Abivax Bolstered by Positive Mid-Stage Results from Ulcerative Colitis Treatment

Shares of Paris-based Abivax have shot up more than 8 percent this morning after the company posted positive mid-stage results for its ulcerative colitis treatment, ABX464.

This morning the company released topline results from its Phase IIa trial showing treatment with the experimental drug resulted in “statistically significant efficacy based on both clinical and endoscopic endpoints in this study.” Abivax’s ABX464 has a mechanism of action that is triggered by an increased expression of miR124, a potent anti-inflammatory microRNA, the company said.

Final eight-week topline data from the Phase IIa study showed 35 percent of patients on the Abivax treatment saw clinical remission, compared to 11 percent of placebo patients. When it came to mucosal healing, 50 percent of ABX464 patients saw a positive response, compared to 11 percent of patients on placebo. Abivax’s ABX464 yielded an overall 70 percent clinical response rate, compared to 30 percent for placebo, results show.

Additionally, the company noted that calprotectin, the best-studied biomarker in ulcerative colitis, was markedly reduced in patients who received ABX464 in comparison to placebo after four weeks of treatment.

For ulcerative colitis patients treated with ABX464 the onset of therapeutic effect was rapid, the company said. Efficacy could be observed following two weeks of treatment and the results “became significant” at eight weeks.

Ulcerative colitis is a debilitating inflammatory bowel disease in adults and children, with limited therapeutic management options for many patients. It is estimated that close to 1 million patients with ulcerative colitis live in the United States.

Jean-Marc Steens, chief medical officer of Abivax, said the results of the Phase IIa trial have exceeded the company’s expectations given the “statistically significant, strong efficacy” that was already observed in the study.

“They validate our hypothesis that ABX464 novel mechanism of action would result in potent anti-inflammatory properties in patients. Like other chronic inflammatory diseases, ulcerative colitis is a debilitating disease that greatly affects patients’ quality of life and warrants expensive innovative therapies. We look forward to developing and potentially market ABX464 as a well-tolerated oral treatment for this large patient population,” Steens said in a statement.

No severe adverse events attributed to ABX464 were reported in the trial. Full clinical trial data will be presented at upcoming international scientific conferences, the company said.

Hartmut Ehrlich, chief executive officer at Abivax, called the Phase II a data impressive. He said it indicates the potential for ABX464 to bring relief to UC patients who “are not adequately helped by currently available therapeutics and are struggling from the devastating consequences of this inflammatory disease.” Based on the results, Abivax will initiate a Phase IIb dose-ranging study for ABX464. Ehrlich added that the data is also leading the company to pursue mid-stage trials in other inflammatory indications, such as Crohn’s disease.

https://www.biospace.com/article/-cp8r-abivax-bolstered-by-positive-mid-stage-results-from-ulcerative-colitis-treatment/

Pfizer Prices $5B Debt Offering

Pfizer Inc. (NYSE: PFE) today announced the pricing of a debt offering consisting of six tranches of notes:

$300,000,000 aggregate principal amount of floating rate notes due 2023

$1,000,000,000 aggregate principal amount of 3.000% notes due 2021

$1,000,000,000 aggregate principal amount of 3.200% notes due 2023

$1,000,000,000 aggregate principal amount of 3.600% notes due 2028

$700,000,000 aggregate principal amount of 4.100% notes due 2038

$1,000,000,000 aggregate principal amount of 4.200% notes due 2048

Pfizer intends to use the net proceeds for general corporate purposes, including to refinance, redeem or repurchase existing debt and to repay a portion of its outstanding commercial paper.

The closing of the offering is expected to occur on September 7, 2018, subject to satisfaction of customary closing conditions.

