drugs in the pipeline

8/20/2019 Drugs in the Pipeline

1/19

Director, Pediatric Programs

National Press FoundationDecember 2, 2015Cape Town, South Arica

Pro!ress in"lobal T#Dru!De$elopment

Cherise P% Scott, Ph%D%, &P'


2/19

2

(ntroduction o Child)

•

T# is a si!ni+cant cause o deathamon! children children aresusceptible to the most se$ere andatal orms o T#

• (n 201-, an estimated 1 million

children became ill with T# and 1-0000 children died o T#

• Children with T# ha$e beenhistoricall* ne!lected . man* !oundia!nosed and untreated

• Child health and sur$i$al isimpro$in!, with ewer deaths romdiseases such as '(/ andpneumonia% #ut T# lin!ers, and lacsattention and resources dedicated tothe problem

Children with T# are the ne!lected o the ne!lected

Childhood T# 'idin! in the shadows


3/19

• (n 2010, 3'4 re$ised dosin! !uidelines or T# dru!s or childrenbased on e$idence that children were not recei$in! enou!h T#medicine

*depending on setting and type of disease

• The !uidance and polic* chan!ed, but the products a$ailable ortreatment o children did not chan!e

• &an* pro$iders must crush or chop a$ailable tablets to achie$e

&aret unresponsi$e to call or new treatments

New !uidelines, old dru!s

Drug Previous WHOGuidance

Current WHOGuidance

Dose and ran!em!6! bod* wei!ht7 Dose and ran!e m!6!bod* wei!ht7

(sonia8id 5 -)97 10 :)157

;iampicin 10 20 15)257 20 15)257


4/19

-


Deli$erin! current treatment is comple? and

burdensome

Challen!es o administerin! treatment

Dosing chart for pediatricTB therapy in the

Philippines. Treatmentadministered via syrups.

Dosing chart for pediatric TBtherapy in South Africa.

Treatment administered via pills.

Pharmacist preparing syrups ofRifampicin for pediatric TB

treatment in Thailand.


5/19

5

/alue o impro$ed, child)riendl* FDCs


6/19

9

Child)riendl* T# medicines in the correctdoses are now a$ailable and can impro$e

adherence and child sur$i$al rom T#

Ad$ocate or Access@

Countries must re!ister and adopt theseproducts immediatel*


7/19:

EVERYCHILD

HASACCESSTO THE

NEWPRODCT

ADVOCATES) =ncoura!e *our

countr* to re!ister andprocure new products

CO!!NITY " CARE GIVERS

) As about a$ailabilit* o newproducts

) earn about T# and its

s*mptoms in children

GOVERN!ENTS"HEALTHPROGRA!S

) Buanti* child T# in countr*

) (mplement current !uidelines

) Scale)up contact in$esti!ation

) Fast)trac product re!istration) (nclude Child T# in #ud!et and

"rants

DONORS

) Fund new products

) ) Fund scale up oeecti$e inter$entions

or child T#

Please si!n the Call to Action and show *our support or

a T#)ree Future b* $isitin! tballiance%or!6si!nthecall

Call to Action

http://www.tballiance.org/signthecallhttp://www.tballiance.org/signthecallhttp://www.tballiance.org/signthecallhttp://www.tballiance.org/signthecall


8/19<

T# Alliance 4$er$iew

• Dedicated to the disco$er* and

de$elopment o better, asterand aordable T# dru!s that area$ailable to all who need them

• Achie$in! maximum impact will

reuire – Short, simple re!imen that is

aordable, a$ailable andaccessible

– Eni$ersal re!imen that is eecti$e

in $irtuall* all people with acti$e T#, erasin! the distinctions o &D;) T# or GD;)T#

– All no$el dru!s, circum$entin!challen!es o resistance to someantibiotics a$ailable toda*

ur s on e er e c nes orAll


9/19H

"lobal T# Dru! Pipeline


10/19

10


T# Alliance is searchin! or the best combinations o no$el dru!s

Acceleratin! Pro!ress From Dru!s to ;e!imens

•

T# must be treated with multi)dru! combinations to pre$ent thede$elopment o resistance

• Toda*Is pipeline o T# dru!s can be tested to!ether, speedin!de$elopment o no$el T# re!imens and reducin! ;JD romdecades to *ears


11/19

Disco$er* and De$elopment Process

T# Dru!6;e!imen De$elopment

Dru!Candidate Pool

Phase (( → Phase (((Sin!le CompoundPreclinical

De$elopment→ Phase ( → =#ACompound 1

Compound -

Compound

Compound 5

Compound 2

Regimen Identifcationin Mice

;e!imen #

(denti+cation o New Dru!Candidates

Selection o Potential New;e!imens

;e!imen C

;e!imen A

Disco$er*

11


12/19

12


• GD;)T# could be a deathsentence% There are no eecti$etreatments% Sal$a!e therap* canlast 2 *ears

• aunched &a* 2015, Ni?)T# is

the +rst clinical trial o an GD;) T# re!imen with minimal pre)e?istin! resistance

• Tests a dru! combination as a 9)H month cure that is all pills, no

inKections

• First participant with GD;)T#completed si?)month stud*treatment in No$ember

•

;e!imen could be +rstLuni$ersalM treatment i sae

Ni?)T# Clinical Trial

A #$%& Lancet stud' (ound near)'

*a)( o( +DR,T- .atients died /it*ina 'ear o( diagnosis0


13/19

1


Ni?)T# Clinical Trial


14/19

1-

"lobal T# Dru! Pipeline


15/19

15


• T-A,12& (nitiated sin!le ascendin! dosePhase 1 stud*% First new dru! to enter theclinic in si? *ears

• O3a4o)idinone and Diar')5uino)ine candidates to mo$e into pre)clinical

de$elopment in 2019

4ptimi8in! nowncompound classes

• D.rE,% in*i6itor 7A8,919%: No$elbactericidal compound with no pre)e?istin!resistance is appro$ed to mo$e into pre)clinical de$elopment

• 2 ead 4ptimi8ation and 2 ead(denti+cation pro!rams initiated

De$elopin! no$elclasses based on

no$el tar!ets

ar *) a!e esearc n! ePipeline

T# Alliance is now a partner in the T# Dru! Accelerator ProKect

T# Alliance le$era!es industr* and other partners to support thecontinued !rowth o the !lobal T# dru! pipeline% &4;= new

dru!s are needed


16/19


17/19

1:


• Please attend (mpro$in! Access to AppropriateChildhood T# &edicines uncheon S*mposium,December , 2015, 11-5)1-00, The Table #a* 'otel,

The #allroom

• /isit us at the T# Alliance booth• Come help us paint a mural or a LT# Free Future%M

Dedication on Frida* at 1215 pm% ocationConerence center, ((4 Ha)) &-

• /isit us online at tballiance%or!

earn more


18/19

1<


T# Alliance Supporters

#ill J &elinda"ates

Foundation

=uropeanCommission

Enited StatesFood and Dru!Administration

(rish Aid ES N(A(D

EQ aidEnited States A!enc*

or (nternationalDe$elopment

"lobal 'ealth(nno$ati$e Technolo!*

Fund

EN(TA(D

AustralianDepartment o

Forei!n Aairs and

Trade )ndonesia ealth

+und


19/19

Than *ou@

drugs in the pipeline

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