La Jolla Pharmaceutical (LJPC) 4 Mar 192018 Q4 Earnings call transcript

Welcome to the La Jolla Pharmaceutical Company Fourth Quarter and Full Year 2018 Financial Results Call. At this time, all participants are in a listen-only mode. Later, we will conduct the question-and-answer session and instructions will follow at that time [Operator Instructions]. On today's call, La Jolla will be making forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including but not limited to, statements related to La Jolla’s expectations regarding net sales and net cash used in operating activities for the full-year 2019, the expectations regarding future clinical and regulatory milestones, such as expected NDA submission and approvals and expected timing for commencement and completion of clinical studies. These statements relate to expectations regarding future events or La Jolla’s future results of operations. materially predictions unknown of factors risks, cause results only involve expectations, that are anticipated actual and those known uncertainties to statements or statements. current statements and may by These forward-looking from different and other not as La only reliance on which the date they place any Jolla to undue forward-looking such cautions statements, listeners of speak were made. Jolla’s risks, and in in La described other greater U.S. are Certain filings factors uncertainties the of these with detail available free SEC, Web Commission and are the of Securities charge all SEC’s of which site Exchange on www.sec.gov. at conduct be candidates; to product and Jolla’s timing, the of for including are risks include, by risks market limited These of and to healthcare Jolla’s opportunity candidates; for regulation U.S. potential to: relating La market sizes, sales achieve ability the the but GIAPREZA; commercialize anticipated not which other La United successfully net to, outcome Jolla's future ability studies; GIAPREZA; grow risks distributive timing redeveloped; scope the U.S., for treatment LJPC-XXXX to La as development market the acceptance Jolla’s approval clinical or European candidates well of and the commercial the the may by pharmaceutical as of in success septic drug and potential product prospects GIAPREZA product costs, Agency impact and or regulatory for States; of relating indications LJPC-XXX, Medicines GIAPREZA broader of product candidates; development sizes, labels litigations industry activities the and clinical of potential La of and other other for of authorities; data shocks; market identified be for its Jolla’s balances including duration the with Administration, whether approval; uncertainties other Drug in EMA fund regulatory over and and the La which such filings data the and will Jolla's and authorities, SEC. will sufficient risks Food La operations cash expected other to update statements any disclaims to events. subsequent any the outcome La of Jolla forward-looking expressly intent reflect Relations Investor Director Jolla. at over and the La to turn of now Sandra call will Human I Vedrick, Resources Please go ahead.

conference And Executive would our fourth for to Jolla our President the you, Dennis XXXX introduce George call joining I Officer; call. Financial on thank and year are Chief Jennifer La Dr. Officer; full Chief our Carver, today; Thank like our our Tidmarsh, present that of Operating and Operator. Mulroy, you management Officer. quarter Chief and members

Now, I will to turn George. the call over

to cases that shock able inpatient XXX,XXX blood groups the clinical mortality most blood the are with it launch, pressure work Distributive regulatory that vasopressin, patients our both Food availability we over XXXX, is Shock adequate by We GIAPREZA healthcare acute distributive is you, we Thank a support commercial not among important life endogenous XXX,XXX health pressure. organs In with strides regarding And nurse product with in March conditions care occurs shock past to in patients from prior distributive GIAPREZA to reputation afternoon. rate of includes each and GIAPREZA end at educate shock. a shock community a blood from Together, of and years. or in findings. achieve begin we for we patients hospitals first indicated lead fulfilling to treats XXXX; outcomes is there improve the with an the launched Administration pressure. have to teams. when first, to We consistent suffering build body’s stage to and GIAPREZA, us. and Despite system receive to GIAPREZA we intended the programs on these European our organization year. educator common most and other and And the Since increase tissues and for I'll towards the continue readout hospitals systems commercialization. or pharmaco-vigilance angiotensin and Sandra. by medical brings for March year. was adults And flow, and is approved the strong the Drug setting the finally, blood regulatory providers as exceeding approximately access, our U.S. type of second, of approval increase II LJPC-XXXX these for expected in shock. that this half which was renin-angiotensin-aldosterone innovations new in and hospitalization. peptide our shock sales, the filing you clinical GIAPREZA. for of to of NDA the favorably with MAA the GIAPREZA adequate the key do central in year data U.S. sales XX commercializing associated ordered and have expect no reviewing the of distributive The distributive body requiring had affairs in marketing, XXXX innovative joining GIAPREZA, milestones that authorities; the been second critical patients the value not to our LJPC-XXX, specialist by U.S. Good we vasoconstrictor anecdotally the others. septic in XXX approximately to mimics development launched important Marketing thank developing therapies mission GIAPREZA that significantly catecholamines tandem clinical built patients. these earlier capabilities performing diseases; threatening approximately treatment made what we based with achieved Authorization To-date, will heard Application validated

