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Diurnal Group plc (AIM: DNL)
A revenue generating European biotech
02 March 2021Date of preparation March 2021 Ref: CORP-GB-0107191
DisclaimerThis presentation does not constitute or form part of an offer or invitation to acquire, purchase, subscribe for, sell or otherwise dispose of, or issue, or any
solicitation of any offer to acquire, purchase, subscribe for, sell or otherwise dispose of, or issue, any shares or other securities of Diurnal Group plc
(“Diurnal”) or to engage in any other investment activities.
The information set out herein may be subject to updating, completion, revision and amendment and such information may change materially. None of
Diurnal, its advisers nor any other person, representative or employee undertakes any obligation to update any of the information contained herein. No
representation or warranty, express or implied, is or will be made by Diurnal, its advisers or any other person as to the accuracy, completeness or fairness
of the information or opinions contained in this presentation and any reliance you place on them will be at your sole risk. Without prejudice to the
foregoing, none of Diurnal, its associates, its advisers nor its representatives accept any liability whatsoever for any loss howsoever arising, directly or
indirectly, from the use of this presentation or its contents or otherwise arising in connection therewith.
The information in this presentation has not been independently verified and it may contain forward-looking statements that may or may not prove
accurate. For example, statements regarding expected revenue growth, trading margins and market trends are forward-looking statements. Phrases such
as "aim", "plan", "intend", "anticipate", "well-placed", "believe", "estimate", "expect", "target", "consider" and similar expressions are generally intended
to identify forward-looking statements. Forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause
actual results to differ materially from what is expressed or implied by the statements. Any forward-looking statement is based on information available
to Diurnal as of the date of the statement. All written or oral forward-looking statements attributable to Diurnal are qualified by this caution. Diurnal does
not undertake any obligation to update or revise any forward-looking statement to reflect any change in circumstances or in Diurnal’s expectations.
No statement in this presentation is intended as a profit forecast or profit estimate.
Past performance is no guide to future performance and persons needing advice should consult an independent financial adviser.
2
3
Company snapshot
Martin Whitaker PhD
Chief Executive Officer
• 20 years working in pharma and biotech
Sam Williams PhD
Interim Chairman
• 20 years working in biotech
Richard Bungay
Chief Financial Officer
• 25 years in senior financial positions
Richard Ross MD
Chief Scientific Officer
• 30 years in senior medical positions
▪ IPO on AIM 24 Dec 2015 144p
▪ Share price (25/02/21) 58p Market Cap (25/02/21) £80m
▪ Cash position (31/12/20) £20.3m No debt
Investment summary
▪ Revenue generating business, initially targeting >$3bn market in cortisol deficiency
▪ First product, Alkindi® (hydrocortisone granules in capsules for opening), launched and generating revenues in Europe (year ended 30 June 2020 £2.4m); approved in Israel, Australia and US; US sales commenced in Q4 2020
▪ Second product, Chronocort® (modified release hydrocortisone), submitted for EMA and MHRA review with approval in Q1 2021(E)
▪ Commercial exclusivity until 2034 – orphan drug, regulatory and patents1
▪ Earlier pipeline maturing; our third product, DITEST™ (oral native testosterone) in clinical development targeting the ca. $4.8bn male hypogonadism market with abbreviated (“505(b)(2)”) route to US approval confirmed
▪ Company has direct sales force in key territories in Europe; potential to leverage through in-licensing agreements; forging commercial partnerships globally
4
Market opportunity numbers based on Datamonitor Report (2015) with exchange rate of £:$1.301. Conditional and subject to grant of market authorisation (and that Diurnal is the first sponsor to obtain market authorisation for the relevant product and
demonstrating significant clinical benefit), on grant of pending patents and ongoing grant
On track to becoming a world-leading endocrinology specialty pharma company focused on ca. $9.5bn market opportunity
To become a world leading endocrinology specialty pharma company
• Establish commercial traction via lead product candidates• Continue to develop specialist products and bring them to market• Potential for targeted M&A to accelerate and leverage international profile
5
The vision
6
The opportunity
Addressing significant unmet medical needs
Endocrine disorders:
▪ Chronic diseases
▪ Life-long treatment
▪ Serious health impact
Pituitary
Thyroid
Adrenal
Gonads
7
A collection of glands that produce hormones to regulate the body – including metabolism, growth and development, tissue and sexual function, reproduction, sleep and behaviour
The endocrine system is critical to life
Note: diagram shows the Company’s view of dominant areas of focus within the endocrinology market
Large pharma Large specialty pharma Small niche endocrinology
Eli Lilly
SanofiNovo Nordisk Pfizer
NovartisAbbott Ipsen FerringEndoCorcept
Strongbridge
Chiasma
HRA Pharma
Diurnal is seeking to build a commercial franchise in niche areas of unmet patient need
8Source: Company estimates based on publicly available information.
