On March 2, 2022 Ascendis Pharma A/S (Nasdaq: ASND) reported financial results for the full year ended December 31, 2021 and provided a business update (Press release, Ascendis Pharma, MAR 2, 2022, View Source [SID1234609372]).

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"2021 was an extraordinary year for Ascendis, and we expect that 2022 will be even stronger. The regulatory approvals of TransCon hGH in the U.S. and Europe were key milestones in achieving our Vision 3×3," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.

"By progressing towards our Vision 3×3, we believe we are moving towards becoming a viable, sustainable, and profitable biopharmaceutical company. We estimate that our first therapeutic area of endocrinology rare disease alone represents a combined US$10 billion global market potential. On top of that, we have a highly differentiated oncology pipeline, and we plan to add a third therapeutic area," continued Mr. Mikkelsen.

Corporate 2021 Highlights & Anticipated 2022 Milestones

TransCon hGH:
In fall-2021, the Company commercially launched in the U.S., TransCon hGH under the brand name of SKYTROFA (lonapegsomatropin-tcgd), the only FDA-approved once-weekly treatment for pediatric patients one year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone. As of February 28, 2022, 708 SKYTROFA prescriptions were written by approximately 263 prescribers, including 44% writing prescriptions for multiple patients.
In January 2022, the Company received marketing authorization for Lonapegsomatropin Ascendis Pharma (developed under the brand name of TransCon hGH) in the European Union as a once-weekly subcutaneous injection for the treatment of children and adolescents ages 3 to 18 years with growth failure due to insufficient secretion of endogenous growth hormone.
Plan to submit a protocol to the FDA to evaluate TransCon hGH for Turner Syndrome in the second quarter of 2022. The Company expects to evaluate higher doses of TransCon hGH and daily growth hormone for Turner Syndrome compared to those doses for pediatric or adult GHD.
The ongoing conflict in the region surrounding Ukraine and Russia has impacted our ability to continue clinical trial activities in those countries. While this may affect our timelines for the foresiGHt Trial, our Phase 3 trial in adult growth hormone deficiency, there is currently no material impact to our business from this situation.
TransCon PTH:
In November 2021, the Company announced top-line results from Week-84 of the Company’s Phase 2 PaTH Forward Trial evaluating the safety, tolerability, and efficacy of TransCon PTH in adults with HP. The week 84 data showed that subjects treated with TransCon PTH had both mean serum calcium levels and urinary calcium excretion that remained stable and in the normal range and that all subjects continued to be free from taking active vitamin D and 93% were taking less than 600 mg/day of calcium supplements.
After two years of treatment in the open label extension portion of the PaTH Forward Trial, as of February 22, 2022, 57 out of 59 original subjects continued in the trial.
Top-line results from PaTHway, a Phase 3 randomized, double-blind placebo-controlled clinical trial in North America and Europe, evaluating the safety, tolerability, and efficacy of TransCon PTH in adults with HP, are expected in the first quarter of 2022.
If the Phase 3 Trial results are positive, the Company plans to submit a New Drug Application to the FDA during the third quarter of 2022 and expects to submit a Marketing Authorisation Application to the European Medicines Agency during the fourth quarter of 2022.
Top-line results from PaTHway Japan, a single-arm Phase 3 trial of TransCon PTH in a minimum of 12 Japanese subjects with HP are expected in the third quarter of 2022.
Initiation of a pediatric HP program is planned for the fourth quarter of 2022.
TransCon CNP:
In December 2021, the Company announced completed enrollment in the ACcomplisH Trial, a Phase 2 randomized, double-blind, placebo-controlled clinical trial in North America, Europe, and Oceania in subjects with achondroplasia (ages 2–10). Forty-two percent of subjects enrolled in the ACcomplisH Trial are between the ages of 2 and 5 years old.
Top-line data from the ACcomplisH Trial are expected in the fourth quarter of 2022.
The Company plans to file an Investigational New Drug (IND) application or similar for the ACcomplisH Infants Trial in subjects with achondroplasia (ages 0–2) during the second quarter of 2022.
TransCon TLR7/8 Agonist:
In December 2021, the Company announced initial first-in-human results from transcendIT-101, a Phase 1/2 study of TransCon TLR7/8 Agonist with or without pembrolizumab in patients with advanced or metastatic solid tumors, which demonstrated early signs of clinical activity in three out of three efficacy-evaluable cancer patients treated with TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab.
transcendIT-101 top-line data from monotherapy and combo-therapy dose escalation expected in the third quarter of 2022.
TransCon IL-2 β/γ
In the third quarter of 2021, the Company submitted an investigational new drug application and initiated IL-βelieγe ("I’ll Believe") Trial, a Phase 1/2 clinical trial to evaluate TransCon IL-2 β/γ in patients with advanced cancer.
Top-line monotherapy data from the IL-βelieγe Trial are expected in the fourth quarter of 2022.
TransCon TLR7/8 Agonist and TransCon IL-2 β/γ Combination:
The Company plans to submit an IND or similar for Phase 2 cohort expansion for TransCon TLR7/8 Agonist and TransCon IL-2 β/γ during the fourth quarter of 2022.
In November 2021, the Company’s shareholders elected Rafaèle Tordjman, Founder & CEO of Jeito Capital, to the board.
Ended 2021 with cash, cash equivalents, and marketable securities totaling €789.6 million.
Full Year 2021 Financial Results