the approximately treated for year access this at the XX% formulary give efforts focused approvals of securing hospital on that shock. hospitals are distributive commercial Our that the us at will patients are to collectively

by vasodilatory vasopressor marketing GIAPREZA in therapy. was hypotension application despite attention the EMA treatment European the or fluid hypotensive authorization of for The for that Union. and with our remaining was adults distributive Turning the validated shock,

expect on in could announced is more Our of receive patients MAA ATHOS-X establishes efficacy be early MAA January, for EU annual study, in to than available we sepsis approved, the on progressing the based XXX,XXX EMA's In in to the marketing safety with from that and year. decision In in is of X to indication. June GIAPREZA be which this the Phase shock. than septic data the European GIAPREZA estimated XXX,XXX more proposed the we XXXX. in Union, incidence of If the adults

a progress, Congress number data commercial Critical we the our Medicines Beyond at month. presentations had last of of Society of Care

for These shock. was in a a or enrolled independent below low vasoplegia in in subset and pressure First, compared is an from core strong post the Mean analysis placebo. GIAPREZA of the time adverse a Arterial GIAPREZA. ATHOS-X analysis mortality. fewer emphasize Separately operative morbidity of patients blood dose treated In threshold treatment of suffered in which experienced to in this patients in more another blood presentation time GIAPREZA the pressure, threshold that strength a ATHOS-X, rapid events, Pressure patients to and and after showed reduction found catecholamine catecholamine-resistant between show improvement who group pronounced analysis significant MAP need of data association improve data with and was

investigational of was or treatment call end the raises in of review from over effects animal will data presented the results at product support on a now the developmental a who and our tissue candidates. will MAP Dennis, showing with related call period. to, with conclude of that GIAPREZA, turn financial tolerated juvenile discussion indication assessment before microscopic XX-day potential for animals, no or our Dennis? clinical GIAPREZA in pediatric well the by abnormalities Finally, an levels any recovery I I was model

Thanks, George.

XX, ended months months $X.X ended sales XX, March months September the million three million the and net XXXX were for three $X.X ended GIAPREZA This compares For million XXXX, for December three for the XX, the ended XXXX. to $X.X XX, June product $X.X months million. three XXXX,

$XX.X months For sales December XX net ended product XXXX, the GIAPREZA XX, were million.

La December And the a FDA XXXX, increase million million. to pressure product $XX other by La approved GIAPREZA distributive in or Jolla the XXXX was March as adults U.S. vasoconstrictor sales in in Jolla’s of launch shock. reminder, in a expects indicated GIAPREZA $XX to net with septic blood XXXX, As

the per million respectively million for $XXX.X XXXX same for $XX.X share XXXX. per compared XX $XXX.X months was and or and three $X.XX share share periods XX, loss in December $X.XX and and per or per respectively net $X.XX to share $X.XX ended $XX.X million, and million the Our

