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Advanced therapies, pricing and reimbursement challenges, oncology breakthroughs and digital technologies are among the key topics for 2019 identified by biopharma industry players and watchers. Scrip spoke to more than 40 executives, experts and investors to find out what they will be watching for. In the first installment of our Scrip Asks series focused on 2019, interviewees share their thoughts on technology and therapeutic progress.

Perhaps reflective of the long timelines in biopharma R&D, few people identified single major events expected to rock the sector in 2019. Nevertheless, many were excited about the opportunities afforded by recent and ongoing advances in therapeutic technologies, understanding of diseases, and artificial intelligence - all of which are expected to help generate a reliable flow of product approvals for next year and beyond.

Summing up his expectations for the industry overall, John Thero, president and CEO of cardiovascular-focused company Amarin Corp. PLC, said "pockets of important innovation and growth" would feature "amongst broader industry contraction as drugs go off patent and the pipeline of new products becomes narrower and more specialized." Also taking a broad view was James Dentzer, CEO of cancer drug developer Curis Inc., who predicts "a year of evolution, not revolution."

Dentzer, focusing on oncology, believes this evolution will involve the continuation of a couple of trends, namely "increases in the ability to target specific patient populations" and "an increasing amount of physicians going toward therapies that combine well." He believes these trends will ultimately lead to cancer becoming "no longer a disease that will kill you: it's a disease that will require chronic treatment," tailored for each patient to enable them to live with their disease rather than die from it.

When it comes to conversion of R&D into marketed drugs, the good news is expected to continue. For example, in the US, the Food and Drug Administration is expected to approve more than 60 new drugs in 2018. For executives at Veeva Systems, this record number is the new normal: the pharma-focused cloud computing firm expects to see an average of 40-60 drugs approved a year. Jim Reilly, vice president of Veeva's Vault Clinical platform, thinks product approvals will also be speedier in 2019. He noted that "early success with partnership programs between health authorities and the industry" – including accelerated approval programs such as the Real Time Oncology Review pilot – helped fuel 2018's record haul. Reilly thinks additional industry/regulator collaboration programs as well as companies "improving R&D operational performance" will "help drive more efficient processes and bring life-changing therapies to patients faster."


A lot of the people we spoke to highlighted advances in therapeutic technologies. "I'm really interested in the expanding biological toolkit – in the last decade we have seen the first approvals for technologies such as bispecific antibodies and antibody-drug conjugates, but recently there has been a proliferation with RNA, gene and cell therapies," said Dan Chancellor, principal analyst for Informa Pharma Intelligence.


"The application of gene therapies in more prevalent diseases is a bigger test of its broader potential." Dan Chancellor, Informa Pharma Intelligence


"Next year will be an important one, as BioMarin Pharmaceutical Inc. completes Phase III development of its hemophilia A gene therapy, val-rox [valoctocogene roxaparvovec], and will file for regulatory approval." Chancellor noted that Informa's Biomedtracker database includes 15 high-impact catalysts expected to occur in 2019 for gene therapies, spanning indications in hematology, ophthalmology, oncology and neurology. While the first gene therapy, Spark Therapeutics Inc./Novartis AG's Luxturna [voretigene neparvovec], was approved in 2017, that was for a rare eye disease. "The application of gene therapies in more prevalent diseases is a bigger test of its broader potential," Chancellor said.

Amanda Micklus, a principal analyst with Informa Pharma Intelligence's Datamonitor Healthcare, added that "we could see at least two new gene therapy approvals next year: Novartis/ AveXis Inc.'s spinal muscular atrophy therapy AVXS-101 in the US and bluebird bio Inc.'s LentiGlobin for transfusion-dependent beta-thalassemia in the EU." Sounding a note of caution, though, Andy Smith, director-analyst at investment research firm Edison Group, said "I don't expect bluebird's gene therapy to have mature enough data to be approved in the EU." On the gene editing side, "we might see some of the earliest data from industry-sponsored trials evaluating CRISPR-Cas9, as Editas Medicine Inc. just recently got IND approval to move forward with its in vivo program for Leber's congenital amaurosis 10 (partnered with Allergan PLC), and CRISPR Therapeutics AG/ Vertex Pharmaceuticals Inc.’s ex vivo gene editing program for sickle cell disease id undergoing a trial in Germany," Micklus noted.

"Advances in cell and gene therapy are expected to break through in 2019," thinks L.E.K. Consulting partner Clay Heskett. "Commercial traction for the CAR-T therapies and clinical advances in higher profile diseases such as hemophilia are coming into focus, and these proofs of concept should excite more of pharma's larger players to make bold moves in these areas."

