endpts.com Open in urlscan Pro
13.224.189.70  Public Scan

Form analysis
1 forms found in the DOM

https://endpts.com/

<form action="https://endpts.com/">
  <input type="text" name="s" placeholder="Search">
  <button class="epn_ux_button epn_white epn_search_submit_button"></button>
</form>

Text Content

 * Channels
   * All News
   * Premium Content
   * In Focus
   * Special
   * AI
   * Bioregnum
   * Biotech Voices
   * Cell/Gene Tx
   * China
   * Coronavirus
   * Deals
   * Diagnostics
   * Discovery
   * FDA+
   * Financing
   * Law
   * Letters to the Editor
   * Manufacturing
   * Marketing
   * Opinion
   * Outsourcing
   * Peer Review
   * People
   * Pharma
   * R&D
   * Startups
   * Weekly
 * Webinars
 * Biopharma Jobs
 * More
   * Work at Endpoints
   * Letter to Editors
   * IPO Tracker
   * Webinars
   * Events
   * Sponsored Posts
   * Advertise
   * Privacy Policy
   * Endpoints Merch
   * About Us
   * Help


 * SIGN UP
 * LOG IN



Emil Kakkis, Ultragenyx CEO
March 2, 2023 04:08 PM EST
Pharma


Q&A WITH UL­TRAGENYX CEO: TIME TO IN­CREASE THE USE OF AC­CEL­ER­AT­ED
AP­PROVALS FOR RARE DIS­EASE DRUGS


ZACHARY BRENNAN

SENIOR EDITOR

Emil Kakkis, CEO of the rare dis­ease drug­mak­er Ul­tragenyx
Phar­ma­ceu­ti­cals, sat down with End­points News to­day in Wash­ing­ton, DC to
dis­cuss in­dus­try’s more re­cent strug­gles around rare and ul­tra-rare
dis­ease drug de­vel­op­ment, the need for an Or­phan Drug Act 2.0, and why the
FDA needs to qual­i­fy more bio­mark­ers and open up the ac­cel­er­at­ed
ap­proval path­way.

At least 30 rare dis­ease drug pro­grams have been can­celed over the last year,
Kakkis said, not­ing that the ap­proval process has be­come more dif­fi­cult in
some cas­es, with FDA rais­ing the bar, or the mon­ey is too much.

Kakkis was on Capi­tol Hill to­day speak­ing to mem­bers of Con­gress and the
Rare Dis­ease Leg­isla­tive Cau­cus about the need for more in­cen­tives like
in­creas­ing the use of the ac­cel­er­at­ed ap­proval path­way to speed the
de­vel­op­ment of rare dis­ease treat­ments.

advertisement

advertisement

“There’s still this on­go­ing anger and re­ac­tion with ac­cel­er­at­ed
ap­provals. It’s very wrong-hand­ed and to­tal­ly off-base,” he said.

Be­low is a light­ly edit­ed and con­densed in­ter­view with Kakkis.

End­points: In what way are you work­ing with FDA and CBER Di­rec­tor Pe­ter
Marks on this new reg­u­la­to­ry frame­work around n-of-1 or ul­tra-rare
dis­eases, and how is that pro­gress­ing, and what do you see com­ing?

Kakkis: Pe­ter does get the sto­ry, and he can see the com­mer­cial out­come is
hard­er to sup­port. You can’t spend $200 mil­lion and have $50 mil­lion
re­turn. It’s not go­ing to work. Ul­ti­mate­ly the FDA has to reg­u­late
dif­fer­ent­ly. You can’t re­quire large stud­ies and a lot of time. I’ve been
talk­ing about the need for bet­ter ac­cess to the ac­cel­er­at­ed ap­proval
path­way.

The FDA needs to im­prove their un­der­stand­ing of the un­der­ly­ing sci­ence
so they can qual­i­fy a bio­mark­er with­out the types of da­ta they usu­al­ly
ex­pect and which is usu­al­ly nev­er at­tain­able.

