Jan 13-16, 2025
The Westin St. Francis Hotel
San Francisco, USA
JPM24, Day 1: Novartis reportedly in late talks for Cytokinetics after nabbing a
Merck KGaA spinoff; J&J, Merck tie up M&A deals
The J.P. Morgan Healthcare Conference in San Francisco is back and here at Fierce
Biotech, we’re going to bring you all the latest updates live from the life sciences event of the year.
This year we’re not just dusting off the New Year’s confetti and trying to contain the outrage
over the price of, well, everything, but also the flurries of M&A and licensing deals that have
blown into life sciences land in the run up to JPM 2024.
Those deals involve a host of Big Pharmas, including the likes of Bristol Myers
Squibb, Boehringer Ingelheim, AstraZeneca, Johnson & Johnson, AbbVie, Novo Nordisk,
Merck KGaA, Roche, as well as for Karuna, Insilico, Galapagos, and Moma, with a particular
focus on experimental research for obesity and cancer.
But on the flip side, we also saw a major pruning in the gene-editing and cell therapy space
as AlloVir, AlloGene, Intellia, and Aera either took the ax to staffers and programs or had a
major re-think on their research direction, as they streamline into 2024.
With such a major series of moves before wheels up for SF, what can we expect from JPM
itself? The Fierce team will be on the conference floor hunting down the biggest
announcements and interviews, so stay tuned.
Check out our daily updates below for all the latest from JPM today and come back each day
this week for your JPM roundups.
Monday 6:00 p.m. ET Jan. 8
CG Oncology and CARGO Therapeutics have each individually drawn partnership
interest, with late-stage programs that have become the apple of Big Pharma's eye,
according to Ally Bridge investor Andrew Lam.
"I think most actionable within our portfolio are things that are going into or in pivotal-stage
clinical development, phase two post-proof of concept," he said. Lam did not say which
potential partners were lining up for each.
CG Oncology was the first biotech to file IPO paperwork in 2024, while cell therapy-focused
CARGO raked in almost $300 million from its IPO in November 2023.
Editor's note: This was updated to clarify that CARGO and CG are not in partnership talks
with each other.
Monday 5:45 p.m. ET Jan. 8
Merck & Co. CEO Rob Davis said the company "is open to deals of any size," aside from
large-scale, expensive acquisitions that would force the company to fuse workforces. His
comments follow the company's $680 million acquisition of Harpoon Therapeutics earlier in
the day. The deal adds further diversity to Merck's oncology portfolio and places it squarely
in T-cell engaged race alongside Amgen. The class has demonstrated an ability to treat
small cell lung cancer, a hard-to-treat lung cancer that's played second fiddle to NSCLC.
Davis and research chief Dean Li, M.D., Ph.D., were consistent with previous comments that
dealmaking decisions are led by the best science, not the best financial opportunity. Davis
says is it just so happens that Merck's "sweet spot" have been deals in the $0-15 billion
range. The New Jersey pharma acquired immunology biotech Prometheus for $10.8 billion
and is paying Daichii Sankyo at least $5.5 billion over two years for a batch of antibody-drug
conjugates.
Monday 1:45 p.m. ET Jan. 8
Novartis is in advanced talks to buy Cytokinetics, the Wall Street Journal reported Monday.
The San Francisco biotech reported in late December that its heart
medication aficamten improved patients' exercise capacity in a phase 3 trial, sending the
company's shares soarings. Rumors have swirled about pharmaceuticals lining up to buy
Cytokinetics in the weeks since.
Monday 1 p.m. ET Jan. 8
Global computing powerhouse Nvidia has announced a series of new initiatives that include
a collaboratiaon with Amgen’s deCode. The Amgen company will power Nvidia's new
genomics foundation models alongside Nvidia’s supercomputer and BioNeMo generative AI
platform. BioNeMo is a suite of programs aimed at accelerating drug discovery by combining
several foundational models that companies can tinker with using their own
data. Recursion is also joining the party and will be the first third-party addition to the
BioNeMo platform.
Monday 7:35 a.m. ET Jan. 8
Where Merck kicks us off with the first official deal of JPM 2024 Johnson &
Johnson swoops in with a $2 billion deal to snap up Ambrx. For its cash, J&J nabs
Ambrx's antibody drug conjugate (ADC) research programs, including ARX517, which is in a
phase 1/2 trial for advanced prostate cancer.
Monday 7:30 a.m. ET Jan. 8
After rumors swirled from Bloomberg this morning, Merck has officially bought out immunooncology
biotech Harpoon Therapeutics for $23 a share, or $680 million. Merck will get its
hands on Harpoon’s lead candidate, HPN328, a T-cell engager targeting delta-like ligand 3
(DLL3), that is in early-to-mid stage tests for small cell lung cancer (SCLC) and
neuroendocrine tumors.
Monday 6:30 a.m. ET Jan. 8
J.P. Morgan’s annual 2023 Biopharma Licensing and Venture Report found that biopharma
licensing partnerships accounted for $63 billion in total value during the fourth quarter from
108 deals. The biggest focus was on antibody-drug conjugates (ADCs) in cancer research,
which drove a fourth-quarter spike in licensing deal. Fierce Biotech's senior editor Annalee
Armstrong drills down into the report and what it means for biopharma.
Monday 2 a.m. ET Jan. 8
The first official M&A deal of JPM ’24 sees Novartis snap up Merck KGaA autoimmune
spin-off Calypso in an upfront deal worth $250 million with an extra $175 million in
biobucks. The Swiss Big Pharma nabs its lead candidate, CALY-002, an experimental
antibody that binds to and neutralizes Interleukin-15.
Monday 1 a.m. ET Jan. 8
Good morning from the Fierce Biotech team in the office and on the ground at San
Francisco. There’s something of a tradition that at least one major M&A deal is announced
over the weekend before we officially kick things off today, but despite whispers and rumors
from some corners, that deal never materialized.
We did however see two separate licensing deals Sunday morning from Alphabet’s
Isomorphic, with Eli Lilly and Novartis, respectively. These pacts are worth nearly $3
billion in combined biobucks, but with relatively low upfronts. Both Big Pharmas will
tap Google DeepMind’s AlphaFold AI tech, the foundation of Isomorphic’s platform.
