Recap of the 2018 Alliance for Regenerative Medicine Cell and Gene Therapy State of the Industry

What happened in the Cell and Gene Therapy sector in 2017, and what should we be looking out for in 2018? Over 500 executives, investors, scientists and patient advocates gathered together yesterday to find out at the Alliance for Regenerative Medicine (ARM) State of the Industry Briefing in San Francisco, California.

ARM Chairman, Robert Preti, and ARM CEO, Janet Lynch Lambert, kicked off the session by discussing how 2017 marked an inflection point for the sector. They underscored the approval of three cell/gene therapies (see slide below) by the U.S. Food and Drug Administration (FDA), a “bright and robust” future pipeline that should yield over 40 approved therapies in the next five years, and an improving regulatory environment that’s accelerating approvals of regenerative medicine therapies. This year alone, the FDA has granted 12 Regenerative Medicine Advanced Therapy (RMAT) designations through the 21st Century Cures Act (see slide below for companies/products that received RMAT in 2017).

In 2017, a total of four cell/gene therapies were approved and the US FDA awarded 12 RMAT designations. This slide is from the 2018 ARM Cell and Gene Therapy State of the Industry Briefing presentation.

Next up was a snapshot of the clinical landscape highlighting a total of 946 ongoing clinical trials at the end of 2017, and their breakdown by disease (see chart below). Oncology (cancer) is the clear winner comprising over 50% of the trials while Cardiovascular (heart) took second with 8.6% and diseases of the central nervous system (brain and spinal cord) took third with 6.5%.

Lambert also gave a brief overview of finances in 2017 and listed some impressive numbers. $7.5 Billion in capital was raised in 2017 compared to $4.2 Billion in 2016. She also mentioned major acquisitions, mergers, partnerships and public financings that paved the way for this year’s successes in cell and gene therapy.

Lambert concluded that while there was significant progress with product approvals, growing public awareness of successes in the sector, regulatory advances and financial maturity, there is a need for further commercial support and a focus on policy making, industrialization and manufacturing.

The Industry Update was followed by two panel sessions.

The first panel focused on cell-based cancer immunotherapies and featured company leaders from Juno Therapeutics, Mustang Bio, Adaptimmune, Novartis, and Fate Therapeutics.

In the cancer field, companies are aggressively pursuing the development of cell-based immunotherapies including Chimeric Antigen Receptor T (CAR-T) cells, modified T-cells and Natural Killer (NK) cells, to name a few. These therapies all involve engineering or modifying human immune cells to identify and target cancer cells that resist first-line cancer treatments like radiation or chemotherapy.

The panelists spoke of a future that involved the development of combination therapies that partner cell-based immunotherapies with other drugs and treatments to better target specific types of cancer. They also spent a significant portion of the panel discussing the issues of manufacturing and reimbursement. On manufacturing, the panel argued that a centralized cell manufacturing approach will be needed to deliver safe products to patients. On reimbursement, they addressed the difficulty of finding a balance between pricing life-saving therapies and navigating reimbursements from insurance companies.

The second panel focused on the state of gene therapy and the outlook for 2018. This panel featured company and academic leaders from CRISPR Therapeutics, Sangamo Therapeutics, BioMarin Pharmaceutical, Adverum Biotechnologies, and the Gladstone Institutes.

ARM Gene Therapy Panel: Martha Rook (MilliporeSigma), Deepak Srivastava (Gladstone Institutes), Amber Salzman (Adverum Biotechnologies), Bill Lundberg (CRISPR Therapeutics), Geoff Nichol (BioMarin Pharmaceutical), Sandy Macrae (Sangamo Therapeutics)

The panel spoke about the difference between gene editing (fixing an existing gene within a cell) and gene therapy (adding a new gene into a cell) technologies and how the delivery of these therapies into tissues and cells is the biggest challenge in the area right now.

Sandy Macrae, President and CEO of Sangamo Therapeutics, made an interesting point when he said that for gene therapy to be successful, companies need to plan two to three years in advance for a phase III trial (the final stage before a product is approved) because manufacturing gene therapies takes a long time. He said the key for success is about having medicines that are ready to launch, not just reporting good results.

Overall, ARM’s State of the Industry provided an exciting overview of the progress made in the Cell and Gene Therapy Sector in 2017 and shared outlooks for 2018 and beyond.

