Post by luxetvox on Sept 5, 2023 20:18:03 GMT
I know this category is "All About Opko Health" but, to me, this info is important because it is impactful for Opko, which took nearly a 4% header today.
Ascendis Pharma Reports Second Quarter 2023 Financial Results
* Expect to resubmit NDA to FDA for TransCon™ PTH in October 2023; EC
decision on MAA for TransCon PTH expected during the fourth quarter of
2023
* U.S. and EU regulatory authorities endorsed ApproaCH as a pivotal Phase 3
trial of TransCon CNP in children with achondroplasia; enrollment
completed, and topline results expected in second half of 2024
* Raising full year 2023 U.S. SKYTROFA^®* revenue expectations to €165 -
€170 million
* Conference call today at 4:30 pm ET
COPENHAGEN, Denmark, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S
(Nasdaq: ASND) today announced financial results for the second quarter ended
June 30, 2023 and provided business updates.
“We have built a fully integrated Endocrinology Rare Disease franchise with a
growing commercial reach and are on our way to achieving our Vision 3x3,
including regulatory approval for three differentiated product candidates by
2025,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive
Officer. “With a proven U.S. and expanding global commercial infrastructure
positioned to capture the multi-billion-dollar opportunities that each
represents, coupled with an R&D engine for label expansion and future
innovation for new patient populations, Ascendis has many routes to realize
long-term profitability and sustainability.”
Corporate Highlights
o TransCon hGH (marketed in the U.S. as SKYTROFA):
o Second quarter 2023 SKYTROFA revenue totaled €36 million, and the
Company is increasing full year 2023 U.S. SKYTROFA revenue
expectations from €150 - €160 million to €165 - €170 million.
o Second quarter 2023 SKYTROFA revenue included a negative adjustment
to provision for estimated sales rebates related to sales from prior
periods of €2.1 million as a result of expanding payer coverage and a
negative foreign currency impact of €0.6 million compared to the
first quarter of 2023, due to a weaker U.S. dollar.
Q2-2022 Q3-2022 Q4-2022 Q1-2023 Q2-2023
SKYTROFA revenue (millions) €4.4 €12.3 €17.1 €31.6 €35.9
o
* Based on reported revenues, SKYTROFA was the leading U.S. growth
hormone product in the second quarter, with a market share of less
than 10% of treated U.S. pediatric GHD patients.
* First commercial launch of SKYTROFA in the EU expected in September
in Germany.
* Received FDA approval of Lonza as a high-capacity drug substance
manufacturing site to support worldwide demand of SKYTROFA. Expect EU
approval in first half of 2024.
* Topline results from Phase 3 foresiGHt Trial in adult growth hormone
deficiency investigating the metabolic impact of TransCon hGH
expected in the fourth quarter of 2023, opening up the first label
expansion opportunity.
o TransCon PTH:
o In June 2023, the Company requested a Type A meeting with FDA and
submitted an updated control strategy. The Company’s Type A meeting
was held with FDA in late August based on the Agency’s availability.
Following a constructive Type A meeting, the Company submitted
additional information to FDA supporting the updated control
strategy. Ascendis believes the materials submitted to FDA combined
with the Type A meeting discussions will position the Company to
resubmit the NDA for TransCon PTH for adults with hypoparathyroidism
in October 2023.
o Remain on track for European Commission decision on Marketing
Authorisation Application (MAA) for TransCon PTH during the fourth
quarter of 2023. If approved, first country launch planned in Germany
in early 2024.
o New post hoc analysis showed TransCon PTH-treated patients
demonstrated substantial increases in estimated glomerular filtration
rate (eGFR), indicating improved kidney function. More information on
this analysis can be found in a separate press release issued today
and available here on the Investors & News section of the Ascendis
Pharma website.
o 145 out of 154 participants continue in the open-label extension
(OLE) portions of the Phase 2 PaTH Forward, Phase 3 PaTHway, and
PaTHway Japan trials.
o The U.S. Expanded Access Program and German Compassionate Use Program
continue to be open for enrollment of eligible patients.
o TransCon CNP:
o U.S. and EU regulatory agencies have endorsed ApproaCH, a global
randomized, double-blind, placebo-controlled trial in children ages
2–11 years with achondroplasia, as a pivotal Phase 3 trial.
