Hookipa, Gilead in pact for HIV, hep B immunotherapies

 Hookipa Biotech AG (“Hookipa”), a clinical-stage biotech company pioneering an innovative class of active immunization therapies for oncology and infectious diseases and Gilead Sciences, Inc., (“Gilead”), a research-based biopharmaceutical company that discovers, develops and commercializes innovative medicines in areas of unmet medical need, today announced that they have entered into a research collaboration and license agreement that grants Gilead exclusive rights to Hookipa’s TheraT^®and Vaxwave^® arenavirus vector-based immunization technologies for two major chronic infectious disease indications, hepatitis B virus (HBV) and human immunodeficiency virus (HIV).

Under the terms of the agreement, Gilead will provide an upfront payment of $10 million. Additionally, Hookipa will be eligible to receive milestone payments based upon the achievement of specified development, regulatory, and commercial milestones up to a total of more than $400 million. Gilead will fund all research and development activities. Hookipa will also be eligible to receive tiered royalties on net sales.

“Gilead, a world leader in innovative therapies against major viral diseases, is the ideal partner for us to drive our pipeline development in this area for the benefit of patients in need. This partnership is strong recognition of our unique immunization technology, and helps us concentrate our own energy and resources on immuno-oncology,” commented Joern Aldag, Chief Executive Officer of Hookipa. “The collaborative HIV and HBV programs nicely complement our significant efforts in the infectious disease area with an exciting proprietary prophylactic CMV vaccine.”

“Gilead is committed to advancing innovative approaches directed at functional cures against HIV and HBV,” said Bill Lee, PhD, Executive Vice President of Research, Gilead. “We are convinced that Hookipa’s unique therapeutic vaccine technology, which has demonstrated excellent safety and immunogenicity in Phase 1 clinical studies, has strong potential to have synergistic effect with other Gilead cure efforts in both of these diseases areas. Our ultimate long-term goal is to eliminate the need for life-long antiviral therapy for millions of patients around the world.”

https://bit.ly/2sI03oI

AbbVie rheumatoid arthritis med meets endpoints in Phase 3 study

AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, today announced positive top-line results from SELECT-EARLY showing that both doses of upadacitinib monotherapy (15 mg and 30 mg) met the primary endpoints of ACR50^a at week 12 and clinical remission^b at week 24 versus methotrexate (MTX).¹ Additionally, all ranked secondary endpoints were met.¹ The ongoing study evaluates upadacitinib, an investigational oral JAK1-selective inhibitor, as a monotherapy treatment compared to methotrexate monotherapy in adult patients with moderate to severe rheumatoid arthritis who were methotrexate-naïve.¹ Upadacitinib is not approved by regulatory authorities and its safety and efficacy have not been established.

“SELECT-EARLY is the fifth pivotal trial that will support regulatory submissions for upadacitinib in rheumatoid arthritis later this year,” said Michael Severino, M.D., executive vice president, research and development and chief scientific officer, AbbVie. “Results from SELECT-EARLY further support our belief that upadacitinib has the potential to be an important new treatment option for patients with rheumatoid arthritis.”

https://bit.ly/2LZz1C0

Nimbus Raises Another $65M from Lilly, Bill Gates, Pfizer, others

Nimbus Therapeutics, headquartered in Cambridge, Massachusetts, raised an additional $65 million in financing. Early investors participated, including Atlas Venture, SR One, Lilly Ventures, Bill Gates, Pfizer Venture Investments, Lightstone Ventures, and Schrodinger.

About two years ago, Nimbus was considering an initial public offering as a way to fund its work in autoimmune and liver diseases. Instead, the company decided to negotiate deals with larger companies. In 2016, Gilead Sciences acquired a Nimbus subsidiary with an upfront payment of $400 million to gain access to a possible drug for nonalcoholic steatohepatitis (NASH). Nimbus could receive up to $800 million in milestone payments on the drug, which is currently in mid-stage clinical trials.

Since then it has signed deals with Monsanto, Genentech, and Celgene The deal with Monsanto is to develop fungicides. It has two programs with Celgene. One is a tyrosine kinase 2 (Tyk 2) program for immunology and oncology, the other a stimulator of interferon genes (STING), for immunology.