You can access the Live Webcast of ARM’s State of the Industry Briefing including both panel sessions on the ARM website. Be sure to check out our blog featuring our 2018 Stem Cell Conference Guide for more ARM events and other relevant stem cell research meetings in the coming year.

Stem cell and gene therapy research gets a good report card from industry leader

arm

Panel discussion at ARM State of the industry briefing: left to Right Robert Preti, Chair ARM; Jeff Walsh, bluebird bio; Manfred Rudiger, Kiadis Pharma; Barbara Sasu, Pfizer;  Thomas Farrell, Bellicum Pharmaceuticals. Photo courtesy ARM.

The state of the regenerative medicine field is strong and getting stronger. That was the bottom line verdict at the 2017 Cell and Gene Therapies State of the Industry briefing in San Francisco.

The briefing, an annual update on the field presented by the Alliance for Regenerative Medicine (ARM), gave a “by the numbers” look at the field and apart from one negative spot everything is moving in the right direction.

Robert Preti, Chair of ARM’s Board, said worldwide there are more than 750 regenerative companies working in the stem cell and gene therapy space. And those companies are increasingly moving the research out of the lab and into clinical trials in people.

For example, at the end of 2016 there were 802 clinical trials underway. That is a 21 percent growth over 2015. Those breakdown as follows:

Phase 1 – 271 (compared to 192 in 2015)

Phase 2 – 465 (compared to 376 in 2015)

Phase 3 – 66 (compared to 63 in 2015)

The bulk of these clinical trials, 45 percent, are focused on cancer. The second largest target, 11 percent, is on heart disease. The number of trials for neurological disorders and rare diseases are also growing in number.

Preti says the industry is at an important inflection point right now and that this growth is presenting new problems:

“The pipeline of products is robust and the technologies supporting that pipeline is even more robust. The technologies that are fueling the growth in clinical activity have accelerated so fast that we on the manufacturing side are playing catchup. We are at a point where we have to get serious about large scale commercial production.”

Preti also talked about “harmonization” of the regulatory process and the need to have a system that makes it easier for products approved for clinical trials in one country, to get approval for clinical trials in other countries.

Michael Werner, the executive director of ARM, said the organization has played a key role in helping promote the field and cited the recently passed 21st Century Cures Act as “a major win and a powerful statement of ARM’s leadership in this sector.”

But there was one area where the news wasn’t all positive, the ability of companies to raise capital. In 2015 companies raised $11 billion for research. In 2016 it was less than half of that, $5.3 billion.

With that somber note in mind it was appropriate that the panel discussion that followed the briefing was focused on the near-term and long-term challenges facing the field if it was to be commercially successful.

One of the big challenges was the issue of regulatory approval, and here the panel seemed to be more optimistic than in previous years.

Manfred Rüdiger of Kiadis Pharma said he was pleasantly surprised at how easy it was to work with different regulatory agencies in the US, Canada and Europe.

“We used them as a kind of free consultancy service, listening to their advice and making the changes they suggested so that we were able to use the same manufacturing process in Europe and Canada and the US.”

Jeff Walsh of bluebird bio, said the key to having a good working relationship with regulatory agencies like the Food and Drug Administration (FDA) is simple:

“Trust and transparency between you and the regulatory agencies is essential, it’s a critical factor in advancing your work. The agencies respond well when you have that trust. One thing we can’t be is afraid to ask. The agencies will tell you where their line is, but don’t be afraid to ask or to push the boundaries. This is new for everyone, companies and regulators, so if you are pushing it helps create the environment that allows you to work together to develop safe therapies that benefit patients.”

Another big issue was scalability in manufacturing; that it’s one thing to produce enough of a product to carry out a clinical trial but completely different if you are hoping to use that same product to treat millions of people spread out all over the US or the world.

And of course cost is always something that is front and center in people’s minds. How do you develop therapies that are not just safe and effective, but also affordable? How do companies ensure they will get reimbursed by health insurers for the treatments? No one had any simple answer to what are clearly very complex problems. But all recognized the need to start thinking about these now, long before the treatments themselves are even ready.

Walsh ended by saying:

“This is not just about what can you charge but what should you charge. We have a responsibility to engage with the agencies and ultimately the payers that make these decisions, in the same way we engage with regulatory agencies; with a sense of openness, trust and transparency. Too often companies wait too long, too late before turning to the payers and trying to decide what is appropriate to charge.”

