Amgen gets FDA OK for osteoporosis med

Amgen (NASDAQ:AMGN) announced that the U.S. Food and Drug Administration (FDA) has approved the use of Prolia^®(denosumab) for the treatment of glucocorticoid-induced osteoporosis (GIOP) in men and women at high risk of fracture, defined as a history of osteoporotic fracture, multiple risk factors for fracture, or patients who have failed or are intolerant to other available osteoporosis therapy. This approval is based on data from a Phase 3 study which showed patients on glucocorticoid therapy who received Prolia had greater gains in bone mineral density (BMD) compared to those who received active comparator (risedronate).

“As a leader in bone health with more than 20 years of osteoporosis research experience, we are pleased that Prolia will now be available for patients at high risk of fracture who are suffering from bone loss due to long-term glucocorticoid treatment,” said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. “This is a serious condition that leads to rapid decreases in bone mineral density and increased risk of fracture. This approval gives patients and physicians a new treatment option.”

“Patients on long-term systemic glucocorticoid medications can experience a rapid reduction in bone mineral density within a few months of beginning treatment¹,” said study lead Kenneth F. Saag, M.D., M.Sc., professor of medicine at the University of Alabama at Birmingham School of Medicine. “With this approval, patients who receive treatment with glucocorticoids now have a new option to help improve their bone mineral density.”

https://bit.ly/2LBADkZ

mAbxience, Amneal in Pact for US Avastin Biosimilar

mAbxience, a company specialized in the research, development, manufacturing and global commercialization of monoclonal antibody biosimilars, part of the Insud Pharma Group and Amneal Pharmaceuticals, Inc. (NYSE: AMRX), an integrated specialty pharmaceutical company powered by a robust U.S. generics business, have signed an exclusive licensing and supply agreement in the United States for mAbxience’s bevacizumab, a biosimilar candidate for Avastin(R).

Under the milestone based commercial agreement mAbxience will lead product, clinical development and subsequent manufacture while Amneal will guide the product through regulatory approval and have exclusive commercialisation rights in the United States.

“We are very proud to deepen our partnership with Amneal with the bevacizumab biosimilar agreement. We are passionate about increasing access and affordability of biologics in all countries around the world and this latest agreement with Amneal will be an important contribution to public health in the United States,” said Emmanuelle Lepine, General Manager of mAbxience.

mAbxience is progressing its international expansion, and recently established partnerships in Europe and Australia for its bevacizumab biosimilar. Building long lasting partnerships is central to the company’s business philosophy. Biosimilars bring patients a safe, effective, efficient and affordable treatment option, which contributes to the sustainability of healthcare systems worldwide. More than 10,000 patients have now been treated with a mAbxience biosimilar.

https://bit.ly/2xp9WwO

Willis Towers Watson: Health programs fragmented, fall short for Asia workers

• Over half of employees in Asia have elevated levels of stress.
• While employers endeavour to engage them in an increasing array of non-traditional benefits, there is still a disconnect with employees.
• Finding the ‘right’ programs and leveraging technology will be key as employers start to take a more strategic approach toward health

By and large, employers in Asia still miss the mark when it comes to their health and well-being benefits, with many employees feeling that their needs are not met, according to research from leading global advisory, broking and solutions company Willis Towers Watson (NASDAQ: WLTW).

According to the Willis Towers Watson 2017/2018 Global Benefits Attitudes Survey, managing their health is a top priority for two-thirds of employees (67%) in Asia. Moreover, over half (56%) say that they suffer from elevated levels of stress. There’s a role for employers, and many are endeavouring to respond by looking beyond traditional benefits and offering behavioural/health management programs (60%), lifestyle risk management programs (44%), and financial well-being programs (33%) to meet the diverse needs of employees.

However, even though 63% of employees say that their employer-sponsored health care plan meets their needs, only 41% say that their employer-sponsored health and well-being initiatives do so.