JPM24, Day 1: Even after M&A deals, BMS CEO focuses on pipeline; Amgen
spotlights obesity prospects
As the J.P. Morgan Healthcare Conference kicked off in San Francisco, EY released its
annual report on the industry's dealmaking firepower. Plus, Regeneron, Moderna, bluebird
and many other companies updated investors on their businesses heading into 2024.
UPDATED: Monday, Jan. 8 at 7:20 p.m. ET
Regeneron is happy with the launch of its high-dose version of Eylea. In Q4, its first full
quarter on the market, sales reached $123 million, the company revealed in an SEC filing on
Monday. Later in the day, at the J.P. Morgan Healthcare Conference, CEO Len Schleifer
called the figure “quite a good achievement by all measures compared to other recent
launches in the space.” Schleifer, of course, was referring to Roche’s Vabysmo, which by
comparison pulled in $112 million in its first full quarter on the market, Q2 of 2022, and was
assured of blockbuster sales in 2023. Vabysmo’s success is attributed to its longer duration
of effectiveness, allowing patients to need injections every three to four months versus
Eylea, which is dosed every two months. The HD version of Eylea is Regeneron and Bayer’s
answer to Vabysmo, showing durability in trials that is superior to that of Vabysmo for some
patients. The successful launch of Eylea HD came despite some adversity. “There’s still
some amount of prescriber hesitancy related to the fact that we don’t have a permanent Jcode,”
Schleifer said, referring to the identifying system that allows pharmacies, hospitals
and physicians to bill for medications. Once Eylea HD receives its J-code, it will facilitate
better access to the drug, said Regeneron’s commercial chief Marion McCourt, who added
that patient feedback has been “very positive.”
UPDATED: Monday, Jan. 8 at 4:18 p.m. ET
After last year’s $7.3 billion buyout of Reata, Biogen CEO Chris Viehbacher touted a
“tremendous” launch of Friedreich's ataxia therapy Skyclarys. The drug is “outperforming”
other rare-disease launch analogues, including Biogen’s own spinal muscular atrophy launch
Spinraza, the CEO said. Because of a prior manufacturing-related delay for the drug, there
had been a buildup of patients eager to start on therapy, Viehbacher explained. Now with
approvals in the U.S. and Europe, Biogen is working to make the most of a medicine it
inherited with the Reata buyout. He noted that the company still needs to conduct a pediatric
study of Skyclarys because many patients are diagnosed with Friedreich's ataxia before the
age of 16.
As Johnson & Johnson moves forward without its consumer healthcare group, the
company is leaning on several new launches to drive growth. In multiple myeloma, J&J
figures Tecvayli, Talvey and Carvkyti can handily beat analyst expectations by 2027, CEO
Joaquin Duato said Monday at the J.P. Morgan Healthcare Conference. Between new
launches and the blockbuster Darzalex, J&J’s multiple myeloma franchise is heading toward
potential annual sales of $25 billion, according to the CEO. In addition, depression nasal
spray Spravato is poised to beat out analyst estimates, according to the company’s
calculations. All this comes as J&J steams toward $57 billion in pharmaceutical sales by
2025.
UPDATED: Monday, Jan. 8 at 3:53 p.m. ET
For Gilead Sciences, 2024 is set to be a “very catalyst-rich” year, according to CEO Daniel
O’Day. With up to two dozen clinical trial readouts and no patent cliffs on the horizon, the
company is moving into a “new era," he explained. Four years ago, the drugmaker pulled in
some $500 million from its oncology offerings. Now armed with antibody-drug conjugate
Trodelvy and cell therapies Yescarta and Tecartus, the company’s oncology portfolio is
generating around $3 billion annually. As O’Day sees it, that performance falls in line with
Gilead’s previously communicated goal of pulling a third of its overall revenues from the
oncology field by 2030. The work with Trodelvy isn't done yet, though, as the company looks
to advance the med into earlier lines of therapy, plus expand into different tumor types. Then
there’s Arcellex-partnered CAR-T anito-cel, which is “well-positioned” for a 2026 launch in
multiple myeloma. While Gilead remains busy with its oncology ambitions, the company
hasn’t forgotten about its bread-and-butter HIV franchise. Gilead is looking at five potential
new launches by 2030 in both treatment and pre-exposure prophylaxis (PrEP), many of
which use the company’s long-acting HIV treatment lenacapivir (currently marketed as
Sunlenca).
UPDATED: Monday, Jan. 8 at 1:35 p.m. ET
2024 looks to be a “busy year” for Amgen, CEO Robert Bradway said during his company's
presentation on Monday. After integrating Horizon Therapeutics and building out its
biosimilar business, the company is putting a particular focus on its obesity program, led by
phase 2 asset MariTide (maridebart cafraglutide). Despite intense competition in the obesity
space, the company says that its contender has a differentiated profile. Amgen plans to
review results from a clinical study of the prospect later this year. Aside from MariTide,
Amgen is working on an oral small molecule that’s currently in phase 1, as well as “half a
dozen or so” preclinical programs, Bradway said. In oncology, Amgen's tarlatamab is up for
an FDA decision by June 12 in certain patients with advanced small cell lung cancer.
Beyond that indication, the company looks to study the med in earlier disease stages. As for
biosimilars, Amgen's next three launches will target Johnson & Johnson’s Stelara,
Regeneron’s Eylea and Alexion’s Soliris. Behind those three meds is a biosimilar candidate
targeting Bristol Myers Squibb’s Opdivo and two other undisclosed medicines.
UPDATED: Monday, Jan. 8 at 12:50 p.m. ET
It took Bristol Myers Squibb’s new CEO Chris Boerner 16 minutes into his 24-minute
presentation to address the big news the company made last month with its
holiday buyouts of Karuna Therapeutics, for $14 billion, and RayzeBio, for $4.1 billion. What
Boerner most wanted to highlight was the company’s pipeline, which could deliver more than
16 new products through 2030. “These products are overwhelmingly first or best in class,”
Boerner said. “And it is the pipeline momentum that supports the growth opportunities that
we see in the back half of the decade.” That period will be crunching time for BMS as it
bears the brunt of patent expirations and Inflation Reduction Act effects for its trio of
blockbusters drugs Revlimid, Eliquis and Opdivo, which accounted for 65% of the company’s
revenue in 2022.