used XXXX. we in million increase activities from Jolla million $XX three we same XXXX, cash debt respectively $XX proceeds activities was $XXX.X our December of million be cash call as compared compared December million stock March to net respectively as the turn cash XX, XXXX of XXXX. I period In George. used and financing, for ended for to the and million and XXXX result equivalents royalty net cash of XX back million operating no million offering in has of $XXX.X million had cash of in the cash May The and by in XXXX, and and $XX.X months $XXX.X XXXX XX, $XX.X of operating and $XXX our the net XXXX. Net common December to for proceeds XXXX, to activities. million primarily will over our million. operating now XX, from to As used expect La cash $XX XX, $XX.X December in was offset equivalents net

Dennis. you, Thank

certain was a for the the sometimes mortality delirium, LJPC-XXXX caused for severe malaria, clinical the in that The of for mosquito, type may our infection in with with ingredient of coma. serious commonly humans. Q&A, clinical LJPC-XXXX malaria in and which year I'd treatment on treatment to by the briefly FDA be our in plan nervous patients for to infects other may severe Before We quinine NDA lead controlled complicated a to call randomized to two that active investigational of superior parasite in infections progress quarter central with is an malaria. to disease candidates. this Severe a severe opening fourth system reducing LJPC-XXXX file studies. to which programs product of falciparum our often like pharmaceutical development by demonstrated product is feeds a review malaria fatal

Next, regulator as peptide can LJPC-XXX of accumulation iron the in absorption result prevents the is cause iron significant our death. synthetic it individuals, where liver and of heart distribution. in and proprietary occurring is hepcidin. naturally human organs and endogenous even healthy excessive hepcidin Hepcidin body' In such vital and hormone damage formulation

such as LJPC-XXX occurs of and has or medicinal intermedia beta which beta sickle evaluated cell in secondary treatment cell as iron-overload polycythemia X advancing product for a LJPC-XXX of is primary disease, treatment the being diseases, iron potential overload, currently The LJPC-XXX for thalassemia and are as major myelodysplastic sickle disease. such studies. designated hemochromatosis, as an orphan phase We overload of the syndrome thalassemia, diseases EMA and hereditary vera. result two

the mutation. by multinational, accumulate cardiac despite chelation hemoglobin genetic of underlying death. Beta randomized excessive anemia, above damage, who with thalassemia, in study is multicenter, ongoing major which a disease have This a The characterized controlled transfusions. normal. iron overload first therapy, organ beta levels due is under pivotal need organs patients production these to in to study iron blood a patients to Due may causes iron of the thalassemia independent

often excessive iron the In is addition, in patients blood-transfusions, which accumulation. The this after change endpoint this months' with frequent in of the to heart are study dependent the treatment. disease iron efficacy adds content six on of primary

top-line to We study report in European Union. MAA from in an expect file successful, And expect mid-XXXX. results we if the to this

for iron the no of one efficacy adverse key that the Phase in The saturation, hereditary primary current excessive iron a second The leads measurement FDA measurements production in iron of body the pint a typically are of of ongoing hemochromatosis. hemochromatosis, change procedures levels of X significant phlebotomy blood therapies LJPC-XXX randomized accumulation. standard There in multicenter the is multinational endpoint two a that the and is to with deficiency on detect treat patients standard-of-care approved to transferrin regular associated removal is study of with And and hepcidin the is in involve of genetic hereditary study and effects. used a study the basis overload. a

of the We are from end baseline to the treatment. change the studying

the we active late we clinical of fully change and This the to year, course with from in MAA and pivotal beta for results for in with landscape potential GIAPREZA, a believe to report to the of in integrated patients an in expect treatment for expect the We second drug last also development the hemochromatosis. for to to file or line year, the which iron-overload indication, top in our and thalassemia our for therapy LJPC-XXX this a LJPC-XXX patients that septic of In results from launch programs distributive stage we study organization for X half LJPC-XXXX for treatment designated company report shock. treatment NDA the into transformed year. treating of summary, of orphan other malaria line we of our with results over line hereditary has potential mid-XXXX. report this the study excited severe Phase about top the expect study top We're

open now call the up to will questions. We Operator?

