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

Editor’s Note

This is the final edition of Biopharmaceutical NewsWeek of 2016. It has been a great, sometimes challenging and even difficult year for some of us and we, therefore, want to thank all of you for your continuous support. We wish all our subscribers and readers a Merry Christmas and a Happy and Successful Year 2017.

Mergers, Acquisitions and Joint Ventures

Johnson & Johnson is again in exclusive talks with Actelion in an attempt to acquire the Swiss company. Just five days after J&J indicated it had given up on making a deal with Actelion, the US giant is back at the negotiation table and sidelining Sanofi from the bidding process. Sources were telling Bloomberg that the contingent value right, or CVR, Sanofi has included is worth about $20 of the $275 price per Actelion share, amounting to a $2 billion bonus if certain pipeline drugs were as successful as Actelion CEO Jean-Paul Clozel suggests.  It was price that led J&J to walk away from negotiations with Actelion last week, giving Sanofi an opening to walk in. But when J&J withdrew, so did Actelion’s share price, which fell about 7%. Sources are telling Reuters that Clozel may now be in a place where he has to make a deal or risk that angry investors will take steps to replace the Actelion board with members that will make a deal. Investors are now hoping to see the deal tied up before New Year’s Eve.

Lonza (Basel, Switzerland) has agreed to acquire Capsugel (Morristown, NJ, USA) for $5.5 billion in cash, an acquisition which will expand the Swiss firm’s drug delivery business. Pfizer sold Capsugel to Kohleberg Kravis Roberts & Co for $2.4 billion back in 2011.

Allergan (Dublin, Irland) acquires Life Cell Corporation (Bridgewater, NJ, USA), a regenerative medicine company for $2.9 billion from Acelity LP in cash. The acquisition combines LifeCell's novel, regenerative medicines business, including its high-quality and durable portfolio of dermal matrix products with Allergan's leading portfolio of medical aesthetics, breast implants and tissue expanders. LifeCell's commercial portfolio features Acellular Dermal Matrices, commonly used in breast reconstruction procedures and complex hernia surgeries to provide soft tissue support such as ALLODERM, REVOLVE, STRATTICE™ and ARTIA™.

Harbour BioMed (Shanghai, China) acquires Harbour Antibodies BV (Rotterdam, The Netherlands) in cash and equity to create a new, global oncology-focused biotechnology company. The acquisition was financed by Harbour BioMed investors and by Advantech Capital and Legend Capital, two leading China-focused investors with a particular emphasis on life sciences. The company is led by Jingsong Wang, and Liang Schweizer, two former Sanofi Asia R&D leaders. Specific terms of the deal were not disclosed.

Northern Biologics (Toronto, Canada) merges with Mosaic Biomedicals (Barcelona, Spain) to become a global cancer company. The company has been backed by Versant Ventures and Celgene which gains rights to MSC-1, a first-in-class antibody that targets leukemia inhibitory factor (LIF), a pleiotropic cytokine that is overexpressed in certain solid tumors. LIF promotes cancer progression by regulating the tumor microenvironment and by inducing self-renewal in tumor-initiating cells. Specific terms of the deal were not disclosed.

Business

Incyte (Wilmington, DEL, USA) enters a collaboration with Merus (Utrecht, The Netherlands) to develop specific antibodies using Merus’ Biclonics technology platform. Biclonics are bispecific, full-length human immunoglobulin G (IgG) antibodies, which Merus claims demonstrate better tumor-killing functionality than conventional monoclonal antibody combinations. Under the terms of the deal, Incyte will get exclusive rights to as many as 11 bispecific antibody programs including two of Merus’ existing preclinical immune-oncology programs, and will pay Merus an upfront fee of $120 million plus an equity investment of $80 million, as well as up to $350 million in milestones tied to development and marketing of eight of the programs.

Editas Medicine (Cambridge, MA, USA), a leading genome editing company, announced it has exclusively licensed intellectual property related to new CRISPR technologies for human therapeutics. These global licensing agreements include intellectual property owned by the Broad Institute of MIT and Harvard (Broad Institute), Harvard University, Massachusetts Institute of Technology (MIT), Wageningen University, the University of Iowa, and the University of Tokyo for the new CRISPR genome editing system known as Cpf1, advanced forms of Cas9, and additional Cas9-based genome editing technologies.