UPDATED: Monday, Jan. 8 at 12:44 p.m. ET
Blueprint Medicines has “decided to discontinue global development and marketing of
Gavreto in territories excluding the U.S. and Greater China,” the company said in a Monday
update. Before this disclosure, Blueprint’s former partner on the targeted cancer medicine,
Roche, opted to end their collaboration. Blueprint cited a “lack of global infrastructure in lung
and thyroid cancer” as its reason for discontinuing certain efforts on the medicine. Besides
the Gavreto disclosure, Blueprint said it’s ending further investment in pipeline meds BLU-
945 and BLU-451. Meanwhile, Blueprint expects Ayvakit’s launch in indolent systemic
mastocytosis to “drive strong revenue growth in 2024,” the company said.
Even after a late-stage trial failure for sparsentan in focal segmental glomerulosclerosis
(FSGS), Travere Therapeutics said it’s conducting additional reviews and “will engage with
regulators to evaluate potential regulatory pathways.” The development comes after the
company last month said it was cutting 20% of its workforce to reduce operating costs in the
wake of the trial failure. Travere’s sparsentan is already approved with the brand name
Filspari to treat primary immunoglobulin A nephropathy. Since the launch in February 2023,
the drug has generated $30 million, the company said on Monday.
As Apellis progresses with the launch of two key brands, the company said it pulled down
roughly $366 million during 2023. Syfovre, Apellis’ treatment for geographic atrophy,
generated around $275 million, while paroxysmal nocturnal hemoglobinuria medicine
Empaveli was responsible for $91 million of the company's haul. Both drugs use the same
active ingredient, pegcetacoplan, which Apellis is also testing in several other rare diseases.
In a statement, Apellis’ CEO Cedric Francois, M.D., Ph.D., said the company’s recent
commercial progress “has positioned us for an even stronger 2024.”
UPDATED: Monday, Jan. 8 at 11:21 a.m. ET
Regeneron hopes its longer-lasting Eylea formulation, Eylea HD, can build on the success
of the company's original blockbuster product. With $123 million in sales during Eylea HD's
first quarter on the market, the eye med is showing early indicators of broad initial uptake,
CEO Leonard Schleifer, M.D., Ph.D., said in a corporate update. But overall Q4 Eylea sales,
at $1.46 billion, missed analyst expectations of $1.49 billion to $1.56 billion, according to
Piper Sandler analyst Christopher Raymond. Meanwhile, Sanofi-partnered immunology
superstar Dupixent posted solid sales growth in 2023 continues to build on its “pipeline in a
product” potential, according to Regeneron. The drugmaker highlighted the biologic's
potential in chronic obstructive pulmonary disease, an indication where Dupixent has picked
up an FDA breakthrough therapy designation. Elsewhere, the company looks to gain ground
in oncology with its Libtayo offering. The med is slated to exceed $1 billion in global net
product sales in 2024, Regeneron said.
UPDATED: Monday, Jan. 8 at 10:35 a.m. ET
Exelixis has preliminarily disclosed 2023 U.S. Cabometyx revenues of $1.63 billion—
compared with $1.4 billion in 2022—and it projects 2024 product sales will land between
$1.65 billion and $1.75 billion. Cabometyx, used in combination with Roche’s Tecentriq,
recently beat a change of hormonal therapy on progression-free survival in metastatic
castration-resistant prostate cancer patients who had tried a hormonal therapy. As the study
continues to evaluate its other primary endpoint of overall survival, Exelixis said it will
continue to talk to the FDA to find a potential regulatory path forward. Another investigatorinitiated
trial also showed Cabometyx’s benefit in advanced neuroendocrine tumors, and
Exelixis is working on a potential FDA filing in that indication this year. Amid uncertainties
related to Cabometyx’s label expansion, Exelixis will launch a restructuring that will cut about
13% of its workforce, or about 175 employees, to focus on its pipeline. The company’s board
also authorized a $450 million stock buyback for 2024.
UPDATED: Monday, Jan. 8 at 10:10 a.m. ET
For all of 2023, PTC Therapeutics brought in $946 million in unaudited revenues, good for a
35% increase from the prior year. Besides product revenues of $661 million, PTC’s
collaboration and royalty revenue associated with Roche’s commercialization of spinal
muscular atrophy therapy Evrysdi came to $278 million, the company said in a release. PTC
is kicking off JPM with a 10:30 a.m. ET presentation today, where it’ll share updates about
its ongoing launches and pipeline advancement efforts. In 2024, the company expects to
finalize FDA and European Medicines Agency filings for sepiapterin for phenylketonuria. The
drugmaker also expects to submit an FDA filing for Upstaza for the treatment of AADC
deficiency during the first quarter of the year, among other planned R&D developments.
After a “transformative year,” Mirum Pharmaceuticals CEO Chris Peetz said the company
is looking toward more milestones in 2024. Specifically, the company projects continued
revenue growth and a potential label expansion for Livmarli in progressive familial
intrahepatic cholestasis by March 13. Further, the company is preparing a regulatory filing
for Chenodal in cerebrotendinous xanthomatosis after a phase 3 trial win. As for 2023, the
company said it generated $186 million to $188 million, but that the figure is unaudited. The
company’s Alagille syndrome therapy Livmarli pulled down $141 million to $143 million last
year, good for around 89% growth from the prior year. Mirum’s full JPM presentation will be
on Wednesday at 1:30 p.m. ET.
UPDATED: Monday, Jan. 8 at 9:10 a.m. ET
With nine late-stage pipeline programs in development and 45 overall, Moderna expects to
have an “exciting year," CEO Stéphane Bancel said in a business update. The mRNA
specialist pulled down $6.7 billion in 2023 revenues, including $6.1 billion in COVID-19
vaccine sales. Moderna expects its Spikevax to remain profitable and projects some $4
billion in 2024 product sales, which includes contributions from an anticipated RSV vaccine
launch. Other than its RSV prospect, the company looks to file its seasonal flu vaccine with
regulators this year and is expecting data from several assets, including its next-generation
COVID vaccine and its cytomegalovirus vaccine. Moderna’s full presentation at the
conference will take place at 6:45 p.m. ET today.
