Roche nears $5-billion deal to acquire Spark Therapeutics: report

UPDATE: Roche entered an agreement to buy gene therapy developer Spark Therapeutics for $114.50 per share in cash, or approximately $4.3 billion, the companies announced Monday.

The Wall Street Journal, citing people familiar with the matter, had recently reported that Roche was close to reaching an agreement to buy Spark for nearly $5 billion, as the Swiss drugmaker seeks to bolster its haemophilia portfolio. Some of the sources noted that as of February 22, there was at least one other undisclosed bidder for Spark, which generated $64.7 million in revenue last year and a net loss of $78.8 million, had a market capitalisation of less than $2 billion on February 22.

Spark partnered with Pfizer in 2014 to develop fidanacogene elaparvovec, formerly known as SPK-9001 and as SPK-FIX, a programme incorporating a bio-engineered adeno-associated virus (AAV) vector for the potential treatment of haemophilia B. Phase I/II results unveiled last year showed that patients with severe or moderately severe haemophilia B who received the investigational gene therapy had discontinued routine factor IX concentrate infusions and experienced no serious adverse reactions. The companies initiated a Phase III study of fidanacogene elaparvovec last July. Spark is also developing SPK-8011 for haemophilia A.

Meanwhile, analysts expect Roche's Hemlibra (emicizumab-kxwh) will generate billions of dollars in annual sales. The therapy initially gained FDA approval in 2017 for patients with haemophilia A who have developed factor VIII inhibitors, and was later expanded to include those without factor VIII inhibitors. Roche recently described Hemlibra as one of its key growth drivers, with sales of 111 million Swiss francs ($112 million) reported in last year's fourth quarter.

In 2017, the FDA approved Spark's gene therapy Luxturna (voretigene neparvovec-rzyl) to treat biallelic RPE65 mutation-associated retinal dystrophy. Spark has indicated plans to sell Luxturna in the US at a cost of $425 000 per eye, or $850 000 in total, raising questions about cost-effectiveness, but the company wants to offer partial refunds if patients do not meet recovery targets.

Other companies developing gene therapies include BioMarin Pharmaceutical, which is developing valoctocogene roxaparvovec, formerly known as BMN 270, for haemophilia A. Meanwhile, uniQure recently presented updated results from its mid-stage trial of the experimental AAV5-based gene therapy AMT-061 for haemophilia B, saying that the first three patients treated had a mean factor IX activity of 38 percent of normal at 12 weeks (for related analysis, see ViewPoints: uniQure finds plenty of space alongside Pfizer's heme B gene therapy).

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