Citigroup Global Markets Inc., Credit Suisse Securities (USA) LLC, Merrill Lynch, Pierce, Fenner & Smith Incorporated and Morgan Stanley & Co. LLC are acting as joint book-running managers for the offering.

https://www.biospace.com/article/releases/pfizer-prices-5-000-000-000-debt-offering/

Tempus Data Analytics Hits $2B Valuation, 1 of Few Chicago ‘Unicorns’

On August 29, Chicago-based Tempus, which focuses on data analytics for molecular and clinical data, announced it had raised $110 million in Series E financing. This brought total money raised so far to $320 million. This also gave the company a valuation of $2 billion. The round included Baillie Gifford, T. Rowe Price, Revolution Growth, New Enterprise Associates (NEA) and other existing investors. A venture-backed private company with a valuation of at least $1 billion is dubbed a “unicorn.” (So far, no one has started calling companies with $2 billion in valuation a “rhinoceros.”)

Tempus is also noted for being founded by Eric Lefkofsky in 2015. Lefkofsky is the co-founder of Groupon. Forbes writes, “Propelled to start Tempus after, Lefkofsky says, he noticed during his wife’s cancer treatment a few years ago that there was a lack of data to inform her treatment options, his company now collects both molecular and clinical data from hospitals around the country and analyzes it within a database with the aim of improving treatment for patients with cancer. So far, they’ve partnered with some 250 hospital systems and have collected 2 million clinical records.”

The company expects to use the Series E funds to expand beyond oncology into healthcare areas like cardiovascular disease and diabetes, as well as to push outside the U.S. into Europe, Australia and Asia.

According to CB Insights, there are only 100 “unicorns” in the U.S. and about 200 worldwide. In the Chicago area, there are only a handful, including Avant, ExteNet, SMS Assist and Uptake Technologies. Uptake was founded by Lefkofsky’s longtime business partner, Brad Keywell.

In biotech, probably the best-known “unicorn” is Cambridge, Massachusetts-based Moderna Therapeutics, which focuses on messenger RNA (mRNA) technology. Another is Ginkgo Bioworks, which hit the “unicorn” status this year.

Tempus employs almost 500 people. Lefkofsky told Crain’s Chicago Business, “We’re in a period of rapid growth and acceleration. This capital allows us to look more broadly than just cancer in the United States. We’ve begun to think about Europe, Asia and Australia, and how our model would extend there. We’ll be cautious in any step we take. These tools and our lab are operating at scale, so we feel like we’re in a unique position to extend our platform into other areas.”

Without disclosing specific data, Lefkofsky told Crain’s, “In general, we’re now sequencing tens of thousands of patients. We hope to shortly be sequencing hundreds of thousands of patients. Clinicians are getting these tests. They’re using it as an important tool in prescribing therapeutic treatments for patients.”

Tempus is working in an area where two other big companies are focused—Flatiron Health, which was acquired by Roche earlier this year, and Foundation Medicine a few months later.It has several areas, including a laboratory that sequences tumor genetics in addition to inherited genes to determine which mutations affect an individual’s cancer. It also collects and analyzes clinical data from doctors, hospitals and clinical trials in an attempt to analyze patterns and standardize the information. Its products and services fall into four broad categories: genomic sequencing, clinical data structuring, image recognition, and biological modeling.

The company’s collaborators include Cleveland Clinic, Mayo Clinic, University of Michigan Comprehensive Cancer Center, Penn Medicine and many others.

Forbes points out that Tempus’ valuation had doubled in the last six months. Per Pitchbook, it was valued at a bit more than $1 billion after its Series D financing in March. The Flatiron deal was for $1.9 billion. Roche picked up Foundation Medicine in June for $2.4 billion.

Mohamad Makhzoumi, general partner and head of New Enterprise Associate’s healthcare services and healthcare IT division, told Forbes, “I don’t know how long this is going to take, and I don’t know what the right kind of business model is going to be, but it’s undeniable that this is going to be a huge chunk of the healthcare economy in the U.S.”

https://www.biospace.com/article/-ip3o-tempus-data-analytics-firm-hits-2-billion-valuation-one-of-a-handful-of-chicago-unicorns-/?s=81

Gilead, Trianni enter licensing pact for use of Trianni transgenic platform

Gilead Sciences and Trianni announced that the companies have entered into a license agreement that grants Gilead the use of the Trianni transgenic human monoclonal antibody discovery platform to support the company’s drug discovery efforts. “We look forward to integrating Trianni’s technology into our research and development program,” said Bill Lee, PhD, Executive Vice President of Research, Gilead. “This platform will help enhance our ability to discover human antibodies and to develop new therapies in areas of unmet medical need.” No financial details were disclosed.

