Author: Jean-Claude Muller, 穆卓Executive Editor at BtoBioInnovation, jcm@btobioinnovation.com

Business

Seattle Genetics (Bothell, WA, USA) acquires rights of Immunomedic’s (Morris Plain, NJ, USA) IMMU-132, as atreatment of solid tumors, in a deal worth up to $1.7 billion. IMMU-132, or sacituzimab govitecan, is an antibody drug conjugate (ADC) that contains SN-338, the active metabolite of irinotecan linked to sacituzumab aimed at treating triple negative breast cancer. Under the terms of the deal Seattle Genetics will make a $250 million upfront payment and an additional $50 million payment for ex-US rights.  In ad­di­tion to the cash and mile­stones, Seat­tle Ge­net­ics is also making an equity in­vest­ment for up to a 9.9% stake in Im­munomedics.

Janssen Pharmaceuticals grants Renova Therapeutics’ (San Diego, CA, USA) rights to stresscopin, a hormone involved in responses to physiological stress, as a treatment for a form of heart failure. “Obtaining this stresscopin program IND helps propel forward the development of our, RT-400, a peptide infusion treatment indicated for acute decompensated heart Failure (ADHF)”, said. Jack W. Reich, Ph.D., Renova CEO. Terms of the licensing deal were not disclosed.

Biotime (Alameda, CA, USA) signs a licensing deal with the University of Pittsburgh Medical Center to acquire worldwide rights to a stem cell-derived retinal repair platform. Biotime intends to use the technology, which generates 3D retinal tissue from human pluripotent stem cells, to produce laminated human retinal tissue for use as implants to help restore vision in patients with advanced retinal degenerative diseases. Terms of the deal were not disclosed.

HealthCare Royalty Partners (HCR) purchases the royalty rights to Portola Pharmaceutical’s (South Francisco, CA, USA) of AndexXa^TM, or andexanet alpha, for $50 million upfront and $100 million upon regulatory approval. Portola indicated that it will use the cash to finance the development of AndexXa, a recombinant human Factor Xa protein, a molecule designed to act as an antidote in patients treated with an oral or injectable Factor Xa inhibitor.  The drug has received US FDA’s Breakthrough Therapy designation. HCR is a private investment firm that purchases royalties and uses debt-like structures to invest in commercial or near-commercial-stage life science assets. HCR has $3.4 billion in cumulative capital commitments.

Lysogene (Paris, France), a gene therapy company, has raised $25 million from an IPO on Euronext Paris and will be able to pursue the de­vel­op­ment of two drugs to treat dis­abling, dev­as­tat­ing and deadly dis­eases of the cen­tral ner­vous sys­tem, which start in in­fancy and lead to the pa­tients pre­ma­ture death,” said CEO Karen Aiach. Lysogen had received US FDA Orphan status for LYS-GM101, as a treatment of GM1 Gangliosidosis last week.

Approval of drugs and vaccines

US FDA approves:

Marathon Pharmaceutical’s (Northbrook, Ill, USA) Emflaza or defalzacort, a corticosteroid, for the treatment of patients at least five years old with Duchenne muscular dystrophy (DMD). It is the first corticosteroid approved in the US to treat DMD, an orphan drug indication. The drug works by decreasing inflammation and reducing the activity of the immune system.

….and Amgen’s Parsabib or etelcalcetide, a calcimimetic drug, as a treatment of sec­ondary hy­per­parathy­roidism (SHPT), a dis­ease that af­fects chronic kid­ney dis­ease pa­tients on he­modi­a­lyis. The ap­proval marks the first new drug for this con­di­tion in 12 years. The agency had rejected the drug last August.

Drugs at clinical stage

US FDA grants orphan status:

to Gensight Biologics (Paris, France) GS030 as a treatment for retinitis pigmentosa. GS030 received an advanced therapy medicinal product classification in Europe in September 2016

…and to Imara’s (Cambridge, MA, USA) IMR-687, an orally-administered selective phosphodiesterase 9 (PDE9) inhibitor as treatment of sickle cell disease, a rare genetic condition. A person with sickle cell disease has red blood cells that are hard, sticky, and C-shaped. These cells clog smaller blood vessels resulting in pain as well as increased risk for infection, acute chest syndrome and stroke.

US FDA grants fast track status to Taxus Cardium’s (San Diego, CA, USA) Generx, a gene therapy product, as a treatment for angina pectoris patients who do not respond to standard therapy.

US FDA grants priority review to Merck’s Keytruda, or pembrolizumab, as a first-line treatment of locally advanced or metastatic urothelial cancer in patients for whom treatment with cisplatin-containing therapy is inappropriate and as a second-line treatment for patients whose disease progressed during or after taking platinum-based chemotherapy.