Portola Pharmaceuticals receives an unsecured $50 million loan from Pfizer and Bristol-Myers Squibb that will fund the continued development of AndeXa, or andexanet alfa, a Factor Xa antidote. Under the terms of the agreement, each company will loan Portola $25M with principal and interest to be repaid through royalties on AndexXa commercial sales.

Approval of drugs and vaccines

US FDA approves Clovis Onclogy’s (Boulder, COL, USA) Rubraca, a PARP Inhibitor, as a treatement of ovarian cancer patients who have the BRCA gene mutation and who’ve already had two or more chemotherapies. The approval came more than two months ahead of the Feb. 23 PDUFA date under its accelerated approval program. Priced at at $6,870 for a 15-day supply of Rubraca at both the 300 mg and 200 mg dosage strengths, the drug will challenge AstraZeneca’s Lynparza, approved in advanced ovarian cancer back in 2014.

The US FDA has removed the black-box warning from Pfizer’s Chantix, or varenicline, a nicotinic receptor partial agonist, after weighing data from the seven years’ EAGLES postmarketing study showing that Chantix was not associated with a higher rate of severe neuropsychiatric event. In its statement, the FDA said the “risk of serious side effects on mood, behavior, or thinking … is lower than previously suspected,” though still present in the stop-smoking drugs. Following the EAGLES analysis, the FDA also removed language detailing “serious mental health side effects” from GSK’ Zyban’s label warning; the drug will nevertheless continue to feature a black box.

Drugs at clinical stage

US FDA grants fast-track designation :

to Astellas’s and US partner Immunomic Therapeutics’ (Lancaster, PA, USA) ASP0892 a new DNA vaccine candidate based on the LAMP-Vax platform for the treatment of peanut allergies. According to a 2011 study, about 1.4% of children in the U.S. are allergic to peanuts, while the rate in adults is 0.6%

…and to Apellis Pharmaceuticals’ (Louisville, KY, USA) APL-2, an investigational treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematopoietic stem cell disorder disorder in which red blood cells break apart prematurely. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation with a particularly high potency.

Medical Devices and Diagnosis News.

US FDA approves Alcon’s AcrySof IQ ReSTRO +3.0D multifocal toric intraocular lens for cataract patients. The product is already approved in the EU, Australia, Canada and certain countries in Central and South America as well as in Asia.

Science and Technology

Researchers from The University of Texas Medical Branch at Galveston have developed the first vaccine for chikungunya fever made from an insect-specific virus that doesn't have any effect on people, making the vaccine safe and effective. The newly developed vaccine quickly produces a strong immune defense and completely protects mice and nonhuman primates from disease when exposed to the chikungunya virus.

Cost, Pricing and Market Access

Intelligence Provider GBI Research (London, UK) predicts that the market for immunotherapies is set to grow more than fourfold from $16.9 billion in 2015 to $75.8 billion by 2022. According to this market report, there are currently 2,037 products in active development in the cancer immunotherapy pipeline, accounting for 37% of the entire oncology pipeline However, "although the cancer immunotherapy pipeline is clearly strong, a significant proportion is made up of early-stage products, namely those from the Discovery stage to Phase I. This is indicative of the difficulty associated with progressing cancer immunotherapies from preclinical laboratory studies to clinical application in humans", noted Yasser Mushtaq, a senior analyst at GBI.

GBI Research also predicts that global revenues for the neurodegenerative disorders market, which covers Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, Huntington’s disease and multiple sclerosis, is set to grow at a compound annual growth rate of 7.42%, from $27.2 billion in 2015 to $45 billion in 2022. The neurodegenerative disorders pipeline is large and diverse, and contains 1,494 products The report states that much of this growth is expected to occur between 2018 and 2022, due to the approval of many pipeline products within these years. Indeed, several late-stage products are expected to be highly commercially successful, including ocrelizumab, ozanimod, solanezumab, ITI-007 and RG-7412.