Enrollment is complete and topline results are expected in the second
half of 2024.
o All 57 patients remain in the OLE portion of the Phase 2 ACcomplisH
Trial, with treatment duration up to 3 years. One-year follow-up data
from OLE expected in the fourth quarter of 2023.
o TransCon TLR7/8 Agonist:
o Enrollment continues in the Phase 2 portion of transcendIT-101 at the
recommended Phase 2 dose (RP2D) in four indication-specific cohorts.
Topline/interim analysis from Phase 2 dose-expansion cohorts expected
in 2024.
o TransCon IL-2 β/γ
o Completed Phase 1 dose escalation in combination with pembrolizumab
in the Phase 1/2 IL-Believe Trial with a total of 21 patients
enrolled. RP2D was determined at 120 µg/kg IV every three weeks. No
dose-limiting toxicity, vascular leak syndrome, or grade 3 or 4
cytokine release syndrome was observed at any dose level evaluated.
o Enrollment initiated in the Phase 2 portion in indication-specific
cohorts, with expected topline/interim analysis from Phase 2
dose-expansion cohorts expected in 2024.
o Expanding leadership in long-acting prodrug technologies with new TransCon
technology carrier platform to unlock large market opportunities where
high volume and low-cost manufacturing is required.
o Proof-of-principle demonstrated for GLP-1 analogs (semaglutide), with
data supporting potential best-in-class weekly and monthly
administration profiles. More information on the pre-clinical data
can be found in a presentation here on the Investors & News section
of the Ascendis Pharma website.
o Ended the second quarter of 2023 with cash, cash equivalents, and
marketable securities totaling €431.1 million.
o Subsequent to the quarter end, entered into a $150 million capped
synthetic royalty funding agreement with Royalty Pharma. More information
on this funding can be found in a separate press release issued today (I've posted the PR below) and
available here on the Investors & News section of the Ascendis. Pharma
website.
Second Quarter 2023 Financial Results
Total revenue for the second quarter of 2023 was €47.4 million compared to
€6.2 million during the same period in 2022. The increase was primarily
attributable to higher SKYTROFA revenue of €35.9 million compared to €4.4
million in the same period last year.
Research and development (R&D) costs for the second quarter were €105.0
million compared to €90.4 million during the same period in 2022. This
increase was primarily due to higher development costs for Ascendis Pharma
Oncology programs (TransCon IL-2 β/γ and TransCon TLR7/8 Agonist), increasing
clinical trial activities for TransCon CNP, and higher employee-related costs,
and was partly offset by lower development costs for TransCon hGH.
Selling, general, and administrative (SG&A) expenses for the second quarter
were €70.3 million compared to €56.6 million during the same period in 2022.
This increase was primarily due to higher external commercial expenses related
to SKYTROFA, pre-launch activities for SKYTROFA outside the U.S., global
pre-launch activities for TransCon PTH, higher employee related expenses, and
an increase in other general and administrative expenses attributable to
organizational growth.
Net finance income was €26.4 million in the second quarter compared to €61.7
million in the same period in 2022.
For the second quarter of 2023, Ascendis Pharma reported a net loss of €121.4
million, or €2.16 per share (basic and diluted) compared to a net loss of
€81.3 million, or €1.46 per share (basic and diluted) for the same period in
2022.
As of June 30, 2023, Ascendis Pharma had cash, cash equivalents, and
marketable securities totaling €431.1 million compared to €742.9 million as of
December 31, 2022. As of June 30, 2023, Ascendis Pharma had 57,335,496
ordinary shares outstanding.
And here's the royalty deal terms for Skytrofa. Once OPK starts recording revenue from the royalty and/or gross profit share arrangement, this may have some relevance regarding a present value of Somatrogon as an Opko asset and source of revenue and profit.
