastine HCL ophthalmic solution 0.05% (Elestat). The topical antihistamine was recently approved by the FDA for the prevention of itching associated with allergic conjunctivitis.

In clinical trials, Elestat demonstrated rapid-onset action with mast-cell stabilizing and anti-inflammatory activity.

The drug was originally licensed from Boehringer-Ingelhein and developed by Allergan to serve a fast-growing ocular allergy market that accounted for about $325 million in U.S. sales in 2003.

Under the terms of the agreement, Inspire will make an upfront payment to Allergan, while Allergan will pay royalties to Inspire based on Elestat sales. The agreement also calls for Allergan to significantly reduce its existing royalty payments to Inspire for Restasis, the dry eye drug covered by an existing partnership agreement between the two companies.

Inspire will have the primary responsibility for sales, promotional and marketing activities related to Elestat, and will incur associated costs. Allergan and Inspire will collaborate on overall product strategy and management.

"With four new Allergan Eye Care products approved in the last 12 months, this partnership will allow Allergan to focus its sales and marketing organization on its key business drivers (Lumigan, Restasis and Acular LS) while maximizing the value of this recently approved technology with a high-quality company such as Inspire," said David Pyott, Allergan's chairman, president and CEO.

IN THE NEWS

New Pfizer unit. Following its acquisition of Pharmacia earlier this year, Pfizer Inc. recently launched Pfizer Ophthalmics, a new business group that combines the ophthalmic products of Pharmacia and Pfizer.

In addition, Pfizer is donating enough doses of its Zithromax antibiotic to treat 135 million people for trachoma, the leading cause of preventable blindness. Pfizer called the gift the largest donation of a patented drug in history. Pfizer has donated about 8 million doses of Zithromax to countries in Africa and Asia over the past 5 years.

Contact lens prescriptions. President Bush has signed the "Fairness to Contact Lens Consumers Act," which gives consumers automatic access to their contact lens prescriptions, allowing them to purchase their contact lenses wherever they choose.

The legislation goes into effect Feb. 4. It prohibits a prescribing eyecare provider from charging a fee to provide a copy of the prescription to the patient, and allows a contact lens provider to fill a prescription if the prescribing doctor fails to verify it within 8 business hours.

Patent dispute. Advanced Medical Optics (AMO) said it has filed a complaint in U.S. District Court charging that Alcon has infringed on two AMO patents that cover advanced fluidics technologies incorporated into AMO's Sovereign and Sovereign Compact phacoemulsification systems. The complaint contends that Alcon's Infiniti and Series 20000 Legacy systems infringe on the patents.

Focus Night & Day marks 2 years. After 2 years in the U.S. marketplace, Focus Night & Day 30-day-and-night silicone hydrogel contact lenses represent the fastest-growing major contact lens category in the United States. CIBA Vision, the maker of Focus Night & Day lenses, predicts that the silicone hydrogel category is likely to exceed 9% of U.S. industry sales in 2004.