ASND halted….news pending

Spoken like a true sarcastic long term sufferer!  I salute you!

But I seriously hope not!

Yeah, that would be smart of them.  To "splain" it first...but whatever they say (to make themselves look/feel better) will not change the reality.  If a CRL is true, PFE will have a huge head start...that was ASND's only advantage really in my opinion.

She asks her husband, "How’s my cat doing?"

The husband says, "The cat’s dead."

The woman’s upset and says, "Well, you could have broken the news to me when I got home. I can’t enjoy my vacation now. You could’ve just said a little white lie, like the cat’s on the roof and you can’t get her down."

"Okay, I’m sorry," says the husband, "I’ll remember that."

The woman says, "Anyway, how’s my mother doing?"

The husband says, "Your mother’s on the roof and we can’t get her down."

---

www.reddit.com/r/Jokes/comments/8wgguv/a_womans_on_vacation_and_calls_home/

Ascendis Pharma A/S Announces U.S. Food and Drug Administration Approval of SKYTROFA® (lonapegsomatropin-tcgd), the First Once-weekly Treatment for Pediatric Growth Hormone Deficiency
- SKYTROFA, the first FDA approved treatment utilizing TransCon™ technology, is a long-acting prodrug of somatropin that releases the same somatropin used in daily therapies –
- Once weekly SKYTROFA demonstrated higher annualized height velocity (AHV) at week 52 compared to a daily growth hormone with similar safety and tolerability –

- Availability in the U.S. expected shortly supported by a full suite of patient support programs –

- Ascendis Pharma to host investor conference call today, Wednesday, August 25 at 4:30 p.m. E.T. -

COPENHAGEN, Denmark, Aug. 25, 2021 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to potentially create new treatments that make a meaningful difference in patients’ lives, today announced that the U.S. Food and Drug Administration (FDA) has approved SKYTROFA (lonapegsomatropin-tcgd) for the treatment of pediatric patients one year and older who weigh at least 11.5 kg (25.4 lb) and have growth failure due to inadequate secretion of endogenous growth hormone (GH).

As a once-weekly injection, SKYTROFA is the first FDA approved product that delivers somatropin (growth hormone) by sustained release over one week.

“Today’s approval represents an important new choice for children with GHD and their families, who will now have a once-weekly treatment option. In the pivotal head-to-head clinical trial, once-weekly SKYTROFA demonstrated higher annualized height velocity at week 52 compared to somatropin i ,” said Paul Thornton, M.B. B.Ch., MRCPI, a clinical investigator and pediatric endocrinologist in Fort Worth, Texas. “This once-weekly treatment could reduce treatment burden and potentially replace the daily somatropin therapies, which have been the standard of care for over 30 years.”

Growth hormone deficiency is a serious orphan disease characterized by short stature and metabolic complications. In GHD, the pituitary gland does not produce sufficient growth hormone, which is important not only for height but also for a child’s overall endocrine health and development.

The approval includes the new SKYTROFA ® Auto-Injector and cartridges which, after first removed from a refrigerator, allow families to store the medicine at room temperature for up to six months. With a weekly injection, patients switching from injections every day can experience up to 86 percent fewer injection days per year.

“SKYTROFA is the first product using our innovative TransCon technology platform that we have developed from design phase through non-clinical and clinical development, manufacturing and device optimization, and out to the patients. It reflects our commitment and dedication to addressing unmet medical needs by developing a pipeline of highly differentiated proprietary products across multiple therapeutic areas,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “We are grateful to the patients, caregivers, clinicians, clinical investigators, and our employees, who have all contributed to bringing this new treatment option to children in the U.S. with GHD.”

In connection with the commercialization of SKYTROFA, the company is committed to offering a full suite of patient support programs, including educating families on proper injection procedures for SKYTROFA as the first once-weekly treatment for children with GHD.

“It is wonderful that patients and their families now have the option of a once-weekly growth hormone therapy,” said Mary Andrews, Chief Executive Officer and co-founder of the MAGIC Foundation, a global leader in endocrine health, advocacy, education, and support. “GHD is often overlooked and undertreated in our children and managing it can be challenging for families. We are excited about this news as treating GHD is important, and children have a short time to grow.”

The FDA approval of SKYTROFA was based on results from the phase 3 heiGHt Trial, a 52-week, global, randomized, open-label, active-controlled, parallel-group trial that compared once-weekly SKYTROFA to daily somatropin (Genotropin ® ) in 161 treatment-naïve children with GHD ii . The primary endpoint was, AHV at 52 weeks for weekly SKYTROFA and daily hGH treatment groups. Other endpoints included adverse events, injection-site reactions, incidence of anti-hGH antibodies, annualized height velocity, change in height SDS, proportion of subjects with IGF-1 SDS (0.0 to +2.0), PK/PD in subjects < 3 years, and preference for and satisfaction with SKYTROFA.