Adrenas
Spruce BioscienceNeurocrine
Alkindi® Chronocort®
Life-long
Licensed & measured dosing Matching circadian rhythm
Building a life-long “Adrenal Franchise”
9
Cortisol – an essential hormoneCortisol deficiency results in fatigue, depression and death through adrenal crisis
Target indications Key products
Adrenal Insufficiency (AI): Patients lose the ability to produce cortisol leading to insufficient cortisol production
Alkindi® (hydrocortisone granules in capsules for opening): Approved by the EMA to treat children <18 years in Europe; approved by the FDA to treat children <17 years in the US
Congenital Adrenal Hyperplasia (CAH): Patients are born without an enzyme that stimulates the adrenal glands to release the hormone cortisol
Chronocort® (modified-release hydrocortisone): Aims to be the first hydrocortisone product that closely mimics cortisol circadian rhythm in adults and adolescents
Diurnal aims to have two products licensed in Europe in a >$2bn* market with ca. 10 years’ exclusivity by end 2023
*Market opportunity numbers based on Datamonitor Report (2015) with exchange rate of £:$1.30
10
Alkindi® (AI & CAH children)
The first product specifically designed for children suffering from AI and CAH
Chronocort® (CAH and AI adults)
Aims to be the first hydrocortisone product that mimics cortisol
circadian rhythm in adults and adolescents
2
1
Pharmacy compounded adult tablets 25% out of specification1
Formulations that are
challenging to administer
Imprecise, inaccurate and highly variable
dosing that risks over- or under-
treatment
Unpalatable, bitter taste
Alkindi®
Chronocort®
Current unlicensed treatments have significant draw backs
2
Alkindi®: A major breakthroughin paediatric AI (including CAH)
111. Out of European Pharmacopeial specification: Neumann et al (2017)
Approved Products
12
Alkindi®
Alkindi® Sprinkle
▪ $66m market opportunity2
▪ Approved May 2018
▪ Children < 18 years old
▪ Hydrocortisone granules in capsules for opening
▪ >$100m market opportunity1
▪ Approved September 2020
▪ Children < 17 years old
1. Based on Eton Pharmaceuticals’ estimates for Alkindi® Sprinkle2. Based on Datamonitor Report (2015) with exchange rate of £:$1.30
Alkindi®
▪ Approved Israel: August 2020
― Children < 18 years old
▪ Approved Australia: August 2020
― No age restriction
Alkindi®Generating revenues with significant exclusivities
131. Alkindi® UK market packs
1
▪ Authorised by the European Medicines Agency in 2018 with 10 years data and market exclusivity (PUMA)
▪ Successfully launched in: UK, Germany, Austria, Sweden, Denmark, Norway, Iceland and Italy
▪ Pricing agreed in: the Netherlands
▪ Market access plans in place for a timely launch across key European territories ― The same commercial infrastructure can be used for future Diurnal products
▪ Distribution agreements in place outside core territories― Frost Pharma (Nordics); Consilient / Goodlife (Benelux); EffRx (Switzerland)
US:
▪ New Drug Application (NDA) approved by the FDA on 29 September 2020
▪ Alkindi® Sprinkle available to US patients in November 2020 through
licensing partner Eton Pharmaceuticals
▪ Eton Q4 2020 earnings update expected during March 2021
RoW:
▪ Alkindi® Sprinkle license extended to Canada with Eton
▪ Alkindi® license for China with Citrine Medicine
▪ Alkindi® marketing and distribution agreement in Turkey with Er-Kim1
14
Establishing a global product presenceRoW
Alkindi®
1. Marketing and distribution agreement with Er-Kim also includes Chronocort® (subject to approved in Europe)
Chronocort®: targeting effective disease control in adults
15Source: Debono et al. JCEM (2009); Mallappa et al JCEM (2014) & company data on file; BID, twice daily; TID, thrice daily
Cortisol circadian rhythm
Current hydrocortisone TID
Chronocort® BID “Toothbrush regimen”
0
100
200
300
400
500
600
700
800
Seru
m C
ort
iso
l n
mo
l/l
23:00 03:00 07:00 11:00 15:00 19:00 23:00Time (24 hour clock)
Early morning rise in cortisol
Too muchdrug
Avoids troughs in Cortisol
10mg HC 5mg HC 2.5mg HC