Total revenue for 2021 was €7.8 million compared to €7.0 million in 2020. Revenues for 2021 include U.S. SKYTROFA sales, and license, clinical supply and services provided to third parties, primarily VISEN Pharmaceuticals.

Research and development (R&D) costs for 2021 were €295.9 million compared to €260.9 million in 2020. Higher R&D costs in 2021 reflect continued advancement of development programs and an increase in personnel-related costs to support them.

Selling, general, and administrative (SG&A) expenses for 2021 were €160.2 million compared to €76.7 million in 2020. Higher SG&A expenses were primarily due to an increase in commercial and administrative personnel as well as an increase in commercial and IT costs.

Net profit of associate was €12.0 million for 2021, compared to a net loss of €9.5 million for 2020. For 2021, the net profit of associate included a non-cash gain of €42.3 million as a result of a financing round in VISEN.

Net finance income was €55.8 million in 2021 compared to a net expense of €79.0 million in 2020.

For the full year 2021, Ascendis Pharma reported a net loss of €383.6 million, or €7.00 per share (basic and diluted) compared to a net loss of €419.0 million, or €8.28 per share (basic and diluted) for the same period in 2020.

As of December 31, 2021, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €789.6 million compared to €834.1 million as of December 31, 2020. As of December 31, 2021, Ascendis Pharma had 56,937,682 ordinary shares outstanding.

Conference Call and Webcast Information

Ascendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its full year 2021 financial results. Details include:

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

On March 2, 2022 Delcath Systems, Inc. (Nasdaq: DCTH), an interventional oncology company focused on the treatment of primary and metastatic cancers of the liver, reported that on February 28 its CHEMOSAT Hepatic Delivery System received Medical Device Regulation (MDR) certification under the new European Medical Devices Regulation [2017/745/EU]. Since April 2012, Chemosat had been certified under a previous regulation, the Medical Device Directive [2007/47/EC] (MDD), which is being replaced by MDR (Press release, Delcath Systems, MAR 2, 2022, View Source [SID1234609371]).

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Achieving MDR certification entails a detailed evaluation from a designated EU Notified Body, including an audit of quality systems and a review of documentation supporting safety and performance claims for the device. MDR greatly expands upon existing MDD requirements including the level of clinical evidence supporting claims, post-marketing surveillance, database traceability, unique device identification (UDI) and increased supply chain oversight. Under MDR, CHEMOSAT’s designation has changed from a Class IIb to a Class lll medical device.

On March 2, 2022 Amgen (NASDAQ:AMGN) reported that its Board of Directors declared a $1.94 per share dividend for the second quarter of 2022 (Press release, Amgen, MAR 2, 2022, View Source [SID1234609370]). The dividend will be paid on June 8, 2022, to all stockholders of record as of the close of business on May 17, 2022.

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On March 2, 2022 Prokarium, a biopharmaceutical company pioneering the oncology field of microbial immunotherapy, reported it exercised its option with Lausanne University Hospital (Centre Hospitalier Universitaire Vaudois — CHUV) for an exclusive, worldwide license for the use of Salmonella immunotherapy in the treatment of bladder cancer (Press release, Prokarium, MAR 2, 2022, View Source [SID1234609369]).