‘The danger is that, although well-intentioned, employers will use a scattergun approach and spend valuable resources on creating standalone and fragmented programs that employees do not value,’ said Dr. Amitabh Deka, Regional Consultant, Benefits & Wellness Advisory, Asia & Australia, at Willis Towers Watson. ‘The reality is that health and well-being has broad reaching business implications – employees in good health are more productive in the workplace, as they are more engaged, less stressed, and less likely to take days off. And as employers increasingly recognise this, they also start to approach health and well-being as a strategic issue.’

Employers start to take more strategic approach

It’s encouraging that the move towards being more strategic has already started among Asia employers. By next year, almost all (97%) of employers will have a health and well-being strategy (compared with 64% today) and almost three-quarters plan to differentiate and customise their strategy to make it a key competitive advantage.

‘Key to customisation of benefits will also be businesses recognising the interconnectedness between health and well-being issues. For example, from our research we know that an individual’s job, relationships, financials, and health are leading sources of stress, and therefore mental health,’ said Cedric Luah, Head of Health & Benefits, Asia and Australasia at Willis Towers Watson.

‘We suggest that employers rethink how their programs are designed and incentivise to create and encourage long-term behavioural changes. Part of this will also be ensuring that they are centred on the employee. Employees do not want to be told what to do; rather, they want to be supported with programs that lead to improved well-being. It’s an essential balance that’s not very easy to achieve.’

What employers can do

What steps can employers take to achieve this balance? We suggest the following:

1. Use medical claims and health risk data to establish a baseline and test the effectiveness of healthcare and well-being programs through financial and non-financial metrics. This approach ensures relevant changes to make programs effective. There’s a positive increase in the percentage of those who plan to use organisational or analytics to test the effectiveness of programs by 2019 (currently 20% to 81% by 2019).
2. It is critical to introduce the ‘right’ programs. Employers should first understand which health and well-being programs are valued by their employees, and what will generate positive outcomes rather than introduce one-off programs which could be costly and ineffective. Once you have a better understanding of your particular workforce, you can design your benefits to be more relevant as well as providing greater flexibility and choice.
3. Employers can leverage technology in their programs, policies and culture, as 62% of employees are already using it to manage their own health. Just over two in five (41%) use wearables to monitor fitness activity or sleep, while 40% use technology to monitor a health condition, and 34% to track eating habits.

‘While online tools help employees make better decisions about health choices, the real change will come from solutions that connect individuals and employers,’ noted Dr. Deka. ‘Technology can be key to integrate these programs that link multiple areas of an employee’s life and help to create a consumer-grade experience that is critical to attracting employees and sustaining employee engagement.’

About the Global Benefits Attitudes Survey

The Willis Towers Watson 2017/2018 Global Benefits Attitudes Survey measured attitudes of over 30,000 private sector employees in 22 countries – including 9,462 responses from Asia Pacific, specifically from Australia, China, Hong Kong, India, Japan, Philippines and Singapore. The survey was conducted in July and August 2017.

About the Asia Pacific Benefit Trends Survey

The 2017/2018 Asia Pacific Benefit Trends survey is the fifth in our biennial series. It focuses on high-level trends around benefit strategy, benefit design, health and wellness, retirement plans, benefit delivery and benefit cost. This year’s survey was conducted between April and June 2017, and received responses from over 1,141 large Asia Pacific employers.

https://bit.ly/2kylsg5

Casma Therapeutics Launches with $58.5 Million

Casma Therapeutics, located in Cambridge, Massachusetts, closed on a Series A round totaling $58.5 million. The company was solely funded by Third Rock Ventures.

The company will focus on stimulating autophagy to stop or reverse rare genetic diseases and other illnesses, including neurodegeneration. Autophagy is a natural process, where extra or abnormal proteins are broken down by the cells. Other materials are also broken down, including organelles like mitochondria, viruses and other pathogens.

The materials are then recycled as a source of energy or as materials for new structures. If autophagy is functioning abnormally, the materials accumulate, causing a cascade of potential problems related to numerous illnesses.