Ascendis Pharma and Royalty Pharma Enter into $150 Million Royalty Funding
Agreement
- Proceeds to support continued development and commercialization of Endocrine
Rare Disease products, and general corporate purposes
COPENHAGEN, Denmark and NEW YORK, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Ascendis
Pharma A/S (Nasdaq: ASND) and Royalty Pharma plc (Nasdaq: RPRX) today
announced that Ascendis has entered into a $150 million capped synthetic
royalty funding agreement with Royalty Pharma based on U.S. net SKYTROFA
revenue.
“This transaction reflects the significant value of SKYTROFA. We are pleased
to partner with Royalty Pharma, a leading funder of innovation across the
biopharma industry, and look forward to partnering with them in the coming
years,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive
Officer. “With this funding, we continue to reduce our cost of capital and
provide added flexibility to support our global commercial capabilities to
bring our TransCon products to patients as fast as possible.”
“We are excited to partner with Ascendis, a global, integrated
biopharmaceutical company focused on endocrine rare disease and oncology,”
said Pablo Legorreta, founder and Chief Executive Officer of Royalty Pharma.
“SKYTROFA, as the first U.S. approved weekly growth hormone therapy for
pediatrics, addresses significant unmet patient need, which is underscored by
its strong launch. We look forward to Ascendis’ continued success in reaching
as many patients as possible with this important therapy as well as the
potential for label expansion in additional indications.”
Under the terms of the agreement, Ascendis Pharma receives an upfront payment
of $150 million in exchange for a 9.15% royalty on U.S. net SKYTROFA revenue,
beginning on January 1, 2025. The royalty payments to Royalty Pharma will
cease upon reaching a multiple of 1.925x, or 1.65x if Royalty Pharma receives
royalties in that amount by December 31, 2031.
As to the terms that trigger a cessation of monies paid to Royalty Pharma, I assume that means that they are capped to receive between $247.5 million and $288.75 million in total royalties, the former if received by 12/31/31.
By the way Royalty Pharma PLC is a public company, whose Class A common stock trades under the symbol RPRX. Having a tough year, but market cap is still healthy at $17.75 billion.
Ascendis Pharma Reports Second Quarter 2023 Financial Results
* Expect to resubmit NDA to FDA for TransCon™ PTH in October 2023; EC
decision on MAA for TransCon PTH expected during the fourth quarter of
2023
* U.S. and EU regulatory authorities endorsed ApproaCH as a pivotal Phase 3
trial of TransCon CNP in children with achondroplasia; enrollment
completed, and topline results expected in second half of 2024
* Raising full year 2023 U.S. SKYTROFA^®* revenue expectations to €165 -
€170 million
* Conference call today at 4:30 pm ET
COPENHAGEN, Denmark, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S
(Nasdaq: ASND) today announced financial results for the second quarter ended
June 30, 2023 and provided business updates.
“We have built a fully integrated Endocrinology Rare Disease franchise with a
growing commercial reach and are on our way to achieving our Vision 3x3,
including regulatory approval for three differentiated product candidates by
2025,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive
Officer. “With a proven U.S. and expanding global commercial infrastructure
positioned to capture the multi-billion-dollar opportunities that each
represents, coupled with an R&D engine for label expansion and future
innovation for new patient populations, Ascendis has many routes to realize
long-term profitability and sustainability.”
Corporate Highlights
o TransCon hGH (marketed in the U.S. as SKYTROFA):
o Second quarter 2023 SKYTROFA revenue totaled €36 million, and the
Company is increasing full year 2023 U.S. SKYTROFA revenue
expectations from €150 - €160 million to €165 - €170 million.
o Second quarter 2023 SKYTROFA revenue included a negative adjustment
to provision for estimated sales rebates related to sales from prior
periods of €2.1 million as a result of expanding payer coverage and a
negative foreign currency impact of €0.6 million compared to the
first quarter of 2023, due to a weaker U.S. dollar.
