These are the biggest biopharma stories we covered in 2018

Any given year tends to see a lot of big news for pharmaceuticals, biotechnology and diagnostics, and 2018 has been no exception. From drugs and diagnostics, to the companies that make them and the policies that govern them, several big events and milestones have occurred this year. And many of these have significant implications going into next year as well.

In the time-honored, journalistic tradition of year-end roundups, here are six of the most important biopharma events and trends that occurred in the 12 months of 2018 that MedCity News has covered. They are presented in no particular order, but are arranged according to the following categories: Regulatory Approval; International Trend; Controversy; Drug Discovery and Development Trend; Financing; and Policy Trend.

Regulatory Approval: Loxo Oncology and Bayer’s Vitrakvi

Ever since the 2002 approval of Novartis’s now generically available Gleevec (imatinib), drugs that zero in on specific mutations – known as targeted therapies – have been a mainstay in oncology.

In the strictest sense, Loxo Oncology and Bayer’s Vitrakvi (larotrectinib) is one of many such drugs. But while it is also the second drug to win a tissue-agnostic label from the Food and Drug Administration – after Merck & Co.’s Keytruda (pembrolizumab) last year – it is the first to win such a label as its sole indication. The drug is approved for solid tumors that express NTRK fusions, regardless of where they occur in the body.

What makes the approval important is that it represents in many ways the future of oncology. Value-based care has become a buzzword and will likely take a long time to become reality, but Vitrakvi’s approval should be seen as a predictor of what is to come. Instead of the traditional approach of defaulting to one-size-fits-all treatments, cancer patients will undergo genomic sequencing and then be treated with therapies that have a high probability of actually being effective. In fact, this is already happening to an extent: testing lung cancer patients for EGFR mutations and ALK fusions is already standard of care, while acute myeloid leukemia patients are routinely tested for FLT3, IDH1 and IDH2 to see if they qualify for drugs tailored to those targets.

But Vitrakvi is a kind of poster child for this trend: NTRK fusions are rare and occur in a rather spotty fashion across different tumor types – ranging from virtually all cases in some to 1 percent or less of others. But when they do occur, the drug produces very high response rates. Patients will eventually become resistant to therapy, but Loxo already has a successor drug, LOXO-195, designed to counter resistance mechanisms. It’s also developing another drug, LOXO-292, which targets RET fusions, while Blueprint Medicines has its own RET inhibitor in development.

Loxo, Bayer’s Vitrakvi wins FDA approval. Now comes the hard part: finding patients

FDA approval puts Loxo, Bayer’s Vitrakvi on the rails to value-based care

Updated ASCO data from Loxo study show more patients responding

International Trend: Brexit

The good news for British Prime Minister Theresa May is that she survived a no-confidence vote in the House of Commons. The bad news, however, is that the UK is still hurdling toward the March 29, 2019 date of Brexit without a deal with the EU, thanks to May’s decision to postpone a vote on one that the two parties had agreed upon when it became clear the vote would fail. Perhaps an apt metaphor is the high-speed Eurostar train zipping through the Channel Tunnel, but with the signals broken down and uncertain as to whether it will end up in Paris, Brussels or simply off the rails.

Where this becomes problematic for biopharma is on the question of whether the UK will remain under the authority of the European Medicines Agency, collaborate with global regulatory authorities or crash out entirely and regulate drugs by itself. Forming a coalition with global regulators would be ambitious, but it would also be complex and time-consuming to implement. Staying under the EMA’s authority would enable the country to take advantage of being part of one of the largest drug markets. The third option is the least attractive, as it would make the UK a second- or third-tier market, meaning one drugmakers consider after they have secured FDA and EMA approval. Meanwhile, although the UK will likely continue to boast a strong research and development infrastructure regardless of whether it is part of the EU, the bigger question is how the country will make up for research funding that the EU has been providing.

Survey: UK life sciences face post-Brexit apocalypse; but expert sees more shades of grey

Report sounds alarm about Brexit risks to UK clinical trials environment

May postpones vote on Brexit agreement that pharma sector had supported

Controversy: The NantHealth-Independence Blue Cross partnership

First, a confession is necessary. The headline of the story, “What ever happened to the NantHealth-Independence Blue Cross sequencing partnership?” was a reference to the campy 1962 thriller, “What Ever Happened to Baby Jane?” The movie, now considered a classic, starred Bette Davis in the titular role of Baby Jane Hudson and Joan Crawford as her sister, Blanche.

That wasn’t meant to allude to the fictional abusive relationship between Jane and Blanche – let alone the infamous, decades-long feud between Davis and Crawford. However, it was meant to ask a similar question to the movie’s title: What, indeed, did happen to the much-heralded deal between Patrick Soon-Shiong’s NantHealth and IBC to provide genomic sequencing to cancer patients? Just as Baby Jane Hudson had gone from a childhood star to being forgotten, the NantHealth-IBC partnership had largely faded from view despite its celebratory unveiling in January 2016.