The company’s focus is built on the work of Tokyo Institute of Technology’s Institute of Innovative Research cell biologist Yoshinori Ohsumi, who won the Nobel Prize in Medicine in 2016. Ohsumi is not one of the company’s founders, but four other experts in autophagy are. They are Beth Levine, director of the Center for Autophagy Research at the University of Texas Southwestern Medical Center; Andrea Ballabio, founder and director of the Telethon Institute of Genetics and Medicine of Pozzuoli; James Hurley, Judy C. Webb Chair and professor of biochemistry, biophysics and structural biology of the University of California Berkeley; and Herbert Virgin, the current chief scientific officer and executive vice president for research of Vir Biotechnology.

The company’s chief executive officer is Keith Dionne, a serial entrepreneur. Leon Murphy,most recently at Novartis Institutes for BioMedical Research, is Casma’s senior vice president, biology. Frank Gentile is the interim chief operating officer. Bob Tepper is the interim chief scientific officer. He is the co-founder of Third Rock Ventures and previous president of R&D at Millennium Pharmaceuticals. Cary Pfeffer is the interim chief business officer, a partner at Third Rock.

Xconomy wrote, “Casma plans to use small molecule drugs to induce autophagy in situations where the process is either slowed down because of disease, or needs to speed up to deal with the buildup of a toxic substance. The goal: stop, or even reverse the course of these diseases, though Casma is a few years from human testing and will have to prove it can interfere with this cellular process without causing other problems. ‘We think we’re working on a fundamentally safe pathway,’ Dionne told Xconomy. ‘We just have to induce it in the correct way.’”

Dionne joined Third Rock Ventures as entrepreneur-in-residence in 2011. Prior to that he led Alantos Pharmaceuticals and Surface Logix, which were both sold. Prior to Casma, he was chief executive officer of Constellation Pharmaceuticals, which was formed by Third Rock in 2008. Dionne took over the job as chief executive in 2012, only two months after it had signed a broad alliance with Genentech that included a buyout option. That deal faded in 2015, with Dionne at the time saying he regretted that the deal with Genentech had ever been signed, because it probably prevented Constellation from launching an initial public offering.

Dionne left Constellation in 2016. The company is still privately held. Since then, Dionne has been incubating Casma. He’s excited to be making the decisions from the beginning, rather than taking over after many critical decisions have been made. He told Xconomy, “I wanted to do this from the beginning, when you really have the chance to mold the company from the very start.” His plan to do that “just took a hiatus during Constellation.”

https://bit.ly/2KWirkU

Escient Pharmaceuticals Launches with $40 Million

Escient Pharmaceuticals launched in San Diego with a Series A financing round worth $40 million. The round was syndicated by The Column Group and 5AM Ventures, and joined by Osage University Partners.

The new company will focus on developing fist-in-class G Protein-Coupled Receptor (GPCR)-targeted drugs. It will look at specific orphan GPCRs, including the family of Mas-Related G-Protein Receptors (Mrgprs) in neuro-immuno-inflammatory and autoreactive diseases.

“GPCR-targeted drugs account for a large percentage of today’s best-selling medicines,” said Alain Baron, the company’s co-founder and chief executive officer, in a statement. “And, yet, this broad success has hinged upon exploiting a relatively small set of GPCRs, leaving significant opportunity to mine the potential of numerous unexplored GPCRs, including the specific receptors we are focused on at Escient. We look forward to building a world-class team in the coming months dedicated to translating our proprietary insights about the functions and biology of these target GPCRs to develop novel therapies that address serious, unserved clinical needs of patients impacted by our lead indications.”

Baron is the chief executive officer, co-founder and president of Elcelyx Therapeutics. He previously was a Venture Partner at Lightstone Ventures.

Marcus Boehm is the company’s chief scientific officer. He was co-founder and former chief technology officer of Receptos until it was acquired by Celgene Corporation in 2015.

The company’s third co-founder is Xinzhong Dong, a Howard Hughes Medical Instituteinvestigator and professor of Neuroscience, Neurosurgery and Dermatology with the Solomon H. Snyder Department of Neuroscience at Johns Hopkins University School of Medicine.Dong will serve on the company’s Scientific Advisory Board.

