ASND: Distr Ptnr Skytrofa Arab Countries

As a fellow investor just said when he sent this to me: "How it should be done".....

ASND is presenting today at JPM. And in preparation for their opening day update to investors at a very important conference, they put out a PR. The PR lays the groundwork of what investors should expect to hear from management today. Very transparent, very deliberate, very professional. They leave no doubts about what they're doing, where they're going, and how they're going to get there. A bit different than the m.o. at Opko. Here's what a real company does in its communications with the street. By the way, the presentation must be going over quite well: the stock is up almost NINE POINTS, about 7%. Adding over FIVE HUNDRED MILLION IN MARKET CAP, just crossing EIGHT BILLION.

Unless Opko reveals some very important information on Wednesday, then the upcoming meeting agenda could be summarized with a single question: Folks, why aren't you doing something like this?

Ascendis Pharma Introduces Vision 2030
2024-01-07 22:45:28.705 GMT

Ascendis Pharma Introduces Vision 2030

o Strategic roadmap to achieve blockbuster status for multiple products and
expand
the Company’s engine for future innovation

COPENHAGEN, Denmark, Jan. 07, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S
(Nasdaq: ASND) today introduced selected 2024 corporate milestones and Vision
2030, its strategic roadmap through 2030. Ascendis President and CEO Jan
Mikkelsen will present the update tomorrow, January 8, at the 42^nd Annual
J.P. Morgan Healthcare Conference.

“With unwavering focus on our values of Patients, Science, and Passion, we
remain on track to fulfill Vision 3x3, with the approval already of two
Endocrinology Rare Disease products and value leadership in the U.S. growth
hormone market for SKYTROFA,” said Jan Mikkelsen, Ascendis Pharma’s President
and Chief Executive Officer. “Today we are introducing Vision 2030, our
strategic roadmap to attain blockbuster status for multiple products and
expand our engine for future innovation.”

Selected Key Updates

o TransCon^™ hGH
(lonapegsomatropin, approved as SKYTROFA^® in the U.S. and EU)

* Following positive topline results from Phase 3 foresiGHt Trial in
adult growth hormone deficiency (GHD), plan to submit a supplemental
Biologics License Application to FDA in the second quarter of 2024.
* Topline results from Phase 2 trial in Turner syndrome expected in the
fourth quarter of 2024.
* Attained U.S. market value leadership in 2023.
* SKYTROFA preliminary unaudited fourth quarter 2023 revenue is
expected to be approximately €64 million.
* Full year 2024 SKYTROFA revenue expected to be €320 million to €340
million (based on average 2023 exchange rates).

o TransCon PTH
(palopegteriparatide, approved as YORVIPATH^® in the EU)

* First launch planned in Germany in January 2024, leveraging the
Company’s existing commercial infrastructure.
* FDA regulatory review for the treatment of adult patients with
hypoparathyroidism continues, with a Prescription Drug User Fee Act
(PDUFA) date of May 14, 2024; if approved, U.S. commercial launch
planned in the third quarter of 2024.

o TransCon CNP
(navepegritide)

* Treatment with TransCon CNP at 100 mg/kg/week for one year in all 57
children with achondroplasia (age 2 to 10 years) in ACcomplisH,
demonstrated growth consistent with results from the blinded period
and achieved improvements in health-related quality of life and
disease impacts assessed with SF-10 and Achondroplasia Child
Experience Measure.
* During the third quarter of 2023, submitted Clinical Trial
Application (CTA) for infant trial (age 0-2 years).
* Topline results from the pivotal ApproaCH Trial expected in the
fourth quarter of 2024.
* During the fourth quarter of 2024, plan to submit a New Drug
Application to FDA for children with achondroplasia (age 2-11 years).
* Week 26 topline data from the COACH Trial (TransCon hGH/TransCon CNP
combination) expected in the fourth quarter of 2024.
* During the fourth quarter of 2024, plan to submit an Investigational
New Drug application or similar in adults with achondroplasia.

o Global commercial presence in Endocrinology Rare Disease

* During the fourth quarter of 2023, entered into an exclusive license
agreement with Teijin Limited for TransCon hGH, TransCon PTH, and
TransCon CNP in Japan. Phase 3 PaTHway Japan Trial of TransCon PTH
completed, and Phase 3 riGHt Trial of TransCon hGH fully enrolled.
* VISEN’s TransCon hGH Phase 3 and TransCon CNP Phase 2 trials in China
completed.
* Expanding global reach through exclusive sales & distribution
agreements with geographic market leaders in International Markets,
with three regional agreements signed to date: Specialised
Therapeutics Asia Pte Ltd, Er-Kim, and Vector Pharma FZCO.

o Oncology

* During the fourth quarter of 2024, expect to complete enrollment in
BelieveIT-201, a Phase 2 trial in advanced head and neck squamous
cell carcinoma (HNSCC).
* During the fourth quarter of 2024, plan to provide a clinical update
from the Phase 2 portion of indication-specific, dose expansion
cohorts in the IL-Believe Trial.

o Ophthalmology

* Creation of Ophthalmology NewCo, financed by institutional investors,
expected during the first quarter of 2024.

o Financial Update and Outlook Based on Current Plans

* Preliminary unaudited December 31, 2023 cash, cash equivalents, and
marketable securities of ~€400 million.
* Full year 2024 SKYTROFA revenue expected to be €320 million to €340
million (based on average 2023 exchange rates).
* Expect to provide YORVIPATH revenue update during 2024.
* Expect total operating expenses (SG&A and R&D) of approximately €600
million for 2024.
* Expect to be operating cash flow breakeven on a quarterly basis by
the end of 2024.

Vision 2030: Ascendis Pharma’s 2025–2030 Strategic Roadmap

Achieve blockbuster status for multiple products and expand our engine for
future innovation.

o Be the Leading Endocrinology Rare Disease Company

o Achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and
TransCon CNP through worldwide commercialization
o Be the leader in growth disorders and hypoparathyroidism, pursuing
clinical conditions, innovative life cycle management, and
complementary patient offerings
o Expand pipeline with Endocrinology Rare Disease blockbuster product
opportunities

o Create Value in Additional Therapeutic Areas through Innovative Business
Models

o Obtain accelerated approval in oncology with registrational trials
ongoing
o Pursue TransCon product opportunities in >$5B indications
o Maximize value creation of these product opportunities through
collaboration with therapeutic area market leaders

o Differentiate with Ascendis Fundamentals

o Outperform industry drug development benchmarks with Ascendis’
product innovation algorithm
o Remain independent as a profitable biopharma through lean and
flexible ways of working
o Let our values Patients, Science, Passion drive our decisions to
success

Presentation at J.P. Morgan Healthcare Conference on Monday, January 8th

A live webcast of the event will be available via the Investors & News section
of the Ascendis Pharma website at investors.ascendispharma.com. The
presentation will begin at 12:00 p.m. Eastern Time / 9:00 am Pacific Time. A
webcast replay will be available for 30 days.

The Company’s slides from the J.P. Morgan presentation will be available on
the same Investor Relations website at investors.ascendispharma.com.
About Ascendis Pharma A/S