I’m talk­ing about the need for an Or­phan Drug Act 2.0 that looks at
ul­tra-rare dis­eases, for less than 2,000 in the US, that should have more
ready ac­cess to the ac­cel­er­at­ed ap­proval path­way based on sol­id sci­ence
… when the treat­ment is di­rect­ed at the un­der­ly­ing cause. If you’re do­ing
en­zyme re­place­ment, gene ther­a­py, siR­NA — if you’re do­ing that and
mea­sur­ing cause, that is a more pre­cise mea­sure of the dis­ease than
clin­i­cal out­comes.

End­points: If the ac­cel­er­at­ed ap­proval path­way does open up, is there
go­ing to be a ma­jor chal­lenge for in­dus­try to con­vert a lot of these
ac­cel­er­at­ed ap­provals to full ap­provals, as FDA’s Wil­son Bryan cau­tioned
re­cent­ly?

Kakkis: I don’t think there is. I don’t think you can use ran­dom­ized
con­trolled tri­als for some dis­eases and in­di­ca­tions. When we formed
Ul­tragenyx, one of the things I want­ed to change was our post­mar­ket
stud­ies. We came up with a Dis­ease Mon­i­tor­ing Pro­gram to mon­i­tor those
who are get­ting the drug com­mer­cial­ly, and the size of it is usu­al­ly four
to five times what you file with; that’s the com­mit­ment. We’ve done it now for
four pro­grams, so it’s not a the­o­ry.

End­points: On or­phan drug ex­clu­siv­i­ty — the FDA lost that big Cat­a­lyst
court case and put out their own FR no­tice on how they were go­ing to stray
from the court’s de­ci­sion. Are you wor­ried about ex­clu­siv­i­ty?

Kakkis: I thought that was bold, ba­si­cal­ly tell the courts you don’t rule me.
And the courts will say, “I’ll show you.” I think the FDA is un­for­tu­nate­ly
play­ing a po­lit­i­cal game. The FDA has this new thing where they’re
age-re­strict­ing la­bels — they used to not do that for rare dis­eases. But no
one thought that left an open­ing for a com­pound. Be­cause it means we have to
fight for a la­bel from birth for every­thing … The lim­it­ing of the age is a
mis­take.

This is a change. For an Al­du­razyme (la­ronidase) tri­al for MPS I — we
stud­ied age 5 and up, and we had a post­mar­ket com­mit­ment for un­der 5
[year-olds], and the la­bel at launch was birth and up be­cause it was go­ing to
work. Every­one knew that.

So in the new world, if some­one comes up with an en­zyme re­place­ment
ther­a­py for a 4-year-old and then breaks the or­phan [ex­clu­siv­i­ty] while
we’re do­ing a post­mar­ket com­mit­ment? Does that make sense?

End­points: FDA is mak­ing the case that they’re try­ing to in­cen­tivize
ad­di­tion­al de­vel­op­ment in a lot of these rare dis­eases.

Kakkis: The whole in­cen­tive is based on false in­for­ma­tion. The false
in­for­ma­tion is that the 1-year-old and 2-year-old and the 8-year old are
ac­tu­al­ly dif­fer­ent in re­spond­ing. The FDA has no proof that that’s true …
what I’m say­ing is his­tor­i­cal­ly the FDA has la­beled for a dis­ease not
cre­at­ed these lit­tle seg­ments of pe­di­atric ages.

They think they’re be­ing rig­or­ous, but there’s ac­tu­al­ly no valid ba­sis
for mak­ing this dis­tinc­tion and it push­es ran­dom­ized con­trolled tri­als
in younger chil­dren which peo­ple don’t want to do and I don’t think are
nec­es­sary. The FDA and EMA are di­vid­ing the pe­di­atric pop­u­la­tions to
cre­ate a leak hole in the Or­phan Drug Act, and all that’s go­ing to mean is
that we’re go­ing to fight hard from birth … I don’t think FDA is think­ing this
through and I don’t think age is the right cri­te­ria.

