Biopharmaceutical News Week 52

CRISP Technology: Breakthrough of the Year 2015

Every December, the staff of Science singles out a significant achievement as the Breakthrough of the Year. This year they have selected CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats), occurring in the genome of certain bacteria, from which the system was discovered. CRISPR has already become a rapid and easy to use gene editing technology that can selectively delete, modify or correct a specific DNA segment. In 2012, Emmanuelle Charpentier and Jennifer Doudna described how the Cas9 endonuclease works together with crRNA and tracrRNA to form functional molecular scissors to cut at a specific DNA sequence in the genome^.Cas9 is a CRISPR-associated endonuclease (an enzyme), the “molecular scissors” that are easily programmed to cut and edit, or correct, disease-associated DNA in a patient’s cell. The location at which the Cas9 molecular scissors cut the DNA to be edited is specified by guide RNA, which is comprised of a crRNA component and a tracrRNA component, either individually or combined together as a “single guide RNA” (sgRNA). For example, a guide RNA can direct the molecular scissors to cut the DNA at the exact site of the mutation present in the genome of patients with a particular genetic disease. Once the molecular scissors make a cut in the DNA, additional cellular mechanisms and exogenously added DNA will use the cell’s own machinery and other elements to specifically “repair” the cut DNA. Earlier this month, at the International Summit on Human Gene Editing held in Washington DC, members of national scientific academies discussed about the ethical questions raised by recent advances in CRISPR-Cas9 mediated human genome editing. In conclusion, they agreed to proceed further with basic and clinical research under appropriate legal and ethical guidelines.

From CRISPR Therapeutics http://crisprtx.com/our-programs/crispr-cas9-gene-editing.php

Acquisitions /mergers/joint-ventures

Porsolt SAS (Le Genest-Saint-Isle, France), a preclinical in vivo efficacy and safety CRO, acquires Fluofarma (Pessac, France) to increase its non-animal cell based disease models for predictive pharmacology and toxicology studies, for an undisclosed amount.

Business

Bayer and CRISPR Therapeutics (Cambridge, CA, USA and Basel, Switzerland) have launched a joint-venture to discover, develop and commercialize new therapies to treat blood disorders, blindness and congenital heart disease. The joint venture is designed to combine CRISPR-Cas9 gene editing technology (see above) with Bayer’s protein engineering and disease expertise. Under the terms of the deal Bayer will pay a minimum $300 million in R&D investment toward the joint venture over five years and invest $35 million in cash to acquire a minority stake in CRISPR Therapeutics. Vertex Pharmaceuticals had already struck a deal and made an equity investment in CRISPR Therapeutics in October 2015.

Novartis has signed up as a strategic investment in Seventure Partner’s (Paris, France) microbiome fund. Seventure, led by Isabelle Cremoux, has raised 33% more than initially targeted and with €160 billion the fund can back up to 25 microbiome companies from across three industries: biopharma, connected health including diagnostics and food and nutrition.

Janssen, a Johnson & Johnson company, has entered a collaboration with Intrexon (Germantown, MD, USA) to use its Actobiotis platform to develop oral delivery systems designed to treat diabetic patients. The pill delivers proteins and peptides to the oral and gastrointestinal tract using food-grade microbes called Lactococcus lactisengineered to contain the ActoBiotics drugs.

Elli Lilly partners with Halozyme Pharmaceuticals (San Diego, CA, USA) to use the Enhanze drug delivery platform which enables subcutaneous injections of drugs that would otherwise require intravenous injections. The Enhanze technology enables subcutaneous delivery by degrading hyaluronan, a component of the subcutaneous space located just under the skin and facilitates the passage of large molecules through the subcutaneous space. According to Halozyme the degradation is temporary because hyaluronan reestablishes itself after several days. Under the terms of the deal, Lilly will make a $25 million upfront payment as well as up to $160 million in milestone payments for each of up to 5 developed drugs using the technology.

Sanofi and MAB Discovery (Neuried, Germany) have entered into a research and license agreement in the field on monoclonal antibodies for selected targets of Sanofi.

Polyphor (Allschwil, Switzerland) and Boehringer Ingelheim extend their macrocycle R&D collaboration to discover new drugs for challenging targets.

Approval of drugs, vaccines, diagnostics and devices

US FDA approves:

AstraZeneca‘s Zurampic or lesinurad in combination with a xanthine oxidase inhibitor (XOI) such as febuxostat for the treatment of hyperuricemia or elevated levels of uric acid associated with gout. Lesinurad inhibits the function of transporter proteins involved in uric acid resorption in the kidneys, thereby helping them excrete uric acid. The approval includes a boxed warning about the risk of kidney failure and requires the company to conduct a postmarketing study to assess the renal and cardiovascular safety of Zurampic.

…..and Actelion’s Uptravi or selexipag to slow down the disease progression of patients with pulmonary arterial hypertension. Selexipag, which was discovered by Nippon Shinyaku, is a potent, orally available, selective prostacyclin IP receptor agonist.