20mg Chronocort® 10mg Chronocort®
$0.4bn* market opportunity (Europe and US) for Congenital Adrenal Hyperplasia$2.8bn* market opportunity (Europe and US) for Adrenal Insufficiency
Chronocort® – innovative drug delivery solution:▪ Delayed release coat allows pH triggered
release in GI tract
Chronocort® – improved disease control:▪ Control of morning 17-OHP
― 94% of patients vs 31% on standard treatment in Phase 2 trial
Alkindi®
Chronocort®
* Based on Datamonitor Report (2015) with exchange rate of £:$1.30
MAA Europe: In final stages of regulatory review
▪ European Medicines Agency (EMA)
― Positive scientific advice meeting with EMA in March 2019
― Marketing Authorisation Application (MAA) submitted December 2019
― MAA validated by EMA in April 2020
― Day 120 questions answered and submitted by Diurnal to EMA on time during November 2020
― Day 180 questions answered and submitted by Diurnal to EMA on time during February 2021
― Anticipated CHMP recommendation for approval in late March 2021
▪ UK Medicines and Healthcare products Regulatory Agency (MHRA)
― MAA submitted in early January 2021 following end of Brexit Transition period
― Anticipated opinion for approval during same timeframe as for the EMA
▪ Data from pivotal DIUR-005 study and DIUR-006 safety extension study published inJournal of Clinical Endocrinology and Metabolism during February 2021
16
MAA based on largest interventional CAH study ever completed and compelling long-term data from safety extension study
Chronocort®
Generating favourable long-term outcomes data
▪ 81% of patients remained in the study at April 2020 data cut
▪ 80% of subjects still have controlled 09:00 hours 17-OHP after 18 months of thesafety extension study (compared to 52% at baseline)
▪ Importantly, this androgen control continues on a lower dose of steroid than inDIUR-005 and lower than in the literature with a median daily hydrocortisone doseof 20mg after 18 months of the safety extension study
▪ New episodes of unexpected therapeutic benefit reported in both studies:
― Restoration of menses in 8 patients (one on standard therapy)
― 3 patient pregnancies and 4 patient partner pregnancies (none on standard therapy)
▪ Adrenal crises with Chronocort® occur at a similar / lower rate than in the literature
▪ Adverse events consistent with the known safety profile of hydrocortisone
▪ No significant changes in secondary endpoints; trends favourable
▪ Data will be used to support pricing and reimbursement discussions
17Source: Adapted from Merke et al. JCEM (2021)
Chronocort®
Anticipated key value inflection points
18
Estimated Approval
Phase III1 (DIUR-014)
Chronocort®
(CAH)
Chronocort®
(CAH)
Chronocort®
(AI)
Phase II (DIUR-010)
Addressable market
Next steps2020 2021 2022 2023 2024
> $1.2bn
Estimated Approval
NDA
CAH MAA Under Reviewby EMA and MHRA
Study Report
Chronocort®
(AI)
DIUR-016
Source: Company estimates, market estimates based on Datamonitor Report (2015), pricing from British National Formulary (No. 76) and GBP:1.30 USD exchange rate1. Subject to further partnering or funding
Phase III1 (DIUR-015)
Line Extension to AI MAA
Estimated Approval
SPA
> $2.1bn
Chronocort®
19
DITEST™
Aims to be the first oral native testosterone product
for male hypogonadism
Targeting a US market of ca. $4.8bn*
3
*Based on Datamonitor Report (2015) with exchange rate of £:$1.30
Positive clinical proof-of-concept
20
Positive headline results from Phase I patient clinical trial
▪ Phase I proof-of-concept study evaluated the pharmacokinetics, safety and tolerability of DITEST™ in 24 adult men with primary or secondary hypogonadism compared to oral modified testosterone (testosterone undecanoate)
▪ Primary endpoint met:
― DITEST™ achieved testosterone levels within the healthy young male adult normal range
― These testosterone levels were less variable than testosterone undecanoate
▪ Secondary endpoints demonstrated:
― DITEST ™ can be taken either with food or in the fasted state, representing a major difference with testosterone undecanoate