EY's Firepower report, which publishes annually on the opening day of the J.P. Morgan
Healthcare Conference, assesses the capacity of the industry’s top 25 companies to execute
M&A deals based on the strength of their balance sheets. EY tabulated the available
firepower of the companies at $1.37 trillion entering 2024, which is the second-highest figure
in the 10 years of the analysis. Another factor that bodes well for M&A in 2024 is the sudden
surge in high-dollar deals at the end of 2023. Several other elements are in play—including
patent expirations, government regulations and the recent failure of several late-stage
assets—which signal urgency by large pharma companies to make deals for late-stage and
commercial assets, EY analysts say.
Busy with three gene therapy launches, bluebird bio on Monday said it has cash on hand to
last until the first quarter of 2025. On the heels of a recent FDA approval for bluebird’s sickle
cell disease gene therapy Lyfgenia, 35 treatment centers are ready to receive referrals for
the medicine, bluebird said in a release. Plus, after recently striking two outcomes-based
deals on Lyfgenia, the company says talks with other payers are in “advanced discussions.”
Besides Lyfgenia, bluebird is also progressing with the launches of beta thalassemia
medicine Zynteglo and cerebral adrenoleukodystrophy medicine Skysona. Bluebird’s full
presentation at the J.P. Morgan Healthcare Conference is on Tuesday at 1:30 p.m.
JPM24, Day 2: GSK, Eli Lilly, Blueprint, SR One and more
Welcome to Day 2 of the J.P. Morgan Healthcare Conference 2024 in San Francisco.
Yesterday "Merger Monday" lived up to its name with deals for Novartis, J&J and Merck, and
today, we’re also starting Tuesday with an early deal. Check out that deal and all the updates
from JPM below.
Tuesday 6:43 p.m. ET Jan. 9
Eli Lilly CEO David Ricks, speaking to the slow and steady progress that brought tirzepitide
to the world, says the Indianapolis pharma is now ready to open up the chocolate factory.
“I know a number of biotechs are in the room at this conference so it's worth mentioning:
we're open for business on external innovation,” Ricks said at the J.P. Morgan Healthcare
Conference Tuesday.
Tuesday 6:10 p.m. ET Jan. 9
SR One CEO and managing partner Simeon George, M.D., said the firm was eyeing some
investments in psychedelic-focused biotechs but is waiting on formally financing one to see
how the space matures. He specifically pointed to the rollout of J&J's Spravato, an
esketamine spray used to treat major depression. The treatment is currently one of J&J's
fastest-growing products and is a pillar in the company's neuroscience portfolio.
"We've been watching, obviously, how that has gone in terms of the launch and again, that
product looks like it's starting to be on a nice ramp there," George said in an interview with
Fierce Biotech on Tuesday. "My sense is there's going to be more than a couple of
companies that will be interesting to look at from a fundraising perspective."
George said there are probably "one or two" companies on the firm's shortlist, though he
wouldn't specify which.
Tuesday 3:45 p.m. ET Jan. 9
"We're seeing companies make investments in traditional pharma assets that are a little
further along in the development pipeline," Sharon Flanagan, managing partner for global
law firm Sidley's San Francisco office, told Fierce Biotech on the conference sidelines.
"ADCs (antibody drug conjugates) are really interesting," she continued, citing Johnson &
Johnson's $2 billion acquisition of ADC-focused Ambrx Biopharma. "So that's a way to do
something that's still pharma, but with something novel and interesting, that has a spin to it. I
do think in markets that are a little uncertain, sometimes the more truly novel types of
products might get less interest."
Tuesday 9:45 a.m. ET Jan. 9
Blueprint Medicines is pulling back from a core focus area, cutting two non-small lung
cancer programs after getting a peak of early data. It was just a year ago that CEO Kate
Haviland touted the EGFR tyrosine kinase inhibitors as the company's path to first-line
treatment. The biotech says it will explore possible out-licensing deals to find a home
for BLU-945 and BLU-451.
Tuesday 3 a.m. ET Jan. 9
GSK is getting in on the M&A action as it snaps up respiratory biotech Aiolos Bio for $1
billion upfront and $400 million in biobucks. The deal sees GSK nab Aiolos’ leading asset
AIO-001, a phase 2-ready mAb, with plans for the drug to also treat chronic rhinosinusitis
with nasal polyps.
JPM24, Day 3: Gene editors talk business development; Roivant CEO has
money to spend
We're off for another day of the J.P. Morgan Healthcare Conference 2024. The flood of
licensing deals and M&A may have slowed, but there's still plenty of action to come as
companies make pitches to their peers, investors and industry watchers.
We start off the day with a recap of the gene editors.
Our Day 1 conference tracker is available, and all the Day 2 action is wrapped. Fierce
Pharma’s take on the action so far can be found.
Check out all the updates for day 3 below.
Wednesday 4:38 p.m. ET Jan. 10
Sanofi CEO Paul Hudson did not explicitly rule out pursuing future business development
centered on Pompe disease, despite the U.S. Federal Trade Commission’s recent challenge
of the pharma company’s deal with Maze Therapeutics.
The north star for Sanofi remains exciting science, Hudson said at a Wednesday briefing at
the J.P. Morgan Healthcare Conference.
“We will do what we always do, which is every time we see something that we think is good
science, that will benefit patients, that we can do better than other people, we will try,” he
said.
Wednesday 4:26 p.m. ET Jan. 10
Roivant's J.P. Morgan has a different vibe this year. After selling
off Telavant to Roche for $7.1 billion last year, the company is flush with cash and not
looking to raise funds, for once.
"One of the things that we're often doing at J.P. Morgan is meeting with investors around
raising money. We don't need to raise any money so we're still meeting with investors, but
that has a different flavor to it," CEO Matt Gline said in an interview on the sidelines of the
conference.
So far, the CEO has been peppered with questions about how he plans to spend that
capital, which sits at $7 billion.
"And that's really what this conference is about for us, is meeting with prospective partners,
Big Pharma, biotech companies, and trying to find our next opportunities now from a position
of some pretty significant strength," Gline said.
Wednesday 10:58 a.m. ET Jan. 10
Intellia Therapeutics Chief Medical Officer David Lebwohl, M.D., says the biotech's work
to assure the FDA that its gene edits do not pass down to a patients' children, aka germline
editing, is going well.
"We feel that the off-target work we have done is satisfying them," Lebwohl said during a
Tuesday presentation.
