Phathom Pharmaceuticals, Inc. (Nasdaq: PHAT), a biopharmaceutical
company focused on developing and commercializing novel treatments
for gastrointestinal (GI) diseases, announced today the U.S. Food
and Drug Administration (FDA) has approved VOQUEZNA® (vonoprazan)
tablets 10 mg and 20 mg, a novel potassium-competitive acid blocker
(PCAB), as a new treatment for adults for the healing of all grades
of Erosive Esophagitis, also known as Erosive GERD
(gastroesophageal reflux disease), maintenance of healing of all
grades of Erosive GERD, and relief of heartburn associated with
Erosive GERD.2
“This approval demonstrates Phathom's commitment to changing the
GI treatment landscape for patients and healthcare providers,
bringing the first major innovation to the U.S. Erosive GERD market
in over 30 years,” said Terrie Curran, President and Chief
Executive Officer at Phathom. “Erosive GERD can be extremely
painful and often has a significant impact on patients. Research
has shown patients and healthcare providers are largely unsatisfied
with current treatments and we are excited about the approval of a
first-in-class treatment option that has the potential to meet a
large unmet medical need.”
Erosive GERD, also referred to as Erosive Esophagitis or Erosive
Acid Reflux, is a major type of GERD that affects approximately 20
million people in the U.S.3,4 In addition to experiencing troubling
heartburn symptoms, patients with inadequately treated Erosive GERD
may develop more severe diseases including Barrett’s esophagus, a
condition in which esophageal tissue changes can progress to
cancer.3
This approval is based on positive results from the Phase 3
PHALCON-EE study (NCT04124926). The pivotal trial was a randomized,
double-blind, multicenter study that enrolled 1,024 patients with
Erosive GERD in the U.S. and Europe and compared VOQUEZNA to the
PPI lansoprazole in the healing and maintenance of healing of
Erosive GERD and associated heartburn symptom relief.1
Results showed that VOQUEZNA 20 mg met the primary endpoint of
non-inferiority (p<0.0001) for complete healing by Week 8 in
patients with all grades of Erosive GERD with a healing rate of 93%
compared to 85% for lansoprazole 30 mg, with superior rates of
healing demonstrated in a secondary endpoint in patients with
moderate-to-severe disease (LA Grade C/D) at Week 2 compared to
lansoprazole (70% for VOQUEZNA 20 mg and 53% for lansoprazole 30
mg) (p=0.0008). VOQUEZNA 20 mg also demonstrated non-inferiority to
lansoprazole 30 mg in the mean percentage of 24-hour heartburn free
days over the healing period. In the maintenance phase of the
trial, VOQUEZNA 10 mg was superior to lansoprazole 15 mg in
maintaining healing at six months in all randomized patients (79%
for VOQUEZNA 10 mg, compared to 72% for lansoprazole 15 mg) as well
as in the subset of patients with moderate-to-severe Erosive GERD
(75% for VOQUEZNA 10 mg, compared to 61% for lansoprazole 15 mg)
(p=0.0490). In addition, VOQUEZNA 10 mg was evaluated as a
secondary endpoint for relief of heartburn in Erosive GERD patients
and demonstrated non-inferiority to lansoprazole 15 mg over six
months.
Adverse event (AE) rates for VOQUEZNA were comparable to
lansoprazole in the trial. The most common AEs in the healing phase
(≥ 2% in the VOQUEZNA treatment arm) were gastritis (3.0% for
VOQUEZNA 20 mg and 2.0% for lansoprazole 30 mg), diarrhea (2.0% for
VOQUEZNA 20 mg and 3.0% for lansoprazole 30 mg), abdominal
distension (2.0% for VOQUEZNA 20 mg and 1.0 % for lansoprazole 30
mg), abdominal pain (2.0% for VOQUEZNA 20 mg and 1.0% for
lansoprazole 30 mg) and nausea (2.0% for VOQUEZNA 20 mg and 1.0%
for lansoprazole 30 mg). The most common AEs in the maintenance
phase (≥ 3% in the VOQUEZNA treatment arm) for VOQUEZNA 10 mg
compared to lansoprazole 15 mg were gastritis (6.0% vs. 3.0%),
abdominal pain (4.0% vs. 2.0%), dyspepsia (4.0% vs. 3.0%),
hypertension (3.0% vs. 2.0%), and urinary tract infection (3.0% vs.
2.0%). These are not all of the potential side effects associated
with the use of VOQUEZNA. Please see Important Safety information
below and the full Prescribing Information for VOQUEZNA for more
information.