End­points: There’s a lot of out­cry about how high these prices are for rare
dis­eases — what role do you see for ICER and oth­er po­ten­tial
cost-con­trol­ling mea­sures? Is rare dis­ease drug de­vel­op­ment fea­si­ble
with­out these high prices?

Kakkis: Pric­ing has to be what it has to be be­cause of the cost spent to make
it rea­son­able. And for a lot of these ul­tra-rare drugs, if we don’t have the
prices, we won’t be able to spend that much mon­ey at-risk.

The prob­lem with high prices is how it’s harm­ing ac­cess. Part of the way
com­pa­nies in this space have en­sured that cost is not an is­sue for ac­cess,
via co­pay sup­port and we pro­vide free drug for those who can’t get it one way
or the oth­er. What’s the right price? You can price low and be very
al­tru­is­tic, but if you don’t earn enough mon­ey and be­come prof­itable, you
go out of busi­ness and sell to some­one who is.

Our pric­ing phi­los­o­phy is dif­fer­ent from oth­er com­pa­nies and I want­ed
to change the equa­tion. Every­one has been go­ing high­er and high­er but I
think that’s had an im­pact on ac­ces­si­bil­i­ty. You can’t be in this space
with­out the re­spon­si­bil­i­ty of tak­ing care of peo­ple cor­rect­ly. Our
pric­ing strat­e­gy has been to mod­er­ate. If you’re hit­ting the $3 mil­lion
mark, you’re not see­ing glob­al ac­cess.

End­points: Thoughts on the cre­ation of a con­sor­tia of reg­u­la­tors who are
mak­ing si­mul­ta­ne­ous ap­proval de­ci­sions to ex­pand some of these rare
dis­ease drugs’ pop­u­la­tions?

Kakkis: I think Pe­ter Marks is try­ing hard to make things bet­ter. But
get­ting reg­u­la­tors around the world to agree, I wor­ry about the worst
com­mon de­nom­i­na­tor. And I don’t want syn­chronic­i­ty be­tween Eu­rope and
the US, be­cause some­times I need to go to Eu­rope to get the an­swer we need
that the US would not have. And if you put them to­geth­er, it’ll make things
hard­er, not bet­ter. Every reg­u­la­to­ry re­gion asks dif­fer­ent things from
our Phase III tri­als — the truth is no one wants to give up pow­er to any­one.

If he can get that, that would be help­ful. But he needs to get the
qual­i­fi­ca­tion of bio­mark­ers for rare dis­eases im­proved in CBER so that
we ac­tu­al­ly get drugs ap­proved in a rea­son­able time­frame and cost
struc­ture. That’s what will re­al­ly jump­start the uti­liza­tion of
pre­ci­sion med­i­cine for a whole bunch of dis­eases.




AUTHOR


ZACHARY BRENNAN

SENIOR EDITOR

zachary@endpointsnews.com @ZacharyBrennan
Zachary Brennan on LinkedIn
SPONSORED
February 27, 2023 06:00 AM EST


DIG­I­TAL THER­A­PEU­TICS: THE KEY TO MAX­I­MIZ­ING THE PO­TEN­TIAL OF
MED­I­C­I­NAL AS­SETS


FISHAWACK HEALTH

Key takeaways:

Digital therapeutics support increased access to safe and effective therapies,
providing an untapped opportunity for biopharmaceutical companies to maximize
the value of medicinal assets and drive differentiation
When developed in combination with a medicinal asset, digital therapeutics
enable significant market differentiation and significant benefits for
stakeholders—from optimizing patient outcomes to supporting more efficient
generation of compelling real-world data
Integrating digital therapeutics early in the asset development process will
allow for gathering of clinical data to drive a clearer value proposition that
maximizes the potential of both products
Early market entry is fundamental, and manufacturers must plan strategically to
consider how digital therapeutics (DTx) are best incorporated into the launch
and go-to-market strategy. Internal digital and data science expertise is
critical to developing a united value proposition that aligns the benefits of
the digital and medicinal product