https://thefly.com/landingPageNews.php?id=2785509

Dova submits application for orphan drug extended indication

Dova Pharmaceuticals announced the submission of a supplemental New Drug Application, sNDAto the U.S. Food and Drug Administration for DOPTELET, avatrombopag, the Company’s second generation, orally administered thrombopoietin receptor agonis, TPO-RA, seeking approval for the treatment of adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment. The FDA previously granted orphan drug designation to avatrombopag for this indication. DOPTELET was recently approved by the FDA in May 2018 for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure. “Following the exciting last few months with the approval and launch of DOPTELET for patients with CLD, Dova continues its forward momentum with the submission of our sNDA for DOPTELET for the treatment of patients with ITP,” said Alex C. Sapir, President and CEO of Dova. “This represents another significant achievement for Dova that has the potential to expand the treatment applications for DOPTELET and validate its use for an additional indication. Despite the availability of two approved TPO receptor agonists for the treatment of chronic ITP, there remains an important unmet medical need.” “Given DOPTELET’s convenient oral route of delivery, combined with its lack of hepatotoxicity or need for strict dietary restrictions with its administration, we believe DOPTELET, if approved by the FDA, is well-differentiated and has the potential to capture a meaningful share of the $1.5 billion global ITP market,” Mr. Sapir added.

https://thefly.com/landingPageNews.php?id=2785537

Tesaro expands to 2nd stage of lung cancer combo trial

TESARO announced it has initiated the second stage of the JASPER study that is designed to assess clinical benefit of ZEJULA in combination with an anti-PD-1 antibody in first-line non-small cell lung cancer, NSCLC, patients. The decision to advance the trial was based on achieving the protocol defined response criteria in the initial cohort of 16 treated patients with high PD-L1 expression, of which 14 were evaluable for a response. Nine of the 14 patients had objective responses by RECIST criteria at the time of the analysis1; with all 14 patients experiencing tumor shrinkage.”These JASPER data provide preliminary evidence that the combination of ZEJULA and an anti-PD-1 antibody could be active as a first-line treatment for patients with non-small cell lung cancer and high levels of PD-L1 expression,” said Mary Lynne Hedley, Ph.D., President and COO of TESARO. “In the second stage of the trial, 36 additional patients will be enrolled and treated with ZEJULA in combination with TSR-042, our anti-PD-1 antibody. TSR-042 is the foundation of our lung cancer strategy, and is also being studied as a monotherapy in our GARNET trial in anti-PD-1 naive patients who have progressed on chemotherapy, and in combination with TSR-022, our anti-TIM-3 antibody, in AMBER, a study in late-line NSCLC patients that have progressed after anti-PD-1 therapy. We look forward to sharing lung cancer data from both GARNET and AMBER at the Society for the Immunotherapy of Cancer, SITC, Annual Meeting in November.”

https://thefly.com/landingPageNews.php?id=2785543

Agios Pharmaceuticals announces Jacqualyn Fause to become new CEO

Agios Pharmaceuticals announced that effective February 1, 2019, David Schenkein, M.D., will transition to the role of executive chairman of the board of directors and serve as a member of the board’s Science & Technology Committee, after a successful decade-long tenure as chief executive officer. Jacqualyn Fouse, Ph.D., a member of the company’s board, has been named as Agios’ next chief executive officer. Prior to joining Agios’ board in December 2017, Dr. Fouse served as president and chief operating officer of Celgene Corporation, a global biopharmaceutical company, until April 2017, and as a member of its board through June 2017. Dr. Fouse joined Celgene in 2010 as chief financial officer and was named president of the company’s global hematology and oncology franchise in 2014. Prior to joining Celgene, Dr. Fouse served as chief financial officer of Bunge Limited, a leading agribusiness and food company. Earlier in her career, she held senior roles at Alcon Laboratories and various international companies.

https://thefly.com/landingPageNews.php?id=2785591