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"This intellectual property is an important milestone for our lead oncology program as it enters clinical development in 2023 and also lays the groundwork for expanding Salmonella immunotherapy into other solid tumors," said Livija Deban, PhD, Chief Scientific Officer of Prokarium. "Microbial immunotherapy is the gold standard-of-care in the treatment of bladder cancer. However, high recurrence rates and limited treatment options are currently available for patients. Prokarium is introducing a differentiated, efficacious microbial immunotherapy to a market that has seen little disruption in over 30 years."

In exchange for the exclusive license, Prokarium will make an upfront payment, as well as additional payments upon achievement of development and commercialization milestones, and, if any product is approved, will pay royalties on net sales.

"Since initiating our collaboration with Prokarium in 2019, our team at the Department of Urology at CHUV has generated a robust preclinical data package demonstrating that Prokarium’s proprietary Salmonella bacteria generates quantitatively and qualitatively superior immune responses with different kinetics compared to the standard-of-care," said Dr Denise Nardelli-Haefliger, Head of the Urology Research Unit. "Together with Prof Patrice Jichlinski, Prof Beat Roth, Dr Ilaria Lucca and Dr Sonia Domingos-Pereira, we are thrilled to see that the results of our collaboration have the potential to expand the disease-free interval and improve quality of life in patients with non-muscle invasive bladder cancer (NMIBC)."

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Bladder cancer represents 5% of all new cancer cases in the US and accounts for 550,000 new cases yearly worldwide. The clinical staging is determined by the depth of invasion into the bladder wall. More than 70% of cases are diagnosed at an early stage, also known as non-muscle invasive bladder Cancer (NMIBC). Despite early diagnosis, the only approved therapies are Bacillus Calmette-Guérin (BCG), which often faces shortages, and chemotherapy, both delivered intravesically. Because of its high incidence and the limited number of treatment options, a huge unmet medical need remains in NMIBC.

On March 2, 2022 Bristol-Myers Squibb Company (NYSE:BMY) ("Bristol Myers Squibb"), with its wholly-owned subsidiary Celgene Corporation ("Celgene") (collectively, the "Offerors"), reported the accepted amounts and pricing terms of their previously announced 22 separate offers to purchase for cash notes issued by the Offerors listed in the tables listed herein (Press release, Bristol-Myers Squibb, MAR 2, 2022, View Source [SID1234609368]).

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The outstanding debt securities listed in (i) the first table below labeled "2025 Pool" are referred to collectively as the "2025 Pool Notes," (ii) the second table below labeled "2026 Pool" are referred to collectively as the "2026 Pool Notes," (iii) the third table below labeled "2027 Pool" are referred to collectively as the "2027 Pool Notes," (iv) the fourth table below labeled "2029 Pool" are referred to collectively as the "2029 Pool Notes," and (v) the fifth table below labeled "High Coupon Pool" are referred to collectively as the "High Coupon Pool Notes." The High Coupon Pool Notes, the 2025 Pool Notes, the 2026 Pool Notes, the 2027 Pool Notes and the 2029 Pool Notes are referred to collectively as the "Notes," and each series of Notes is referred to as a "series." We refer to each offer to purchase a series of Notes for cash as an "Offer," the offers to purchase the 2025 Pool Notes collectively as the "2025 Pool Offers," the offers to purchase the 2026 Pool Notes collectively as the "2026 Pool Offers," the offers to purchase the 2027 Pool Notes collectively as the "2027 Pool Offers," the offers to purchase the 2029 Pool Notes collectively as the "2029 Pool Offers," the offers to purchase the High Coupon Pool Notes collectively as the "High Coupon Pool Offers," and all the offers to purchase Notes are referred to collectively as the "Offers."

The table below indicates, among other things, the aggregate principal amount of Notes tendered as of 5:00 p.m. (New York City time) on March 1, 2022 (the "Early Tender Deadline") and accepted in each Offer, the Offer Yield (each as defined below), the proration factor, if any, and the total consideration for each $1,000 principal amount of each series of Notes validly tendered at or prior to the Early Tender Deadline and accepted for purchase (the "Total Consideration"), as calculated at 9:00 a.m. (New York City time) today, March 2, 2022 in accordance with the terms of the Offer to Purchase dated February 15, 2022 (as it may be amended or supplemented from time to time, the "Offer to Purchase"), as amended by Bristol Myers Squibb’s press release dated March 1, 2022:

(1)

The "Offer Yield" is equal to the applicable Reference Yield (as defined in the Offer to Purchase), which is based on the bid-side price of the applicable Reference U.S. Treasury Security (as specified in the Offer to Purchase)) plus the applicable Fixed Spread (as specified in the Offer to Purchase).