At week 52, the treatment difference in AHV was 0.9 cm/year (11.2 cm/year for SKYTROFA compared with 10.3 cm/year for daily somatropin) with a 95 percent confidence interval [0.2, 1.5] cm/year. The primary objective of non-inferiority in AHV was met for SKYTROFA in this trial and further demonstrated a higher AHV at week 52 for lonapegsomatropin compared to daily somatropin, with similar safety, in treatment-naïve children with GHD.

No serious adverse events or discontinuations related to SKYTROFA were reported. Most common adverse reactions (≥ 5%) in pediatric patients include: infection, viral (15%), pyrexia (15%), cough (11%), nausea and vomiting (11%), hemorrhage (7%), diarrhea (6%), abdominal pain (6%), and arthralgia and arthritis (6%) i i . In addition, both arms of the study reported low incidences of transient, non-neutralizing anti-hGH binding antibodies and no cases of persistent antibodies.

Conference Call and Webcast Information

Date Wednesday, August 25, 2021
Time 4:30 p.m. ET/1:30 p.m. Pacific Time
Dial In (U.S.) 844-290-3904
Dial In (International) 574-990-1036
Access Code 8553236
A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com . A webcast replay will be available on this website shortly after conclusion of the event for 30 days.

Ascendis Pharma A/S Reports Second Quarter 2021 Financial Results
2021-08-25 20:12:26.22 GMT

Ascendis Pharma A/S Reports Second Quarter 2021 Financial Results

– Announced U.S. Food and Drug Administration Approval of SKYTROFA^®
(lonapegsomatropin-tcgd), the First Once-weekly Treatment for Pediatric Growth
Hormone Deficiency –

– Exceeded target enrollment in Phase 3 PaTHway Trial for TransCon PTH
(palopegteriparatide) in adults with hypoparathyroidism (HP); top-line results
expected in Q1 2022 –

– Initiated combination therapy arm in transcendIT-101; TransCon TLR7/8
Agonist used in combination with a check point inhibitor (CPI) –

– Conference call today at 4:30 p.m. Eastern Time –

COPENHAGEN, Denmark, Aug. 25, 2021 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S
(Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative
TransCon™ technologies to potentially create new treatments that make a
meaningful difference in patients’ lives, today announced financial results
for the second quarter ended June 30, 2021.

“We are actively preparing for the U.S. commercial launch of SKYTROFA for the
treatment of children with GHD, which is now the first FDA-approved
once-weekly treatment for pediatric GHD. SKTROFA is also the first
FDA-approved product utilizing our innovative TransCon technology. Our pivotal
heiGHt Trial demonstrated that once-weekly TransCon hGH increased annualized
height velocity in treatment-naïve subjects at 52 weeks compared to a daily
growth hormone with comparable safety and tolerability,” said Jan Mikkelsen,
Ascendis Pharma’s President and Chief Executive Officer. “We see this
approval as the first step in creating a market leading product and building a
fully integrated global biopharmaceutical company guided by our values of
patients, science, and passion.”

Company Highlights & Progress

o TransCon hGH (lonapegsomatropin)

o TransCon hGH is now FDA approved in the U.S. under the brand name
SKYTROFA. Continued preparation for commercial launch for the
treatment of pediatric patients with GHD in the U.S.
o European Commission decision on the company’s Marketing Authorisation
Application (MAA) for the treatment of pediatric patients with GHD is
anticipated in the fourth quarter of 2021.
o Ongoing enrollment in the foresiGHt Trial, a global phase 3 trial in
adults with GHD, and the riGHt Trial, a phase 3 trial in Japan in
pediatric patients with GHD.
o Patient follow-up continues in enliGHten, a multi-center phase 3,
long-term open-label trial investigating safety and efficacy of
SKYTROFA in pediatric patients with GHD.
o Comprehensive results from the heiGHt Trial recently published
on-line in the Journal of Clinical Endocrinology & Metabolism, an
official journal of the Endocrine Society.

o TransCon PTH (palopegteriparatide)

o Exceeded target enrollment in the PaTHway Trial, a phase 3 trial
evaluating the safety, tolerability, and efficacy of
palopegteriparatide in adult subjects with hypoparathyroidism with
similar demographics as enrolled in the phase 2 trial including broad
representation of different non-surgical disease etiologies and
leading influential clinical sites balanced between North America and
Europe.
o On track to announce 84-week top line results from the open label
extension (OLE) portion of the PaTH Forward Trial in the fourth
quarter of 2021. Continued strong long-term subject retention with 58
out of the 59 randomized subjects continuing in the OLE portion of
the trial as of August 23, 2021.
o Clinical trial notification for the PaTHway Japan Trial was accepted
by the Japanese Pharmaceuticals and Medical Device Agency. The
single-arm, phase 3 study will enroll a minimum of 12 Japanese
subjects with HP.
o Received Orphan Drug Designation (ODD) from the Japanese Ministry of
Health, Labor and Welfare.
o VISEN Pharmaceuticals (VISEN) obtained investigational new drug (IND)
approval to initiate the phase 3 PaTHway China Trial.

