Ascendis Pharma A/S : TransCon PTH PaTH Forward Trial 58-Week OLE Clinical Update Presentation

Ascendis Pharma A/S

TransConTM PTH - 58-WeekOpen-Label

Extension (OLE) Update

May 10, 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 business strategy, prospective products, 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, timing and likelihood of success, plans and objectives of management for future operations and future results of current and anticipated products 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 March 10, 2021 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 TransCon are trademarks owned by the Ascendis Pharma Group. © May 2021

2 Ascendis Pharma A/S.

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

Preliminary PaTH Forward Open-Label Extension (OLE) Data at 58 Weeks

• 58 subjects continue in the open-label extension beyond 58 weeks*
• Continued treatment with TransCon PTH demonstrated that:
• • 91% of subjects were off standard of care therapy**
  • Urinary calcium maintained in the normal range
  • Bone markers trended towards the mid-normal levels
  • Quality of life benefits measured by SF-36 continued within normal range
• TransCon PTH was well-tolerated at all doses administered
• • No treatment-related serious or severe adverse events occurred, and no treatment-emergent adverse events (TEAEs) led to discontinuation of study drug
  • No change to the safety profile in the OLE portion of the study

Preliminary data support TransCon PTH as a potential hormone replacement therapy for adult HP

Preliminary PaTH Forward OLE week 58 data from live database snapshot. Data on file. *As of May 7, 2021.

3 **Not taking active vitamin D and taking ≤600 mg/day of calcium supplements.

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

PaTH Forward OLE Mean Active Vitamin D Dose

	1.8	OLE starts											TransCon PTH
	1.6
												Placebo
Mean	1.4												All TransCon PTH
1.2
Active
Vitamin D	1.0
Dose	0.8
µg/day	0.6
(± SE)
0.4
	0.2
	0.0
	0	4	8	12	16	20	24	28	32	36	40	44	48	52	56	60
								Weeks

TransCon PTH enabled discontinuation of active vitamin D within two weeks of treatment initiation

4 Preliminary PaTH Forward OLE week 58 data from live database snapshot. Data on file.

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

PaTH Forward OLE Mean Calcium Supplement Dose

	3,000	OLE starts											TransCon PTH
	2,500												Placebo
													All TransCon PTH
Mean	2,000
Calcium	1,500
Dose
mg/day	1,000
(± SE)
	500
	0
	0	4	8	12	16	20	24	28	32	36	40	44	48	52	56	60
								Weeks

TransCon PTH enabled rapid and continuous calcium supplement reduction;

40 of 58 subjects were taking 0 mg, and 53 of 58 subjects were taking 0 to 600 mg at week 58

5 Preliminary PaTH Forward OLE week 58 data from live database snapshot. Data on file.

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