― No serious adverse events in the DITEST™ arm of the study
― Levels of the potent testosterone derived androgen, dihydrotestosterone (DHT), were lower than with testosterone undecanoate
DITEST™
Abbreviated 505(b)(2) route to New Drug Application submission
21
▪ Positive pre-IND Meeting with FDA― A pre-Investigational New Drug (IND) meeting was successfully concluded with the FDA in June
2020 which confirmed that DITEST™ can progress to an NDA via the abbreviated 505(b)(2) route
▪ On track to IND submission during Q2 2021― IND package preparation, including non-clinical studies, in progress
― Manufacturing is located entirely within the US
― IND submission anticipated during Q2 2021 to allow a Phase 1 multiple ascending dose study to progress in the US; CRO appointed
▪ Commercial preparations in progress― US pricing and market activities ongoing to allow commercial preparations to begin
Phase 32MAD1 Study
Next steps2020 2021 2022 2023 2024
End-of-Phase 2 Meeting with FDA
OpenIND
Study ReportNon-Clinical
1. Multiple Ascending Dose study 2. Subject to further FDA approval and to partnering or further funding
DITEST™
Indications
Annual Addressable
Market (Europe & US)
Product
Thyroid Hypothyroidism $1bn ▪ Modified-release T3 (preclinical)
AdrenalCAH
AICushing’s
$0.5bn$2.8bn$0.5bn2
▪ Alkindi® (completed)▪ Chronocort® (Phase 3)▪ Oligonucleotide therapy (preclinical)
Gonads Hypogonadism $4.8bn▪ DITEST™ (positive Phase 1 study in
hypogonadal subjects)
Pipeline – a ca. $9.5bn market opportunity leading to strategic opportunities
Building an earlier stage pipeline
22
Source: Company estimates based on Datamonitor Report (2015), pricing from British National Formulary and exchange rate of £:$1.301. Indicates areas of potential focus beyond the Company’s existing pipeline2. Includes Cushing’s syndrome and disease
In-licensing
M&A
Partnerships
Summary and outlook
23
Continuing to meet key milestones
Conclude US partnering discussions for Alkindi® (Chronocort®) H1 2020✓
Confirmation of regulatory pathway for DITEST™ from FDA Mid-2020✓
Approval of Alkindi® in Australia Mid-2020✓
Approval of Alkindi® in Israel Mid-2020✓
Approval of Alkindi® Sprinkle in US 29 Sep 2020✓
Outlook for 2021
Agree Special Protocol Assessment (SPA) for Chronocort® in US Q1 2021
Recommendation for approval of Chronocort® in Europe Q1 2021
Submission of DITEST™ IND in US Q2 2021
Start DITEST™ Multiple Ascending Dose Study in US Q3 2021
First commercial Chronocort® launch in Europe 1 Q3 2021
Start Chronocort Phase 3 Study in US 2 H2 2021
1. Subject to regulatory approval by the EMA2. Subject to agreeing SPA with FDA and further partnering or funding
Strong team with ability to deliver
Opportunities to broaden the offering
Credible market access
strategy
Strong base position in
orphan diseases
Building a global endocrinology
specialty pharma
24
Appendix
25
Diurnal – Company history
26
2004Diurnal founded as a spin-out from the University of Sheffield
2005
Diurnal gains EU orphan drug designation for Chronocort® for Congenital Adrenal Hyperplasia (CAH)
2013Successful completion of Phase I Alkindi® trials in adults, enabling the Company to progress to Phase III paediatric trials
Chronocort® Phase II trial begins in adult CAH patients at the National Institutes of Health (US)
2014Positive results from the Chronocort® Phase II trial published in Journal of Clinical Endocrinology and Metabolism
2014-15Diurnal secures further institutional funding to initiate Phase III registration trials of both Alkindi® and Chronocort® in Europe and to further strengthen the management team
2004 - 08Chronocort® intellectual property licensed to Phoqus plc
2007EU orphan drug designation approved for Chronocort® for Adrenal Insufficiency (AI)
2008 -12Chronocort® licence repurchased from Phoqus plc1 administrators
Institutional shareholder base established and partners engaged to develop Chronocort®
New Chronocort® formulation Phase I trials successfully completed
2004 Present
2015Chronocort® gains US orphan drug designation for both AI & CAH.
