January 15, 2017 (Vol. 37, No. 2)

Industry Watch: New York State of Mind: $1B+ for Biotech

Biopharma and other life sciences employers have more than a billion reasons to thank the dueling politicians who lead New York City and New York state.

New York Gov. Andrew Cuomo (D) on December 12 unveiled $650 million in incentives intended to correct the Empire State’s shortcomings in attracting life-sci companies and their jobs.

A day later, NYC Mayor Bill de Blasio (D), who faces re-election this year, announced the Big Apple’s almost-as-big incentive package—a $500 million, 10-point initiative dubbed LifeSci NYC.

The combined $1.15 billion shows that while de Blasio and Cuomo have long been rivals, the two agree that when it comes to New York’s biocluster, the whole is less than the sum of its parts.

Despite the presence of top-tier academic medical institutions and Wall Street, New York has lagged behind the nation’s top clusters—Boston/Cambridge, MA, and the San Francisco Bay Area—in commercializing academic discoveries and supporting companies and the talent necessary to sustain them.

Take venture capital, for example. The New York region drew $142.935 million in 25 deals during the first nine months of 2016, according to the quarterly MoneyTree Report by PwC with Thomson Reuters data. San Francisco generated $1.713 billion in 79 deals; Boston/Cambridge, $2.386 billion in 80 deals.

 “New York has all the ingredients necessary to build the world’s foremost life science cluster,” Nathan Tinker, executive director of NewYorkBIO, the state life-sci industry group, said in a statement. “The initiatives launched this week by Governor Cuomo and Mayor de Blasio offer a roadmap to engage these assets in exciting ways.”


Discovery & Development: Computational Discovery Starts to Add Up

Drug discovery efforts that take place inside computers are cleaner than those that take place inside the lab—except for the overstuffed databases and seemingly endless calculation cycles. These digital messes can be as confounding as the chemical messes associated with combinatorial assays.

New approaches, however, are tidying up computational drug discovery. For example, a new approach developed by scientists at the University of Barcelona amounts to a labor-saving device. It is called dynamic undocking, or DUck. Rather than attempt the difficult chore of predicting binding affinities for protein-ligand complexes, DUck calculates the work necessary to reach a quasi-bound state at which the ligand has just broken the most important native contact with the receptor.

To be fair, DUck complements existing structure-based computational methods, which focus on docking. With the docking-undocking combination, researchers not only identify the most promising interactions, but also the most stable complexes. This combination, recently described in Nature Chemistry, constitutes an exceptionally efficient screen, one that the researchers used to evaluate candidates against an oncology target while obtaining a hit rate approaching 40%.

Besides structure-based methods, pathway- or network-based methods are being refined. At the Institute for Research in Biomedicine (IRB Barcelona), scientists conducted in silico analyses to identify drug combinations that could interrupt cell signaling pathways associated with breast cancer. This approach, detailed in Cancer Research, found that raloxifene and cabozantinib exert a joint effect that is “much much greater” than the effect of either drug alone. The drug combination could permit lower doses to be administered, mitigating side effects. Also, by attacking tumors on multiple fronts, the combination could make it harder for the tumors’ cells to resist treatment.

Advances such as these are contributing to the growing market for drug discovery informatics. Globally, the market is estimated to reach $6.5 billion by 2022, up from $1.7 billion in 2014. These figures come from Grand View Research, which notes that costly drug development failures are driving a shift from traditional in vitro development to computer-aided design, as well as the growing use of in silico models for early ADME/Toxicity screening.


Combined therapy using two drugs emerges as a promising approach to tackle cancer resistance. For example, biocomputational analyses have revealed new synergistic combinations, such as raloxifene and cabozantinib, for the treatment of breast cancer. [Samira Jaeger, IRB Barcelona]

Genomics & Proteomics: Patent Concerns Don’t Stop CRISPR Collaborations

The start of oral arguments surrounding the cutting-edge genome editing technology CRISPR-Cas-9, at the Patent Trial and Appeal Board within the United States Patent and Trademark Office (USPTO) in early December, has seemingly not dampened either parties’ claim, as the Broad Institute (along with MIT and Harvard), as well as the University of California (UC), have brokered IP collaborations amid patent concerns.

CRISPR Therapeutics, Intellia Therapeutics, and Caribou Biosciences (co-founded by one of the original CRISPR researchers Jennifer Doudna, Ph.D.) recently teamed up with ERS Genomics and entered into an agreement with CRISPR IP co-owners, the Regents of UC, Emmanuelle Charpentier, Ph.D. (original CRISPR researcher with Dr. Doudna), of the Helmholtz Centre for Infection Research, and the University of Vienna.