US FDA extend the approval of Merck’s Keytruda or pembrolizumab as a first-line treatment for unresectable or metastic melanoma.

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended 93 new medicines in 2015.

Drugs at clinical stage

US FDA grants breakthrough status to Boehringer Ingelheim’s BI 1482694, a third generation an epidermal growth receptor mutation-specific tyrosine kinase inhibitor, as a treatment for advanced and pretreated EGRF mutation-positive non-small cell lung cancer.

ImmuPharma (London, UK and Mulhouse, France) announced the start of a phase 3 clinical trial with IPP-201101 or Lupuzor for the treatment of patients with systemic Lupus Erythematosus.

Science and Technology

Zika fever is a mosquito-borne viral disease caused by Zika virus (ZIKV), consisting of mild fever, rash, headaches, arthralgia, myalgia, asthenia, and non-purulent conjunctivitis, occurring about three to twelve days after the mosquito vector bite. Given the recent massive increase of congenital anomalies, Guillain-Barré syndrome, and other neurological and autoimmune syndromes in areas where Zika virus is circulating and their possible relation to the virus, the Pan American Health Organization / World Health Organization (PAHO/WHO) recommends its Member States establish and maintain the capacity to detect and confirm Zika virus cases and prepare healthcare facilities for the possible increase in demand at all healthcare levels and specialized care for neurological syndromes, and to strengthen antenatal care. Brazil has recently declared itself in an emergency situation after a massive raise in neurological syndromes in newborns in the last months.

A German-Dutch team of scientists has succeeded in immunizing dromedaries against the recently discovered coronavirus referred to as MERS (for Middle East Respiratory Syndrome) virus which causes an often fatal infectious respiratory disease in humans. As the camels appear to be the major reservoir of the virus, the vaccine should also reduce the risk of future outbreaks of the disease in humans.

Cost, Pricing and Market Access

According to an analysis by the Institute for Clinical and Economic Review (ICER), a US based cost watchdog, GlaxoSmithKline’s Nucala, an injectable drug indicated for severe asthma patients with eosinophilic inflammation, approved by the US FDA in November 2015, should cost between $7,800 and $12,000 per year, which is as much as 76% lower than the $32,500 price it bears right now.

Miscellaneous

The Innovative Medicines Initiative (IMI) the world largest public-private partnership in life sciences, structured by the European Federation of Pharmaceutical Industries and Associations (EFPIA), has launched two new calls. Call 7 with focus on clinical endpoints in dry age-related macular degeneration as well as targets to modulate misfolded proteins in Alzheimer’s and Parkinson’s diseases. Call 8 will focus focuses on proposals in the area of Ebola and other filoviral haemorrhagic fevers. The IMI will provide a budget of €70 million for the Ebola Call, while both the IMI and EFPIA companies will contribute €46.8 million towards Call 7.

Under the new legislation FDA will receive a $2.7 billion budget in discretionary funding, a $132 million increase over the FY2014. Specifically the bill includes a funding of $8.7 million for combating resistant bacteria, $5 million for the Precision Medicine Initiative, $2.5 million for orphan product development grants and $2.4 million for foreign inspections. Under the same bill the National Institutes of Health (NIH) will receive a $32 billion budget, $2 billion above the enacted 2015 level. The increase are specially focused on Alzheimer’s disease research, brain research, antibiotic research and the Precision Medicine Initiative.

Bioevents

BIOTech ShowCase 2016 on January 11-13 in San Francisco (USA)
JP Morgan 33^rd Annual Healthcare Conference on January 12-16 in San Francisco (USA)
BIO CEO Investor Conference on February 8-9 in New York City (USA)
BIO Asia 2016 on March 15-16 in Tokyo (Japan)
9^th European Life Science CEO Forum and Exhibition on March 15-16 in Zürich (Switzerland)
Global Healthcare Forum on March 16-17 in Singapore (Singapore)
BIOPharma Asia Convention on March 22-24 in Singapore (Singapore)
BIO Europe Spring on April 4-6 in Stockholm (Sweden)
BIO-IT World Conference & Expo’16 on April 5-7 in Boston (USA)
BIO Vision on April 13-14 in Lyon (France)
BIO Trinity on April 25-27 in London (UK)
BIOEquity Europe 2016 on May 10-11 in Copenhagen (Denmark)
Strategic Alliance Management Congress on May 16-18 in Philadelphia (USA)
China BIO Partnering on May 18-19 in Suzhou (China)
BIO International Convention on June 6-9 in San Francisco (USA)

And later in the year

BIO Pharm America on September 13-15 in Boston (USA)
7^th Innovation Days on October 3-4 in Paris (France)
Biofit 2015 on November 30-December 1 in Lille (France)

IMPORTANT NOTICE: 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’seditor at the time of writing and are subject to change. btobioinnovation has no obligation to update its opinions or the information in this document.

Author: Jean-Claude Muller, Special Advisor at I&IR, jcm@btobioinnovation.com

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