The technical journal Nature indicates that about one third to half of all marketed drugs act by binding to GPCRs. Nature says, “GPCRs are a large family of cell surface receptors that respond to a variety of external signals. Binding of a signaling molecule to a GPCR results in G protein activation, which in turn triggers the production of any number of second messengers. Through this sequence of events, GPCRs help regulate an incredible range of bodily functions, from sensation to growth to hormone responses.”

Escient believes that it can develop drugs that target GPCRs whose function isn’t yet known. The company is staying mum about what specific targets or diseases it is targeting, although Baron told FierceBiotech, “We are excited about being able to pursue a novel class that has been quite mysterious. … We are excited to crack this nut and bring forth novel therapies for serious diseases.”

The company’s board of directors will include Robert Tjian, Discovery Partner at The Column Group and professor of Biochemistry and Molecular Biology at the University of California, Berkeley; JJ Kang, Principal at The Column Group; Andrew Schwab, founder and Managing Partner at 5AM Ventures; and Charles Zuker, professor of Biochemistry, Molecular Biophysics and Neuroscience at Columbia University and a Howard Hughes Medical Institute investigator.

“We quickly recognized the tremendous opportunity of the orphan GPCRs that Escient is targeting,” Tjian said in a statement. “Alain and Marcus’ GPCR-specific experience and previous entrepreneurial successes, coupled with Xinzhong’s indisputable scholarship in this area, well position Escient in this historically productive area of drug discovery and development.”

https://bit.ly/2L12tGo

$87M in Hand, Beam Therapeutics Launches CRISPR Base Editing Plan

Beam Therapeutics, a new precision genetic medicines company, launched this morning with $87 million in a Series A funding round backed by F-Prime Capital Partners and ARCH Venture Partners.

Although Beam did not lay out any specifics regarding its primary disease research, the company said it will focus its research on multiple DNA base editor platforms that were developed in the Harvard University lab of David Liu, as well as on the RNA base editor platform developed by Feng Zhang at the Broad Institute of MIT and Harvard. Beam said it will be the first company to pursue development of new therapies using CRISPR base editing technology.

DNA, Beam said, is made up of billions of nucleobases, or “bases,” each represented by a single letter (A, G, T, C). These letters are subsequently encoded in RNA messages for expression by the cell. Base editors are capable of precisely targeting and directly editing just one base out of billions within the genome, without cutting the DNA or RNA, the company said. The method is more precise than the more common CRISPR programs that cut and edit, or correct, disease-associated DNA in a cell. The base editing technology allows for the rewriting of a mutation in the DNA.

In its coming out announcement, Beam said it will use the different technologies to “generate a broad pipeline of precision genetic medicines that repair disease-causing point mutations, write in protective genetic variations, or modulate the expression or function of disease-causing genes.”

Chief Executive Officer John Evans, most recently head of Corporate Development and Portfolio Leadership at Agios Pharmaceuticals, said base editors are capable of making single-base changes with high efficiency and unprecedented control. Beam, Evans said, has put together the base editing technologies and intends to establish them as a “new therapeutic option for patients with serious diseases.”

Out of the gate Beam Therapeutics has established several licensing agreements. Its first agreement is with Harvard that covers two base editing platforms developed in Liu’s lab. The first is the C base editor, which features Cas9 linked to a cytidine deaminase to deliver programmable C-to-T or G-to-A edits in DNA. The second is the A base editor, which features Cas9 linked to an evolved form of adenosine deaminase capable of editing DNA to deliver programmable A-to-G or T-to-C edits.

The second license is with the Broad Institute for RNA base editing technologies from Zhang’s lab. This includes the RNA editor platform, which features Cas13 linked to an adenosine deaminase to deliver single base A-to-G editing of RNA transcripts, the company said.

Additionally, Beam entered into a third licensing and option agreement with Editas Medicine. Under terms of this agreement Beam secured an exclusive sublicense to patent filings by Harvard for base editing technologies and patent filings by MGH for CRISPR technology. The company also gained an exclusive option for future sublicensing of additional Cas9 patent families and Cpf1 patent families in the field of base editing. In return, Editas Medicine has received an equity stake in Beam and will be eligible for royalties on medicines utilizing the related intellectual property and technologies.