The FDA requested that Intellia conduct a new preclinical test on the impact that NTLA-
2002, a CRISPR-based treatment in hereditary angioedema, may have on a developing
fetus. Intellia has already cleared this hurdle for its NTLA-2001 program for transthyretin
(ATTR) amyloidosis, Lebwohl said.
So Intellia has launched a preclinical reproductive toxicity study, which Lebwohl said is not
expected to have any meaningful surprises, based on earlier preclinical evidence.
The company has benefited from a regenerative medicine advanced therapy designation in
this regard, which has allowed more free communication with the FDA as the off-target
possibilities are studied, according to Lebwohl.
Wednesday 10:36 a.m. ET Jan. 10
CRISPR Therapeutics, hot off the heels of the historic approval of sickle cell gene therapy
Casgevy, doesn't need cash. But CEO Sam Kulkarni, Ph.D., says there's still room for
business development in the gene editing biotech's future, and he's having a lot of meetings
at J.P. Morgan that might one day blossom into those partnerships.
"This is something that we've always been good at, is doing very good BD deals. Our
partnership with Vertex has been an excellent one. We had other partnerships as well. The
question now, is we have the money so we don't need the money for our programs. But I
think it does make sense to look at potential partners because if all seven of our trials
succeed, we may not have the bandwidth and capacity to prosecute all of them all the way to
the finish, in which case we would start looking at potential partners," Kulkarni said during a
Tuesday presentation.
And that could include Big Pharmas, who had tended to stay away from cell therapy but are
now coming around to the table.
"Interestingly, at this meeting here at J.P. Morgan, we had to rejigger our schedule because
we ended up having a lot more BD meetings than we anticipated outside of investor
meetings," Kulkarni said. "Because one of the things that people have been complaining
about in cell and gene therapies is pharma is not jumping in, but in fact, I do think that
pharma's interest in cell and gene therapies is increasing rapidly. Almost every pharma
company now wants to have a cell and gene therapy strategy."
Another huge opportunity for pharma is the rapid expansion of CAR-T into auto-immune
disease.
"So we're continuing to have BD discussions. Nothing impending but I think we'll carefully
think about what to partner and what not to partner based on how the data develop over the
next three to four months," the CEO concluded.
Wednesday 9:15 a.m. ET Jan. 10
In response to a question about the long-standing CRISPR patent battle, Editas
Medicine's Chief Financial Officer Erick Lucera said, "These are foundational patents, we
believe everyone that's working in this area is going to have to have a conversation with us."
Just last month, Vertex Pharmaceuticals entered a deal worth up to $100 million—plus
potential licensing fees—for rights to Editas’ Cas9 gene-editing tech, an agreement that
encompasses freshly approved Casgevy.
In terms of different potential deals, the CFO said Editas "can be creative and structure
something so we can have a win-win scenario." The scenario Lucera detailed yesterday
during the company's presentation allows potential partners to use the geneediting
technology but also gives Editas either access to technology that other companies
have via licensing deals or provides non-dilutive capital to continue to fund Editas' product
development.
JP Morgan Healthcare Conference, Day 3: Grail, Myriad Genetics, Bio-Techne,
Quanterix
Jan 11, 2024
SAN FRANCISCO – It was another busy day on Wednesday at the 42nd annual JP Morgan
Healthcare Conference with one day left.
Below are brief reports on individual presentations from the conference. A report from Day 1
of the conference can be found, and news from Day 2 can be found.
Grail
Grail CEO Bob Ragusa said the company generated full-year 2023 revenues of $93 million,
up 68 percent year over year and in line with revenue guidance provided by Illumina, which
plans to divest the firm by the end of the second quarter of this year. Grail notched $30
million in revenue in Q4 2023.
Ragusa said that to date, the company has performed 150,000 of its Galleri multi-cancer
early detection (MCED) tests in the commercial setting and counts more than 9,000 ordering
providers. He added that Grail has performed roughly 350,000 Galleri tests across both the
commercial and research settings.
Ragusa highlighted several near-term growth drivers for the company, including plans by the
UK's National Health Service this year to review early data from the NHS Galleri trial. If the
results are favorable, the NHS "may initiate a pilot of up to 1 million tests over a two-year
period starting in 2024," he said, noting that this would mark "the first national [health]
system adoption," which could help drive adoption in other single-payor systems.
He said that the NHS Galleri study is on track to complete the final round of subject visits in
Q3 2024 and anticipates having final results in 2026.
Ragusa said that Grail has enrolled 25,000 participants in its Pathfinder 2 study, which he
noted will be the primary study supporting its US Food and Drug Administration premarket
approval submission for Galleri.
He also highlighted the recently announced REACH/Galleri-Medicare study, in which the
company plans to compare 50,000 Medicare beneficiaries receiving usual care plus an
annual Galleri test to a similarly sized synthetic cohort.
Ragusa also pointed to Grail's activities beyond MCED, noting that it sees opportunities for
its methylation-based technology in areas including minimal residual disease detection,
patient stratification, and monitoring of patient response to treatment.
He did not comment on Illumina's planned divestment of the company except to note that
Illumina has publicly announced the divestment and to say that Grail has operated as a
standalone company since Illumina announced its acquisition of the firm three years ago.
Myriad Genetics
Myriad Genetics has completed its turnaround after years of struggle, but "the work is never
done," CEO Paul Diaz said at the JP Morgan Healthcare Conference. After describing the
growth of each of its product lines in 2023 and laying out its product roadmap for the
following year, Diaz said that he is "quite confident that the market's going to see … the
opportunity here that we have to grow and expand multiple and create shareholder and
patient value."
Diaz also provided preliminary financial results during his presentation. Preliminary quarterly
revenue is expected to be between $196 million and $197 million, slightly above analysts'
consensus estimate of $194.4 million and putting Myriad at the high end of its fiscal year
2023 guidance of between $747 million and $753 million. Adjusted earnings per share is
expected to be between $.02 and $.03 for Q4, beating analysts' consensus estimate of $.01.
Diaz said he sees a large market opportunity across all of its products and expects to gain
more market share as smaller players struggle with the infrastructure investments needed in
the space, such as integrating with electronic medical records and dealing with
reimbursement and regulatory challenges.
In pharmacogenomics, although payor coverage for its GeneSight test has "been some slow
going in some regards," it has been an "underappreciated growth engine for the company,
and we see a lot of opportunity to improve payor coverage and improve [average selling
price]," Diaz said.
