Bio space article re:Benitec and PSS

https://www.biospace.com/News/benit...ership-with-patrick/464958/source=TopBreaking

August 2, 2017
By Alex Keown, BioSpace.com Breaking News Staff

SYDNEY, Australia – For the past two years, tiny Benitec Biopharma (BLT.AX) has undergone a transformation with a new board of directors, new executives and a renewed prioritization on its RNA interference treatment pipeline and strategic partnerships.



Now the company, which has a 20-person research facility in the Bay Area, has its eyes on taking four programs into the clinic over the next year or so. The company has a focus on four main target areas, oncology, orphan diseases, hepatitis B and ophthalmology. Benitec is banking on its investigational programs that use its patented gene-silencing technology called ddRNAi or expressed RNAi.

“What we’ve done over the past two years is validate and prioritize our pipeline and have set a path for strong shareholder value… I think people have seen that what we say we’re going to do, we do,” Greg West, chief executive officer of Benitec, said in an exclusive telephone interview from his Australia office.

The company’s lead product is BB-401, a recombinant DNA construct that produces an antisense RNA with specificity against Epidermal Growth Factor Receptor (EGFR). The investigational therapy, licensed from NantWorks, is expected to enter Phase II trials for patients with head and neck cancer squamous cell carcinoma (HNSCC). West said BB-401’s target is overexpressed in up to 90 percent of all HNSCC cases. West expects that BB-401 will advance into Phase II trials in early 2018. In addition to BB-401, the company is developing a follow-on compound using ddRNAi technology called BB-501. The in-house developed compound will be designed to silence EGFR, West said. Early stage iterations of this candidate are being tested in preclinical, he said.

[img src="http://enewsletters.biospace.com/images/news/biospace-news-400x450-benitec-david-suhy.jpg" >

David Suhy, Benitec’s chief scientific officer, said unlike many investigational gene-editing technologies, Benitec’s BB-401 platform is not designed as a “one-and-done” therapy. He said patients will receive four treatments over the course of four weeks. Because cellular structure in tumors can vary, Suhy said the additional treatments are to ensure the tumor is thoroughly targeted and eliminated by the gene-silencing program.

“We want to make sure we’re hitting all the cells we want to,” Suhy said.

The company is also developing a treatment for oculopharyngeal muscular dystrophy (OPMD), a rare progressive muscle-wasting disease caused by mutation in the poly (A)-binding protein nuclear 1 (PABPN1) gene. Suhy said the company is using what it calls a “silence and replace” approach of the mutant PABPN1 into a single vector.

“This is different than anything else being done in gene therapy,” Suhy said. “With CRISPR, you can silence a gene, but the replacement isn’t there for that one vector.”

BB-301 has received orphan drug designation in Europe and the company intends to seek the same designation in the United States.

Additionally, the company is also developing therapies for wet AMD (Age Related Macular Degeneration) and hepatitis B. The wet AMD program is still in preclinical development, but Suhy said it’s possible the company could be ready for that program to enter the clinic by the end of 2018.

“We expect a data readout later this year and we think if the data is positive, it will not only validate the genetic target for wet AMD, but also validate the delivery mechanism as a whole,” Suhy said.

For hepatitis B, the company is harnessing the remnants of a hepatitis C program Benitec shuttered last year due to the intense competition in the area, as well as a shrinking patient population. Suhy said company researchers changed the payload to attack HBV.

While the company readies its pipeline for the clinic, Benitec leadership has been busy forging relationships with multiple partners. One of the company’s key partners is with NantVentures, the private investment arm of biotech entrepreneur Dr. Patrick Soon-Shiong. Under the 2016 investment agreement with NantVentures, Soon-Shiong owns 28 percent of Benitec, West said. It was through the relationship with NantWorks that Benitec gained access to its lead oncology program, BB-401, he added.

Soon-Shiong’s involvement with Benitec has been fairly low-key. Greg attributed that to Jerel Banks, one of Soon-Shiong’s chief lieutenants and the chief investment officer at Nant Capital as having a more visible relationship with the company, rather than the world’s richest doctor.

“Dr. Soon-Shiong has been a big proponent of setting up companies. He didn’t have a genetic-focused company, which is how he hooked up with Benitec,” West said.
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Suhy said Soon-Shiong sees potential with combining BB401 with NantWorks’ GPS Cancer, a molecular profile analysis, which integrates whole genome sequencing, whole transcriptome (RNA) sequencing and quantitative proteomics through mass spectrometry.

Benitec is listed on both the Nasdaq and the Australian Stock exchanges. Shares of Benitec hit a one-year high of $4.10 on April 4, but fell to a low of $1.85 in June.

As Benitec races toward the clinic, West said the company will remain focused on developing its products and believes the next several months will be a time when the company flourishes and becomes more well-known.

“Over the past few years, people are seeing that we do what we say we can do,” West said.