(2)

Includes the Early Tender Premium (as defined in the Offer to Purchase). Payable per each $1,000 principal amount of each specified series of Notes validly tendered at or prior to the Early Tender Deadline and accepted for purchase.

*

Denotes a series of Notes for which the Total Consideration, the Tender Consideration (as defined in the Offer to Purchase), and the Offer Yield were determined taking into account the par call date, instead of the maturity date, of the Notes of such series in accordance with standard market practice.

The Offerors’ obligations to accept Notes tendered in the Offers are subject to the terms and conditions described in the Offer to Purchase, as amended by Bristol Myers Squibb’s press release dated March 1, 2022, which sets forth a detailed description of the Offers, including: (a) the Acceptance Priority Procedures (as defined in the Offer to Purchase), and (b) the following Amended Maximum Acceptance Amounts (as defined in the Offer to Purchase):

all of the 2025 Pool Notes tendered as of the Early Tender Deadline (the "2025 Pool Maximum");
$500 million maximum aggregate principal amount of the 2026 Pool Notes (the "2026 Pool Maximum");
all of the 2027 Pool Notes tendered as of the Early Tender Deadline (the "2027 Pool Maximum");
$1.6 billion maximum aggregate principal amount of the 2029 Pool Notes (the "2029 Pool Maximum"); and
$1.5 billion maximum aggregate principal amount of the High Coupon Pool Notes (the "High Coupon Pool Maximum").
The withdrawal rights for the Offers expired at 5:00 p.m. (New York City time) on March 1, 2022. The Offers will each expire at 11:59 p.m. (New York City time) on March 15, 2022, unless extended or earlier terminated by the Offerors (such date and time with respect to an Offer, as the same may be extended with respect to such Offer, the "Expiration Date").

The withdrawal rights for the Offers expired at 5:00 p.m. (New York City time) on March 1, 2022. As previously announced, all conditions of the Offers were deemed satisfied or waived by the Offerors by the Early Tender Deadline. As previously announced, the Offerors have elected to exercise their Early Settlement Right (as defined in the Offer to Purchase). The Early Settlement Date (as defined in the Offer to Purchase) will occur on March 4, 2022.

Since the aggregate principal amount of the 2025 Pool Notes, 2026 Pool Notes, 2027 Pool Notes, 2029 Pool Notes, and High Coupon Pool Notes validly tendered and not validly withdrawn at or prior to the Early Tender Deadline meets or exceeds the 2025 Pool Maximum, 2026 Pool Maximum, 2027 Pool Maximum, 2029 Pool Maximum, or High Coupon Pool Maximum, as applicable, the Offerors will accept such Notes for purchase according to the Acceptance Priority Procedures and proration steps as described in the Offer to Purchase. Holders of Notes (each, a "Holder" and collectively, "Holders") validly tendered at or prior to the Early Tender Deadline that are accepted for purchase by the Offerors will receive the applicable Total Consideration, in cash. Because the aggregate principal amount of Notes validly tendered and not validly withdrawn at or prior to the Early Tender Deadline exceeded the Amended Maximum Acceptance Amounts, there will be no Final Settlement Date (as defined in the Offer to Purchase) and no additional tenders of Notes will be accepted for purchase by the Offerors after the Early Tender Deadline. As described in the Offer to Purchase, Notes tendered and not accepted for purchase will be promptly returned to the tendering Holder’s account.

The Total Consideration that will be paid on the Early Tender Deadline for each series of notes accepted for purchase does not include a cash payment equal to accrued and unpaid interest (the "Accrued Coupon Payment") on such Notes to, but not including, the Early Settlement Date. For the avoidance of doubt, interest will cease to accrue on the Early Settlement Date for all Notes accepted in the Offers. Under no circumstances will any interest be payable to Holders because of any delay on the part of Global Bondholder Services Corporation, as the tender agent and information agent, The Depository Trust Company or any other party in the transmission of funds to Holders. See the Offer to Purchase for additional information.

All Notes accepted in the Offers will be cancelled and retired and will no longer remain outstanding obligations of the relevant Offeror. Following the Price Determination Date (as defined in the Offer to Purchase), the Offerors may elect to redeem all or a portion of Bristol Myers Squibb’s 3.875% Notes due 2025 or 3.200% Notes due 2026 or Celgene’s 3.875% Notes due 2025 that are not tendered and accepted in the Offers in accordance with the terms of the optional redemption provisions in the indentures governing such Notes.