Its oncology franchise is poised for additional growth as it prepares for the Precise Liquid
launch in Q3 2024, a liquid-based version of its Precise tumor profiling test.
A commercial launch of the firm's minimal residual disease monitoring test, Precise MRD, is
expected in the second half of 2025. The test is currently undergoing studies with
researchers at MD Anderson Cancer Center and Memorial Sloan Kettering Cancer Center in
renal cancer and breast cancer, respectively. The whole-genome sequencing-based assay
is built on Myriad's proprietary technology, and Diaz said the firm is "quite confident in our
freedom to operate" regarding intellectual property. The MRD space has seen a spate of
litigation in recent years, including a recent preliminary injunction against NeoGenomics'
MRD test and permanent injunctions against Invitae and ArcherDx in litigation brought
by Natera.
"The IP related to this and the chemistry and the proprietary workflows on this wholegenome
assay are differentiated from some of the other assays that are in controversy in the
marketplace," he said.
The company is also working on expanding its MyChoice CDx test for ovarian cancer to
other indications, such as breast and prostate cancer, Diaz said.
In its women's health business, Diaz said the Prequel prenatal screening test and the
Foresight carrier screening test "see a lot of runways." The firm expects strong growth this
quarter in the prenatal business, he added. The company plans to launch in Q1 2024 the
Foresight Universal Plus assay, an expanded carrier screening panel of 274 genes.
The FirstGene multiple prenatal screening test, meantime, is expected to have a soft launch
in Q3 2024, with a full commercial launch the following quarter. The test is an integrated
assay for noninvasive prenatal screening, carrier screening, fetal recessive status, and fetomaternal
blood compatibility.
Bio-Techne
At the 42nd annual JP Morgan Healthcare Conference, Kim Kelderman, Bio-Techne's chief
operating officer and incoming CEO, discussed what to expect when he takes control of the
company on Feb. 1. Although he said there is no need to change Bio-Techne's strategy in
the short term, because of the downturn in the macroeconomy, the firm would be looking
internally at its footprint and efficiencies.
Bio-Techne is "making sure that we get ready to come out stronger than anybody else when
the headwinds subside," he said.
According to CFO Jim Hippel, the firm's fiscal second quarter was negatively impacted by
issues including softness in biotechnology funding, which Bio-Techne had forecast in its
fiscal Q1 earnings call. The firm has seen "more conservatism of our pharma customers,
particularly in North America," he said, while the second quarter played out as the company
expected.
Hippel noted that Bio-Techne has heard from customers that demand for its products,
particularly for larger purchases of reagents and instrumentation, is "as high as it's ever
been." While new instrument placements have declined due to budgetary constraints, use of
existing instruments has reached record highs, he said, which "speaks to the insatiable
demand of our products and what we hope will be a pent-up demand once budget starts to
get released."
In China, Hippel previously predicted that market would get worse before it improved. The
first part of his prediction has happened, but he is hopeful the market will stabilize in January
and funding may begin flowing again after the Chinese New Year.
Kelderman also laid out some of the firm's pipeline plans in his presentation. The
Minneapolis-based company plans to launch a spatial biology assay that will include 12 RNA
probes and 20 antibodies in one workflow. "That combination … is a big step towards our
mission in spatial, which is to have a multiomic view [on the] same slide [with a] full
assessment of your tissue," he said.
Bio-Techne has multiple plans for the liquid biopsy and molecular diagnostics market, as
well. Kelderman cited the development of a colorectal cancer test, for which the firm is
currently generating data, and exosome-based solid tumor mutation assays. The company
would "like to partner" on the colorectal cancer assay with another firm, he noted.
Kelderman discussed the competitive landscape in the markets Bio-Techne plays in in light
of two major acquisitions last year, saying that Danaher's $5.7 billion acquisition of
Abcam wouldn't change Bio-Techne's position in protein sciences. "I think we're ready to
compete, and I'm very confident that we will continue to be successful," he said.
Also, he said that although Bio-Techne would've liked to acquire Olink, which is in the
process of being bought for $3.1 billion by Thermo Fisher Scientific, that deal could still work
out for Bio-Techne, which provides "a lot of content" for Olink's platform.
"If it goes to a good company that can place many instruments, it will be for our benefit
because we'll see more pull-through," he said
M&A will continue to be a priority for Bio-Techne, Kelderman added
Quanterix
Quanterix CEO Masoud Toloue said that with the company's corporate reorganization and
assay redevelopment program complete, it is now in a position to accelerate its assay
development and expects to produce on the order of tens of new assays in 2024 and
beyond.
Citing the impact of the reorganization plan, he noted that Quanterix has reduced its cash
burn by threefold since 2022 and delivered double-digit revenue growth for full-year 2023.
The firm also expects to report fourth quarter revenue of $30 million, up 16 percent year over
year and above analysts' consensus estimate of $28.6 million.
Neurology remains central to the company's plans, with Toloue saying Quanterix believes
the space is poised to grow over the next decade in much the way areas like oncology and
immunology have in recent years.
He pointed to the large number of central nervous system (CNS) drug programs ongoing,
noting that there are currently more than 120 CNS programs and that Quanterix is involved
in a substantial percentage of these programs.
Alzheimer's disease is at the center of Quanterix's work within neurology. In October, the
company launched its LucentAD p-Tau 217 lab-developed test for assessing brain amyloid
pathology in patients suspected of having Alzheimer's disease.
Toloue said Quanterix is currently running a prospective clinical trial for the assay with the
goal of submitting it to the US Food and Drug Administration for regulatory approval. He said
readout from that trial is expected in 2024.
Quanterix is also working to develop the LucentAD p-Tau 217 assay for use with dried blood
spot samples, which Toloue said could help improve accessibility to the test globally.
Toloue said that in addition to assay development, the company is also investing in the
development of a next-generation platform with a focus on combining single-molecule
sensitivity with higher levels of multiplexing and throughput. Key to that process will be
reducing the size of the instrument in order to meet what Toloue said is the company's goal
of having Simoa, the firm's digital biomarker platform, not just in specialty labs but in all
laboratories.
He said the company expects to provide more details on this instrument development work
in 2024.
