Participating Companies

3D Bio
De­vel­op­ing scal­able bio­print­ed car­ti­lage ther­a­peu­tics to ad­dress un­met med­i­cal needs in re­con­struc­tive and or­tho­pe­dic in­di­ca­tions with rapid reg­u­la­to­ry path­way to mar­ket. Fil­ing IND for or­phan in­di­ca­tions in 2018. The plat­form in­cludes pro­pri­e­tary GM­Print­bio­print­ing hard­ware/soft­ware sys­tem and Col­Vi­vo col­la­gen, which is 20x stronger than any­thing com­mer­cial­ly avai­l­able. [more in­for­ma­tion]
4D Molecular Therapeutics [FDMT] $535 MM MCap
Fo­cused on the dis­cov­ery and de­vel­op­ment of pro­pri­e­tary tar­get­ed AAV gene ther­a­py vec­tors and ther­a­peu­tic prod­ucts through a ro­bust dis­cov­ery plat­form, termed Ther­a­peu­tic Vec­tor Evo­lu­tion. Th­ese cus­tomized gene de­liv­ery ve­hi­cles can de­liv­er genes to any tis­sue or or­gan in the body, by op­ti­mal clin­i­cal routes of ad­min­is­tra­tion and evad­ing pre-ex­ist­ing anti­bodies to treat both rare ge­net­ic dis­eas­es and com­plex large mar­ket dis­eas­es. In­ter­nal pipe­line and part­n­er­ships in­clud­ing: Pfiz­er, Roche, uniQure, AGTC and Benitec. [more in­for­ma­tion]
Ablynx nv [ABLX]
Nano­body plat­form with over 45 pro­pri­e­tary and part­nered pro­grams across in­di­ca­tions in­clud­ing aTTP, RSV, SLE and I-O. In Oc­to­ber 2017 re­port­ed pos­i­tive Phase III da­ta with ca­pla­cizumab for aTTP, a rare clott­ing dis­porder. Ca­pla (fast track) BLA planned for 1H 2018, with po­ten­tial Eu­rope launch in 2H 2018 and US 1H 2019. Oc­to­ber 2017 US IPO. [more in­for­ma­tion]
Acceleron Pharma Inc [XLRN] $10,932 MM MCap
Ac­celeron fo­cus­es its re­search, de­vel­op­ment, and com­mer­cial­iza­tion ef­forts in pul­mo­nary and he­ma­to­log­ic dis­eas­es. In pul­mo­nary, Ac­celeron is de­vel­op­ing so­tater­cept for the treat­ment of pul­mo­nary hy­per­ten­sion, cur­rent­ly in Phase 3 de­vel­op­ment, as well as ACE-1334 in SSc-ILD, which will be in the clin­ic by year-end. In he­ma­tol­o­gy, RE­BLOZYL® (lus­pa­ter­cept-aamt), part of a glob­al col­lab­o­ra­tion part­n­er­ship with BMS, is the first and on­ly ery­throid ma­t­u­ra­tion agent ap­proved in the Unit­ed States, Eu­rope, and Ca­na­da for the treat­ment of ane­mia in cer­tain blood di­s­or­ders. [more in­for­ma­tion]
AcelRx Pharmaceuticals, Inc. [ACRX] $9 MM MCap
Launched DSU­VIA (sufen­tanil sublin­gual tablet 30 mcg) in the US in 2019 for acute pain se­vere enough to re­quire opi­oid anal­gesic (adult pa­tients in cer­ti­fied med­i­cal­ly su­per­vised health­care sett­ings). Al­so de­vel­op­ing Za­lvi­so (Ph 3 com­plet­ed) in the US as an in­no­va­tive pa­tient-con­trolled anal­ge­sia sys­tem for mod­er­ate-to-se­vere acute pain in med­i­cal­ly su­per­vised sett­ings. [more in­for­ma­tion]
Achillion Pharmaceuticals, Inc. [ACHN]
Pipe­line of com­ple­ment fac­tor D in­hibi­tors to treat rare dis­eas­es such as PNH and C3G. In Novem­ber 2017, re­port­ed pre­lim­i­nary proof-of-con­cept with ACH-4471 in C3G. show­ing greater than 50% im­prove­ment in pro­tein­uria at 14 days. [more in­for­ma­tion]
Adamas Pharmaceuticals, Inc. [ADMS] $376 MM MCap
Late-stage pipe­line in­cludes ADS-5102 in P3 for MS walk­ing im­pair­ment and ADS-4101, high-dose, mod­i­fied re­lease la­cosamide, which com­plet­ed P1 for par­tial onset seizures in pa­tients with epilep­sy. [more in­for­ma­tion]
Adaptimmune Therapeutics [ADAP] $176 MM MCap
Mul­ti­ple trials on­go­ing in both solid tu­mors and he­ma­to­log­ic can­cer types with pro­pri­e­tary T-cell en­gi­neer­ing plat­form. Da­ta read­out from NY-ESO SPEAR T-cells in NS­CLC and ovarian can­cer and da­ta from MAGE-A10 Spear T-cells in NS­CLC, blad­der, me­lano­ma, head and neck can­cer ex­pect­ed in 2017. Piv­o­tal study of NY-ESO SPEAR T-cells in syn­ovial sar­co­ma Mid-2017. [more in­for­ma­tion]