Q2-2022 Q3-2022 Q4-2022 Q1-2023 Q2-2023
SKYTROFA revenue (millions) €4.4 €12.3 €17.1 €31.6 €35.9
o
* Based on reported revenues, SKYTROFA was the leading U.S. growth
hormone product in the second quarter, with a market share of less
than 10% of treated U.S. pediatric GHD patients.
* First commercial launch of SKYTROFA in the EU expected in September
in Germany.
* Received FDA approval of Lonza as a high-capacity drug substance
manufacturing site to support worldwide demand of SKYTROFA. Expect EU
approval in first half of 2024.
* Topline results from Phase 3 foresiGHt Trial in adult growth hormone
deficiency investigating the metabolic impact of TransCon hGH
expected in the fourth quarter of 2023, opening up the first label
expansion opportunity.
o TransCon PTH:
o In June 2023, the Company requested a Type A meeting with FDA and
submitted an updated control strategy. The Company’s Type A meeting
was held with FDA in late August based on the Agency’s availability.
Following a constructive Type A meeting, the Company submitted
additional information to FDA supporting the updated control
strategy. Ascendis believes the materials submitted to FDA combined
with the Type A meeting discussions will position the Company to
resubmit the NDA for TransCon PTH for adults with hypoparathyroidism
in October 2023.
o Remain on track for European Commission decision on Marketing
Authorisation Application (MAA) for TransCon PTH during the fourth
quarter of 2023. If approved, first country launch planned in Germany
in early 2024.
o New post hoc analysis showed TransCon PTH-treated patients
demonstrated substantial increases in estimated glomerular filtration
rate (eGFR), indicating improved kidney function. More information on
this analysis can be found in a separate press release issued today
and available here on the Investors & News section of the Ascendis
Pharma website.
o 145 out of 154 participants continue in the open-label extension
(OLE) portions of the Phase 2 PaTH Forward, Phase 3 PaTHway, and
PaTHway Japan trials.
o The U.S. Expanded Access Program and German Compassionate Use Program
continue to be open for enrollment of eligible patients.
o TransCon CNP:
o U.S. and EU regulatory agencies have endorsed ApproaCH, a global
randomized, double-blind, placebo-controlled trial in children ages
2–11 years with achondroplasia, as a pivotal Phase 3 trial.
Enrollment is complete and topline results are expected in the second
half of 2024.
o All 57 patients remain in the OLE portion of the Phase 2 ACcomplisH
Trial, with treatment duration up to 3 years. One-year follow-up data
from OLE expected in the fourth quarter of 2023.
o TransCon TLR7/8 Agonist:
o Enrollment continues in the Phase 2 portion of transcendIT-101 at the
recommended Phase 2 dose (RP2D) in four indication-specific cohorts.
Topline/interim analysis from Phase 2 dose-expansion cohorts expected
in 2024.
o TransCon IL-2 β/γ
o Completed Phase 1 dose escalation in combination with pembrolizumab
in the Phase 1/2 IL-Believe Trial with a total of 21 patients
enrolled. RP2D was determined at 120 µg/kg IV every three weeks. No
dose-limiting toxicity, vascular leak syndrome, or grade 3 or 4
cytokine release syndrome was observed at any dose level evaluated.
o Enrollment initiated in the Phase 2 portion in indication-specific
cohorts, with expected topline/interim analysis from Phase 2
dose-expansion cohorts expected in 2024.
o Expanding leadership in long-acting prodrug technologies with new TransCon
technology carrier platform to unlock large market opportunities where
high volume and low-cost manufacturing is required.
o Proof-of-principle demonstrated for GLP-1 analogs (semaglutide), with
data supporting potential best-in-class weekly and monthly
administration profiles. More information on the pre-clinical data
can be found in a presentation here on the Investors & News section
of the Ascendis Pharma website.
o Ended the second quarter of 2023 with cash, cash equivalents, and
marketable securities totaling €431.1 million.
o Subsequent to the quarter end, entered into a $150 million capped
synthetic royalty funding agreement with Royalty Pharma. More information
on this funding can be found in a separate press release issued today (I've posted the PR below) and
available here on the Investors & News section of the Ascendis. Pharma
website.
