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Pfizer has spent $800 million to build the leading production platform for gene therapies, the company said on Tuesday in its second day of talks to show how it plans to become a fast-growing innovator. Within three years, the drug giant hopes to launch three potential cures for hemophilia and muscular dystrophyworth as much as $4 billion in annual revenue.

We are in an unrivaled position to go to market, said the head of Pfizers rare-disease business, Suneet Varma, in his Tuesday morning presentation. In another Tuesday talk, the companys immunology leaders highlighted coming breakthroughs for the tens of millions who suffer miserable autoimmune skin disorders.

Pfizer (ticker: PFE) is eager to recast itself as a high-multiple growth stock. By year-end, its slower-selling products will be spun off into a business run by Mylan (MYL). From that point, Pfizer thinks it can boost revenue by 6% a year, despite the patent expirations of nearly $20 billion worth of its products in the latter part of this decade. The expected growth will be fired up by 25 product launches, which Pfizer is explaining in two days of online talks.

After rising Monday, Pfizer stock was down 0.3%, at $36.91, in recent trading. The S&P 500 was up 0.4%.

A rare disease may affect only a few hundred thousand people, but there are a lot of these diseases. Cumulatively, they add up to 400 million people worldwide, said Varma, with fewer than 5% having approved treatments. Pfizer believes the rare-disease market will grow at a double-digit annual rate.

Clinical trials are under way for treatments for the bleeding disorders known as hemophilia A and hemophilia B. After treatment, none of the enrolled patients have had bleeding problems, including some in which more than a year has passed since dosing.

Pfizer hopes for approval and launch of its hemophilia B treatment in 2022. Peak annual sales could hit $500 million to $1 billion, says the company. The hemophilia A launch could happen in 2023 and eventually exceed $1 billion in annual sales.

The gene-therapy treatments can require a short hospital stay. But the possibility of a cure will inspire 30% to 40% of hemophilia patients to seek treatment, Pfizer predicts.

A rival in hemophilia A gene therapy, BioMarin Pharmaceutical (BMRN), had a head start over Pfizer in clinical trials. But last month, the U.S. Food and Drug Administration rebuffed BioMarins application for approval and demanded additional follow-up of its patients for a period that will extend through the end of 2021.

The FDA hasnt asked Pfizer to change its design for the hemophilia A trial that the company will begin in a few weeks, said the chief medical officer of the rare-disease unit, Brenda Cooperstone,

There were some worrisome immune reactions among boys in the continuing clinical trial for Pfizers gene therapy for Duchenne muscular dystrophy. But after adjustments in the trials preparatory regime, Pfizer says there have been no more of those events. The treatment showed promising efficacy in early phase trials.

The pivotal Phase 3 muscular dystrophy trial will begin within weeks, with the first data expected in 2022. Pfizer hopes for a launch in 2023 of a muscular dystrophy therapy that would be used by 30,000 people a year in the U.S. and Europe, generating over $2 billion in annual revenue. to reach that goal, Pfizer is racing with Sarepta Therapeutics (SRPT).

In addition to its rare-disease treatments, Pfizer plans to launch four products for autoimmune disorders by 2025. Two of the products would treat atopic dermatitis, which causes painful itching in up to 30 million Americans. Only a portion of those patients get any treatment today.

Based on successful clinical trials, sufferers of the skin disease can look forward to breakthrough treatments, similar to those launched in recent years for psoriasis, says Pfizers inflammation and immunology business head, Richard Blackburn.

Corrections & Amplifications

Pfizers gene-therapy treatments can require short hospital stays. An earlier version of this article incorrectly said the treatments are like bone-marrow transplants, which require months of grueling hospital care.

Write to Bill Alpert at william.alpert@barrons.com

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How Pfizer Plans to Lead the Industry in Gene Therapies - Barron's

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