The Offerors have retained Deutsche Bank Securities Inc., BofA Securities, Inc. and Goldman Sachs & Co. LLC as dealer managers for the Offers. Questions regarding terms and conditions of the Offers should be directed to Deutsche Bank Securities Inc. at (866) 627-0391 (toll-free) or (212) 250-2955 (collect) or BofA Securities, Inc. at (888) 292-0070 (toll-free) or (980) 683-3215 (collect) or Goldman Sachs & Co. LLC at (800) 828-3182 (toll-free) or (212) 902-5962 (collect). Global Bondholder Services Corporation is acting as the tender agent and the information agent for the Offers (the "Tender and Information Agent").

Offer and Distribution Restrictions

This announcement is for informational purposes only. This announcement is not an offer to sell or purchase, a solicitation of an offer to sell or purchase, or the solicitation of tenders with respect to any of Notes described herein. The Offers are being made solely pursuant to the Offer to Purchase. The Offers are not being made to Holders of Notes in any jurisdiction in which the making or acceptance thereof would not be in compliance with the securities, blue sky or other laws of such jurisdiction. In any jurisdiction in which the securities laws or blue sky laws require the Offers to be made by a licensed broker or dealer, the Offers will be deemed to be made on behalf of the Offerors by the dealer managers or one or more registered brokers or dealers that are licensed under the laws of such jurisdiction.

United Kingdom

The Offer to Purchase is only addressed to Holders where they would (if they were clients of the Offerors) be per se professional clients or per se eligible counterparties of the Offerors within the meaning of the rules of the Financial Conduct Authority (" FCA "). Neither the Offer to Purchase nor any other related documents or materials are addressed to or directed at any persons who would be retail clients within the meaning of the FCA rules and any such persons should not act or rely on them. Recipients of the Offer to Purchase and any other documents or materials relating to the Offer should note that the Offerors is acting on its own account in relation to the Tender Offer and will not be responsible to any other person for providing the protections which would be afforded to clients of the Offerors or for providing advice in relation to the Offer.

This announcement, the Offer to Purchase and any other documents or materials relating to the Tender Offer are not being made and such documents have not been approved by an authorized person for the purposes of section 21 of the Financial Services and Markets Act 2000. Accordingly, such documents and/or materials are not being distributed to, and must not be passed on to, the general public in the United Kingdom. The communication of such documents and/or materials as a financial promotion is only being made to persons outside the United Kingdom and to those persons in the United Kingdom falling within the definition of investment professionals (as defined by Article 19(5) of the Financial Services and Markets Act 2000 (Financial Promotion) Order 2005 (" Financial Promotion Order ")) or persons who are within Article 43 of the Financial Promotion Order or any other persons to whom they may otherwise lawfully be communicated under the Financial Promotion Order and should not be relied on or acted on in the United Kingdom by any other persons.

EEA

In the EEA, this announcement and the Tender Offer will not, directly or indirectly, be made to, or for the account of, any person other than to qualified investors within the meaning of Article 2(e) of the Prospectus Regulation. Neither this announcement nor the Offer to Purchase, nor any other documentation or material relating to the Tender Offer, has been or will be submitted to a competent authority in the EEA for approval. Therefore, neither the Offer to Purchase nor any other documentation or material relating to the Tender Offer qualifies as an approved prospectus as meant in Article 6 of the Prospectus Regulation.

Accordingly, in the EEA, the Tender Offer may not be made by way of an "offer of securities to the public" within the meaning of Article 2(d) of the Prospectus Regulation and the Offer may not be promoted and is not being made to, any person in the EEA (with the exception of "qualified investors" within the meaning of Article 2(e) in conjunction with Article 1(4)(a) of the Prospectus Regulation). This announcement, the Offer to Purchase and any other documentation or materials relating to the Tender Offer (including memoranda, information circulars, brochures or similar documents) have not been forwarded or made available to, and are not being forwarded or made available to, directly or indirectly, any such person.

With regard to the EEA, this announcement and the Offer to Purchase have been transmitted only for personal use by the aforementioned qualified investors and only for the purpose of the Tender Offer. Accordingly, the information contained in this announcement and the Offer to Purchase may not be used for any other purpose or be transmitted to any other person in the EEA.