 

 

What Went Down at ARM’s Regenerative Medicine State of the Industry

Every January, downtown San Francisco is taken over by a flock of investors, bankers, biotech companies, and scientists attending the annual JP Morgan Healthcare Conference. This meeting looks at the healthcare advancements over the past year and predicts the disease areas and technologies that will see the most progress and success in 2016.

According to some of the experts at the event, regenerative medicine and stem cell research are experiencing impressive, accelerated advancements, which has peaked the interest of investors, biotech, and pharmaceutical companies.

Because these are such fast paced fields, the Alliance for Regenerative Medicine (ARM) hosts the Annual Regenerative Medicine and Advanced Therapies State of the Industry Briefing during JP Morgan to discuss the recent progress and outlook for the industry in the coming year.

Screen Shot 2016-01-11 at 4.03.30 PM

What happened in 2015 and what’s next?

ARM’s  6th Annual Briefing was open to the public and drew over 300 people on Monday morning. The meeting opened with an industry update from Edward Lanphier, ARM Chairman and President/CEO of Sangamo BioSciences.  Then two panels featuring top leaders from biotech and pharmaceutical companies discussed the 2016 clinical data forecast and the promise of regenerative medicine and advanced therapies in oncology (cancer).

With an upbeat attitude, Lanphier gave an overview of clinical development progress in 2015, with 20 approved products worldwide and over 600 clinical trials both from academia and industry. More than 40% of these ongoing clinical trials are in cancer while approximately 12% are in heart disease/injury. These trials are not limited to Phase 1 either. In 2015, there were 376 in Phase 2 (compared to 200 in 2014) and 64 in Phase 3 (compared to 39 in 2014).

Edward Lanphier

Edward Lanphier

Two other areas Lanphier emphasized were CAR-T and other cell-based immunotherapies and gene therapy programs for rare diseases. He ended with 2015 statistics on clinical milestones in various disease and therapy programs, key company IPOs, the financial landscape, and predictions of major anticipated data from clinical trials in 2016.

It was a lot to take in, but this was definitely a good thing and a sign that the areas of regenerative medicine and advanced therapies are thriving. If you want more details, you can check out ARM’s State of the Industry presentation.

Major Theme: Data is King

The major theme that cropped up during the industry update and panel discussions was the importance of producing meaningful clinical data to get positive outcomes in regenerative medicine.

This was succinctly put by panelist Sven Kili, head of Gene Therapy Development at GlaxoSmithKline:

“I would say “Data is King”. A great idea is fantastic, passion is wonderful, and most companies will buy into a strong management team, but that only gets you so far. After that you need to have data, and you need to have a good plan for going forward.”

Kill added that there’s the need to work with the FDA to change the regulatory process, saying the FDA is, understandably, cautious about working with therapies that can alter a person’s genome permanently. However, he said there needs to be serious discussions with the FDA about how to speed up the process, to make it easier for the most promising projects to get approval.

Edward Lanphier also talked about the industry’s new focus on clinical data and the questions that arise when trying to advance regenerative medicine research into approved treatments and cures for patients:

“How do we communicate the value of curing blindness? How do we think about pricing that? What do we think about [drug] reimbursement?  For rare diseases, we aren’t trying to talk about acute treatments – we are talking about one-time, curative outcomes. And the value and benefit to patients in this is enormous. This is what we are trying to do, and on the cusp of, in terms of generating both approvable data and also the proof of concept data that then allows us to drive that next value inflection point in terms of financings.”

The Future Looks Good

After listening to the briefing, the future of regenerative medicine and advanced therapies certainly looks bright. As Jason Kolbert, head of Healthcare Research at the Maxim Group, said:

“This industry is now rapidly maturing and regenerative medicine and gene therapy have great things in store for the next decade.”

Usman Azam, Global Head of Cell and Gene Therapies at Novartis, had a similar outlook:

“We now are going from proof of concept to commercial availability of a disruptive innovation within seven years. If somebody had said that to me four years ago, I would have said, not possible. But that gives you a sense of how quickly this field is moving.”