US Senate Democrats urge Trump to work with them on drug prices: In a letter dated dated December 20, the 19 senators named five areas for cooperation: allowing the Medicare program to negotiate prescription prices, increasing transparency, stopping abusive pricing, passing reform on incentives for innovation and supporting generic competition for branded drugs.
 

Miscellaneous

The Japanese Pharmaceutical and Medical Devices Agency (PDMA) will share API manufacturing facility inspection data with European regulators in order “to allow more sites to be monitored and reduce unnecessary duplication”.

People on the move:

Biogen is promoting Chief Commercial Officer Michel Vounastos as its new CEO replacing outgoing CEO George Scangos. Vounastos, a French native arrived at Biogen in March after a stay of 20 years at Merck.

Innate Pharma (Marseille, France) announces key leadership and corporate governance changes:

• Mondher Mahjoubi, former AstraZeneca’s head Oncology is appointed as the new CEO
• Hervé Brailly, co-founder of Innate and current CEO is appointed Chairman of the Supervisory Board replacing Gilles Brisson who remains at the Board
• Philippe Pouletty resigns from the Board
• Laure-Hélène Mercier is appointed as Chief Financial Officer.

Abivax (Paris, France), an innovative biotechnology company targeting the immune system to eliminate viral diseases, appoints Didier Blondel as Chief Financial Officer, effective January 2, 2017. Mr Blondel was CFO at Sanofi Pasteur MSD in Lyon and held senior finance position at Sanofi in Commercial Operations and R&D.

Bioevents

9^th Biotech Showcase Investor Conference on January 9-11, in San Francisco (USA)

JP Morgan Health Conference on January 9-12, in San Francisco (USA)

BIO CEO & Investor Conference on February 13-17, 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)

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

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)

This document has been prepared by btobioinnovation and is provided to you for information purposes only.  The information contained in this document has been obtained from sources that btobioinnovation believes are reliable but btobioinnovation does not warrant that it is accurate or complete. The views presented in this document are those of btobioinnovation’s editor at the time of writing and are subject to change.  btobioinnovation has no obligation to update its opinions or the information in this document.

Biopharmaceutical News Week # 2016.51

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

Editor’s Note

This is the final edition of Biopharmaceutical NewsWeek of 2016. It has been a great, sometimes challenging and even difficult year for some of us and we, therefore, want to thank all of you for your continuous support. We wish all our subscribers and readers a Merry Christmas and a Happy and Successful Year 2017.

Mergers, Acquisitions and Joint Ventures

Johnson & Johnson is again in exclusive talks with Actelion in an attempt to acquire the Swiss company. Just five days after J&J indicated it had given up on making a deal with Actelion, the US giant is back at the negotiation table and sidelining Sanofi from the bidding process. Sources were telling Bloomberg that the contingent value right, or CVR, Sanofi has included is worth about $20 of the $275 price per Actelion share, amounting to a $2 billion bonus if certain pipeline drugs were as successful as Actelion CEO Jean-Paul Clozel suggests.  It was price that led J&J to walk away from negotiations with Actelion last week, giving Sanofi an opening to walk in. But when J&J withdrew, so did Actelion’s share price, which fell about 7%. Sources are telling Reuters that Clozel may now be in a place where he has to make a deal or risk that angry investors will take steps to replace the Actelion board with members that will make a deal. Investors are now hoping to see the deal tied up before New Year’s Eve.

Lonza (Basel, Switzerland) has agreed to acquire Capsugel (Morristown, NJ, USA) for $5.5 billion in cash, an acquisition which will expand the Swiss firm’s drug delivery business. Pfizer sold Capsugel to Kohleberg Kravis Roberts & Co for $2.4 billion back in 2011.

Allergan (Dublin, Irland) acquires Life Cell Corporation (Bridgewater, NJ, USA), a regenerative medicine company for $2.9 billion from Acelity LP in cash. The acquisition combines LifeCell's novel, regenerative medicines business, including its high-quality and durable portfolio of dermal matrix products with Allergan's leading portfolio of medical aesthetics, breast implants and tissue expanders. LifeCell's commercial portfolio features Acellular Dermal Matrices, commonly used in breast reconstruction procedures and complex hernia surgeries to provide soft tissue support such as ALLODERM, REVOLVE, STRATTICE™ and ARTIA™.