As it turns out, the results were apparently not consistent with the hype. Although NantHealth had been – in the words of one expert – aggressive in promotion, IBC had only ended up paying for a small number of NantHealth tests.

In the meantime, a number of companies have risen to dominate the next-generation sequencing field, which is growing in importance for cancer care. These include Foundation Medicine, now owned by Roche, Illumina, Thermo Fisher and up-and-comers in the diagnostics space like Guardant Health and Grail.

What ever happened to the NantHealth-Independence Blue Cross sequencing partnership?

Drug Discovery and Development Trend: Blockchain and AI/ML

Watch any science fiction that offers a positive vision of the future, and you’ll likely see two predictions: Computers will become sentient, and science will conquer disease and master biology. “Star Trek” comes to mind as a good example, or even the dystopian “Blade Runner.”

As cliché as it sounds, science fiction is becoming reality. Although research on blockchain and artificial intelligence and machine learning in healthcare predates this year, 2018 seems to be when it started to truly grow in importance, and that is likely to carry into next year.

A number of startups that employ AI/ML and blockchain in drug development have launched or raised decent sums of money this year, and research institutions have gotten in on the action as well.

At the same time, both technologies remain in their infancy, as far as drug discovery and development are concerned. Although Salt Lake City-based Recursion Pharmaceuticals received authorization in July from the FDA to start a Phase I clinical trial REC-994, a potential medicine for the genetic disease cerebral cavernous malformation discovered via machine learning, no AI/ML-discovered drug has entered clinical testing yet.

Meanwhile, blockchain technology holds a lot of promise for use in clinical trials. Potential applications include ensuring the safety and security of data, improving reproducibility of data and providing a “shared source of truth” for how different stakeholders in a trial handle things like patient consent when there are changes to protocol.

That doesn’t mean there isn’t skepticism around the idea. For example, many of the functions of a shared ledger could also be achieved with a centralized server and database. However, that would have the potential drawback of requiring a centralized organization to manage and pay into that business, and blockchain could create utility across an organization.

All the same, as one industry insider said, 2019 will likely be a year that sees significant progress in the development of blockchain technology in biopharma.

Blockchain becomes a ‘source of truth’ for biopharma

Embleema emerges from stealth mode, launches blockchain-based health record system

AI/ML-based drugmaker gets FDA green light for Phase I study

Financing: The biggest-ever biotech IPO

2018 saw a string of eye-popping fund raises by biopharma companies. The most recent was discovery-stage Relay Therapeutics, which on Dec. 20 announced it raised $400 million in a Series C round for a technology platform that involves finding targets through the folding and unfolding of proteins. For comparison, its Series B round last December amounted to $63 million.

The most dramatic financing of the year was less than two weeks earlier, on Dec. 7, when Moderna raised more than $600 million in what was the biggest-ever initial public offering by a biotechnology company. Indeed, the $500 million it had initially set out to raise would already have been the largest. That was not long after another biotech mega-IPO, by Allogene, which makes allogeneic CAR-T cell therapies and went public only months after its launch earlier this year.

But perhaps the most surprising news came in the hours following the IPO, when the company’s stock fell 19 percent on the Nasdaq. It has since fallen further, and at the time of this writing was trading at $14.07 per share. Citing an unnamed investor familiar with the company, Business Insider reported that investors feared Moderna’s valuation was too high and would likely be more optimistic if it were more typical of a biotech company, in the billion-dollar range. As of early afternoon Friday, Moderna’s market cap was $4.63 billion.

Moderna raises more than $600M in biggest-ever IPO

Moderna Therapeutics seeks $500M for IPO

Policy Trend: Drug pricing

With insulin prices so high that Type 1 diabetes patients are resorting to potentially lethal rationing, while others pursue DIY drugs, and the scandal around Mylan jacking up the price of the life-saving EpiPen, drugmakers have come under increasing criticism for the prices they charge.

Enter the Trump administration, which has made drug prices a key part of its agenda. While drug prices are not part of the FDA’s mandate, Commissioner Scott Gottlieb has been unusually aggressive, at least to the extent he can be. In May, the agency published a list of branded drugmakers that have been the subject of complaints from generic manufacturers for making it difficult for the latter to obtain the product samples they need to produce generics. And this month, Gottlieb proposed a pathway for biosimilar competition for branded insulin products.

The Department of Health and Human Services has also made a number of proposals in its “blueprint” for lowering drug prices that it released earlier this year. However, while some of the proposals – like reference pricing, cracking down on deals between pharmacy benefit managers and manufacturers and requiring drugmakers to include list prices in ads – have generated discussion, many experts have doubts about whether it will change very much.

FDA’s Gottlieb proposes biosimilar pathway to tackle skyrocketing insulin prices

FDA is throwing shade on these drugmakers

Trump’s drug cost cutting plans include transparency, but critics doubt much will change

Photo: Stuart Ritchie, Getty Images