Biopharmaceutical News Week # 17-18.2017
Author: Jean-Claude Muller, 穆卓Executive Editor at BtoBioInnovation, firstname.lastname@example.org
Mergers, Acquisitions and Joint Venture
Sosei (Tokyo, Japan) announces its plan to acquire MiNA Therapeutics (London, UK) in buying 25.6% of its equity and an exclusive option to acquire the company for $45 million in cash. Right now MiNA is testing its first saRNA product in a phase 1/2a trial in lung cancer. If successful Sosei can complete the deal for another $180 million in cash plus $309 million in milestones.
Seattle Genetics (Bothwell, WA, USA) stops its intention to acquire Immunomedics (Morris Plain, NJ, USA) in a deal worth up to $2 billion under the strong pressure of venBio an activist investment group. The settlement is linked to the immediate resignation of Immunomedic’s CEO, Cynthia Sullivan and Immunomedic’s CSO, David Goldenberg, who both led the counterattack against venBio.
Shire has been granted exclusive global rights to Parion Sciences’ (Durham, NC, USA) P-321, an epithelial sodium channel inhibitor (ENaC) as a treatment of dry-eye disease. Under the terms of the deal Shire will make a $20 million upfront payment followed by a $20 million near-term milestone as well as up to $495 million milestones and royalties on future sales. In 2015, Parion granted Vertex Pharmaceuticals rights to a portfolio of ENaC inhibitors to treat cystic fibrosis and other pulmonary disorders.
AstraZeneca enters a R&D collaboration with Pieris Pharmaceuticals (Boston, MA, USA) to develop inhaled Anticalins in the respiratory field with PRS-060, a first lead drug. PRS-060 serves to treat asthma through direct delivery of a potent Interleukin (IL4Ra) targeting Anticalin protein to the lungs. Under the terms of the deal AstraZeneca will make a $45 million upfront payment followed by a $12.5 million milestone when PRS-O60 enters phase 1. The total deal has been valued at $2.1 billion. Earlier this year Pieris entered a partnership with Servier to develop PRS-332 a next-generation PD-1 drug as well as four other immune-oncology drugs.
Novartis gets two license deals to expand its long-term investment in CAR-T therapies. A first one with Celyad (Mont-Saint-Guibert, Belgium) for non-exclusive rights to its allogeneic CAR-T, off-the-shelf cell therapies for various cancers in a deal worth $96 million. A second one with BlueBird (Cambridge, MA, USA) with a non-exclusive license covering certain bluebird patent rights related to lentiviral vector technology for CAR-T cells. Financial terms were not disclosed. In October 2014 Novartis had entered an exclusive license deal on lentiviral vector technology with Oxford Biomedica.
…and extends its interest in NASH in concluding an exclusive worldwide deal with Conatus (San Diego, CA, USA) for emricasan in paying an additional $7 million. Both companies had announced their collaboration in late 2016.
Zai Lab (Shanghai, China) signs a deal with Paratek Pharmaceuticals (Bosto, MA, USA) to develop and commercialise omadacycline in greater China. Omadacycline is a once-daily, oral and intravenous broad-spectrum antibiotic designed to be used as empiric monotherapy in patients suffering from serious community-acquired bacterial infections. Zai Lab will make a $7.5 million upfront and additional development triggered milestones as well as royalties on sales within the licensed territory.
Takeda Pharmaceuticals enters a collaboration with Harrington Discovery Institute at University Hospitals in Cleveland to advance the discovery and development of rare diseases and complement Takeda’s internal R&D efforts.
Approval of drugs and vaccines
The European Commission approves Janssen Biotech’s Darzalex, or daratumumab, as part of combination therapy for previously treated patients with relapsed or refractory multiple myeloma. Darzalex was granted accelerated approval by the US FDA in November 2015, as monotherapy in patients with multiple myeloma who have received at least three prior lines of therapy. Full FDA clearance, including the combination therapy indication, was granted at the end of last year.