Alkindi® gains US orphan drug designation for paediatric AI
Diurnal Group plc lists on AIM as DNL.L
2017Alkindi® receives positive opinion for approval from European Medicines Agency
Alkindi® and Chronocort® patents granted in the US
2016Positive results from Alkindi® Phase III trial announced
First patient dosed in Chronocort® Phase III European trial
First patient dosed in testosterone Phase I proof-of-concept trial
2018Alkindi® receives market authorisation approval from European Commission
Alkindi® launched in Germany and UK – first revenues generated
1. Phoqus plc’s version of Chronocort® was different to Diurnal’s new version of Chronocort® due to: i) a different formulation; ii) a different manufacturing process which could not be scaled; iii) a different dosing regimen (once daily); and iv) being in tablet rather than capsule form
Marketing authorisation and launch of Alkindi® in Europe and US and submission of Chronocort® MAA in Europe achieved since IPO
2019Alkindi® NDA submitted in US
Chronocort® MAA submitted in Europe
Positive headline results from DITEST™ testosterone Phase I proof-of-concept trial announced
2020Alkindi® approved by FDA and launched by partner Eton in the US
Alkindi® approved in Israel and Australia
Positive pre-IND meeting with the FDA confirms 505(b)(2) route to NDA for DITEST ™
Experienced leadership team
27
Martin Whitaker PhD
Chief Executive Officer
Sam Williams
Interim Chairman
• Over 20 years working in biotech
• Previous experience as a top-ranked equity analyst and subsequently, as an entrepreneur and CEO
• Managing Partner of Life Sciences at IP Group plc and Non-Executive Chairman and/or Director for several portfolio companies
• 20 years working in pharma and biotech
• Previous positions at Critical Pharmaceuticals, Pfizer
• Founder at D3 Pharma which successfully commercialised Plenachol®, a high dose Vitamin D product
• Over 25 years in senior financial positions
• Non-Executive Director at Cambridge Cognition
• Previous CFO positions at Mereo Biopharma, Glide Technologies, Verona Pharma and Chroma Therapeutics
• Previous positions at Celltech and AstraZeneca
Richard Ross MD
Chief Scientific Officer
• Over 30 years in senior medical positions
• Diurnal founder, world leading endocrinologist & industry key opinion leader
• Professor of Endocrinology at the University of Sheffield
• Recognised by the Endocrine Society’s 2021 Laureate Awards for Outstanding Innovation
Richard Bungay
Chief Financial Officer
Key investors
28
ShareholderAs at 30 November 2020
Number of shares % holding
IP Group 44,085,999 31.9%
Polar Capital 12,613,378 9.1%
Finance Wales 11,534,888 8.3%
Amati Global Investors 9,500,000 6.9%
Chelverton Asset Management 5,800,000 4.2%
BGF 4,400,000 3.2%
Walker Crips 4,237,910 3.1%
Management and Board 3,687,490 2.7%
Others 42,469,274 30.6%
Total 138,328,939 100.0%
Endocrine focussed development pipeline
29
In Progress / Completed Line Extension Key:
Source: Company market estimates based on Datamonitor Report (2015), pricing from British National Formulary 76 and GBP:1.30 USD exchange rate
Annual Addressable
Market (Europe & US)
Estimated
Approval
$1,033mTBC
$463mTBC
$4,830mTBC
2025
Therapy Area
Cushing’s DiseaseOligo-nucleotide
