The year 2019 has been a busy year for global pharma and biotech companies involved in M&A, option & licensing agreements and gaining approvals. Our team has compiled a list of 20 most read life sciences news on PharmaShots in 2019.

1. Celltrion Signed an Exclusive Co-Marketing Agreement with Juno in Australia

• Juno provided sales promotion and hospital contracting services to Australian Hospital market customers and will co-market Herzuma (biosimilar, trastuzumab) and Truxima (biosimilar, rituximab) with Celltrion in Australia
• The focus of the agreement was to market globally biosimilar brands to Australian Healthcare professionals and patients
• Herzuma (CT-P6, biosimilar, trastuzumab) is a mAb targeting the extracellular domain of HER2 receptor and has received MFDS, EC and the FDA's approval in Jan'2014, Feb'2018 and Dec'2018 respectively. Truxima is a mAb (CT-P10, biosimilar, rituximab) is a mAb approved in Korea in 2016, EU in Feb 2017 and is a novel biosimilar approved by Health Canada for NHL, CLL, and RA

2. ONO Signed a Research Collaboration with Repare Therapeutics for its Pol Inhibitor Program

Published: Jan 31, 2019 |Tags: Collaboration, Inhibitor, ONO, Pol, Program, Repare Therapeutics, Research, Signed

• Repare received $15M upfront, $160M milestones, and royalties on sales of products from ONO. Repare got exclusive development and commercialization rights for Repare's Pol? Inhibitor Program in Japan, South Korea, Taiwan, Hong Kong, Macau and ASEAN countries
• Repare will retain development and commercialization rights (ex- ONO's territory) for Pol? Inhibitor Program in the US, Canada, and EU
• Pol? inhibitor is a DNA Polymerase targeted to repair DNA breaks in homologous recombination deficient (HRD) cells and has the ability to play a vital role in breast and ovarian cancer including prostate and pancreatic in combination with chemotherapy, radiotherapy, and Immuno-Oncology agent

3. Novo Nordisk Signs Non -Exclusive Agreement with Abbott for its FreeStyle Libre Mobile App

• The companies entered into an agreement to assess insulin dose data from Novo Nordisk pre-filled insulin pen connected to the digital health tool marked with Abbott's FreeStyle Libre system (FreeStyle LibreLink mobile app and LibreView cloud-based system)
• The focus of this agreement was to advance management techniques for diabetes with the combination of the two technologies CGM & connected insulin pen, enabling healthcare professionals to monitor glucose and insulin data
• In H1'17, Novo signed a collaboration with Glooko for its platform connected to insulin pens for uploading data by any means of communication-capable Android phone. Abbott's FreeStyle Libre system uses sensors allowing the monitor glucose levels in real-time with an eight-hour glucose history & change in glucose level

4. Nektar and BMS Received the US FDA's Breakthrough Designation for Bempegaldesleukin (NKTR-214) + Opdivo (nivolumab) to Treat Advanced Melanoma

• The FDA's BT designation was based on the ongoing P-I/II PIVOT-02 study assessing the doublet therapy in patients with metastatic melanoma. FDA's BT designation is to expedite the development of medicine targeting life-threatening diseases
• In Feb 2018, BMS signed a WW development and commercialization agreement with Nektar for its Bempegaldesleukin
• Nektar's Bempegaldesleukin is a therapy targeting CD122 receptors, activating CD8+ effector T cells and NK cells and is being evaluated in P-III study in combination with Opdivo vs Opdivo in patients with 1L advanced melanoma. Opdivo is a PD-1 immune checkpoint inhibitor harnessing the body's immune system by restoring anti-tumor response

