Oncopeptides : Citi’s 15th Annual BioPharma Virtual Conference – Presentation

THE TRANSFORMATION OF ONCOPEPTIDES

Citi's 15th Annual BioPharma Virtual Conference

MARTY J DUVALL

Chief Executive Officer, CEO

JAKOB LINDBERG

Chief Scientific Officer, CSO

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TRANSFORMATION INTO A FULLY INTEGRATED BIOPHARMA COMPANY

GROWTH STRATEGY SUPPORTED BY STRATEGIC INITIATIVES

Discovery and IND

generation

• Unique proprietary PDC platform

Portfolio Development and Life Cycle			Launch investment and
Management			geographic expansion
• Initial focus on $ 23 B MM market with a broad		•	Management team strengthened
supportive clinical program		• Ramping up for US launch around
• Priority review of first-in class RRMM drug		•	year-end
melflufen ongoing		Cash position ~180 MUSD after

landmark directed share issue

3

MARTY J DUVALL

PROFESSIONAL EXPERIENCE

• Executive Leadership experience from public and private companies; CEO, CCO, SVP, Global Commercial and Marketing roles
• Pharma and biotech experience across geographies; Aventis (Sanofi), MGI (Eisai), Abraxis (Celgene), Merck (MSD), ARIAD (Takeda) and Tocagen (Forte)
• Broad and deep oncology experience including; hematology (e.g. MDS, CTCL, CML, AML, MM, etc.), and solid tumors (e.g. breast, lung, prostate, H/N, gastric, GBM, etc.), biologics, small molecules, gene therapy, supportive care
• Launch experience; Taxotere (US, Europe and Asia), Abraxane (China), Dacogen (US and Europe), Sylatron (Global), Iclusig (US, Europe, and Asia) and Alunbrig

4

MELFLUFEN IS A FIRST IN CLASS PRODUCT

GENERATED FROM OUR PDC PLATFORM

Melflufen is a first in class peptide-drug

conjugate (PDC) that targets

aminopeptidases and rapidly releases

alkylating agents into tumor cells

5

RECENT EVENTS

VALUE GENERATION AND RISK REDUCTION

6

REGULATORY TIMELINES

NDA REVIEW PROCESS FOR MELFLUFEN

•

•

•

•

•

US FDA Submission* - June 30

Priority Review ** - August 29

No ODAC committee planned

90-day Safety Update - September

PDUFA Date - February 28th, 2021

• Evaluation underway for regulatory timelines in other key geographies **Mid-Cycle Review Meeting in October is driving our "launch readiness" work in order to be ready in November

7

MELFLUFEN CLINICAL DEVELOPMENT PROGRAM

FULL CLINICAL DEVELOPMENT PROGRAM IN RRMM

O-12-M1

HORIZON

OCEAN

ANCHOR

Phase 1

Phase 2

BRIDGE

AL-AMYLOIDOSIS

LIGHTHOUSE

PORT

2016

2017

2018

2019

2020

2021

2022

Phase 1 and 2:

Phase 2:

Phase 3:

Phase 1 and 2:

Phase 2:

Phase 1 and 2:

Phase 3:

Phase 2:

single-arm study (O-

single-arm study in

randomized head-

triple-combination

study in renally

single-arm study in

randomized

open-label,

12-M1)

late stage RRMM

to-head study vs

study (ANCHOR)

impaired patients

AL-amyloidosis

daratumumab

randomized, cross-

(HORIZON)

pomalidomide

(BRIDGE)

combination study

over study (PORT)

(OCEAN)

(LIGHTHOUSE)

The arrows show estimated Last Patient In, in the studies

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COMPETITIVE MELFLUFEN DATA

TRIPLE CLASS REFRACTORY MULTIPLE MYELOMA PATIENTS

	Melflufen		Xpovio		Blenrep
	Oncopeptides		Karyopharm		GSK
	US NDA, June 30, 2020	US approval, July 2019		US Approval, Aug 6, 2020
Number of patients studied	119		122		95
Overall Response/Clinical Benefit Rate	26%/39%		25%/39%		31%/36%*
mDOR / mPFS responders	5.5m / 8.5m		3.8m / 4.0m		NR/NR
Progression-free survival	3.9 months		3.7 months		2.8 months*
Overall survival	11.2 months		8.0 months		14.9m*
Share of patients with EMD	42%		22%		20%*
Serious Adverse Event Rate	51%		58%		40%
Non-hematologic toxicity (grade 3/4)	Pneumonia	9%	Fatigue	25%	Keratopathy	44%
reported in >5% of patients			Hyponatremia	20%	Decreased Visual
			Nausea	10%	Acuity	28%
			Pneumonia	9%	Pneumonia	7%
			Diarrhea	7%	Pyrexia	6%
			Sepsis	6%
			Hypokalemia	6%
			Mental status	6%
			General det.	6%

9 Source: Submission for melflufen, FDA Label documents for Xpovio and Blenrep (items marked with '*' is data from DREAMM-2 as published in Lancet).