Experts Panel

ARM Panel: 2016 Sector Forecast: Upcoming Clinical Data Events

Creative partnerships that promote progress

Lewis and Clark: great partnerships can change the world

Lewis and Clark: great partnerships can change the world

Having a good partner can turn something good into something truly memorable. Where would Laurel be without Hardy, Lewis without Clark, Butch Cassidy without the Sundance Kid. That’s why the stem cell agency has partnerships on a number of different levels as part of our mission of accelerating the development of stem cell cures to patients with unmet medical needs.

Our latest partnership is with RegMedNet which, in its own words, “provides a unique and unparalleled platform for the regenerative medicine community to share insights, discuss the latest research, and help move the field forward.” With a goal like that why would we not want to support them?

Like us RegMedNet believes that regenerative medicine is going to completely change the way we treat disease, even the way we think about disease. They also believe that progress of the kind we all want is only going to come by bringing together all the key players from the researchers and manufacturers, to the government regulators and, of course, the patient advocates. Each has a vital role to play in moving the field forward and RegMedNet reflects that in both the content it posts online and in the contributors, who represent institutions and companies worldwide.

One of the most important elements in any partnership is understanding, and RegMedNet does a great job of trying to raise awareness about the field, the challenges we all face, and the progress being made. Bringing together so many different perspectives in one spot really helps create a much deeper understanding of regenerative medicine as a whole.

In a few short years regenerative medicine has gone from a relatively small field to a global industry. Our hope is that creating partnerships with like-minded groups around the world, is going to help it get even bigger and, even better.

How one strong ARM can create a community

I spent the last two days at the annual Washington meeting of the Alliance for Regenerative Medicine (ARM), the advocacy organization that CIRM became a founding member of in 2009. Having been CIRM’s representative at that first organizing meeting it has been a pleasure to see the organization mature into an effective advocacy group for our field. It has lived up to its goal of creating a community where all the stakeholders in the field, from academic and industry leaders to patient advocates and investors, can come together in a coordinated front.

ARM and CIRM share the goal of accelerating the development of regenerative therapies to patients with unmet medical needs. The organization also dovetails well with our effort to inform the public about the great hope in the field. To quote ARM’s website: “ARM also works to increase public understanding of the field and its potential to transform human healthcare.”

But that transformation can be fostered or impeded by actions in our nation’s capital, both regulatory and legislative, the main thrust of the past two days’ activities.

While the iconic Capitol building is the most recognized footprint of our Congress, it is the House and Senate office buildings that ring three sides of the Capitol where most of the work gets done, like in the Rayburn building, which houses the office of Dianna DeGette, the Colorado congresswoman and champion of regenerative medicine.

While the iconic Capitol building is the most recognized footprint of our Congress, it is the House and Senate office buildings that ring three sides of the Capitol where most of the work gets done, like in the Rayburn building, which houses the office of Dianna DeGette, the Colorado congresswoman and champion of regenerative medicine.

ARM members presented three specific proposals for advancing the field to members of congress and their staffs. These would:

  • Create a center of excellence to develop technical and process standards for regenerative medicine. Not very sexy on the surface, but agreement in advance on what regulators will accept in creating a new product can shave months or years off the development of needed therapies.
  • Create a special pathway within the Food and Drug Administration—much like the one created for orphan diseases—for “Qualified Regenerative Medicine Products (QRMPs). These products would have shown potential to change the course of a disease with currently unmet medical needs and the FDA would be required to meet with their sponsors to discuss expedited review of the product.
  • Advocate for the adoption of a national regenerative medicine strategy that includes federal agency coordination, support for research and regulatory reform to create a clear and predictable pathway that enables quick approval of safe and effective products. To accomplish that ARM has promoted the establishment of a Regenerative Medicine Coordinating Council within the U.S. Department of Health & Human Services.
Jamie Goldfarb with her son Kai and husband Jeff. Photo courtesy Melanoma Research Alliance

Jamie Goldfarb with her son Kai and husband Jeff.
Photo courtesy Melanoma Research Alliance

Jamie Goldfarb, who beat back melanoma with the help of a cell-based immune therapy, made a clear and passionate case for the urgency of making it easier to get these therapies to patients at the ARM member dinner Tuesday night:

“Enhanced awareness for the power of regenerative medicine means a world of difference. It means less suffering, less pain, less fear, less expense, less hardship, less loss. It also means more hope, more determination, more love, more strength for individuals and for society as a whole. Every person in this room and those organizations you represent are improving lives.”

Don Gibbons