5. Zai Lab Received Priority Review for Zejula's (niraparib) NDA from NMPA (CFDA) in China

• The priority review has been granted to Zai Lab's Zejula as a maintenance treatment for the recurrent epithelial ovarian, fallopian tube, or primary peritoneal ovarian cancer which were in complete or partial response to Pt-based CT in adults
• The NMPA's Priority Review is granted to the drugs to accelerate drug's registration and approval in China for multiple therapy areas
• Zejula (niraparib,ZL-2306) is a qd poly (ADP-ribose) PARP1/2 inhibitor and received FDA & EMA approval for women with recurrent epithelial ovarian, fallopian tube, primary peritoneal cancer in Mar,2017 & Nov,2017 respectively. In Sept, 2016 Zai Lab in-licensed Zejula from Tesaro to commercialize it in China

6. Paige.AI Received FDA's Breakthrough Designation for its Artificial Intelligence in Pathology

• FDA granted BT designation to Paige.AI to develop AI in computational pathology for the diagnosis and treatment of cancer including prostate cancer
• The FDA's BT designation is granted for technologies that provide effective diagnosis and treatment for life-threatening diseases
• Paige.AI is based on Fuch's technology, works with the de-identified dataset to develop AI portfolio targeting multiple cancer across the world

7. Lupin and YL Biologics Etanercept Biosimilar (YLB113) Received PMDA's Approval for Moderate-to-Severe Rheumatoid Arthritis (RA) and Juvenile Idiopathic Arthritis (JIA) in Japan

• The approval was based on global P-III trial results assessing etanercept biosimilar (YLB113) vs Enbrel in 500 patients with RA across 11 countries including Japan, EU, and India, evaluating its efficacy and safety
• In Mar, 2018 Lupin ad YLB filled NDA based on the above study results to Pharmaceutical and Medical Devices Agency (PMDA) in Japan
• YLB113 is a biosimilar of Amgen's Enbrel, targeted globally for severe autoimmune disorders including RA & JIA. Lupin also has two oncology biosimilars Lupifil (filgrastim) and Lupifil-P (peg-filgrastim)

8. Novartis Mayzent (siponimod) Received FDA's Approval for Patients with Secondary Progressive Multiple Sclerosis (SPMS)

• The approval was based on P-III EXPAND study results assessing Mayzent vs PBO in 1,651 patients with SPMS from 31 countries aged 48yrs.
• P-III study results: reduction in risk of three-month & six-month confirmed disability progression (CDP) 21% & 26%, 33% reduction with relapse activity in the two years, 55% reduction in annualized relapse rate (ARR), limited increase of T2 lesion volume by ~80, 89% free from gadolinium-enhancing lesions

9. Roche Acquired Spark Therapeutics for $4.3B

• Roche acquired Spark Therapeutics, in an all-stock transaction for $114.50 /share in cash, making the total deal value $4.3B. Spark to receive a premium of 122% based on its last day closing share price value i.e., 22 February 2019
• The focus of the acquisition was to expand Roche's footprints in genetic disorders including blindness, hemophilia, lysosomal storage disorders, and neurodegenerative diseases
• Spark Therapeutics portfolio included SPK-8011 & SPK-8016 for hemophilia A, SPK-9001 for haemophilia B, SPK-3006 for Pompe disease, SPK-1001 for CLN2 disease and Luxturna (voretigene neparvovec'rzyl) approved for confirmed biallelic RPE65 mutation-associated retinal dystrophy. The transaction is expected to close in the Q2'19    

10. Merck Acquired Immune Design for its Technologies for $300M

• Merck acquired Immune Design, in all stock transaction for $5.85/share in cash making total deal value ~ $300M. The transaction was expected to close in Q2'19
• The focus of the agreement was to develop and commercialize immune vaccines using Immune Design's in vivo technologies i.e., GLAAS and ZVex against cancer and infectious diseases
• GLA Adjuvant Systems (GLAAS) is an in vivo Glucopyranosyl Lipid A (GLA) based platform that binds TLR4 receptor activating DCs to produce cytokines and chemokines activating Th1-type immune response. ZVex is a delivery system designed for developing T-Cells targeting tumor cells 