Editor’s Corner: Driverless cars and doorless planes at JPM24
The biotech industry has a love-hate relationship with the J.P. Morgan Healthcare
Conference in San Francisco.
By Annalee ArmstrongJan 12, 2024 11:02am
The biotech industry has a love-hate relationship with the J.P. Morgan Healthcare
Conference in San Francisco. Every year, thousands gather on the Golden Gate City and
battle rain, hail, floods, what have you, just to make billion-dollar deals.
This year, flight disruptions were the key challenge for conference goers, spurred by Alaska
Airlines’ accident days before the industry was set to descend on San Francisco. One of the
company’s Boeing 737 Max 9 aircraft flying from Portland, Ore., to Ontario, Calif.
experienced a “rapid depressurization event.”
Or to be clear, a plugged exit door blew out mid-flight, sucking out bits of seat, people’s
clothing and belongings. Cell phones were found scattered about the countryside. The
oxygen masks dropped. The cockpit door sprung open. It was, by all accounts, every flyer’s
worst nightmare.
No one was seriously injured in the incident, luckily, although there were reports of
concussions, ear bleeding (sounds serious to me) and headaches. But this is the event that
preceded all J.P. Morgan travel this past weekend. It was top of mind, ripe for dark jokes
about the conference everyone loves to hate.
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For Roviant CEO Matt Gline, J.P. Morgan is squarely in the hate-hate category. He told
Fierce Biotech on the sidelines that, while he didn’t fly Alaska on the way to J.P. Morgan, he
did sit in the exit row. He was of course consumed by thoughts of the blowout. But for him,
what would have been worse would be attending the conference first, then experiencing a
“rapid depressurization event.”
He couldn’t imagine anything worse than that.
Now, this accident happened days before I was set to fly Alaska to San Francisco. I picked
the flight because it was non-stop, Washington Reagan to SFO. Easy peasy. Our reporter
Max Bayer, too, opted for the ease of the one-and-done flight.
I was never on a MAX plane. But Max, fittingly, was. He managed to take off no problem out
of New York, an hour before me. With work to do on the plane, we both bought a WiFi pass
and were able to chat. As Max neared landing in San Francisco, he told me that he was
happy the door to his plane was still attached.
The blood drained out of my face. I still had three hours to go.
Anyway, I made it safe via my trusty Boeing 737-800. So did Max on his MAX. Many
members of the Fierce crew began receiving notifications Wednesday that flights,
particularly on United Airlines, were being canceled due to the grounding of the MAX 8/9
fleet. One of our team members got rerouted home via a fun night at the Las Vegas Airport.
Hesitant flier, Publisher Rebecca Willumson, offered to rent a bus with me and drive the
2,813 miles to Washington, DC. As much as I love a road trip, I opted to take the four-and-ahalf-
hour flight home.
We began asking each of our interviewees if they had flight disruptions on Wednesday
afternoon, spurring panic as people realized the issue was still ongoing. We hope everyone
made it out safely.
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ready to deal again
Another key safety concern of J.P. Morgan was the driverless cars taking the San Francisco
streets by storm. Everywhere you went, San Franciscans were grumbling about the bulky
CUVs with spinning sensors pasted onto all angles of the car.
This reporter encountered one on her way back from an interview to Fierce HQ. Leaving a
hotel, I looked both ways and boldly stepped out into the street after one had passed by,
only to hear a friendly bellhop behind me shout “Whoa whoa whoa” and chase after me.
“Be careful of those driverless cars, Ma’am! They don’t like pedestrians!” he cried.
What do you mean, they don’t like pedestrians?! I thought. Turns out a driverless car from a
previous company had tragically killed a pedestrian on a right turn. The new company,
Waymo, had returned the modality to the streets; however, San Franciscans don’t seem to
be all that welcoming of their new rides.
Maybe the friendly bellhop was looking for a tip—I had none to offer as I had been fleeced of
my only $40 in cash by a rather crabby cabby a day earlier who grumbled when I pulled out
my credit card—but he escorted me across the road and opened the door of my Uber for
me, ensuring I got in safely.
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choice
We then set off following the driverless car, which had found some riders, and came to a
right turn. My driver began grumbling. “Oh, they don’t like right turns,” he said. Due to the
pedestrian fatality, Waymo had put in new safety checks for right turns, making the new cars
much more hesitant and cautious. We waited through two turns of the light—my Uber driver
fuming—until it finally lurched forward.
The car sped off ahead, leaving us at a red light. We watched it drive up to a delivery van,
stop suddenly and seem to struggle to navigate the lane change needed. Three cars
hurriedly got out of its way as the Waymo car navigated the lane change and drove up
towards Nob Hill and out of sight.
JPM24: Roche's pharma BD chief explains renewed ADC interest
but is spoilt for choice
By Angus LiuJan 12, 2024
As one of the first Big Pharmas to embrace antibody-drug conjugates (ADCs), Roche plans
to make further bets on the popular cancer modality, according to the company's global head
of pharma partnering, James Sabry, Ph.D. But biotechs looking for an alliance must first
meet an invisible bar.
“We plan to do more deals, and we plan to do internal work in ADCs,” Sabry said in an
interview with Fierce Biotech on the sidelines of the 2024 annual J.P. Morgan Healthcare
Conference.
Sabry’s comments—and a recent licensing deal—marked a change of public tone at Roche
toward ADCs. Back in mid-2022, chairman and then-CEO Severin Schwan told
reporters that Roche had “rather limited interest” in ADCs despite other companies’ growing
investments in the field.
Still, getting on Roche’s good side won’t be an easy task for prospective ADC partners—
Sabry’s team reviews around 6,000 opportunities across all disease areas each year.
“For Roche, what the biotech companies are competing with is our internal work,” he said.
But that internal work isn’t necessarily visible to outsiders. When asked what internal
capabilities Roche has built around ADCs, Sabry said, “You’ll see when the products come.”
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Roche currently has two commercial ADCs, both built with other companies’ technologies.
HER2-targeted Kadcyla was made with ImmunoGen’s platform, and large B-cell lymphoma
drug Polivy has Seagen to thank. But since forming those partnerships over a decade ago,
the Swiss company had stayed quiet on the ADC deal-making scene, while the likes of
Merck & Co., AstraZeneca, Eli Lilly and AbbVie recently bought into the field.
