Ascendis Pharma A/S : 39th Annual J.P. Morgan Healthcare Conference Presentation

Ascendis Pharma A/S

39th Annual J.P. Morgan Healthcare Conference January 11, 2021

		All product candidates are investigational. For investor communication
1		only. Not for use in promotion or product commercialisation.

Cautionary Note On Forward-Looking Statements

This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, such as statements regarding our future results of operations and financial position, including our business strategy, prospective products, availability of funding, clinical trial results, product approvals and regulatory pathways, collaborations, licensing or other arrangements, the scope, progress, results and costs of developing our product candidates or any other future product candidates, the potential market size and size of the potential patient populations for our product candidates, timing and likelihood of success, plans and objectives of management for future operations, the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates, future results of current and anticipated products, and the future operations of VISEN Pharmaceuticals are forward-looking statements. These forward-looking statements are based on our current expectations and beliefs, as well as assumptions concerning future events. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the results discussed in the forward- looking statements. These risks, uncertainties and other factors are more fully described in our reports filed with or submitted to the Securities and Exchange Commission, including, without limitation, our most recent Annual Report on Form 20-F filed with the SEC on April 3, 2020 particularly in the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". In light of the significant uncertainties in our forward-looking statements, you should not place undue reliance on these statements or regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified timeframe, or at all.

Any forward-looking statement made by us in this presentation speaks only as of the date of this presentation and represents our estimates and assumptions only as of the date of this presentation. Except as required by law, we assume no obligation to update these statements publicly, whether as a result of new information, future events, changed circumstances or otherwise after the date of this presentation.

This presentation concerns product candidates that are or have been under clinical investigation and which have not yet been approved for marketing by the U.S. Food and Drug Administration, European Medicines Agency or other foreign regulatory authorities. These product candidates are currently limited by U.S. Federal law to investigational use, and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated.

	Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and	All product candidates are investigational. For investor communication
	TransCon are trademarks owned by the Ascendis Pharma Group. © January 2021
2	Ascendis Pharma A/S.	only. Not for use in promotion or product commercialisation.

Company Overview

• Create best-in-class products addressing unmet medical needs by applying TransCon™ technologies to parent drugs with clinical proof-of-concept or clinically validated pathways
• Endocrinology rare disease
• • TransCon hGH:
  • • Pediatric growth hormone deficiency (GHD): BLA and MAA submitted, phase 3 trials in China1 ongoing and Japan initiated
    • Adult GHD: Global phase 3 foresiGHt Trial ongoing
  • TransCon PTH: Adult hypoparathyroidism (HP) phase 3 PaTHway Trial in North America and Europe ongoing
  • TransCon CNP: Achondroplasia phase 2 trials: ACcomplisH Trial ongoing and ACcomplisH China Trial1 initiated
• Oncology
• • TransCon TLR7/8 Agonist: IND filed
  • TransCon IL-2β/γ: IND filing or similar expected in Q3 2021
• As of September 30, 2020, cash, cash equivalents and marketable securities of €957.5 million

	1 Conducted by VISEN Pharmaceuticals.		All product candidates are investigational. For investor communication
3	BLA = Biologics License Application. MAA = Marketing Authorisation Application.		only. Not for use in promotion or product commercialisation.

Vision 3x3: Building a Leading BioPharma Company

Our Goal is to Achieve Sustainable Growth through Multiple Approaches

• Obtain regulatory approval for three independent Endocrinology Rare Disease products
• • TransCon hGH for pediatric growth hormone deficiency
  • TransCon PTH for adult hypoparathyroidism
  • TransCon CNP for achondroplasia
• Grow Endocrinology Rare Disease pipeline through

• • Global clinical reach
  • Pursuing 9 total indications, label optimization, and life cycle management
  • New endocrinology products
• Establish global commercial presence for our Endocrinology Rare Disease area

• • Build integrated commercial organization in North America and select European countries
  • Establish global commercial presence through partners with local expertise and infrastructure
• Advance a high value oncology pipeline with one IND or similar filing each year
• Create a third independent therapeutic area with a diversified pipeline

		All product candidates are investigational. For investor communication
4		only. Not for use in promotion or product commercialisation.