Not to be outmaneuvered by this newly formed IP coalition, Editas Medicines hammered out a deal a few days later with Broad Institute, Harvard University, MIT, Wageningen University (The Netherlands), the University of Iowa, and the University of Tokyo for the new CRISPR genome editing system known as Cpf1.

Cpf1 is a protein that was brought to the forefront by Feng Zhang, Ph.D., and his colleagues and has stirred interest by appearing to be a more versatile tool than Cas9, as it requires only one RNA guide molecule to replace target DNA, rather than two for Cas9.

Currently, the courts show little signs of whether they will lean toward Dr. Zhang’s claim that he conceptualized using CRISPR well before Dr. Doudna’s 2012 seminal science paper describing the gene-editing tool or if they will rule for Drs. Doudna and Charpentier’s work that described the fundamentals of the molecular technique. What is clear, however, is that each party seems determined to move forward with their paths, all of which are geared toward clinical use in humans as soon as possible.

In a recent interview with Precision Medicine World Conference, Dr. Doudna concluded that “the power and versatility of CRISPR will undoubtedly positively impact healthcare. The technology is already taking drug discovery and development to a higher level and opening up new and wide-ranging possibilities to treat and cure diseases.”


Bioprocessing: Lonza Agrees to Acquire Capsugel for $5.5B

Lonza agreed to acquire Capsugel from KKR for $5.5 billion in cash. The deal will be financed with a combination of debt and equity financing.

A Lonza official told GEN that Capsugel is a leader in innovative dosage forms.

“It’s a ‘perfect fit’ for Lonza, which contributes a strong CDMO [contract development and manufacturing organization] business with a growing product business. Capsugel is strong on the product side of the business and has a growing CDMO business,” noted the Lonza spokesperson.

The acquisition is designed to create a value-added solutions provider in drug development, formulation, delivery technologies, and manufacturing for the global pharma, consumer healthcare, and nutrition industries, according to Richard Ridinger, CEO of Lonza.

 “It will provide additional value by offering an integrated portfolio of industry-leading technologies, from active pharmaceutical ingredients through excipients to dosage forms and delivery technologies,” he said. “The combined technologies and offerings will provide customers innovative solutions in both large and small molecules, and solidify Lonza’s position as the partner best able to support the pharma industry by bringing new, differentiated medicines to market, rapidly and efficiently.”

Ridinger added that the new integrated approach will benefit customers, who will gain from “the simplicity and efficiency of working with one company that can provide world-leading support from APIs to excipients and dosage forms.”

This acquistion comes just one week after Lonza sold off its peptides operations to PolyPeptide Group.


Molecular Diagnostics: PGDx Expands Pan-Cancer Tumor Profiling Offerings

Personal Genome Diagnostics (PGDx) wrapped up a busy year with the mid-December launch of its CancerSELECT™ 125 test for pan-cancer tumor profiling. CancerSELECT 125 identifies clinically actionable and functionally important sequence mutations and structural alterations across multiple cancer types.

In October, the company launched PlasmaSELECT™ 64, a targeted panel for pan-cancer tumor profiling without the need for invasive biopsies. 

“The landscape of clinically actionable biomarkers is evolving rapidly as approved cancer therapeutics, ongoing clinical trials, and the scope of scientific inquiries change. PGDx routinely investigates and updates its tissue- and plasma-based pan-cancer assays to ensure that we are offering the most clinically relevant assays as defined by an associated clinical guideline, available drug therapeutic, or an active clinical trial,” Mark Sausen, vp, R&D, explained.

“CancerSELECT 125 incorporates the most recent information on these parameters,” he continued. For example, it includes MET-exon 14 skipping as well as 17 new genes covering over 30 new clinical trials across multiple indications [including NTRK2 &3, AXL, MEK2 (MAP2K2), and PIK3CB], as more actionable information about these genes was published.

 According to Sausen, “Competitive assays are not updated routinely to ensure clinically actionability. As a result, while these assays contain a large number of genes, the panels may have limited clinical utility and/or may be very expensive as a result of the number of genes included (some labs have reportedly made the panels large in order reduce the need to provide updates, or for specific clinical research/biomarker discovery purposes).”


PGDx’ CancerSELECT 125 identifies clinically actionable and functionally important sequence mutations and structural alterations across multiple cancer types.


























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