Read More
DL Hughley headlines Janssen's multiple myeloma campaign to drive better
outcomes for the Black community.
March 3, 2023 12:48 PM EST
Pharma
Marketing


J&J’S JANSSEN CALLS ON CO­ME­DI­AN TO UN­DER­SCORE MUL­TI­PLE MYELO­MA
IN­EQUITIES IN THE BLACK COM­MU­NI­TY


BETH SNYDER BULIK

SENIOR EDITOR

Comedian and radio show host D.L. Hughley is known for his quick wit and bold
humor, but his new campaign for J&J’s Janssen has a more serious mission.
“That’s My Word” is tackling disparities in multiple myeloma diagnosis and
treatment in the Black community. The effort centers on pledges, or people
giving their word, to spread information and increase awareness.

While Hughley is not a patient, he’s connected to cancer through his father and
sister, and he’s also a vocal advocate for equitable health care.


KEEP READING ENDPOINTS WITH A FREE SUBSCRIPTION

Unlock this story instantly and join 161,800+ biopharma pros reading Endpoints
daily — and it's free.

SIGN UP LOG IN

March 2, 2023 07:00 AM EST
R&D
Pharma


NO­VO NORDISK TOUTS EX­PAN­SION IN BOSTON, CUTS ABOUT 100 JOBS IN TWO OTH­ER US
CITIES


KATHERINE LEWIN

NEWS REPORTER

Novo Nordisk is expanding its R&D and lab-based activities in Boston while
adding jobs — and cutting some elsewhere, too.

The company announced today it’s adding more than 200 new jobs, with about 150
of them focused on data science, biology or chemistry research and ribonucleic
acid interference (RNAi) R&D in Lexington, Cambridge and Watertown, MA.

But now that Boston will be the “principal” R&D site in the US, about 20
positions in Indianapolis and 80 positions in Seattle will be cut as lab-based
work in Seattle is moved to other locations globally and closer to the R&D
facility in Indianapolis.


KEEP READING ENDPOINTS WITH A FREE SUBSCRIPTION

Unlock this story instantly and join 161,800+ biopharma pros reading Endpoints
daily — and it's free.

SIGN UP LOG IN
ENDPOINTS CAREERS


HEMOLYTIC ANEMIA SALES SPECIALIST, RARE GENETIC DISEASES

AGIOS PHARMACEUTICALS

REMOTE

view job offer post your job now
March 2, 2023 03:03 PM ESTUpdated 04:48 PM
Pharma


US SPENT NEAR­LY $32B IN PUB­LIC FUNDS ON MR­NA VAC­CINE DE­VEL­OP­MENT, STUDY
FINDS


NICOLE DEFEUDIS

EDITOR

The US government contributed at least $31.9 billion in public funding to the
development of mRNA-based Covid-19 vaccines, according to a new BMJ study.

That includes at least $337 million in the three decades prior to the pandemic,
seven researchers from Brigham and Women’s Program On Regulation, Therapeutics,
And Law (PORTAL) wrote.

For co-author and primary care physician Hussain Lalani, the study began as a
way to understand the origins of mRNA vaccines and convey to patients that the
Covid shots were not created overnight.

Read More
March 3, 2023 11:26 AM EST
R&D


PRAX­IS STOCK TANKS AF­TER MID-STAGE MOVE­MENT DIS­OR­DER TRI­AL MISS


PAUL SCHLOESSER

ASSOCIATE EDITOR

Praxis Precision Medicines has missed its goal in a Phase IIb trial after
rolling out positive Phase IIa results last year.

