Regeneron’s Rare Disease Drug Shows Zero New Abnormal Bone Growth In Extended Trial – REGN Stock Heads For Best Day In Over A Month

• The Phase 3 Optima trial enrolled 63 adults who received Pasatru or a placebo every four weeks.
• A higher dose recorded one disease flare-up, compared with 17 in the placebo group.
• The findings were presented at the American Society for Bone and Mineral Research.

Regeneron Pharmaceuticals (REGN) reported encouraging long-term results for its recently approved rare disease drug on Monday, with patients showing no new abnormal bone growth during an extended Phase 3 trial.

REGN shares were up about 2.8% and heading for their best day in over a month.

What Did The Trial Show?

Regeneron’s Pasatru treats fibrodysplasia ossificans progressiva (FOP), an extremely rare condition in which muscles, tendons and other soft tissues gradually turn into bone.

The Phase 3 Optima trial enrolled 63 adults who received Pasatru or a placebo every four weeks. Between weeks 56 and 84, patients taking Pasatru developed zero new abnormal bone lesions, compared with nine in the placebo group.

The findings were presented at the American Society for Bone and Mineral Research (ASBMR).

Regeneron Plans New Study For Children

The FDA approved Pasatru in August, following earlier trial results showing at least a 90% reduction in new abnormal bone lesions after 56 weeks. European regulators are currently reviewing Pasatru.

The drug works by blocking Activin A, a protein involved in abnormal bone formation. Regeneron plans to begin another Phase 3 study, Optima 2, involving children and adolescents later this year.

REGN’s Pipeline Includes Cancer, Eye Disease Treatments

Beyond Pasatru, Regeneron is expanding its broader drug pipeline, which includes treatments for cancer, eye diseases and inflammatory conditions.

The biotech firm recently expanded its partnership with Sanofi (SNY) to develop four new long-acting antibody treatments for inflammatory diseases. Under the deal, Regeneron will receive $1 billion upfront and could earn an additional $7 billion if the treatments meet certain development, regulatory, and sales targets. The companies will share development costs and split future profits equally.

The company reported about $17.8 billion in cash and marketable securities as of June 30.

Separately, BofA raised its price target on Regeneron to $908 from $891 and kept a ‘Buy’ rating.    This implies a 19% upside from current levels.

Retail Remains Bullish On REGN

Despite the recent run, retail sentiment on Stocktwits surrounding REGN remained ‘bullish’ over the past 24 hours.

The stock is down around 1.4% so far this year, underperforming the broader State Street SPDR S&P Biotech ETF’s (XBI) roughly 25% gains.

Also read: Microsoft Looks In-House To Power Its AI Ambitions – CEO Nadella Calls It ‘Our Biggest Bet Yet On Our Own Silicon’

For updates and corrections, email newsroom[at]stocktwits[dot]com.