Harbour BioMed (Shanghai, China) acquires Harbour Antibodies BV (Rotterdam, The Netherlands) in cash and equity to create a new, global oncology-focused biotechnology company. The acquisition was financed by Harbour BioMed investors and by Advantech Capital and Legend Capital, two leading China-focused investors with a particular emphasis on life sciences. The company is led by Jingsong Wang, and Liang Schweizer, two former Sanofi Asia R&D leaders. Specific terms of the deal were not disclosed.

Northern Biologics (Toronto, Canada) merges with Mosaic Biomedicals (Barcelona, Spain) to become a global cancer company. The company has been backed by Versant Ventures and Celgene which gains rights to MSC-1, a first-in-class antibody that targets leukemia inhibitory factor (LIF), a pleiotropic cytokine that is overexpressed in certain solid tumors. LIF promotes cancer progression by regulating the tumor microenvironment and by inducing self-renewal in tumor-initiating cells. Specific terms of the deal were not disclosed.

Business

Incyte (Wilmington, DEL, USA) enters a collaboration with Merus (Utrecht, The Netherlands) to develop specific antibodies using Merus’ Biclonics technology platform. Biclonics are bispecific, full-length human immunoglobulin G (IgG) antibodies, which Merus claims demonstrate better tumor-killing functionality than conventional monoclonal antibody combinations. Under the terms of the deal, Incyte will get exclusive rights to as many as 11 bispecific antibody programs including two of Merus’ existing preclinical immune-oncology programs, and will pay Merus an upfront fee of $120 million plus an equity investment of $80 million, as well as up to $350 million in milestones tied to development and marketing of eight of the programs.

Editas Medicine (Cambridge, MA, USA), a leading genome editing company, announced it has exclusively licensed intellectual property related to new CRISPR technologies for human therapeutics. These global licensing agreements include intellectual property owned by the Broad Institute of MIT and Harvard (Broad Institute), Harvard University, Massachusetts Institute of Technology (MIT), Wageningen University, the University of Iowa, and the University of Tokyo for the new CRISPR genome editing system known as Cpf1, advanced forms of Cas9, and additional Cas9-based genome editing technologies.

Portola Pharmaceuticals receives an unsecured $50 million loan from Pfizer and Bristol-Myers Squibb that will fund the continued development of AndeXa, or andexanet alfa, a Factor Xa antidote. Under the terms of the agreement, each company will loan Portola $25M with principal and interest to be repaid through royalties on AndexXa commercial sales.

Approval of drugs and vaccines

US FDA approves Clovis Onclogy’s (Boulder, COL, USA) Rubraca, a PARP Inhibitor, as a treatement of ovarian cancer patients who have the BRCA gene mutation and who’ve already had two or more chemotherapies. The approval came more than two months ahead of the Feb. 23 PDUFA date under its accelerated approval program. Priced at at $6,870 for a 15-day supply of Rubraca at both the 300 mg and 200 mg dosage strengths, the drug will challenge AstraZeneca’s Lynparza, approved in advanced ovarian cancer back in 2014.

The US FDA has removed the black-box warning from Pfizer’s Chantix, or varenicline, a nicotinic receptor partial agonist, after weighing data from the seven years’ EAGLES postmarketing study showing that Chantix was not associated with a higher rate of severe neuropsychiatric event. In its statement, the FDA said the “risk of serious side effects on mood, behavior, or thinking … is lower than previously suspected,” though still present in the stop-smoking drugs. Following the EAGLES analysis, the FDA also removed language detailing “serious mental health side effects” from GSK’ Zyban’s label warning; the drug will nevertheless continue to feature a black box.

Drugs at clinical stage

US FDA grants fast-track designation :

to Astellas’s and US partner Immunomic Therapeutics’ (Lancaster, PA, USA) ASP0892 a new DNA vaccine candidate based on the LAMP-Vax platform for the treatment of peanut allergies. According to a 2011 study, about 1.4% of children in the U.S. are allergic to peanuts, while the rate in adults is 0.6%

…and to Apellis Pharmaceuticals’ (Louisville, KY, USA) APL-2, an investigational treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematopoietic stem cell disorder disorder in which red blood cells break apart prematurely. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation with a particularly high potency.