“For many GERD patients with Erosive Esophagitis, the response
to current treatment is suboptimal, leaving them with incomplete
healing and ongoing symptoms5,” said Colin W. Howden, MD, Professor
Emeritus, University of Tennessee College of Medicine. “The FDA
approval of VOQUEZNA (vonoprazan) provides healthcare providers
with a new first-in-class therapeutic option that demonstrated
faster healing in the more difficult to treat GERD patients with
Erosive Esophagitis. In addition, VOQUEZNA (vonoprazan) provided
superior maintenance of healing in all grades of Erosive
Esophagitis, compared to lansoprazole, a commonly prescribed PPI,
and provided 24-hour heartburn relief on most days in the
trial.”
VOQUEZNA is expected to be available in the U.S. in December
2023 and will be marketed exclusively by Phathom Pharmaceuticals,
Inc.
Based on the terms of Phathom’s revenue interest financing
agreement, the FDA approval of VOQUEZNA for Erosive GERD also
entitles the company to receive a $175.0 million payment. This
non-dilutive capital will help fund the commercial launch.
Conference Call and Webcast on November 2, 2023, at
11:00 a.m. ETPhathom will host a live conference call and
webcast on Thursday, November 2, 2023, at 11:00 a.m. ET to discuss
the FDA approval and the Company’s U.S. commercialization plans for
Erosive GERD and H. pylori infection. The conference call will be
available via a listen-only webcast on the investor page of the
Company’s website at
https://investors.phathompharma.com/news-events/events-and-presentations.
About VOQUEZNA®VOQUEZNA® tablets, VOQUEZNA®
TRIPLE PAK® (vonoprazan, amoxicillin, clarithromycin), and
VOQUEZNA® DUAL PAK® (vonoprazan, amoxicillin) contain vonoprazan,
an oral small molecule potassium-competitive acid blocker
(PCAB). PCABs are a novel class of medicines that block acid
secretion in the stomach, and VOQUEZNA is the first PCAB to be
approved in the U.S. Vonoprazan has shown the potential to provide
acid suppression that can achieve pH levels that are important in
enhancing treatment effectiveness. Phathom in-licensed the U.S.,
European, and Canadian rights to vonoprazan from Takeda, which
markets the product in Japan and numerous other countries in Asia
and Latin America.
About Erosive GERDErosive GERD is a major type
of gastroesophageal reflux disease (GERD) characterized by erosions
in the gastric mucosa caused by acidic reflux of stomach contents
into the esophagus.3 There are estimated to be over 65 million
individuals with GERD in the U.S., of which approximately 30% have
Erosive GERD.4,6,7 In addition to experiencing troubling heartburn
symptoms, patients with inadequately treated Erosive GERD may
progress to more severe diseases including Barrett’s esophagus and
esophageal cancer.4
About Phathom Pharmaceuticals, Inc.Phathom
Pharmaceuticals is a biopharmaceutical company focused on the
development and commercialization of novel treatments for
gastrointestinal diseases. Phathom has in-licensed the exclusive
rights in the United States, Europe, and Canada to vonoprazan, a
first-in-class potassium-competitive acid blocker (PCAB). For more
information about Phathom, visit the Company’s website
at www.phathompharma.com and follow the
Company on LinkedIn and Twitter.
Please see Patient Information
and full Prescribing Information
for VOQUEZNA.
Visit www.voquezna.com to learn more about VOQUEZNA.
INDICATION AND IMPORTANT SAFETY INFORMATION
What is VOQUEZNA?VOQUEZNA® (vonoprazan) is a
prescription medicine used in adults:
- for 8 weeks to heal acid-related
damage to the lining of the esophagus (called Erosive Esophagitis
or Erosive Acid Reflux) and for relief of heartburn related to
Erosive Acid Reflux.
- for up to 6 months to maintain
healing of Erosive Acid Reflux and for relief of heartburn related
to Erosive Acid Reflux.
It is not known if VOQUEZNA is safe and effective in
children.
Do not take VOQUEZNA if you:
- are allergic to vonoprazan or any
of the other ingredients in VOQUEZNA. Allergic reaction symptoms
may include trouble breathing, rash, itching and swelling of your
face, lips, tongue or throat.
- are taking a medicine that contains rilpivirine (EDURANT,
COMPLERA, JULUCA, ODEFSEY, CABENUVA) used to treat HIV-1 (Human
Immunodeficiency Virus).
Before taking VOQUEZNA, tell your healthcare provider
about all your medical conditions, including if you:
- have low magnesium, calcium, or potassium in your blood, or you
are taking a medicine to increase urine (diuretic).