But for David Lucchino, president, co-founder and CEO of Frequency Therapeutics (which is developing small molecule candidates to activate progenitor cells) and chairman of MassBio, while there has been a "fundamental shift in R&D where more companies are targeting ways to utilize the body to heal itself instead of simply creating therapeutics to cure disease," simplifying these approaches will be important too. "I expect market investors to show more support for biotech that could have a similar impact [to modalities like CRISPR gene editing] but in a much less complex manner and without a lot of ethical concerns."

Ultan McDermott, chief scientist – oncology at AstraZeneca PLC, is excited about the expanding utility of CRISPR gene editing and single-cell RNA sequencing. "There have been a few papers released over the past couple of months beginning to show that whereas we could already use CRISPR in cancer relatively easily, doing this in immune cells, which has been quite difficult, is now beginning to become easier." For McDermott, this opens up the possibility of understanding why immune therapies don't always work in cancer, and how cells become resistant to immune therapies, which in turn may enable such therapies to be made more effective and stop resistance emerging.

"In the same way that we can talk about using CRISPR to hit the cancer cells, we can begin to identify the genes within immune cells to help immune therapies work better."

On single-cell RNA sequencing, McDermott– notes that the technology is able to analyse cancer cells that survive treatment to identify the pathways that enable them to do so, "and if those pathways are druggable and we could destroy those cells, that might change the whole paradigm of how resistance emerges."



For Cameron Turtle, chief business officer of Eidos Therapeutics Inc., "2019 will see advancement of precision medicine approaches beyond oncology." He noted the "application of genetically targeted therapeutics within cardiology" which signals a shift from a historical "all-comers approach" in cardiology drug development to defined sub-populations of broader heart diseases based genetic source, "allowing us to more precisely design treatments, raising the probability of success."

"The biggest challenge the industry will face is the ability to effectively maximize outcomes from rapid advances in research, which will continue to accelerate." Mark Straley, Agendia

Meanwhile, Vedanta Biosciences Inc.'s CEO Bernat Olle believes the microbiome is on a par with gene editing for its importance in advancing the understanding of human biology. "2019 will be an important year for the microbiome, clinically at least, with many data readouts expected." He believes there is growing evidence to support the role of the microbiome across a range of immune-mediated diseases, including cancers.

For Mark Straley, CEO of oncology genomic testing firm Agendia BV, genomic testing will be an expanding area. "Genomic testing in oncology will continue to accelerate in order to garner more valuable information about the genetic and molecular biomarkers that drive certain cancers." This will enable better treatment decisions for individual cancers. But, he says, "the biggest challenge the industry will face is the ability to effectively maximize outcomes from rapid advances in research, which will continue to accelerate."

Therapeutic Innovation: Oncology

Oncology accounts for around a third of biopharma development work, and many of the people we spoke to highlighted aspects of cancer R&D. "I think what you'll see in 2019 are new modalities that are used in combination to really make a significant improvement in patients’ longevity and also in their quality of life," commented Bill Newell, CEO of Sutro Biopharma Inc., which is developing a range of targeted cancer therapies. "We want to see in 2019 better responses for patients but also combinations such as a CAR-T and an antibody-drug conjugate [such as Sutro is developing] that really have a much more impactful and sustained response for patients and allow them to enjoy a better quality of life, rather than the constant therapy administrations that sometimes happen with chemotherapy and with antibodies, because that can be very debilitating for a cancer patient even if it extends their life."




Cyclacel Pharmaceuticals Inc. CEO Spiro Rombotis agreed, expecting there to be "a continuing stream of FDA approvals for targeted therapies emerging out of a variety of technologies, including small molecules, antibodies and cell engineering", while the evaluation of immuno-oncology combinations, especially with small molecules, will continue to be a priority. Like AstraZeneca's McDermott, Rombotis also expects "an increased emphasis on therapies targeting cancer-resistance mechanisms in patients who fail approved agents."  He is hopeful for research into add-on combination drugs that may be able to suppress the pro-survival protein pathways that are often highly expressed in resistant cancer cells.




Rombotis believes that addressing resistance to single-agent therapies by using new combination therapies to extend drug treatment periods without relapse "can create enormous value for patients, payers and society." He said: "Clinical trial data comparing such combination strategies to historical standard-of-care treatments demonstrated longer disease-free periods and substantial reductions in the risk of death."

Meanwhile, resistance is something of a moving target, noted Curis's Dentzer, who pointed out that data on the use of new immuno-oncology agents show that new resistance mutations are emerging as a result of their use. "It's like whack-a-mole, where you give something to a patient and something else pops up."