“We’re not a fashion company,” Sabry said. “We don’t just jump on a bandwagon because
everyone else is doing it. We do things because we really believe that an innovative product
will bring unusual value to patients.”
Roche broke the silence at the beginning of 2024 by in-licensing a c-MET ADC from China’s
MediLink Therapeutics. The deal came more than two years since AstraZeneca and Daiichi
Sankyo’s Enhertu trounced Kadcyla in a head-to-head breast cancer trial.
“Enhertu broke every rule that we thought about ADCs,” Sabry said. Roche once thought
certain rules were true around the target’s overexpression level on cancer tissue, the drugto-
antibody ratio, and the stability of the linker. In the wake of the head-to-head fail against
AstraZeneca's drug, Roche went back to the drawing board to renew its understanding of
the modality.
“There’s a lot of internal research we were doing in order to support deals like the MediLink
deal,” Sabry said.
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It doesn’t mean Roche has fully understood the new rules of ADCs, Sabry said. “When you
don’t know the rules, then you look for clinical data.”
“The nice thing about ADCs is that you know clinically usually within phase 1 whether the
drug is safe, and whether it has some effect,” he said. “So that’s what we’re looking for. It
can be very early-stage clinical data.”
Cautious approach to platform deals
As to whether Roche would buy out an ADC specialist similar to Pfizer’s acquisition of
Seagen, AbbVie’s proposed purchase of ImmunoGen and Johnson & Johnson’s Ambrx
takeover, Sabry said only when the external technology is “so different that we don’t have it
on the inside” will Roche make platform deals. And even with platforms, Roche would
typically form collaborations to generate multiple products.
Case in point: Last week Roche put down $66 million upfront to access Moma Therapeutics’
platform to identify and develop “a certain number of” novel drug targets involved in cancer
cell growth and survival. In a separate deal, Roche tapped Remix Therapeutics’ drug
discovery engine to develop small molecules that target RNA processing.
In the few cases where Roche has bought a company outright in the past, such as
Genentech and Spark Therapeutics, the business would often be kept as a separate unit,
Sabry observed.
But Roche’s $4.3 billion takeover of Spark in 2019 hasn’t produced many results, leading to
740 million Swiss francs ($870 million) worth of impairment charges for 2022.
“When we did the Spark deal, it was based on an enthusiasm around gene therapy,” Sabry
said. “As you can imagine, that enthusiasm is still there, we still believe gene therapy will
become a major type of therapy in the future. What we mean is that it’s going to take time for
that to evolve.”
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In October, Roche penned a deal potentially worth $216 million with SpliceBio for a gene
therapy against an inherited retinal disease, which is Spark’s sweet spot thanks to FDAapproved
Luxturna.
The gene therapy world just celebrated a major breakthrough with the approval of Vertex
and CRISPR Therapeutics’ Casgevy, the first gene-editing therapy based on CRISPR
technology. Still, Sabry argued that the main limitation for gene therapy is the lack of ability
to target specific cells effectively.
“We would consider CRISPR, like, eventually,” Sabry said.
How the MediLink, Moma and Remix announcements appeared in close succession
raises the question of whether Roche has shifted into deal-making mode. But Sabry assured
Fierce that “that was just the way they lined up.” The number of deals Roche negotiated last
year, including the flurry of pacts announced in the last few days, was similar to the number
in previous years, he said.
Roche did appear to have spent more on later-stage candidates in 2023,
including purchasing Roivant and Pfizer’s Televant for over $7 billion to get its hands on a
phase 3-ready anti-TL1A antibody in bowel disease. Roche also splashed $310 million
upfront to in-license Alnylam’s RNA interference hypertension candidate zilebesiran, which
in September reported positive blood pressure data from a phase 2 trial.
A few trial failures have left some room in Roche’s late-stage pipeline and created an
urgency to fill it, Sabry acknowledged. Besides, the biotech winter also opened projects up
for the Swiss conglomerate to grab.
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drugs
But Roche didn’t start the year by setting a quota to balance drug discovery pacts and latestage
buys, Sabry said. The decisions are based on science.
“We believe that the most interesting companies are the ones that dramatically change the
way we think about what a medicine is,” Sabry said. He noted how Alnylam’s zilebesiran is
the first to go further upstream of a signaling cascade than other existing hypertension meds.
Roche’s pharma division currently doesn’t have any commercial presence in cardiovascular
or metabolic diseases—although that looks set to change. When asked if the Alnylam deal
indicates a new push into that area, Sabry said: “Yes, absolutely, we’re gonna be evolving
into new medicines in metabolic disease [and] in cardiovascular disease, for sure.”
All eyes on China
Another thing that Roche seems to be doing differently is that it’s recently started to partner
with biotechs from China. In addition to MediLink, Roche’s Genentech paid $60 million
upfront in August 2022 and committed $590 million in biobucks to in-license an androgen
receptor degrader from Jemincare. By May 2023, the Swiss drugmaker was laying out $70
million in upfront and near-term payments to obtain a brain-penetrating HER2 molecule from
Zion Pharma.
Sabry called those candidates “world-class drugs” that can compete with others from
Western companies. Roche has also set up a business development team based in
Shanghai led by Harm-Jan Borgeld, head of Asia partnering.
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partners
China has become a hotbed for partnering, especially in ADCs. MediLink has also outlicensed
its ADC candidate to BioNTech, for example, while Hansoh holds two ADC deals
with GSK. Merck also has a multi-program ADC collab with Kelun Biotech, and in December
Bristol Myers Squibb shelled out $800 million upfront in a deal worth up to $8.4 billion for an
EGFRxHER3 bispecific ADC from SystImmune, a subsidiary of China’s Biokin
Pharmaceutical.
“I believe China will become as powerful as America and Europe in terms of invention,”
Sabry said.
The biotech winter was also one of reasons behind the torrent of deals in China as local
firms partner up to survive. “China has not matured fully into the late stages of development
yet, but it will,” Sabry predicted.
“The issue in China is that they don’t have mature management teams for these biotech
companies, because they are still a young industry,” he said. Once Chinese biotechs and
their management develop, they will find other resources to take them forward because “the
capital flows where the innovation is,” Sabry observed.
As for the potential risk of heightened geopolitical tensions, Sabry said: “When we go to
China, we hope that the Chinese look at us and [think of us as] a Swiss company, not an
American company.”