T3 modified release
DITEST™
$351m2021
2025
$2,767m2023TBC
Congenital Adrenal Hyperplasia (CAH) (Adult)
Chronocort®
Classical Hypogonadism
Hypothyroidism (T4 non-responders)
Adrenal Insufficiency (AI) (Adult)
MAA /
NDAPIIPIPre- PIII
Robust product protection in key markets
30
Intellectual property
Alkindi®
Chronocort®
Oral Native Testosterone
DITEST™
USEuropean
Patent
Regulatory exclusivity
USEU
Orphan 7 years1
Orphan 7 years1
Orphan 10 years1
PUMA
10 years
Hypogonadism not an orphan
disease
2034
2032
Composition of matter
Method of treatment (x2)
Composition of matter
Medical use
2034
2033
Composition of matterComposition
of matter & medical use
2030Composition of matter2029
Composition of matter
2034
1. Conditional and subject to grant of market authorisation (and that Diurnal is the first sponsor to obtain market authorisation for the relevant product) and on demonstrating significant benefit;
2. GB patent application PCT/EP2021/050930
Under review2
Medical use
2032
2034
2033
Hypogonadism not an orphan
disease
The cortisol deficiency market opportunity
31
Prevalence (Estimated no. of patients) Total Addressable Market Size1
Total Total
Paediatric AI(inc CAH)
10,268 4,126 14,394 $63m $100m2 $163m
CAH 40,970 16,231 57,201 $251m $100m $351m
AI 297,159 153,604 450,763 $1,824m $943m $2,767m
Total 348,397 173,961 522,358 $2,138m $1,143m $3,281m
Source: Datamonitor Healthcare Consulting, 20151. Based on Plenadren® price point of approximately $6,138 per patient per annum 2. Based on Eton Pharmaceuticals estimates
Competitive landscape; differentiated products
32
ProductMimics
circadian rhythm
IndicationCountries PriceAI/CAH
paediatricCAH AI
Hydrocortisone(generic)
✓Manipulated
✓ ✓ ca. $3k p.a.
Plenadren®(modified release)
Not Licensed
Not Licensed ✓1
ca. $6k p.a.
Diurnal“Adrenal Franchise”
✓2
Chronocort®
✓Alkindi®
✓2
Chronocort®
✓2
Chronocort®
Targeting Chronocort®
$6k+ p.a.
▪ Generic hydrocortisone provides no circadian release & achieves poor disease control
▪ Plenadren® is a modified release hydrocortisone, does not provide overnight coverage of cortisol and has not demonstrated disease control in congenital adrenal hyperplasia
▪ There are a handful of competitor products vying for exclusive marketing rights in CAH in the US –these products are designed to reduce androgen levels but not to replace cortisol:
1. Over 18 years in Europe only 2. PlannedSource: British National Formulary (No. 76); Han et al (2014); EMA Assessment Report for Plenadren® (21 July 2011)
Chronocort®Diurnal
CrinecerfontNeurocrine Biosciences
TildacerfontSpruce Biosciences
BBP-631Adrenas Therapeutics
Phase II completed in USPhase III completed in Europe
Phase II completed in USRecruiting to Phase III trial in adults
Phase IIa pediatric trial ongoing Recruiting to Phase IIb trial in adults
Pre-clinical
Pivotal study DIUR-005 summary
▪ Evidence of better biochemical control with Chronocort® with lower 17-OHP SDS at 4weeks (p=0.007) and 12 weeks (p=0.019), and between 07:00 to 15:00 hours (p=0.044) at24 weeks compared to standard of care
▪ 91% of subjects had controlled 09:00 hours serum 17-OHP (<1,200 ng/dl) at 24 weekswith Chronocort® compared 71% (p=0.002) to standard of care (and 52% at baseline)