ENROLLMENT COMPLETED IN PHASE 3 OCEAN STUDY

495 PATIENTS RECRUITED - TOP-LINE RESULTS IN H1 2021

Head to head study versus standard of care

N = 495

Lenalidomid-

refractory multiple myeloma patients

Randomization

Melflufen +		Primary
dexamethason		endpoint:
		PFS
		Secondary
Pomalidomid +
	endpoint:
dexamethason		ORR, OS

RRMM data from pomalidomide FDA label and O-12-M1 study

Treatment	ORR	CBR	Median PFS	Median DOR	Median OS
Melflufen + Dexamethason	31%	49%	5.7 months	8.8 months	20.7 months
Pomalidomid + Dexamethason	24%	NR	3.6 months	7.0 months	12.4 months

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NEWER PRODUCTS USED IN ON TOP OF OLD AS SURVIVAL IMPROVES

NEED OF NEW MoA's

US MM # of Total Patients by Product

70000

60000

50000

40000

30000

20000

10000

0 Jul-17Oct-17Jan-18Apr-18Jul-18Oct-18Jan-19Apr-19Jul-19Oct-19Jan-20Apr-20

Revlimid

Velcade

Darzalex

Pomalyst

Kyprolis

Ninlaro

Cytoxan

Empliciti

melphalan

Farydak

Xpovio

Source: Intrinsiq MAT, Jun 2020

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SIGNIFICANT OPPORTUNITIES BASED ON DEVELOPMENT PROGRAM

US MARKET

New data to drive label expansion

3x RRMM

20,000+

1-2x RRMM	1-2x RRMM
Single drug use	Comb. use
25,000	20,000+

EMD/High-risk

18,000

Anticipated label in triple-class refractory patients

Head-to-head superiority study with the most used regimen in RRMM

Combination with PI or anti-CD38 opens up 2L+ combination treatment

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PAVING THE WAY FOR A SUCCESSFUL LAUNCH

GLOBAL ORGANIZATION WITH SIGNIFICANT LAUNCH EXPERIENCE

Mohamed Ladha, General Manager US Business Unit

17 years of industry experience with extensive oncology and launch expertise

Led/built commercial functions at 7 pharma or biotech companies for in-line/ launch products

Sarah Donovan, Head of Marketing

20 years of industry experience in sales, analytics; patient advocacy, US and Global Marketing , 10 years of experience in oncology

Led and built marketing functions for launches and inline brands

Paula O'Connor, MD, Head of Medical Affairs US

17 years industry experience with 30 years oncology experience

Led Clin Dev programs at 3 companies and established Med Affairs at 3 companies

An accomplished US Medical Affairs and Commercial Team with nearly 100 oncology product launches

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PASSIONATE TO MAKE A DIFFERENCE FOR PATIENTS

BUILDING A PATIENT FOCUSED ORGANIZATION

National Medical

Accounts & Science

Reimburs. Liaisons

(~5) (~10)

Oncology

Account (~40) Patient (~10) Nurse

ManagersEducators

(~5)	(~2)
Area	Key Customer
Business
Marketers
Directors

"Ensuring that every patient who

potentially could benefit from melflufen gains access"

Marty J Duvall

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DISEASE AWARENESS AND EDUCATION

PAVE THE WAY FOR A NEW CLASS OF DRUG

15

MELFLUFEN AWARENESS CONTINUES TO INCREASE IN THE US

MOMENTUM IS GROWING FOLLOWING SCIENTIFIC MEETINGS

Awareness of Melflufen in the US

50%

20%

10%

% MDs Likely or Very Likely to use Product

X by line of therapy

(7-point scale from "Very Unlikely" to "Very Likely")

53%

49%

38%

38%

20%

11%

EHA 2019 (Jun 2019) ASH 2019 (Dec 2019) EHA 2020 (Jun 2020)

2L

3L

4L

5L

3x refractory EMD

Source: US physician market research, n=100

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CONTINUOUS NEWSFLOW

MAJOR EVENTS OVER THE NEXT 12 MONTHS

Q2 2020		Q3 2020

Q4 2020	H1 2021

EHA data update

First patient in

Potential accelerated

Top-line results

Amyloidosis study

First patient in PORT

approval in US	OCEAN
Potential launch in	Last patient in

NDA submission

study

the US

ANCHOR

Priority review -

PDUFA date

First patient in sEAPort

program (US)

