SHANGHAI – In China's evolving regulatory and pricing environment, Sciclone Pharmaceuticals Inc., of Foster City, Calif., has managed to keep growing its bottom line with lead drug Zadaxin (thymalfasin), an imported drug manufactured in Italy for the treatment of hepatitis B, hepatitis C, certain cancers and as a vaccine adjuvant. In the first quarter, the firm raked in $33.6 million, growing 7 percent from the year prior and beating China's average pharma growth rate by 1 percent (estimated by Sciclone to be 6 percent).

It has not been easy. The company is facing strong headwinds for imported drugs in China. The provincial tenders have been getting much tougher, eating away at the preferential pricing that "originator" drugs, or off-patent imported drugs, once enjoyed.

The short-term solution is to go after volume selling and there the company has done well. Last quarter, Sciclone managed to increase market share by 17 percent and has 40 percent of the market by value. It is experimenting with fresh sales techniques like telemedicine and e-commerce initiatives in partnership with tier-1 distributor Sinopharm Group. It also managed to get Sinopharm to absorb half the price cut that arose from a recent tender in Zhejiang province – a price cut that other provinces are likely to enforce as well.

In addition, Sciclone is looking to expand indications and is sponsoring a clinical trial with Sun Yat-sen University in Guangdong to determine if there is a survival advantage for sepsis patients treated with Zadaxin. Patients are expected to start enrolling immediately.

But those initiatives will only go so far. China has made it clear that it wants more than imported differentiated specialty drugs; it wants to encourage innovation and local manufacturing. And Sciclone has responded to the message in a bid to stay relevant over the long term.

The firm announced two in-licensing deals for early stage oncology products, one with Ability Pharmaceuticals SL, of Barcelona, Spain, and the other with Phosplatin Therapeutics LLC, of New York.

Sciclone is hopeful that fresh crop of cancer assets will qualify under the CFDA's class I regulatory approval pathway for new drugs and benefit from accelerated review and approval. The firm also chose the assets for their suitability to be manufactured in China, another key requirement for green channel assessment.

"We are quite excited strategically about this. We hope with China opening up and supporting more class I pathways, we will be able to build a more fully integrated company," Friedhelm Blobel, Sciclone CEO, told BioWorld Asia.

PREPPING FOR INDS

Sciclone's current development pipeline contains seven drugs and devices already approved in Western markets, covering oncology, cardiovascular and infectious diseases. In the past, that seemed like a safer bet than taking on risky, unapproved assets, but the long approval timelines those class III assets face has an opportunity cost of its own.

The deal with Ability Pharma is valued at $20 million, with an undisclosed up-front payment, described as modest by Blobel, and potential regulatory and sales milestones along with royalties. Sciclone gains exclusive rights for Greater China (as well as Taiwan and Vietnam) to develop ABTL-08, a first-in-class PI3K/Akt/mTOR signaling pathway inhibitor for the treatment of solid tumors. ABTL-0812 up-regulates TRIB3, leading to inhibition of Akt phosphorylation, which results in decreased activity of mTORC1.

Ability Pharma has completed a phase I/Ib trial of orally administered ABTL-0812 in 29 advanced cancer patients with solid tumors and presented the study results in December 2015, showing the inhibition of the PI3K/Akt/mTOR pathway based on biomarkers, correlating with ABTL-0812 plasma levels. The first in-human signal study was designed to explore ABTL-0812 safety and efficacy profile.

A full phase II program is now being designed in several advanced tumor indications, including endometrial and lung cancer by Ability Pharma. ABTL0812 was granted orphan drug status for pediatric cancer neuroblastoma by the FDA and EMA.

The deal with Phosplatin Therapeutics hands over exclusive commercialization and development rights to oncology candidate PTE-112 for Greater China and Vietnam (and option rights in South Korea and Vietnam). Financial terms were undisclosed.

PTE-112 is a platinum agent that has the potential to be first-in-class. Blobel said it was rationally designed to solve toxicity issues and drug resistance historically associated with that therapeutic class.

"Preclinically, PTE-112 has been shown to be highly active in mice," said Blobel. "Furthermore, it demonstrates less toxicity and overall grade of tolerability than prior platinum agents. Its unique mechanistic profile demonstrates activity on multiple anticancer signaling pathways as well as evidence of introduction of the key cellular factors that lead to a immunogenic cell death or ICD, a very rare property among chemotherapies."

Phosplatin said its study on human colon cancer cells demonstrated that PT-112 treatment simultaneously regulates multiple cellular targets, including apoptosis, cell survival, tumor suppressor and cell cycle proteins and pathways in a manner that is clearly differentiated from oxaliplatin.

With those two fresh candidates in its pipeline, Sciclone is preparing to file investigational new drug applications for China by the end of the year.

No Comments