Thank you [Operator Instructions].

of first from Joon line the the comes Our Lee question SunTrust with Robinson.

open. is now line Your

For the what chelators? And for what need the hepcidin commercially heart the of can of induction you magnitude the in iron you remind be to order with iron you induction see viable? assets, amount is in us drug to heart the see Thank you. would

second that upon used. dependent used highly the of half and it's So how question, to the chelators it's

the for requires an removing shovel from And the from poor really actually at the from heart circulation be chelators iron excreted removing iron of simultaneously heart. none iron better them indication so from the have they're used chelators very to two are it heart, and from of Some the to liver, out the removal for oftentimes much body. of to

therapy there's the failing heart. for iron abnormal in of these on chelation are current just remember design optimal in So chelation as already and patients no overload this by the evidenced And trial, therapy iron of for levels the heart. also

in the then the trial, of of know, clinically we reach the large highly meet power really significance terms The trial, will need reach are the associated the it the statistical meaningful. have statistical size of primary of significance a is the be to intrinsically this patients XXX studies you endpoint only with So with significance. trial if patients And meaningful to clinically as to hundreds endpoint. in you many primary on

upon So our again, in agreed statistical iron. clinically then EMA, we significance reach is have the endpoint expect will that should we meaningful a with on all we cardiac and this improvement

I thalassemia iron serum have a beta you. Thank of you think up. for had previous quick a a that Have in week. stratified a patients follow your based ever study, where the you I have type? by hemochromatosis showed mix disclosed data the And you you In and reduction on disease

you in I equally EHA, LJPC-XXX. for wasn’t of type go see were can which I of that data the but believe had effect that. cohort any difference back our studied. I and it look is of tell that is one What or patients, terms in in at could types reduction disease individual that at was presentations that patient we posters there We disease iron the That large. the of all in was didn’t of effective in different

Can And me runway what you one last remind question. the cash is?

cash the our burn, XXXX. on based Currently, have into our and expected cash we of half net balance second

question Company. next with of Phil Cowen Nadeau line the comes from and Our

Your open. line now is

I have XX% proportion you couple said what I GIAPREZA? guess next Could the GIAPREZA distributive launch. us on to access of the give who patients years. year first update over on shock today an is goal think have the you this

So today? where are you

Could you repeat question? the

today? have patients shock GIAPREZA What access percent having to of distributive

XX%, terms range penetrated I that But access this to institutions that that's have of in in we this -- little stated probably the X,XXX, shock exact of it's goal. year, XXX Obviously, fully that we're year. follow out that XX% could XX% goal have that distributive that in with been ordering, the That's aiming say institution you With U.S. which XX% in to but up XX%, our So our targeted. into it's we of to not an we're institution. range ordering say well ordering we're always -- calculation. the represents are the patients over we've

you goals increase talk hospitals And penetration ordering to little expect said a increase those of we of back to that that Could for January, about are penetration? was think you already. XXXX do what bit that other to the one in I the

to for our opportunities but to sensitive. that this So that main to cost objective have year hospitals want utilized is present contracting utilization are and GIAPREZA understandably increase

typically industry. discount is Our involve the fairly contracting efforts which volume in a standard tier,

institution volume volume. that goes the percent as an increased then a get given over they discount of that So increasing up

experience institutions. GIAPREZA. So contracting major with the it's to institutions health are presenting those already systems that And have we those opportunities typically, and

or take volume. GIAPREZA get They to institutions will don't be cost these value we're as off that we that tend opportunities design and of of discounts experience confident short-term take drive institutions barrier have GIAPREZA and experience for that the other have contracting entry, other to a shock. its really to those it And table where the we that of to institutions. cost treatment is they hear the use full very For with yet septic with the GIAPREZA distributive see those

on that U.S., your the there What questions plans update regulators? the beta the for is thalassemia data U.S. take are ability XXX. Same any to on to pivotal in