In addition to Evans, Beam’s leadership team includes Chief Scientific Officer Giuseppe Ciaramella, who most recently served as CSO of Moderna Therapeutics’ Infectious Disease division.

https://bit.ly/2kvxRkU

Celsius Therapeutics Launches with $65 Million Series A Financing

Celsius Therapeutics launched in Cambridge, Massachusetts with a Series A financing round worth $65 million. The financing was led by Third Rock Ventures with participation from GV(formerly Google Ventures), Heritage Provider Network, Casdin Capital, Alexandria Venture Investments and others.

The new company will focus on developing single-cell genomic research into precision therapeutics for autoimmune diseases and cancer. It links computational algorithms in order to identify first-in-class precision therapies. The company indicates it will begin with single-cell sequencing on defined patient samples to identify individual cells and their interactions that cause disease.

Celsius was co-founded by Aviv Regev, director of the Klarman Cell Observatory at the Broad Institute of MIT and Harvard, professor of Biology at MIT and an investigator at the Howard Hughes Medical Institute.

Jeffrey Bluestone is the co-founder, president and chief executive officer. He is with the Parker Institute for Cancer Immunotherapy, University of California, San Francisco and the A.W. and Mary Margaret Clausen Distinguished Professor.

Vijay Kuchroo is a co-founder. Kuchroo is the Samuel L. Wasserstrom professor of neurology, Harvard Medical School, senior scientist in neurology at Brigham and Women’s Hospital, associate member of the Broad Institute, and director of the Evergrande Center for Immunologic Diseases at Harvard Medical School and Brigham and Women’s Hospital.

Christoph Lengauer is a co-founder and president. A venture partner at Third Rock Ventures, he is also adjunct associate professor, Sidney Kimmel Comprehensive Cancer Center at the Johns Hopkins University School of Medicine.

Ramnik Xavier is also a co-founder. Xavier is chief of gastroenterology, Massachusetts General Hospital, an institute member with the Broad Institute, and the Kurt Isselbacher professor of medicine, Harvard Medical School.

“When first meeting with Aviv and her colleagues and learning about the significance of this new technology, we knew this had to become a company,” said Lengauer, in a statement. “We formed Celsius with the goal of bringing a novel precision medicine approach to underserved patients with autoimmune diseases and certain cancers. With the new level of clarity provided by single-cell sequencing, our team will be able to address many of the challenges of the current treatments and introduce a new class of medicines that will lead to better outcomes and potential cures.”

Celsius licensed technologies from the Broad Institute based on work by Regev and Kuchroo, as well as non-exclusive licenses to single-cell technologies. It also obtained an exclusive license to early-stage therapies.

“Each of us is made up of tens of trillions of cells,” said Alexis Borisy, partner at Third Rock Ventures and chairman of Celsius Therapeutics, in a statement. “At the core of founding Celsius was the new ability to see something we could not see before. We can now see the dysfunction of key cells and their interactions within their neighborhood. Diseases that we have struggled to understand now can become crystal clear. With that clarity, we hope to create novel precision medicines.”

What differentiates Celcius’ approach is that is believes it has a way of isolating single cells and their interactions with each other. A more typical approach is to study aggregates of cells. Or as Xconomy writes, traditionally a bunch of cells are stuck in a blender, ground up into a slurry, then the DNA and RNA is extracted, which provides an average of the group. “Details about differences between individual cells, and how those cells interact, get lost in the blender.”

Celsius will use high-resolution sequencing technologies to evaluate the RNA of an individual cell. By doing so, the company believes it can identify so-far unidentified genes and biological pathways involved in various diseases.

Regev told Xconomy, “One key challenge is the analysis of data sets of unprecedented size and complexity to identify key cells and genes.”

The company hopes the $65 million will be enough to take the company into clinical trials in the next five years.

https://bit.ly/2GUz1Q5