For Hardik Patel, therapeutic area director, oncology, Datamonitor Healthcare, 2019 will see further advances in the development of oncology drugs for tumor-agnostic indications following the approval of Merck & Co. Inc.'s Keytruda   (pembrolizumab) for MSI-H advanced solid tumors and Loxo Oncology Inc.'s Vitrakvi (larotrectinib) for solid tumors with NTRK gene fusions. Such an approach helps get drugs to patients with mutations that may "otherwise be overlooked due to their rarity in individual tumor types," and "clinically, because development for tumor agnostic indications revolves around a specific biomarker, these drugs are highly effective within their targeted patient groups and physicians can have confidence in prescribing them to patients eligible for treatment. Furthermore, with the continued uptake of diagnostic tools such as next-generation sequencing, identifying patients with addressable genetic aberrations is becoming easier and more common-practice."

Patel also expects the expansion of PD-1/PD-L1 therapies into further indications, such as breast, ovarian, esophageal, endometrial cancer, brain cancer, mesothelioma, or prostate cancer, as well as growth of such therapies within indications for which they have already been approved, especially through the approval of new combination regimens. In a recent research note, Credit Suisse analyst Vamil Divan highlighted triple negative breast cancer as the next key battleground for immuno-oncology, while further down the line "earlier lines of therapy" will be "the next big test" since "virtually all I-O data so far has been in the metastatic setting."

When it comes to specific pathways to target, Shafique Virani, CEo of CoA Therapeutics and Navire Pharma, believes the SHP2 pathway will be increasingly of interest in 2019, for its potential to modulate solid tumor resistance. "Exciting new biology emerged in 2018 on the SHP2 pathway, indicating a key role for allosteric inhibitors in suppressing tumor growth in KRAS mutant and NF-1 loss solid tumors," he noted, adding that he expects there to be a partnering boost "as companies look to combine SHP2 inhibitors with other targeted oncology therapies such as MEK inhibitors." Navire's focus is developing SHP2 inhibitors.


"It is not hard to envision a future where patients benefit at the expense of CAR-T franchises." – Anand Mehra


For Anand Mehra, general partner with venture capital firm Sofinnova Ventures, enthusiasm for cell therapy has somewhat dampened interest in dual targeting modalities such as bispecific antibodies. Nevertheless, "a core set of believers in companies like Amgen Inc.,Roche/Genentech Inc.Regeneron Pharmaceuticals Inc.,Xencor Inc. and Merus NV have continued to plug away with bispecific CD3 engagers – and slowly made progress without the fanfare and recognition visited upon their colleagues in the cell therapy space." The Amercian Society for Hematology meeting in early December "may be the start of their moment," he said. Citing data on CD3-engagers and in the CD20 space in myeloma and other cancers presented at ASH, he noted bispecifics "walk into the ring with a formidable set of intrinsic advantages ranging from rapid speed in reaching very sick patients, ‘off the shelf’ manufacturing, titratable dose, and the ability to chase durability by redosing." In Mehra's view, "it is not hard to envision a future where patients benefit at the expense of CAR-T franchises."


"CNS is an area with unaddressed needs but poor return on investment in dementia treatments. There will be attempts to find pharmaceutical means to address mental illnesses. I don’t know how successful it’ll be, but understanding the brain and how to modulate it is a good challenge for the future,"Kevin Bottomley, partner with consulting firm Results Healthcare, told Scrip.

Judy Adair, associate director, CNS/AI with Informa Pharma Intelligence's Trialtrove, said: "There is hope for disease modifying therapy in Parkinson’s disease with 28 companies developing alpha synuclein inhibitors." She highlighted the Michael J Fox Foundation's Alpha-Synuclein Clinical Path Working Group having recognized the need for diagnostic biomarkers to ensure patients can be accurately recruited and monitored, which led MJFF to support AC Immune SA in its first human trials on an alpha-synuclein imaging agent scheduled to start in early 2019.  "This is a trial which will definitely be worth watching," Adair said.

Also excited about alpha-synuclein-focused R&D in Parkinson's was Gene Kinney, CEO of neuroscience-focused Prothena Corp. PLC. "Recent advances have brought about a better understanding of disease pathophysiology, new tools for imaging the brain, decoding DNA, and evaluating and monitoring symptoms.  As a result, I believe we will see tremendous improvements in quality of life for patients with neurodegenerative diseases and as our understanding continues to evolve, I anticipate that one day, the scientific and medical communities will be able to effectively identify and stop these types of diseases even before symptoms emerge." He believes "we are on the verge of substantive advances in neuroscience."

Anirvan Ghosh, senior vice-president, head of research and early development at Biogen Inc., also listed progress on synuclein trials in Parkinson's (both in-house and by Roche) as an area to watch in 2019. Ghosh listed gene therapy, antisense oligonucleotides in amyotrophic lateral sclerosis (in which Biogen is carrying out a pivotal trial) and in the pipelines of Wave Life Sciences Ltd. and Ionis Pharmaceuticals Inc., and progress on tau-related clinical studies in Alzheimer's disease among key areas of interest.