o TransCon CNP

o Continued execution in the ongoing phase 2 ACcomplisH Trial and
ACcomplisH China Trial to evaluate the safety and efficacy of
TransCon CNP in children ages two to ten years with achondroplasia.
o Clinical program update planned for the fourth quarter of 2021.

o TransCon TLR7/8 Agonist

o Initiated combination therapy arm in transcendIT-101 with TLR7/8
Agonist and a CPI.

o TransCon IL-2 ß/y

o IND filing on track for this quarter.

o Ended the second quarter of 2021 with cash, cash equivalents and
marketable securities totaling €641.3 million.

Second Quarter 2021 Financial Results

For the second quarter, Ascendis Pharma reported a net loss of €134.4 million,
or €2.50 per share (basic and diluted) compared to a net loss of €94.9
million, or €1.97 per share (basic and diluted) for the same period in 2020.

Revenue for the second quarter was €1.0 million compared to €1.4 million in
the same quarter of 2020. The decrease was due to a lower amount of license
revenue being recognized, partly offset by higher sale of clinical supplies
and services to VISEN and recognition of revenue from services rendered to
another collaboration partner.

Research and development (R&D) costs for the second quarter were €83.3 million
compared to €63.6 million during the same period in 2020. Higher R&D costs in
2021 reflect an increase in external development costs of the company’s
product candidates and an increase in personnel-related costs.

Selling, general and administrative expenses for the second quarter were €35.3
million compared to €20.8 million during the same period in 2020. The increase
is primarily due to higher personnel-related costs and an increase in IT
costs.

Net loss of associate for the second quarter was €4.8 million compared to a
net loss of €1.9 million in the same quarter of 2020. The net loss of
associate represents our share of the net result from VISEN.

As of June 30, 2021, Ascendis Pharma had cash, cash equivalents and marketable
securities of €641.3 million compared to €771.1 million as of March 31, 2021.
As of June 30, 2021, Ascendis Pharma had 53,900,990 ordinary shares
outstanding.

Conference Call Details

Date Wednesday, August 25, 2021
Time 4:30 p.m. ET/1:30 p.m. Pacific Time
Dial In (U.S.) 844-290-3904
Dial In (International) 574-990-1036
Access Code 8553236

A live webcast of the conference call will be available on the Investors and
News section of the Ascendis Pharma website at www.ascendispharma.com. A
webcast replay will be available on this website shortly after conclusion of
the event for 30 days.

About Ascendis Pharma’s Pipeline

Ascendis Pharma currently has three product candidates in clinical development
in rare endocrine diseases and one oncology product candidate in clinical
development:

* TransCon hGH (lonapegsomatropin-tcgd), an investigational long-acting
prodrug of somatropin (human growth hormone or hGH) that releases
somatropin with the identical amino acid sequence and size as daily growth
hormone, is designed as a once-weekly treatment for GHD and is approved
for pediatric GHD by the U.S. Food and Drug Administration and under
review by the European Medicines Agency.
* TransCon PTH (palopegteriparatide), an investigational long-acting prodrug
of parathyroid hormone (PTH) in phase 3 development as a once-daily
replacement therapy for adults with hypoparathyroidism designed to replace
PTH at physiologic levels for 24 hours, and address both short-term
symptoms and long-term complications of the disease.
* TransCon CNP, an investigational long-acting prodrug of C-type natriuretic
peptide (CNP) in phase 2 development as a therapy for children with
achondroplasia (ACH), the most common form of dwarfism, for which there is
no FDA-approved treatment. TransCon CNP is designed to provide continuous
exposure of CNP at safe, therapeutic levels via a single, weekly
subcutaneous dose.
* TransCon TLR7/8 Agonist is an investigational long-acting prodrug of
resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8.
Administered as an intratumoral injection, TransCon TLR7/8 Agonist is
designed to provide sustained activation of intratumoral antigen
presenting cells driving tumor antigen presentation and induction of
immune stimulatory cytokines in the tumor.
* TransCon IL-2 ß/y is an investigational long-acting prodrug of IL-2 ß/y
designed for optimized IL-2R ß/y bias and potency, combined with low Cmax
and long exposure.