US FDA has approved Cellectis’ (Paris, France) new drug application (NDA) for UCART123, an allogeneic CAR T-cell treatment for the treatment of acute myeloid leukemia and blastic plasmacytoid dendritic cell neoplasm. The approval marks the first time that U.S. regulators have approved clinical testing of an allogeneic, or "off-the-shelf", gene-edited CAR T cell treatment. Drugs of rivals such as Novartis, Juno and Kite have treatments that use modified T cells extracted from individual patients, Cellectis products are derived from healthy donors and aim to be universal.

Innate Pharma (Marseille, France) and partner Bristol-Myers Squibb announced that lirilumab, which is being investigated in six trials across a range of solid and blood cancer indications in combination with other agents, has failed to hit key targets points in elderly patients with acute myeloid leukemia (AML).

Lundbeck and Otsuka announce the failure of two pivotal phase 3 studies with idalopridine a 5HT6 antagonist as a treatment of Alzheimer’s disease. “The efficacy with idalopridine in these studies and in the previous one do not demonstrate efficacy to support a regulatory approval” said the Danish company. Failures with other 5-HT6 antagonists had occurred at Pfizer and GlaxoSmithKline

Science and Technology

Inspite of enormous effort, the World Health Organization estimates that a Zika vaccine for women of childbearing age may not be available before 2020.

A re­search team at Boston Chil­dren’s, headed by Gwenaëlle Géléoc, has tested out a new vec­tor for a gene ther­apy that sig­nif­i­cantly im­proved hear­ing in mouse mod­els for Usher syn­drome, the most com­mon ge­netic dis­ease that trig­gers deaf­ness as well as blind­ness in children. Géléoc’s team say that the re­search also has pos­i­tive im­pli­ca­tions for new gene ther­a­pies to cor­rect blind­ness, pos­si­bly set­ting up a dual ap­proach that can ad­dress both blind­ness and deaf­ness at the same time.

A new study published in the journal Nature Medicine found that older people with low levels of inflammation had something surprising in common: they were all caffeine drinkers. “The more caffeine people consumed, the more protected they were against a chronic state of inflammation,” says study author David Furman, consulting associate professor at the Institute for Immunity, Transplantation and Infection at Stanford University.

Cost, Pricing and Market Access

The UK National Institute for Health and Care Excellence has rejected AstraZeneca’s Daxas for use in certain patients with chronic obstructive pulmonary disease (COPD).

Earlier this week, White House press briefing Sean Spicer confirmed that US President Donald Trump remains committed to Medicaire drug price negotiations.

Miscellaneous

Gilead Sciences (Foster City, CA, USA) said earlier this week that total products sales could decline, as much as 22%, in 2017. Gilead is forecasting hepatitis C drug sales in 2017 to be in the range of $7.5 billion to $9 billion. At the midpoint, that's a 44% drop off from 2016 sales of $14.8 billion.

A US appeal court decided earlier this week to let Sanofi and Regeneron’s  PCSK9 cholesterol drug remain on pharmacy shelves while it sorts out an appeal in a patent fight with Amgen, which markets Repatha a rival drug.

The nonprofit Chan Zuckerberg Biohub, announced today that 47 faculties at three Californian research universities will get no-strings-attached awards to delve into risky new directions. Biohub is the first concrete piece of the Chan Zuckerberg Initiative’s foray into science, launched last September with a commitment of $3 billion over 10 years from Zuckerberg and Chan, a pediatrician.

Erze Vigodman, Teva’s CEO is stepping down as the result of a “mutual agreement” between himself and the Board. Teva Chairman Yitzhak Peterburg will act as interim CEO. The move comes a few days after the Israeli group made a sales guidance lowering of more than $1 billion and after a US court tossed out four patents on long acting Copaxone putting several $billions at risk.

Bioevents

BIO CEO & Investor Conference on February 13-17, in New York City (USA)

European Life Sciences CEO Forum & Exhibition on March 6-7, Zurich (Switzerland)

BIO Asia on March 14-15, in Tokyo (Japan)

BIO-Europe Spring on March 20-22, in Barcelona (Spain)

BIOVISION on April 4-6, in Lyon (France)

Strategic Alliance Management Congress on May 1-3, in Philadelphia (USA)

BIOTrinity on May 8-10, in London (UK)

9^th Annual China BIO Partnering Forum on May 10-11 in Shunde (China)

2017 BIO-IT World Conference & Expo on May 23-25 in Boston (USA)

BIO International Convention on June 19-22 in San Diego (USA)

BIOPharm America on September 26-27 in Boston (USA)

BIO Latin America on October 26-28 in Sao Paulo (Brazil)

BIO Europe on Novmber 6-8 in Berlin (Germany)

BIOFIT on November 28-29 in Strasbourg (France)

http://btobioinnovation.com/events/pharma-biotech-events/

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