Business Corpus: Why Hemophilia Patients Could Pay $1.5 Million For Gene TherapyBioMarin, Spark and UniQure are duking it out for share in hemophilia gene therapies — a market that could generate north of $1.5 million per patient. The post Why Hemophilia Patients Could Pay $1.5 Million For Gene Therapy appeared first on Investor's Business Daily.

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BioMarin Pharmaceuticals (BMRN), Spark Therapeutics (ONCE) and UniQure (QURE) are racing to be the first to market with a gene therapy that treats hemophilia — a prospect that could offer revenue north of $1.5 million per patient, an analyst said Monday.

Leerink analyst Joseph Schwartz boosted his projections for hemophilia gene therapies to $1.5 million from earlier assumptions for $350,000-$500,000 per patient. Some treatments could go as high as $2 million per patient on a gross basis, he said.

Schwartz's analysis is based on pricing for Luxturna, a gene therapy from Spark that treats an ultrarare blindness disorder. Luxturna goes for $425,000 per eye. This "sets a useful precedent" for pricing other gene-based treatments, such as hemophilia gene therapies, he says.

"We believe the manufacturers are testing the waters currently as they embark on Phase 3 trials in order to determine where payers push back significantly," he said in a note to clients. "Based on our checks, a price somewhere in the range of $1 million to $2 million per patient seems most likely."

Luxturna Pricing Precedent

The challenge with pricing gene therapies is that these treatments are being billed as one-time cures, which could warrant a higher price. But most analysts doubt that will actually be the case. Further, they expect payers to push back on high prices without data to back up the therapies' durability.

An agreement with Harvard Pilgrim Health Care to cover Luxturna is based on 30 months of durability data. That sets an important precedent, Schwartz said. By the time they come to market, the slew of hemophilia gene therapies currently under investigation will have 30-50 months of durability data.

Schwartz expects BioMarin to lead Spark in treating hemophilia A. Hemophilia A is a genetic deficiency in a key blood-clotting protein known as Factor VIII. BioMarin is in Phase 3 testing of a gene therapy called valoctocogene roxaparvovec, or Valrox. Spark's therapy, SPK-8011, is in Phase 1 and Phase 2 studies.

"We believe an effective gene therapy for hemophilia could garner at least 2.5 times the annual cost of Factor (VIII) replacement therapy based on durable (effectiveness) data," Schwartz said.

Hemophilia Gene Therapies

BioMarin could enter the hemophilia A market in 2022 and hit peak sales of $4.5 billion in 2030, he said. Spark will likely enter the market a year later with SPK-8011. Schwartz calls for SPK-8011 to garner $4 billion in peak sales in 2030.

Spark also is working on a hemophilia B gene therapy called SPK-9001, under a partnership with Dow Jones stock Pfizer (PFE). There, it trails UniQure's therapy called AMT-061. AMT-061 is expected to begin Phase 3 studies in the third quarter. SPK-9001 is in Phase 1 and Phase 2 studies.

Schwartz models peak sales of SPK-9001 at $1 billion in 2027 with Spark receiving $134 million in peak royalties. He boosted his price target on Spark to 74 from 55, and kept his market perform rating. He also raised his price target on BioMarin to 145 from 132, and reiterated his outperform rating.

In midday trading on the stock market today, UniQure popped 7.4%, near 31.10. Spark advanced 3.9%, near 73.40. BioMarin lifted a fraction, near 85.80.

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