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

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.

Business

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.

Miscellaneous

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

Bioevents

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

China BIO Partnering Forum on May 10-11 in Suzhou (China)

9^thAnnual 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)

5^thAntibody 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)

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

http://btobioinnovation.com/events/in-vitro-diagnostic-events/

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