Presented by Biovays

Bayhill Therapeutics announced today that it entered into a license agreement with Genentech worth $325M, including potential development, regulatory and sales milestones, and royalties. Bayhill gets $25M of that upfront.

Bayhill just announced positive data at ADA (American Diabetes Association) from its lead drug candidate, BHT-3021, in treating patients with Type 1 diabetes.

BHT-3021 is an immuno-based therapy using a plasmid to encode proinsulin. The plasmid DNA tolerizes the immune system to proinsulin, thereby turning off the self directed immune attack which leads to glucose dysregulation, and diabetes.

The recent data at ADA was interim results from a randomized, blinded, placebo-controlled, dose escalation trial in type 1 diabetic patients. Here’s what excited Genentech:

• Patients were randomized 2:1 to receive weekly intra-muscular injection of BHT-3021 (0.3 mg, 1 mg, 3 mg or 6 mg) or placebo for 12 weeks;

• Measured outcome was pancreatic function as assessed by C-peptide levels (C-peptide is a peptide that is made when proinsulin is split into insulin and C-peptide. A high, or normal, C-peptide level indicates that insulin is being produced because of proinsulin cleavage);

• Data from the 1 mg dose cohort was available out to 12 months, and data from the 0.3 mg, 3 mg, and 6 mg dose groups were available out to six months;

• C-peptide levels were maintained at six months after randomization in patients treated with 1 mg (mean increase of 2 pmol/L from baseline) and with 3 mg of BHT-3021 (mean increase of 34 pmol/L from baseline).

• In contrast, C-peptide levels declined in placebo treated patients (mean decrease of -127 pmol/L from baseline).

• Corresponding decline in mean HbA1c levels of 0.13% Hb with 1 mg and of 0.44% Hb with 3 mg of BHT-3021 fifteen weeks after randomization.

• Patients on placebo had no “substantial change” in HbA1c levels at 15 weeks.

• Drug safe and well-tolerated with no serious AEs

Here was Bayhill CEO Mark Schwartz’s thoughts:

“BHT-3021 represents a truly novel approach to treating type 1 diabetes. We believe this collaboration with Genentech reflects the significant potential of our approach to antigen specific tolerance, and will allow us to accelerate and expand the development of BHT-3021, while continuing to develop additional products from our BHT-DNA platform.”

Assessment

The data looks positive and we like that the C-peptide AUC (the primary endpoint used by other sponsors and by FDA) was the primary outcome measure. The maintenance of C-peptide is encouraging (it even increased some), however we’d like to see more C-peptide AUC data, as well as some glucose and insulin parameters. The decrease in HbA1c is also encouraging.

The drug appears safe and having multi-dose safety data is always crucial.

Strong watch, although the fact that the technology is plasmid/DNA based warrants caution.

Here’s today’s press release.

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