The CNS-focused biotech announced Friday morning that the mid-stage trial did
not reach statistical significance in the study’s primary endpoint. The Phase
IIb trial, examining drug candidate PRAX-944 in patients with a condition called
essential tremor, showed the drug induced an average 3.01-point reduction from
baseline in a daily living activity score, compared to a 1.44-point reduction
from placebo after eight weeks.

Read More

March 3, 2023 11:24 AM EST
News Briefing


SUM­MIT CLOS­ES $500M RAISE, DE­SPITE LOW­ER MAR­KET CAP; REV­O­LU­TION RAIS­ES
$300M IN STOCK SALE


MAX GELMAN

SENIOR EDITOR


LEI LEI WU

NEWS REPORTER

Bob Duggan’s Summit Therapeutics has completed its $500 million raise — even
though its market cap is short of that figure.

Summit, which as of Thursday evening had a valuation of roughly $431 million,
sold 476.2 million shares of its stock at $1.05 apiece to bring in the $500
million. The raise comes a few months after Duggan and co-CEO Maky Zanganeh
loaned the company $520 million to finance a deal with China’s Akeso
Therapeutics.

Read More
March 3, 2023 09:15 AM EST
People
In Focus



‘HARD­ER TO FIND HOMES’: LAT­EST WAVE OF BIOTECH LAY­OFFS COULD TEST THE
IN­DUS­TRY


KYLE LAHUCIK

ASSOCIATE EDITOR

A quarters-long flurry of layoffs has caused thousands of workers in life
sciences to go hunting for their next position sooner than expected, and the
latest wave could make it more difficult to get new offers in hand in short
order.

At least three dozen biotechs let employees go so far this year, based on
Endpoints News reporting, with more having done so under the radar. More than
100 drug developers thinned their organization charts last year.


PREMIUM SUBSCRIPTION REQUIRED

Unlock this article along with other benefits by subscribing to one of our paid
plans.

SIGN UP LOG IN
ENDPOINTS CAREERS


HEMOLYTIC ANEMIA SALES SPECIALIST, RARE GENETIC DISEASES

AGIOS PHARMACEUTICALS

REMOTE

view job offer post your job now
Mark Zuckerberg and his wife, Priscilla Chan (Jeff Chiu/AP Images)
March 3, 2023 07:13 AM ESTUpdated 07:28 AM
Discovery
Pharma


CHAN ZUCKER­BERG INI­TIA­TIVE AN­NOUNCES CHICA­GO BIO­HUB TO STUDY TIS­SUE
IN­FLAM­MA­TION


KATHERINE LEWIN

NEWS REPORTER

A new biomedical research hub is coming to Chicago through an expansion of the
CZ Biohub Network, a part of the Chan Zuckerberg Initiative.

The hub will be a collaboration between the University of Chicago, Northwestern
University and the University of Illinois Urbana-Champaign to research and
develop new technology to study inflammation in tissues. It will be funded with
$250 million over the next decade from CZI, Forbes reported. Illinois Gov. JB
Pritzker also committed $25 million in state money to support the hub.


KEEP READING ENDPOINTS WITH A FREE SUBSCRIPTION

Unlock this story instantly and join 161,800+ biopharma pros reading Endpoints
daily — and it's free.

SIGN UP LOG IN

March 3, 2023 07:10 AM EST
R&D


ROCHE, EX­ELIX­IS' TECEN­TRIQ/CABOME­TYX COM­BO FLUNKS SEC­OND PHI­II


AMBER TONG

SENIOR EDITOR

Roche and Exelixis are reporting a second flop out of a trio of Phase III trials
designed to test a combination of their PD-L1 drug and tyrosine kinase
inhibitor.

Adding Tecentriq, Roche’s blockbuster PD-L1 antibody, to Exelixis’ Cabometyx did
not meet the primary endpoint of progression-free survival for a group of
patients with kidney cancer who have already received checkpoint therapy,
Exelixis wrote in a terse update on the CONTACT-03 study.