11. Astellas Signed a Research and License Agreement with Xencor for Bispecific Antibody Program in Oncology 

• Merck acquired Immune Design, in all stock transaction for $5.85/share in cash making total deal value ~ $300M. The transaction is expected to close in Q2'19
• The focus of the agreement was to develop and commercialize immune vaccines using Immune Design's in vivo technologies i.e., GLAAS and ZVex against cancer and infectious diseases
• GLA Adjuvant Systems (GLAAS) is an in vivo Glucopyranosyl Lipid A (GLA) based platform that binds TLR4 receptor activating DCs to produce cytokines and chemokines activating Th1-type immune response. ZVex is a delivery system designed for developing T-Cells targeting tumor cells    

12. Fujifilm Acquired Biogen's Danish Biologics Manufacturing Facility for $890M

• Fujifilm acquired Biogen's subsidiary having the production capacity of 90,000L biologics, in all-stock transaction for $890M. The acquisition is expected to be complete in H2'19
• The focus of this acquisition was to expand Fujifilm's bio CDMO business. It will provide Biogen a tax relaxation. As a part of the transaction, Biogen also signed a service agreement with Fujifilm to develop products for Biogen including Tysabri
• Biogen developed a new manufacturing facility in Solothurn, Switzerland by H2'20 after the closing of this transaction

13. Gilead Reported Results of Biktarvy (Bictegravir, Emtricitabine, Tenofovir Alafenamide) in P-III Study 1490 for Patients with HIV-1

• The P-III 1490 Study involved an assessment of Biktarvy (BIC 50/FTC 200/TAF 25mg tablets) vs dolutegravir and emtricitabine/tenofovir alafenamide (50 mg) (DTG+FTC/TAF) in ratio (1:1) patients with HIV-1 naive to HIV therapy in adults for 96 wks.
• P-III Study 1490 results: HIV-1 RNA levels less than 50 copies/mL 95%; well-tolerated with low discontinuations @48 wks. & 96wks. (1%, 2% vs 4%, 2%) presented at HIV Glasgow 2018. The ongoing 1490 trial will continue for 144 wks.
• Biktarvy includes a boxed warning regarding its discontinuation in the US product label for risk of severe acute exacerbations of hepatitis B in patients with HIV-1 and HBV and is available in Australia, Canada, EU, Hong Kong, and the US

14. Takeda Signed a Multi-Target Agreement with StrideBio to Develop Gene Therapies for Neurological Disorders

• StrideBio received $30M upfront & near term pre-clinical milestones, $680M as development and commercial milestones & royalties on global sales of products. Takeda to get rights for clinical development and commercialization of selected products developed
• StrideBio will take care of preclinical development & manufacturing of in vivo AAV based gene-therapies for Takeda's three targets including Friedreich's Ataxia (FA)
• StrideBio's gene therapy technology is used to develop adeno-associated viral (AAV) therapies for rare diseases able to evade pre-existing antibodies. In 2018, Takeda Ventures funded Stride Bio for the development of AAV technology

15. Immunophotonics Signed a Research Agreement with Clinical Laserthermia Clinical Laserthermia Systems for Cancer Patients with Solid Tumors

• Immunophotonics and CLS collaborated to evaluate the safety & tolerability of Immunophotonics IP-001 in P-Ib/IIa study for cancer patients with solid tumors
• The collaboration will combine CLS Immunostimulating Interstitial Laser Thermotherapy (imILT) method & TRANBERG Laser Thermal Therapy System with intra-tumoral injection of IP-001 
• Immunophotonics IP-001 is designed to be injected intratumorally followed by an interventional oncology procedure i.e., tumor ablation, inducing a polyclonal T cell response against the cancer

