Thursday, Benitec Biopharma Inc ( BNTC ) released interim clinical data from the 90-day timepoint following the administration of BB-301 to the study’s first subject (Subject 1) treated in the BB-301 Phase 1b/2a study in Oculopharyngeal Muscular Dystrophy (OPMD).

“To date, no clinical studies have systematically demonstrated a clinical improvement in OPMD patients across both objective and subjective measures of swallowing. We are, therefore, pleased to report positive interim clinical data from multiple radiographic measures as well as subject-reported outcome measures from the first subject treated with BB-301,” said Jerel Banks, Executive Chairman and Chief Executive Officer of Benitec. 

During the OPMD Natural History Study, which represents the pre-dose observational period for each subject, Subject 1 experienced progressive worsening of dysphagia (difficulty swallowing) as demonstrated by the results of the videofluoroscopic swallowing studies (VFSS), the cold water timed drinking test.

At the 90-day timepoint following the administration of BB-301, Subject 1 demonstrated improvements in key videofluoroscopic assessments.

The results of many assessments completed at the 90-day timepoint demonstrated improvements over the initial measurements assessed at the subject’s first visit for the natural history observational study, which occurred more than 12 months before the 90-day assessment.

The most significant VFSS improvements at Day 90 were observed for swallowing tasks centered on evaluating pharyngeal constrictor muscle function and swallowing efficiency in consuming thin liquids, solid foods, and thick, non-solid foods. 

So far, no severe side effects have been noted in the safety profile of BB-301. Two subjects experienced temporary Grade 2 (mild) Gastroesophageal Reflux Disease (GERD), which was treated with standard prescription medication.

OPMD is a rare progressive muscle-wasting disease caused by a poly(A)-binding protein nuclear 1 gene mutation. The disease is characterized by swallowing difficulties (dysphagia), limb weakness, and eyelid drooping (ptosis). Dysphagia worsens over time.

Concurrently, Benitec Biopharma ( BNTC ) announced a $40 million private investment in public equity (PIPE) financing from the sale of 5.7 million shares at $4.80/share and in lieu of shares of common stock, pre-funded warrants to purchase up to an aggregate of 2.58 million shares at a price per pre-funded warrant of $4.7999, to certain institutional accredited investors.

Price Action: BNTC shares are up 24% at $5.95 on the last check Thursday.

Illustration of Phrama lab worker created with MidJourney.