The US FDA approves:
Sanofi’s Thymoglobulin, in conjunction with concomitant immunosuppression as a treatment for prevention of acute kidney transplant rejection.
BioMarin’s (Novato, CA, USA) Brineura, or cerliponase alfa, as a treatment for symptomatic pediatric patients with late infantile neuronal ceroid lipofuscinosis type 2, an ultra-rare type of Batten disease that is also called tripeptidyl peptidase 1 deficiency. The drug’s cost is listed at $702,000 pear year and averages $486,000 after Medicaid discounts are factored.
Novartis’ Rydapt or midostaurin, a multi-target protein kinase inhibitor, in combination with chemotherapy as a treatment of patients with newly diagnosed acute myeloid leukemia (ALL) who have a genetic mutation called FLT3.
AstraZeneca’s Imfinzi, or durvalumab, a PD-L1 checkpoint inhibitor, as a treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression following platinum-containing chemotherapy.
Roche’s Ventana (Tucson, AZ, USA) PD-L1 (SP263) assay label as a complementary diagnostic for the assessment of the PD-L1 protein to predict a urothelial carcinoma patient’s response to Imfinzi.
…and Takeda’s Alunbrig or brigatinib, as a treatment of patients with ALK-positive non-small cell lung cancer. The drug had been discovered and developed by Ariad which was acquired by Takeda earlier this year.
Drugs at clinical stage
Abivax (Paris, France) announces early report of an HIV breakthrough with its ABX 464 drug after claiming it’s gathered evidence of success in eliminating a significant amount of the reservoirs of HIV that linger in patients. The results were obtained in a small number of 30 HIV patients. “This is the first time we see a signal with any therapeutic candidate that it may be possible to reduce HIV reservoirs in patients,” said Professor Linos Vandekerckhove, Head HIV Cure Research Center at the Department of Internal Medicine, at the University of Ghent, Belgium, a principal investigator involved in the study.
Moderna Therapeutics (Cambridge, MA, USA) reports promise with H10N8, its experimental messenger RNA approach to treat avian flu. The trial was performed in a small group of 31 subjects, looking at their response in two different measures. All demonstrated a sufficient immune response to fight off the virus in the first measure, and all but 3 in the second, for a total of 23 who received the vaccine. None of the 8 subjects who received a placebo responded The data “provide important validation of our core mRNA platform, as we continue to advance our development pipeline tackling more complex vaccines including personalized cancer vaccines, and moving our mRNA therapeutics into clinical development.” said Tal Zaks, Chief Medical Officer of Moderna.
Lundbeck and Otsuka expect to get approval for Rexulti or brexpiprazole, already approved for schizophrenia and major depression, for agitation triggered by Alzheimer’s dementia. Results of two late-stage studies showed positive results from baseline in the Cohen-Mansfield Agitation Inventory total score in one study, but not in the other.
Roche/Genentech halts further clinical development and ongoing studies evaluating GDC-0810, acquired from Seragon in 2014, and will evaluate strategic options for the Selective Estrogen Receptor Degraders (SERDs) program.
Medical Devices and Diagnosis News.
Roche’s Ventana (Tucson, AZ, USA) PD-L1 (SP263) Assay gains CE label expansion to inform treatment decisions in lung cancer patients being considered for Keytruda or pembrolizumab immunotherapy
Researchers have created a $65 test designed to show which prostate cancer patients will respond to Pfizer’s Xtandi and Johnson & Johnson’s Zytiga. Retrospective analysis of plasma DNA using the droplet digital PCR assay suggest the drugs are less effective in patients with multiple copies of the androgen receptor gene. Xtandi and Zytiga have established themselves in prostate cancer treatment pathways by inhibiting the androgen receptor. However, the drugs work better in some patients than others. As there is no approved test to show who is likely to respond, some patients are currently taking medicines that are unlikely to tackle their cancers, resulting in poorer outcomes for them and wasting money for the healthcare system. Researchers have found a way to stratify patients by the likelihood they will respond to Xtandi and Zytiga. “We have developed a robust test that can be used in the clinic to pick out which men with advanced prostate cancer are likely to respond to Zytiga and Xtandi, and which men might need alternative treatments. Our method costs less than £50 ($65), is quick to provide results, and can be implemented in hospital laboratories across the National Health Service,” Gerhardt Attard, M.D., consultant medical oncologist at The Royal Marsden, said in a statement.
