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IMS COM PANY PRO FILES NOVARTIS
Clin i cal Data: In De cem ber 2008 Novartis re ported the first re sults from the TRANS FORMS phase III
trial which showed that FTY 720 had su pe rior ef fi cacy to a cur rent stan dard of care for pa tients with re -
laps ing mul ti ple scle ro sis. Pa tients on oral FTY 720 ex pe ri enced sig nif i cantly fewer re lapses than those
treated with the in ject able med i cine in ter feron beta-1a and the study met its pri mary end point for both
doses of FTY 720. The an nu al ized re lapse rate at one year for pa tients given FTY 720 0.5 mg was 0.16,
rep re sent ing a 52% re duc tion com pared to a re lapse rate of 0.33 for in ter feron beta-1a. The FTY 720
1.25 mg dose also showed a sig nif i cant re duc tion in re lapses with a rate of 0.20 rep re sent ing a 38% re -
duc tion against in ter feron beta-1a. No sta tis ti cally sig nif i cant dif fer ence was seen be tween the two
FTY720 doses. Novartis noted “We are en cour aged by the early re sults from TRANS FORMS, which rep re -
sent a ma jor step to wards de liv er ing an ef fec tive oral treat ment for peo ple with re laps ing-re mit ting MS.”
The one year head-to-head trial TRANS FORMS (TRial As sess ing in ject able in ter feroN vS FTY720 Oral in
RrMS) is the first of three stud ies to re port re sults in one of the larg est phase III clin i cal pro grams ever
con ducted in MS, in volv ing more than 3,400 pa tients around the world. Re sults from the FREE DOMS and
FREE DOMS II phase III tri als are ex pected at the end 2009 and 2011, re spec tively. Results from the
INFORMS phase III trial, in patients with primary progressive MS, are expected in 2013.
Com pe ti tion: An oral treat ment for MS would make a ma jor dif fer ence to treat ing the dis ease. Novartis
says that sec ond to lim ited ef fi cacy, the need to in ject daily is one of the main ob sta cles to ini ti at ing and
main tain ing MS ther apy. Fear of nee dles, in con ve nience and in jec tion site re ac tions are all as so ci ated
with non-com pli ance. For this rea son, other com pa nies are also de vel op ing oral treat ments. An other
lead ing com pany in the field of MS, Merck KGaA has an oral ver sion of cladribine, Mylinax (li censed
from Ivax, now part of Teva), in phase III tri als and is also study ing other novel oral treat ments for MS.
Novartis’ de lay in start ing phase III tri als, orig i nally due to be gin in 2005, means Mylinax has the lead on
fingolimod. Mylinax en tered a two-year phase III trial in 2005. Merck Serono, the pharma arm of Merck
KGaA, plans to sub mit oral cladribine tab lets for EMEA and FDA approval in 2009/2010 for the treatment
of RMS.
Sales/An a lyst Com ment: Mor gan Stan ley an a lysts (De cem ber 2008) noted that they viewed the re -
sults from the TRANS FORMS study as pos i tive, since ef fi cacy far ex ceeded their ex pec ta tions. How ever,
they do note that there are clearly is sues re lated to tox ic ity, with two fa tal her pes in fec tions and three
cases of mel a noma, but in their opin ion ef fi cacy su pe rior to the cur rent stan dard of care com bined with an
ad verse event pro file that ap pears com pa ra ble to Tysabri means that FTY 720 stands a good chance of
gain ing FDA ap proval. As the first oral agent for MS they be lieve it could be ca pa ble of achiev ing rev e nues
of more than $2 bil lion, but con sen sus re sides in the $500 mil lion to $1 bil lion range. “As a once-daily pill
for a fa tigued pa tient pop u la tion cur rently de pend ent on in ject able ther a pies, there is likely to be sig nif i -
cant pa tient de mand, as sum ing the ad verse event pro file is rea son able,” note the an a lysts. The an a lysts’
ma jor con cern is the mel a noma ad verse events, but they note that the longer term FREEDOMS study
reporting in 2009 could better characterize this risk.
Cowen & Co an a lysts (Au gust 2008) fore cast ini tial sales of $50 mil lion in 2010 ($450 mil lion in 2013).
They are of the opin ion that adop tion will be slow be cause of safety con cerns.
MIDOSTAURIN (PKC 412), a pro tein kinase C in hib i tor and an an a logue of staurosporine, en tered piv -
otal phase III tri als in the sec ond quar ter of 2008. The trial is eval u at ing the po ten tial sur vival ben e fit of
midoastaurin in com bi na tion with che mo ther apy com pared to the use of che mo ther apy alone in treat ing
pa tients with acute myeloid leu ke mia (AML). Stud ies have also been re ported in co lon, lung and breast
can cer and CLL. It may also have po ten tial in di a betic retinopathy and other ischemic retinopathies,
choroidal neovascularization due to macular de gen er a tion, oc u lar histoplasmosis and other dis eases.
Stud ies have dem on strated that midostaurin is able to com bat re sis tant tu mors, and it could be an al ter -
na tive to Glivec (imatinib) in GIST pa tients or used in com bi na tion with it. Novartis forecasts filing for
AML in 2011.
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