Xconomy notes, “But Nimbus retains the right to develop drugs that activate the STING pathway, an approach that other companies have since taken up as a potential way to treat cancer. Activating STING may also have applications in virology. A potential Nimbus STING cancer immunotherapy is in preclinical development. The company is looking for a partner for that compound.”

The company was founded in 2009 as Nimbus Discovery. It utilizes chemical-simulation software developed by Schrodinger, a software company that is also an investor in Nimbus.

Nimbus is structured as a series of independent C corporation. Each C corp houses distinct research and development programs focused on a specific disease target. This allows the company to make investment and partnership decisions based on an asset, as opposed to an entire pipeline. In 2016, it sold one of those C Corps, Nimbus Apollo, to Gilead Sciences for an upfront payment of $400 million with a potential for another $800 million in milestones. The Nimbus Apollo program included NDI-010976, an ACC inhibitor to treat NASH.

Of this new financing, Don Nicholson, Nimbus’ chief executive officer, stated, “The continued support from our world-class investor base is testament to our team’s repeated success in designing and developing promising candidates through our unique combination of massive computational-chemistry horsepower with founding partner, Schrodinger, coupled with additional cutting-edge technologies in structural biology, cryo-EM, and machine learning-augmented ADMET prediction to rapidly advance our pipeline to the clinic. We have made substantial progress across our entire portfolio, including inhibitor of Tyk2 (tyrosine kinase 2) and antagonists of STING (stimulator of interferon genes), both under our immunology alliance with Celgene. Additionally, our wholly-owned STING agonist program has generated novel, highly potent, bona fide small molecules with compelling preclinical data.”

Although the company is set up in a way that makes it fairly straightforward to sell drugs it develops, it is interested in keeping some of its internal programs. This new financing will help in that process.

“This most recent infusion of capital from our investors, together with proceeds from business development activity, enables Nimbus to remain a privately held LLC organization, which has allowed us to transact multiple deals with world-leading partners such as Gilead, Celgene, and Genentech,” said Jeb Kelper, Nimbus’ chief financial officer and chief business officer, in a statement. “Nimbus’ success has built a nine-figure balance sheet of resources for the rapid advancement and expansion of our pipeline and technology, which will allow us to develop several other undisclosed target programs forward to the clinic in the next few years.”

https://bit.ly/2xMjrqe

Roche rushes to up Herceptin supply in China after insurance OK spurs shortage

When Roche got Herceptin onto China’s national insurance program, it didn’t expect demand to surge so quickly, overwhelming its production capacity there. Now, to cope with a nationwide shortage, it’s shifting to a different manufacturing site to amp up supplies of the cancer therapy.

Roche applied to change Herceptin’s existing production site for China to a higher-capacity plant soon after it received shortage reports and received that approval on May 30, Roche spokesperson Anja von Treskow told FiercePharma. She didn’t specify the locations of those sites.

Von Treskow said it might take time to completely recover Herceptin supplies in the Chinese market, because imported biologics need to pass inspection by the appointed institute of drug control, a process that could take months.

Herceptin—together with three other Roche cancer drugs, Avastin, Rituxan and Tarceva—was among 36 drugs added to China’s National Reimbursement Drug List last July. To get on the roster, Roche cut the breast cancer medication’s price by about 70% to CNY7,600 ($1,186) per bottle. And because patients only need to pay a small portion of that out of pocket, it means a huge economic relief for them.

Roche is counting on the coverage to help it reach more patients, aiming for volumes that can eventually make up for the huge discount. What it didn’t foresee was the ultrafast uptake.

During an interview last October, Roche Pharmaceuticals China General Manager Hong Chow told FiercePharma almost all provinces had already implemented the insurance switch—in just three months. By contrast, it took about three years to implement the list’s previous update in 2009.

“The much shorter implementation period and significant reduction of patients’ out-of-pocket payments led to rapid increase of demand for Herceptin,” said von Treskow.

Roche then maxed out its production capacity at the plant manufacturing Herceptin for China and tweaked its supply distribution across provinces, but still couldn’t meet the surge in demand.