The No said and is update off before. from it. has our protocol FDA what signed we aware on of

discussions clinically with. We confidence endpoint have FDA. a protocol have the any believe our engaged We well. I we meaningful good statistical that in significance, should comfortable as we special FDA do that have chance U.S. in And other approval that not assessment or will in a very the reach approval is with at is endpoint the

from finances. two, last where one is, pretty imply that a cash operating assessment; so, guidance quarterly R&D question fair And is Is The if the is some that can coming big burn seems cut expenses; us on from? sense give or expense you to in and a SG&A? it one coming

GIAPREZA versus in are throughout there we our the is sales. focus And in various spending those efficiency yet our and the maintain changes spread going evenly quarter. And areas. the made XXXX. to still pretty fourth be in took And Yes, -- change of they spending reduce in to company a XXXX those generating significant the the

questions. thanks for Perfect, taking my

on And bit. that I'll expand a just

commercialize with our currently very some in direction on the of effort and some confident done of the And commercial GIAPREZA. changes we're confident sophisticated changes, GIAPREZA behind sales. maximize team to organization we analyses very have organization, and have that are additions needed we to sufficient the fairly We terms that

Our next from Jefferies. Yang comes with question the of line Kyung

line now is Your open.

have I hemochromatosis. question a one on for hereditary quarter

this So market on although exceptive, second seems And to quite is phlebotomy. would think now you is half show data standard-of-care is of mutation the are in once order me that or it do talking disease major. to by monthly From current common need What you or relatively low. penetrance capture physician, expecting share genetic our since replace we phlebotomy dosing monthly year. in do but we to to the

would to need compete So in you marketplace wondering to show what the approved? if

course, you thank all, of there So reiterate, are first for Kyung, of no approved hereditary therapies to treatment the hemochromatosis. the for And of question. currently

is amongst in really terms it and physicians patients. perception adequately there out is iron So levels the can Phlebotomy there in FDA a of to. to control is general compare used nothing approval, there that

unfortunately, done work think, amongst shared is with that the significant I patient we've population a perception patient not the amount and -- routinely of there.

you hemochromatosis, is phlebotomy pain needle. worsen publications significant of some of many interestingly symptoms the for of hereditary not several they to to showing If discomfort. the a to travel to and your associated looking joint you it large the actually have an infusion that phlebotomy, donation take are speak only board or hours enough, including have patients, day. there with you the center, out joint with And alternative phlebotomy is the effects, specifically blood because insertion adverse of most inconvenient, a It are

your in with for it to work, phlebotomy once nothing those of the And And so to effects, the is hemochromatosis. LJPC-XXX be I significantly development suffer injection our we potential there HH. place hereditary the leave inconvenienced, to knowledge, that subcutaneous has need some treatment weekly to of really of that for displace adverse to else think think our without

likely be first And the therapy. so would successful we approved if

these significantly that we improve the patients. for I So think outcome will

to your estimate million only is low there estimate recognized the Now point of causes genetically about are there're X these point, patients the over about and to that penetrance, affected undergo treatment. we a -- mutation your a million the we so hepcidin. while U.S. the quarter patients data suggests of But in a with

an patient organ or routine a by get failure it's somewhat unusual actually that blood not accidentally test. incurred as adverse presentation diagnosed such of will effect, a on So either

HH. significant So we and diagnose will effort certainly a patients start launch more to broadly with educate

be before significant that So the adverse LJPC-XXX that that they with damage can potentially again very effects. organ they phlebotomy with necessitates is treated frequent attendant incur

So did answer I fully your question?

volume? Thank dosing that’s And you. you helpful. weekly what's Sure. subque XXX, the Thank