- have kidney or liver problems.
- are pregnant, think you may be pregnant, or plan to become
pregnant. It is not known if VOQUEZNA will harm your unborn
baby.
- are breastfeeding or plan to breastfeed. It is not known if
VOQUEZNA passes into your breast milk. You and your healthcare
provider should decide if you will take VOQUEZNA or breastfeed. You
should not do both.
Tell your healthcare provider about all the medicines
you take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements. Keep a list of them to
show your healthcare provider and pharmacist when you get a new
medicine.
VOQUEZNA may affect how other medicines work, and other
medicines may affect how VOQUEZNA works. Especially tell your
healthcare provider if you take medicine that contains rilpivirine
(EDURANT, COMPLERA, JULUCA, ODEFSEY, CABENUVA).
What are the possible side effects of VOQUEZNA?
VOQUEZNA may cause serious side effects
including:
A type of kidney problem (acute
tubulointerstitial nephritis): Some people who take
VOQUEZNA may develop a kidney problem called acute
tubulointerstitial nephritis. Call your healthcare provider right
away if you have a decrease in the amount that you urinate or if
you notice blood in your urine.
Diarrhea caused by an infection
(Clostridioides difficile) in
your intestines: Call your healthcare provider right away
if you have watery stools, stomach pain, and fever that does not go
away.
Bone fractures (hip, wrist, or spine): Bone
fractures in the hip, wrist, or spine may happen in people who take
multiple daily doses of another type of medicine that reduces acid
in your stomach known as proton pump inhibitors (PPI medicines) for
a long period of time (a year or longer). Tell your healthcare
provider if you have a bone fracture, especially in the hip, wrist,
or spine.
Severe skin reactions: VOQUEZNA can cause rare,
but severe skin reactions that may affect any part of your body.
These serious skin reactions may need to be treated in a hospital
and may be life threatening:
- Skin rash which may have blistering, peeling, or bleeding on
any part of your skin.
- You may also have fever, chills, body aches, shortness of
breath, or enlarged lymph nodes.
If you experience any of these symptoms, stop taking VOQUEZNA
and call your healthcare provider right away. These symptoms may be
the first sign of a severe skin reaction.
Low vitamin B-12 levels: VOQUEZNA lowers the
amount of acid in your stomach. Stomach acid is needed to absorb
Vitamin B12 properly. Tell your healthcare provider if you have
symptoms of low vitamin B12 levels, including irregular heartbeat,
shortness of breath, lightheadedness, tingling or numbness in the
arms or legs, muscle weakness, pale skin, feeling tired, or mood
changes. Talk with your healthcare provider about the risk of low
vitamin B12 levels if you have been on VOQUEZNA for a long
time.
Low magnesium levels in the body can happen in
people who take VOQUEZNA. Tell your healthcare provider right away
if you have symptoms of low magnesium levels, including seizures,
dizziness, irregular heartbeat, jitteriness, muscle aches or
weakness, or spasms of hands, feet, or voice.
Stomach growths (fundic gland polyps): A
certain type of stomach growth called fundic gland polyps may
happen in people who take another type of medicine that reduces
acid in your stomach known as proton pump inhibitors (PPI
medicines) for a long time. Talk with your healthcare provider
about the possibility of fundic gland polyps if you have been on
VOQUEZNA for a long time.
The most common side effects of VOQUEZNA for treatment
of Erosive Acid Reflux include:
- stomach inflammation
- diarrhea
- stomach bloating
- stomach pain
- nausea
- indigestion
- high blood pressure
- urinary tract infection
These are not all the possible side effects of VOQUEZNA. For
more information, ask your healthcare provider or pharmacist. Call
your healthcare provider for medical advice about side effects.
You are encouraged to report suspected adverse reactions
by contacting Phathom Pharmaceuticals at 1-888- 775-PHAT (7428) or
FDA at 1-800-FDA-1088 or
www.fda.gov/medwatch.