16. Mitsubishi Tanabe Pharma (MTPA) Collaborated with Massachusetts General Hospital (MGH) for ALS Biomarker Study

• Mitsubishi Tanabe Pharma (MTPA) entered a collaboration with Massachusetts General Hospital (MGH) Neurological Clinical Research Institute (NCRI) to conduct ALS biomarker study for identification of biomarkers in patients with amyotrophic lateral sclerosis (ALS)
• The ALS biomarker study evaluated 4 biomarkers enrolling 200 patients receiving Radicava (edaravone) for 24wks. across 40 sites and was conducted & sponsored by MGH & MTPA respectively with its expected interim analyses in H2'19
• RADICAVA (edaravone, IV) is a neuroprotective agent indicated for the treatment of ALS and received approval in the US, Japan, and South Korea in May'17, Jun'15 & Dec'15 respectively for ALS

17. Gilead's Biktarvy (Bictegravir, Emtricitabine and Tenofovir Alafenamide) Received MHLW's Approval for HIV-1 Infection in Japan

• The approval was based on four P-III 1489 and 1490 & 1844 and 1878 studies result assessing Biktarvy vs PBO in 2,415 naive patients with HIV-1 infected & virologically suppressed adults respectively
• The P-III 1489 and 1490 & 1844 and 1878 studies results: @48wks. met its 1EPs; no treatment-emergent virologic resistance; no discontinuation; no cases of proximal renal tubulopathy/Fanconi syndrome; the high barrier to resistance
• Biktarvy (qd) is an INSTI-based triple-therapy STR combining bictegravir 50mg/emtricitabine 200mg/tenofovir alafenamide 25mg (BIC/FTC/TAF) and has a boxed warning for its discontinuation of post treatment acute exacerbation of Hepatitis B

18. Apple Launched its Watch Series 4 Enabled with ECG App and Irregular Rhythm Notification in Europe and Hong Kong

• The ECG app on Apple Watch Series 4 was evaluated in 600 patients resulted in 98.3% sensitivity in classifying AFib, 99.6% specificity in classifying sinus rhythm, 87.8% of recordings classified by the ECG app
• The irregular rhythm notification function was assessed in the Apple Heart study enrolled with 400,000 candidates and demonstrated 0.5% irregular pulse notifications, identified heart rate irregularities with confirmed AFib 
• Watch Series 4 with OS 5.2 possess health app with irregular rhythm notification enables customers to produce ECG on demand & check user's heart rhythm using the optical heart sensor, available in 19 EU countries and has received FDA's De Novo clearance in the US

19. AstraZeneca Signed a Worldwide Development and Commercialization Agreement with Daiichi Sankyo for $6.9B

• Daiichi Sankyo received $1.35B upfront, $5.55B as regulatory & commercial milestones making the total deal value $6.9B. AstraZeneca got WW co-development and co-commercialization rights for Daiichi's trastuzumab deruxtecan (Ex- Japan) with equal cost & profit sharing
• The focus of the collaboration was to develop trastuzumab deruxtecan for HER2+ breast and gastric cancer with the expansion of AstraZeneca's Oncology portfolio
• Trastuzumab deruxtecan (DS-8201) is ADC designed to deliver CT to cancer cells and has received FDA's BT designation & MHLW's Sakigake designation for HER2+ advanced or m-breast cancer & HER2-positive advanced gastric or gastroesophageal junction respectively with its expected FDA filling in Q2'19

20. Evolus Jeuveau (prabotulinumtoxinA-xvfs) Received FDA Approval for Moderate-to-Severe Glabellar Lines in Adults

• The approval was based on four P-III trials including two (EV-001 & EV-002) US trials and European & Canadian i.e., TRANSPARENCY study results assessing Jeuveau vs PBO & Botox respectively in 2,100 patients with glabellar lines due to corrugator or procerus muscle activity in adults
• The TRANSPARENCY study collectively resulted in meeting 1EPs (67.5%, 70.4% vs 1.2%, 1.3%) & demonstrated safe and effective results
• Jeuveau (prabotulinumtoxinA-xvfs) injection is a neurotoxin, Type A formulation of 900 kDa purified botulinum toxin developed using Hi-Pure technology with its expected marketing in H1'19