ASH data update	Last patient in
BRIDGE
	EHA data update

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18

MELFLUFEN AND THE

PDC PLATFORM

JAKOB LINDBERG

Chief Scientific Officer

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MELFLUFEN - A FIRST IN CLASS DRUG CANDIDATE

A PEPTIDE-DRUG CONJUGATE TARGETING AMINOPEPTIDASES

Amino-peptidase	Alkylating payload
binding domain

AminoAmino Alkylating

AcidAcidpayload

• Increased potency of linked toxin due to aminopeptidase targeting with subsequent hydrolysis
• Potency increase over the course of disease, i.e. with degree of malignancy
• Circumvent significant amount of transport associated resistance development
• Circumvent significant amount of programmed cell-death related resistance developed, e.g. p53 deletion or mutation
• Aminopeptidase targeting enables additional

beneficial activity to direct cytotoxic effect, e.g. anti-angiogenesis and metastatic process

20

AMINOPEPTIDASES ARE EXCELLENT CANCER TARGETS

KEY ROLE IN CANCER CELL SURVIVAL, PROLIFERATION AND MIGRATION

Amino-peptidases play a key role in protein homeostasis, and in other critical functions such as cell-cycle progression, programmed cell death and cell migration

1. Amino-peptidasesare over-expressed in cancer cells

II

Amino-peptidase expression is increased between diagnosis and relapse in patient cancer samples

III

Amino-peptidase expression correlates with mutational burden and poor clinical outcome

21 Note: Structure from Kochan et al, PNAS 108 (19): 7745-50.

PDC PLATFORM

THERAPEUTIC ACTIVITY IN MOST CANCERS

PDC Potentiation

• Melflufen is focused on multiple myeloma and AL-amyloidosis

• New molecules are based on PDC platform

• Potential broadening of indications in AML, Non-Hodgkin Lymphoma and breast cancer

22

PEPTIDE DRUG CONJUGATE TECHNOLOGY

VERSATILE PLATFORM WITH MULTIPLE VENUES FOR FUTURE DEVELOPMENT

Amino	Amino	Toxic	•	Alternate toxic payload
• Alternate reactivity of payload
Acid	Acid	payload
			•	Change membrane
				permeability of payload

• Modify amino-peptidase binding domain to alter
  specificity for different amino-peptidases

23

PDC PIPELINE

FROM PRE-CLINICAL TO CLINICAL DEVELOPMENT 2020/21

EXPLORATORY	LATE PRECLINICAL	PHASE 1	PHASE 2	PHASE 3	REGISTRATION	MARKET
DEVELOPMENT	DEVELOPMENT

Melflufen

OPD 5

OPS 2

• OPD5 - High-dose treatment in i.e. bone-marrow transplantation ready for clinical development late 2020
• OPS2 - Second generation PDC candidate with alkylating payload potentially ready for clinical development in 2021

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FINAL DATA IN TRIPLE CLASS REFRACTORY MULTIPLE MYELOMA

INDEPENDENT REVIEW COMMITTEE DATA

Primary End-Point	Investigator	IRC Data	Incl. unconfirmed
	Assisted Data	Jan14th	responses Jan 14th
	Jan 14th
Overall Response Rate (ORR) - ITT n=157	29%	30%	31% (inv. and IRC)
ORR - 3x RRMM n=119	26%	26%	27% (inv. and IRC)
ORR - EMD n=55	24%	27%	NA

Note: Two unconfirmed responses on January 14th have later been confirmed.

Safety profile demonstrates that hematological toxicities were common but manageable, and non- hematological toxicities were infrequent

25

STRONG ACTIVITY IN HIGHLY REFRACTORY MM PATIENTS

RESPONDING PATIENTS PROGRESSION FREE FOR 8.5 MONTHS

26

27

FINANCIAL RESULTS H1 2020

WELL FINANCED WITH MANY DEVELOPMENT COMMITMENTS

Operating Costs Jan-Jun

800

700

87,2

600

	500	148,9
M
400
SEK

300 27,4

44,3

200	441,4

100 239,4

G&A

M&S

R&D

• Operating loss increased to SEK 696.2 M (loss:305.6)
• • R&D increase primarily due to increase in clinical & drug supply: SEK 332.5 M (193.9)
  • • OCEAN SEK 177.2 M (110.7)
  • Build-upof commercial and medical affairs explains increase in M&S
• Cash flow from operating activities neg. SEK 598.5 M (neg. 265.8)
• Cash position was SEK 937.8 M (626.8) as of Jun 30, 2020
• • Directed share issue raising SEK 1,413.9 M before issue costs of SEK 85.2 M in May 2020 closed in two steps in May and July

• Second step of SEK 673.5 M after issue costs not included in cash

0

as of Jun. 30

20192020

28