Ghosh's colleague, Samantha Budd Haeberlein, vice-president for clinical development, Biogen, noted that in Alzheimer's disease "2019 will be a year of anticipation" as the last year for a number of long and large studies testing anti-beta-amyloid monoclonal antibodies. "For scientists the data that will come from the thousands of patients who have dedicated their time and lives to these trials will be unparalleled for the volume, complexity and richness of biomarkers as never before. So much will be learnt from these trials." But we will have to wait until 2020 for read-outs.


Novo Nordisk AS's chief science officer, Mads Krogsgaard Thomsen, hopes that 2019 will see progress in treating obesity, and importantly, in getting the condition officially acknowledged as a serious, chronic, but treatable disease.

He told Scrip: "Where we stand today with regards to understanding obesity and its global impact is similar to where our understanding of diabetes was twenty years ago, when the landmark UK Prospective Diabetes Study (UKPDS): clinical and therapeutic implications for type 2 diabetes came out." That 1998 studied renamed non-insulin dependent diabetes as type 2 diabetes, acknowledging it as a "a real, chronic and progressive disease that society subsequently began to pursue big time by showing that intensive intervention with medicines would actually improve outcomes," Thomsen said. "That changed the whole outlook for diabetes, and also for innovation. Obesity is a ticking time bomb. But there are very few societies in the world that accept obesity is a serious progressive, chronic disease.”



Noting that there is as yet no equivalent to the UKPDS report for obesity to show a drug "reduces stroke by X%, myocardial infarction by Y%, and expand life by Z%," Thomsen highlighted Novo Nordisk's Phase III SELECT trial of semaglutide's effects on cardiovascular outcomes in 17,500 overweight or obese people with cardiovascular disease. "We're doing it not only for the sake of semaglutide but also for the sake of getting society to acknowledge obesity as a disease," he said. However, SELECT only began in 2018 and will not be complete until 2023. Semaglutide is also under development for weight loss.  

Infectious Disease And Vaccines

The coming year could see a breakthrough in the hunt for a universal seasonal influenza vaccine, with data potentially coming out of the first ever Phase III trial of such a product, noted Michael Haydock, Datamonitor Healthcare therapeutic area director for cardiovascular and metabolic, and infectious diseases. BiondVax Pharmaceuticals Ltd. is evaluating its M-001 vaccine through the 2018/2019 season and has not said it will release preliminary efficacy/immunogenicity data, "but if they are particularly positive it may well do," Haydock commented. 

Haydock also flagged up anticipated data from Novavax Inc.'s trial of its ResVax vaccine in pregnant women to protect infants from severe respiratory syncytial virus in the first months of live, which would address a "major unmet need."

Elsewhere in infectious disease, Haydock noted that ViiV Healthcare is awaiting regulatory approval decisions (including in the US in the second quarter) for its two-drug regimen of Tivicay (dolutegravir) and Epivir(lamividine) in treatment-naïve HIV patients, with further expansion into the maintenance setting anticipated for 2020. "This combination is the cornerstone of ViiV's strategy to capture market share from Gilead Sciences Inc. by marketing simplified treatment options with the potential for superior long-term safety in a market dominated by three-drug regimens," he said. "Should Tivicay/Epivir gain approval, it is expected to generate blockbuster sales." He expects ViiV to price it at a significant discount to Gilead's rival single-tablet regimens in a bid to gain preferential formulary placement and to incentivize physicians to shift from the three-drug regimens they have used for almost two decades. However, take-up may be muted for the first year or so by "hypothetical concerns about the emergence of resistance after long-term treatment", he warned.



As for bacterial infections, David Roblin, chief operating officer, chief medical officer and president of R&D at Summit Therapeutics PLC, is "optimistic about 2019 for antibiotic development." He listed the increasing availability of diagnostics to rapidly and accurately identify specific bacteria, which "will drive demand for targeted antibiotics to improve patient outcomes", as well as "growing awareness of the key role played by the microbiome in health issues from diabetes to CNS disorders", which he thinks will "put a premium on the creation of targeted antibiotics which allow the preservation of healthy gut bacteria," and also "evidence that regulators and payers are becoming more active in incentivizing innovation" on both sides of the Atlantic. He added that "for the first time in decades we will have two novel antibiotics in Phase III development: Summit's ridinilazole, targeting Clostridium difficile, and Polyphor Ltd.s murepavadin, targeting resistant pseudomonas infections."

Future installments of this Scrip Asks... series will address topics including the growing importance of digital technology and artificial intelligence, business trends in fund-raising, deal-making and R&D and operational strategy, clinical trial trends and broader global themes.

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