▪ 17-OHP control achieved with lower median daily hydrocortisone dose of 30mg(compared to 31mg for the standard of care)
▪ No adrenal crises in the Chronocort® arm of the study (compared to 3 adrenal crises inthe standard of care)
33
Chronocort®
Source: Adapted from Merke et al. JCEM (2021)
34
15:00 19:00 23:00 03:00 07:00 11:00 15:000
20
40
60
80
0
500
1000
1500
2000
2500
17
-OH
P n
mo
l/l 1
7-O
HP
ng
/dl
Baseline on standard GC
Chronocort at 24 weeks
Achieved better hormonal control at 24 weeks compared to baseline
Source: Adapted from Merke et al. JCEM (2021)
REFERENCE RANGE
Chronocort®
35
15:00 19:00 23:00 03:00 07:00 11:00 15:000
10
20
30
0
200
400
600
800
1000
24 hour clock
17
-OH
P n
mo
l/l 1
7-O
HP
ng
/dl
Chronocort at 24 weeks
Standard GC at 24 weeks
Source: Adapted from Merke et al. JCEM (2021)
Achieved significantly better control of 17-OHP in the period 07:00-15:00
REFERENCE RANGE
Chronocort®
3 6 9 12 15 180
10
20
30
40
50
0
500
1000
1500
Months on MR-HC
09
:00
h 1
7-O
HP
nm
ol/
l 09
:00
h 1
7-O
HP
ng
/dl
Baseline
36
Median Dose (mg)
Achieved sustained control of androgens for 18 months
Chronocort® achieved sustained control of androgens for 18 months on lower doses of steroid than normally used to treat Congenital Adrenal Hyperplasia
30 26 25 20 20 20
Chronocort®
Source: Adapted from Merke et al. JCEM (2021)
REFERENCE RANGE
Favourable competitive landscape
37
Product Company Active ingredient Delivery Dosing frequency
DITEST™ Diurnal TestosteroneOral
(softgel capsule)Expected twice-daily
Androgel® Abbvie (+ generics) Testosterone Gel Once-daily
Jatenzo® Clarus TherapeuticsTestosterone undecanoate
Oral (softgel capsule)
Twice-daily
Xyosted® Antares PharmaTestosterone
enanthateSC Injection Weekly
Aveed® ENDOTestosterone undecanoate
IM Injection 4 – 10 Weekly
Natesto® Acerus Testosterone Nasal Three times daily
Androderm® Abbvie TestosteronePatch
(transdermal)Once-daily
▪ DITEST™ is expected to have a competitive profile compared to legacy testosterone replacement therapy products and recent market entrants
DITEST™
Selected historical financial information1
381. Half year results to 31 December 2020
£000 6 months to31 Dec 2020
6 months to31 Dec 2019
Revenue 1,214 1,147
Cost of sales (347) (303)
Gross profit 867 844
Research and development expenditure (2,633) (2,388)
Selling and distribution expenses (2,457) (1,977)
Administrative expenses (1,629) (1,054)
Other gains - net 590 -
Operating loss (5,262) (4,575)
Net financial income 56 31
Taxation 545 509
Loss for the period (4,661) (4,035)
Net cash used in operating activities (4,169) (4,957)
Net cash generated by financing activities 9,136 68
Closing cash and cash equivalents 20,344 4,625
Selected historical financial information1
391. Half year results to 31 December 2020
£000 31 Dec 2020 30 Jun 2020 31 Dec 2019
Non current assets 2,434 1,770 144
Inventories 1,666 1,241 764
R&D tax credit receivable 542 1,194 2,614
Trade and other receivables 1,378 1,337 1,410
Cash and cash equivalents 20,344 15,434 4,625
Trade and other payables (3,225) (2,591) (2,124)
Net assets 23,139 18,385 7,433
Share capital and premium 66,185 57,049 46,476
Group reconstruction reserve (2,943) (2,943) (2,943)
Accumulated losses (40,103) (35,721) (36,100)
Total equity 23,139 18,385 7,433