Cost, Pricing and Market Access
The UK National Institute for Health & Care Excellence (NICE) has now published a Final Appraisal Determination (FAD) recommending:
Janssen’s Stelara or ustekinumab as a treatment for patients with moderate to severe active Crohn’s disease who cannot be treated with a conventional drug or with a TNF alpha inhibitor. Ustekinumab is the first human monoclonal antibody for Crohn’s disease to target the interleukin (IL)-12 and IL-23 cytokines, which play a key role in inflammatory and immune responses, giving it a different mode of action from other approved therapies.
Incyte’s (Wilmington, DL, USA) Inclusig, or ponatinib, to treat adult patients with chronic phase, accelerated phase, or blast phase of chronic myeloid leukemia, who are resistant to dasatinib or nilotinib, who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate, or who have the T315I mutation
… but rejects Orexigen’s Mysimba (naltrexone/bupropion) for managing overweight and obesity in adults with a Body Mass Index (BMI) of 30 or more, or 27 to 30 with if the patient has one or more weight-related co-morbidities.. NICE’s Committee said while Mysimba provides “an innovative option after lifestyle measures have failed, and where orlistat is the only pharmaceutical alternative,” there were concerns “about the validity of the economic analysis and the robustness of the results”.
Pfizer offers its breast cancer Ibrance, or palbociclib, a drug which increase the effect of aromatase inhibitors and was approved last November, for free in the UK, while cost regulator NICE appraises the full value of the drug.
The Institute for Clinical and Economic Review (ICER) a Boston based cost watchdog, reviewed three new osteoporosis drugs: Radius Health’s Tymlos, Eli Lilly’s Foreto and Amgen’s romosumab and stated that the new medications are much too pricey to be considered cost-effective. According to ICER, which compared each medication with generically available zoledronic acid, each of the three drugs spurred an increase in both life years and quality-adjusted life years, but each of them also increased costs, and they “far exceeded the commonly-cited cost effectiveness threshold” of $150,000 per quality-adjusted life year, ICER concluded.
Quintiles IMS analysts forecast an important number of new US FDA drug approvals by 2022 dominated by cancer and orphan disease therapies suggesting that the low approval rate of 2016 was an aberration which will not occur over the next five years. Their forecast is a 40-45 new drug approvals per year for the next five years.
New Active Substances (NAS) Launched in the U.S. 2007-2021
They also forecast an increase number of oncology drugs and orphan disease drugs approvals
Late Phase R&D Pipeline by Top Therapy Areas
BIOTrinity on May 8-10, in London (UK)
China BIO Partnering Forum on May 10-11 in Suzhou (China)
9thAnnual China BIO International Partnering Forum on May 31-June 1 in Zhuhai (China)
The Health Industry Sumitt (tHIS) 2017 on May 15-18 in Shanghai (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)
5thAntibody Industrial Symposium, on June 27-28, in Tours (France)
MedFIT on June 28-29 in Grenoble (France)
Nordic Life Science Days (NLS) 2017 on September 12-14 in Malmö-Copenhagen (Sweden and Denmark)
BIOPharm America on September 26-27 in Boston (USA)
BIO Latin America on October 26-28 in Sao Paulo (Brazil)
BIO Europe on November 6-8 in Berlin (Germany)
World Congress on Biomedical Engineering 2017 on November 9-11 in Xian (China)
BIOFIT on November 28-29 in Strasbourg (France)
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