Forward Looking StatementThis press release
contains forward-looking statements. Investors are cautioned not to
place undue reliance on these forward-looking statements, including
statements about the timing of a U.S. commercial launch for
vonoprazan for Erosive GERD, the use of proceeds from the revenue
interest financing agreement, the size of the Erosive GERD patient
population and the potential of vonoprazan to satisfy the large
unmet medical need. The inclusion of forward-looking statements
should not be regarded as a representation by Phathom that any of
its plans will be achieved. Actual results may differ from those
set forth in this press release due to the risks and uncertainties
inherent in Phathom’s business, including, without limitation: we
may not be able to successfully commercialize VOQUEZNA which will
depend on a number of factors including coverage and reimbursement
levels from governmental authorities and health insurers as well as
market acceptance by healthcare providers; the Company has broad
discretion in the use of proceeds from the revenue interest
financing agreement; future data generated from our stability
program may be different from the data submitted to the FDA to date
and may not demonstrate that our mitigation efforts will continue
to maintain the level of the nitrosamine impurity below the
acceptable intake (AI) level throughout the shelf life of products
containing vonoprazan, which could result in market action or shelf
life reduction; risks associated with product manufacturing or
formulation changes required to be made in connection with
achieving the AI; the inherent risks of clinical development of
vonoprazan; Phathom’s dependence on third parties in connection
with product manufacturing, research and preclinical and clinical
testing; regulatory developments in the United States and foreign
countries; unexpected adverse side effects or inadequate efficacy
of vonoprazan that may limit its development, regulatory approval
and/or commercialization, or may result in recalls or product
liability claims; Phathom’s ability to access additional capital
under its term loan facility and royalty interest finance
agreements is subject to certain conditions; Phathom’s ability to
obtain and maintain intellectual property protection for
vonoprazan; Phathom’s ability to comply with its license agreement
with Takeda; and other risks described in the Company’s prior press
releases and the Company’s filings with the Securities and Exchange
Commission (SEC), including under the heading “Risk Factors” in the
Company’s Annual Report on Form 10-K and any subsequent filings
with the SEC. You are cautioned not to place undue reliance on
these forward-looking statements, which speak only as of the date
hereof, and Phathom undertakes no obligation to update such
statements to reflect events that occur or circumstances that exist
after the date hereof. All forward-looking statements are qualified
in their entirety by this cautionary statement, which is made under
the safe harbor provisions of the Private Securities Litigation
Reform Act of 1995.
REFERENCES
- Laine L, DeVault K, Katz P, et al. Vonoprazan Verses
Lansoprazole for Healing and Maintenance of Healing of Erosive
Esophagitis: A Randomized Trial. Gastroenterology. October 10,
2022. https://doi.org/10.1053/j.gastro.2022.09.041
- VOQUEZNA Prescribing Information, Phathom Pharmaceuticals,
2023.
- Dickman R, Maradey-Romero C, Gingold-Belfer R, Fass R. Unmet
Needs in the Treatment of Gastroesophageal Reflux Disease. J
Neurogastroenterol Motil. 2015 Jul 30;21(3):309-19. doi:
10.5056/jnm15105.
- Savarino E, de Bortoli N, De Cassan C, et al. The natural
history of gastro-esophageal reflux disease: A comprehensive
review. Dis Esophagus. 2017;30(2):1-9.
- Vaezi MF, Brunton S, Mark Fendrick A, et al. Patient journey in
erosive oesophagitis: real-world perspectives from US physicians
and patients. BMJ Open Gastroenterology. 2022;9:e000941. doi:
10.1136/bmjgast-2022-000941
- Machicado J.D., Greer J.B., Yadav D. (2020) Epidemiology of
Gastrointestinal Diseases. In: Pitchumoni C., Dharmarajan T. (eds)
Geriatric Gastroenterology. Springer, Cham.
https://doi.org/10.1007/978-3-319-90761-1_7-1.
- U.S. Census Bureau. U.S. and World Population Clock. Accessed
May 2022. https://www.census.gov/popclock.
MEDIA CONTACTNick
Benedetto1-877-742-8466media@phathompharma.com
INVESTOR CONTACTEric
Sciorilli1-877-742-8466ir@phathompharma.com
© 2023 Phathom Pharmaceuticals. All rights reserved.
VOQUEZNA, Phathom Pharmaceuticals, and their respective logos
are registered trademarks or trademarks of Phathom Pharmaceuticals,
Inc.
11/23 US-VPZ-22-0331
PDFs accompanying this announcement are available at the links
belowVOQUEZNA for Erosive GERD - Fact SheetBreaking Down Erosive
GERD - Fact Sheet
Photos accompanying this announcement are available
athttps://www.globenewswire.com/NewsRoom/AttachmentNg/7895e481-ed42-46dd-8881-b06895518153https://www.globenewswire.com/NewsRoom/AttachmentNg/dd0696f6-1062-4616-8c8a-1e49b788529f
Phathom Pharmaceuticals (NASDAQ:PHAT)
Historical Stock Chart
From Apr 2024 to May 2024
Phathom Pharmaceuticals (NASDAQ:PHAT)
Historical Stock Chart
From May 2023 to May 2024