Read More


Q&A WITH UL­TRAGENYX CEO: TIME TO IN­CREASE THE USE OF AC­CEL­ER­AT­ED
AP­PROVALS FOR RARE DIS­EASE DRUGS


DIG­I­TAL THER­A­PEU­TICS: THE KEY TO MAX­I­MIZ­ING THE PO­TEN­TIAL OF
MED­I­C­I­NAL AS­SETS


J&J’S JANSSEN CALLS ON CO­ME­DI­AN TO UN­DER­SCORE MUL­TI­PLE MYELO­MA
IN­EQUITIES IN THE BLACK COM­MU­NI­TY


NO­VO NORDISK TOUTS EX­PAN­SION IN BOSTON, CUTS ABOUT 100 JOBS IN TWO OTH­ER US
CITIES


US SPENT NEAR­LY $32B IN PUB­LIC FUNDS ON MR­NA VAC­CINE DE­VEL­OP­MENT, STUDY
FINDS


PRAX­IS STOCK TANKS AF­TER MID-STAGE MOVE­MENT DIS­OR­DER TRI­AL MISS


SUM­MIT CLOS­ES $500M RAISE, DE­SPITE LOW­ER MAR­KET CAP; REV­O­LU­TION RAIS­ES
$300M IN STOCK SALE


‘HARD­ER TO FIND HOMES’: LAT­EST WAVE OF BIOTECH LAY­OFFS COULD TEST THE
IN­DUS­TRY


CHAN ZUCKER­BERG INI­TIA­TIVE AN­NOUNCES CHICA­GO BIO­HUB TO STUDY TIS­SUE
IN­FLAM­MA­TION


ROCHE, EX­ELIX­IS' TECEN­TRIQ/CABOME­TYX COM­BO FLUNKS SEC­OND PHI­II

Bioscience & Technology Business Center
The University of Kansas
Lawrence, Kansas




LATEST

 * All News
 * Premium Content
 * Don't Miss
 * Special
 * In Focus
   


CHANNELS

 * AI
 * Bioregnum
 * Biotech Voices
 * Cell/Gene Tx
 * China
 * Coronavirus
 * Deals
 * Diagnostics
 * Discovery
 * FDA+
 * Financing
 * Law
 * Letters to the Editor
 * Manufacturing
 * Marketing
 * Opinion
 * Outsourcing
 * Peer Review
 * People
 * Pharma
 * R&D
 * Startups
 * Weekly


MORE

 * Work at Endpoints
 * Letter to Editors
 * IPO Tracker
 * Events
 * Webinars
 * Sponsored Posts
 * Advertise
 * Endpoints Merch
 * About Us
 * Help


WORK IN BIOTECH

 * Endpoints Careers

© Endpoints Company 2023

 * Help
 * Advertise
 * Privacy Policy
 * Business Model


LOG IN TO YOUR ACCOUNT


E-MAIL




PASSWORD


Change my password Get a magic email link
LOG IN


REQUEST MAGIC LINK

If you're already an Endpoints subscriber, enter your email below for a magic
link that lets you log in quickly without using a password. Please note the
magic link is one-time use only and expires after 24 hours.


EMAIL


BACK
REQUEST


RESET PASSWORD

We'll e-mail you a link to set a new password. Please note this link is one-time
use only and is valid for only 24 hours.


EMAIL


BACK
RESET


ABOUT YOU







SUBSCRIPTIONS

ENDPOINTS NEWS Daily at 11:30 AM ET



EARLY EDITION Daily at 7:15 AM ET



ENDPOINTS PHARMA Daily at 2 PM ET



ENDPOINTS MARKETING RX Tue at 2 PM ET



ENDPOINTS FDA+ Wed at 2 PM ET



ENDPOINTS MANUFACTURING Thu at 2 PM ET



ENDPOINTS WEEKLY Sat at 6 AM ET


INSTANT SIGN UP