Medical Devices and Diagnosis News.

US FDA approves Alcon’s AcrySof IQ ReSTRO +3.0D multifocal toric intraocular lens for cataract patients. The product is already approved in the EU, Australia, Canada and certain countries in Central and South America as well as in Asia.

Science and Technology

Researchers from The University of Texas Medical Branch at Galveston have developed the first vaccine for chikungunya fever made from an insect-specific virus that doesn't have any effect on people, making the vaccine safe and effective. The newly developed vaccine quickly produces a strong immune defense and completely protects mice and nonhuman primates from disease when exposed to the chikungunya virus.

Cost, Pricing and Market Access

Intelligence Provider GBI Research (London, UK) predicts that the market for immunotherapies is set to grow more than fourfold from $16.9 billion in 2015 to $75.8 billion by 2022. According to this market report, there are currently 2,037 products in active development in the cancer immunotherapy pipeline, accounting for 37% of the entire oncology pipeline However, "although the cancer immunotherapy pipeline is clearly strong, a significant proportion is made up of early-stage products, namely those from the Discovery stage to Phase I. This is indicative of the difficulty associated with progressing cancer immunotherapies from preclinical laboratory studies to clinical application in humans", noted Yasser Mushtaq, a senior analyst at GBI.

GBI Research also predicts that global revenues for the neurodegenerative disorders market, which covers Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, Huntington’s disease and multiple sclerosis, is set to grow at a compound annual growth rate of 7.42%, from $27.2 billion in 2015 to $45 billion in 2022. The neurodegenerative disorders pipeline is large and diverse, and contains 1,494 products The report states that much of this growth is expected to occur between 2018 and 2022, due to the approval of many pipeline products within these years. Indeed, several late-stage products are expected to be highly commercially successful, including ocrelizumab, ozanimod, solanezumab, ITI-007 and RG-7412.

US Senate Democrats urge Trump to work with them on drug prices: In a letter dated dated December 20, the 19 senators named five areas for cooperation: allowing the Medicare program to negotiate prescription prices, increasing transparency, stopping abusive pricing, passing reform on incentives for innovation and supporting generic competition for branded drugs.
 

Miscellaneous

The Japanese Pharmaceutical and Medical Devices Agency (PDMA) will share API manufacturing facility inspection data with European regulators in order “to allow more sites to be monitored and reduce unnecessary duplication”.

People on the move:

Biogen is promoting Chief Commercial Officer Michel Vounastos as its new CEO replacing outgoing CEO George Scangos. Vounastos, a French native arrived at Biogen in March after a stay of 20 years at Merck.

Innate Pharma (Marseille, France) announces key leadership and corporate governance changes:

• Mondher Mahjoubi, former AstraZeneca’s head Oncology is appointed as the new CEO
• Hervé Brailly, co-founder of Innate and current CEO is appointed Chairman of the Supervisory Board replacing Gilles Brisson who remains at the Board
• Philippe Pouletty resigns from the Board
• Laure-Hélène Mercier is appointed as Chief Financial Officer.

Abivax (Paris, France), an innovative biotechnology company targeting the immune system to eliminate viral diseases, appoints Didier Blondel as Chief Financial Officer, effective January 2, 2017. Mr Blondel was CFO at Sanofi Pasteur MSD in Lyon and held senior finance position at Sanofi in Commercial Operations and R&D.

Bioevents

9^th Biotech Showcase Investor Conference on January 9-11, in San Francisco (USA)

JP Morgan Health Conference on January 9-12, in San Francisco (USA)

BIO CEO & Investor Conference on February 13-17, 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)

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

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)

This document has been prepared by btobioinnovation and is provided to you for information purposes only.  The information contained in this document has been obtained from sources that btobioinnovation believes are reliable but btobioinnovation does not warrant that it is accurate or complete. The views presented in this document are those of btobioinnovation’s editor at the time of writing and are subject to change.  btobioinnovation has no obligation to update its opinions or the information in this document.