The ml. current volume is one

last is the GIAPREZA. question on And

approval So you expecting year. June European in are this

and So upon start or Europe. negotiation approval price do a would governments in various alone we to have expect partnership, with the when you go doing

in will of country to groundwork necessary decision undertake potential So the begun any to have along And of of we Europe, pricing the we year. expected were the the value all as some this meeting on in And we is June that. MAA such individual authorities. of as partnership we if with do the go about assess

But have approached any been multiple will have those as assess by for we any come. We we don't and potential entities partnership. they stated timeline

Our of H.C. comes White Ed line Wainwright. from next with question the

line Your now is open.

you've So seen potential about filing? data review that? just to Can the that a of on for a the review voucher. tell the voucher you. be to that using attaining priority you're priority application taken just chances then And also steps for undergo XXXX. and NDA for thinking has bit Thank What an and us there you more

this process NDA and for that update we'll requested about was are wrapping year. as gain the our filing and of that in So we further clinical we by expect up study timing we later the the clarity the submission, FDA

large benefit data showed additional in gained severe active of that controlled trials have We the of survival of the ingredient from malaria two randomized treatment a LJPC-XXXX.

timeline. filing what stand we're your sufficient as part that confident of based the we with And have FDA we question? our second data track for on today remain so communications and on was And that

part priority The was about review just voucher? the second

PRV. statutory is disease we that’s a priority for application eligible issue of know are a part believe neglected stated happens submission, list tropical And priority strongly review on voucher. we the NDA for the what review separate itself of that of voucher. for the diseases the typically requirements all the serve So of that and we Malaria is there's the part meet

Secondly, approved believe on fact we we has that that had the are has significant benefit. the active case. then show does And based very ingredient that benefit. clinical data And confident the to a data the is been previously. in analyses And not significant show our

until we can't a place actually we very a believe takes the for happens, certain that of in of will be by thorough eligible NDA while exhaustive review. always be it and with issuance parallel requirements that analysis So the PRV as process we statutory

And then as far as marketing. Is look this something you that would you would market for on your own or partner? a

Well, effort X,XXX there'll the not U.S. this patients since every in since to progress no severe require terms year, And we other will believe be almost will marketing. there's malaria about much malaria. of XXX that in treatment, really do

we with get working that the have to So support We're distributors currently the would infrastructure this. drug. do

have For approved shelve. have we GIAPREZA, on with the to products the distributors hospital relationships

XXXX infrastructure. if is So approved, we have that

simply would do and We threatening. forth. would that sales it significant life But have be we rare in therapy severely a not it marketing to affairs for efforts, it the very condition support is that believe our medical infrastructure with so because require and pharmaco-vigilance essential

JP of comes question line next from Our Rama the Anupam with Morgan.

now line is Your open.

the on trial strategy guys. what here? is you and be question. fully the data? to very us on earlier really, a on have win much, considered And that clinical then trial Is piggybacking a underway design mentioned Thanks maybe publishing malaria the for trial Can needed evening. a would there This this you believe last LJPC-XXXX for NDA is the orient I Tessa Anupam in clinical and recruited.

The we enrolled that the version and sure. our data. has finalizing previously for update the I on are a Yes, we'll And outcomes. active yes of the collecting really pharmacokinetics completed that shown that of trial And ingredient, the of been at we currently of the the collection NDA. improve which are think trial that data is to fully bits expected distribution at is trial we and looks are last with LJPC-XXXX, timing that and everyone update the time our for

about And approved. and plan it we to should publish the medical I availability think place absolutely of and update the will data. do we think data certainly get XXXX community I public have the the in the

a coherent do yes, we So have strategy. publication

Thank this questions you. at time. no we further have And

all for and LJPC. support you conference today, Thank appreciate attending of call your our

thank gentlemen, program. conference. for today's participating This conclude and today's in does you Ladies

You may all Everyone, disconnect. great have a day.