Second Quarter 2023 Financial Results
Total revenue for the second quarter of 2023 was €47.4 million compared to
€6.2 million during the same period in 2022. The increase was primarily
attributable to higher SKYTROFA revenue of €35.9 million compared to €4.4
million in the same period last year.
Research and development (R&D) costs for the second quarter were €105.0
million compared to €90.4 million during the same period in 2022. This
increase was primarily due to higher development costs for Ascendis Pharma
Oncology programs (TransCon IL-2 β/γ and TransCon TLR7/8 Agonist), increasing
clinical trial activities for TransCon CNP, and higher employee-related costs,
and was partly offset by lower development costs for TransCon hGH.
Selling, general, and administrative (SG&A) expenses for the second quarter
were €70.3 million compared to €56.6 million during the same period in 2022.
This increase was primarily due to higher external commercial expenses related
to SKYTROFA, pre-launch activities for SKYTROFA outside the U.S., global
pre-launch activities for TransCon PTH, higher employee related expenses, and
an increase in other general and administrative expenses attributable to
organizational growth.
Net finance income was €26.4 million in the second quarter compared to €61.7
million in the same period in 2022.
For the second quarter of 2023, Ascendis Pharma reported a net loss of €121.4
million, or €2.16 per share (basic and diluted) compared to a net loss of
€81.3 million, or €1.46 per share (basic and diluted) for the same period in
2022.
As of June 30, 2023, Ascendis Pharma had cash, cash equivalents, and
marketable securities totaling €431.1 million compared to €742.9 million as of
December 31, 2022. As of June 30, 2023, Ascendis Pharma had 57,335,496
ordinary shares outstanding.
And here's the royalty deal terms for Skytrofa. Once OPK starts recording revenue from the royalty and/or gross profit share arrangement, this may have some relevance regarding a present value of Somatrogon as an Opko asset and source of revenue and profit.
Ascendis Pharma and Royalty Pharma Enter into $150 Million Royalty Funding
Agreement
- Proceeds to support continued development and commercialization of Endocrine
Rare Disease products, and general corporate purposes
COPENHAGEN, Denmark and NEW YORK, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Ascendis
Pharma A/S (Nasdaq: ASND) and Royalty Pharma plc (Nasdaq: RPRX) today
announced that Ascendis has entered into a $150 million capped synthetic
royalty funding agreement with Royalty Pharma based on U.S. net SKYTROFA
revenue.
“This transaction reflects the significant value of SKYTROFA. We are pleased
to partner with Royalty Pharma, a leading funder of innovation across the
biopharma industry, and look forward to partnering with them in the coming
years,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive
Officer. “With this funding, we continue to reduce our cost of capital and
provide added flexibility to support our global commercial capabilities to
bring our TransCon products to patients as fast as possible.”
“We are excited to partner with Ascendis, a global, integrated
biopharmaceutical company focused on endocrine rare disease and oncology,”
said Pablo Legorreta, founder and Chief Executive Officer of Royalty Pharma.
“SKYTROFA, as the first U.S. approved weekly growth hormone therapy for
pediatrics, addresses significant unmet patient need, which is underscored by
its strong launch. We look forward to Ascendis’ continued success in reaching
as many patients as possible with this important therapy as well as the
potential for label expansion in additional indications.”
Under the terms of the agreement, Ascendis Pharma receives an upfront payment
of $150 million in exchange for a 9.15% royalty on U.S. net SKYTROFA revenue,
beginning on January 1, 2025. The royalty payments to Royalty Pharma will
cease upon reaching a multiple of 1.925x, or 1.65x if Royalty Pharma receives
royalties in that amount by December 31, 2031.
As to the terms that trigger a cessation of monies paid to Royalty Pharma, I assume that means that they are capped to receive between $247.5 million and $288.75 million in total royalties, the former if received by 12/31/31.
By the way Royalty Pharma PLC is a public company, whose Class A common stock trades under the symbol RPRX. Having a tough year, but market cap is still healthy at $17.75 billion.