Last year, China returned CHF1.80 billion ($1.83 billion) in sales for Roche’s pharmaceuticals division, a 6% jump year over year. That was a slowdown from 11% growth for the first half of 2017. Chow said during the October interview that she expected the national insurance scheme listing—and its attendant discount—would cut into sales growth short term, but as more patients use the therapies, volume will grow, benefiting the Swiss pharma in the long run.

The rising demand in China also comes as those Roche cancer stalwarts have started facing biosimilar competition in developed markets in the EU. And the U.S. market will see biosims soon, despite the fact that many drugmakers have had their Herceptin copycats rejected or delayed by the FDA, including a partnership between Amgen and Allergan, Pfizer, Mylan and partner Biocon, and Celltrion and Teva.

https://bit.ly/2HlXrSx

Tricida files for $150M IPO to fund kidney disease drug approval

Tricida has filed to raise $150 million to bring a chronic kidney disease drug to market. Proceeds from the planned IPO will set Tricida up to file for FDA approval of TRC101 and start building up the 80- to 100-person sales team it wants to commercialize the drug.

TRC101 is a polymer designed to selectively bind to hydrochloric acid in the gastrointestinal tract and take it out of the body in feces. In doing so, Tricida thinks TRC101 can decrease acid levels and boost the amount of blood bicarbonate. By triggering the changes, TRC101 could treat metabolic acidosis, a condition characterized by the accumulation of acid that contributes to the progression of chronic kidney disease.

Tricida has phase 3 data suggesting aspects of the idea work in practice. In a 217-patient phase 3, blood bicarbonate levels increased by a certain amount or normalized in 59.2% of participants who received TRC101, as compared to 22.5% of people in the placebo cohort. That resulted in the trial hitting its primary endpoint. The study also aced a secondary endpoint that looked at change in blood bicarbonate from baseline.

Armed with the data, Tricida plans to file for FDA approval under the accelerated approval program in the second half of next year. The IPO haul will equip Tricida to file for approval while manufacturing TRC101 and building out its commercial infrastructure ahead of the anticipated U.S. launch.

While Tricida has data from a phase 3 trial, its ability to secure approval on the strength of the results depends on other factors. The FDA has told Tricida it needs to almost finish enrolling a postmarket trial before filing its NDA. Tricida thinks a 1,400- to 1,600-patient study will be sufficient to meet the FDA’s request, but the final sample size is still subject to discussions with the agency.

Tricida sees progress in those discussions and enrollment in the trial leading to a NDA filing next year. At that point, attention will turn to the FDA’s acceptance of the phase 3 endpoint. Tricida looked at blood bicarbonate in a belief it is a surrogate for clinical benefits and has discussed the design with the FDA. However, the agency may question the validity of the surrogate endpoint or the level of change needed to achieve clinical benefits when it reviews the NDA.

Even accounting for those issues, Tricida has a far clearer path to market than many other biotechs that have filed for IPOs this year. While its peers have headed to public markets before generating clinical data, Tricida has relied on the support of backers including OrbiMed and, more recently, a loan from Hercules Capital to get within a stone’s throw of an NDA before looking to go public.

https://bit.ly/2sIV9aK

David Koch to retire from Koch Industries due to poor health

Conservative billionaire industrialist David Koch is retiring from his roles at Koch Industries and associated companies due to poor health, according to an internal memo seen by Reuters.

David Koch, 78, along with his elder brother Charles, owns the second largest private U.S. company, whose operations range from refining and chemicals to ranching and forest products.

The brothers are known to spend heavily on conservative initiatives and to oppose government intervention in business.

David, who was diagnosed with prostate cancer almost two decades ago, is the executive vice president at Koch Industries. The brothers have donated several millions to doctors and researchers working towards a cancer cure.

“David has always been a fighter and is dealing with this challenge in the same way,” Charles Koch wrote in the memo dated June 4.

https://bit.ly/2szBKdb

Genentech gets FDA priority review for hemphilia A med

Genentech, a member of the Roche Group, announced tody that the FDA has accepted the company’s supplemental Biologics License Application, or sBLA, and granted Priority Review for Hemlibra for adults and children with hemophilia A without factor VIII inhibitors. The sBLA is based on data from the Phase III HAVEN 3 study. The FDA is expected to make a decision on approval by October 4.

https://bit.ly/2kNJ6p5