Adverum Biotechnologies [ADVM] $151 MM MCap
Oc­u­lar / rare dis­ease gene thx. AD­VM-022 in P1 OP­TIC study in wAMD. 24-wk da­ta from co­hort 1 (6 pts) re­port­ed in Sep (0 res­cue in­jec­tions). 52-wk da­ta from co­hort 1 and 24-wk da­ta from co­hort 2 exp 1H20. [more in­for­ma­tion]
Affimed [AFMD] $71 MM MCap
On­col­o­gy ther­a­peu­tics de­vel­op­er with most ad­vanced AFM13 for r/r pe­ripher­al T-cell lym­pho­ma com­plet­ing Ph2 en­roll­ment. AFM24 for mul­ti­ple solid tu­mors is in Ph 2a as monother­a­py. [more in­for­ma­tion]
Aileron Therapeutics, Inc. [ALRN] $7 MM MCap
Clin­i­cal stage on­col­o­gy com­pany con­duct­ing 3 clin­i­cal trials for its lead can­di­date, AL­RN-6924, a first-in-class/best-in-class drug that tar­gets the p53 path­way. AL­RN-6924 is from the Sta­pled pep­tide plat­form and is the on­ly clin­i­cal drug can­di­date that binds equipo­tent­ly to both of the p53 sup­pres­sor pro­teins, MD­MX and MD­M2. This com­pany has now ini­ti­at­ed of a Phase 1 / 1b in Acute Myeloid Leukemia (AML) and a Phase 2a in Pe­ripher­al T-Cell Lym­pho­ma. [more in­for­ma­tion]
Akari Therapeutics Plc [AKTX] $20 MM MCap
Cont­in­ues to en­roll pa­tients in Ph 2 study of Parox­ys­mal noc­tur­nal he­mo­globin­uria (PNH), and ex­pects to ini­ti­ate Ph 3 study of PNH and Ph 2 study in atyp­i­cal He­mo­lyt­ic Ure­mic Syn­drome (aHUS) in Q4 2017. [more in­for­ma­tion]
Amarin Corporation [AMRN] $375 MM MCap
Com­mer­cial­iz­ing Vas­ce­pa, the first and on­ly pure EPA ome­ga-3 fat­ty acid ap­proved to low­er trig­lyc­eride lev­els in adults. Pre­sent­ed (+) da­ta from RE­DUCE-IT CV Out­comes trial at AHA. First 9mo rev’s of 2020 ~$450M. [more in­for­ma­tion]
AngioGenex [AGGX] $0 MM MCap
ef­fi­ca­cy in breast can­cer, lung can­cer, col­orec­tal can­cer, AMD, and ROP. Sup­port from by Dr. Lar­ry Nor­ton at MSK & Dr. [more in­for­ma­tion]
Apricus Biosciences, Inc. [APRI]
Ad­vanc­ing medicines in urol­o­gy and rheu­ma­tol­o­gy, with two prod­uct can­di­dates in de­vel­op­ment. Fo­cused on U.S. ap­pro­val of Vi­taros, a top­i­cal gel for erec­tile dys­func­tion; NDA re-sub­mitt­ed in Q3 2017 and PD­U­FA goal date Fe­bruary 17, 2018. Ray­Va, for Ray­naud's Pheno­menon, has de­layed its Ph2b clin­i­cal trial ini­ti­a­tion to fo­cus on or­phan desig­na­tion and is seek­ing ex-US part­n­er­ship. [more in­for­ma­tion]
Aquinox Pharmaceuticals, Inc. [AQXP]
Dis­cov­er­ing and de­vel­op­ing tar­get­ed ther­a­peu­tics in in­flam­ma­tion and im­muno-on­col­o­gy. Lead as­set, Rosip­tor, is an oral, once dai­ly, small molecule ac­ti­va­tor of SHIP1. In Ph3 for the treat­ment of Blad­der Pain Syn­drome/In­ter­sti­tial Cys­ti­tis (BPS/IC) with to­p­line da­ta ex­pect­ed in 3Q 2018. Plans to ini­ti­ate Ph2 in Chron­ic Pro­s­tati­tis/Chron­ic Pelvic Pain Syn­drome (CP/CPPS) in 2Q 2018. [more in­for­ma­tion]
Arena Pharmaceuticals [ARNA] $6,165 MM MCap
Etrasi­mod for ul­cer­a­tive coli­tis with Phase 2 da­ta ex­pect­ed in late 2017, ra­line­pag for pul­mo­nary ar­te­rial hy­per­ten­sion with Phase 2 da­ta ex­pect­ed in mid-year 2017, and ini­ti­a­tion of Phase 2 trial of APD371 for pain as­so­ci­at­ed with Crohn’s dis­ease in ear­ly 2017. Has suf­fi­cient cash to get to da­ta for all three Phase 2 trials. [more in­for­ma­tion]
Arrowhead Pharmaceuticals [ARWR] $2,880 MM MCap
RNA in­ter­fer­ence, or RNAi, is a mech­anism pre­sent in liv­ing cells that in­hibits the ex­pres­sion of a spe­cif­ic gene, there­by af­fect­ing the pro­duc­tion of a spe­cif­ic pro­tein. Ar­row­head’s RNAi-based ther­a­peu­tics lev­er­age this na­t­u­ral path­way of gene si­lenc­ing. [more in­for­ma­tion]
Asklepion Pharmaceuticals
First in­di­ca­tion, the pre­ven­tion of sig­ni­f­i­cant se­que­lae of pe­di­a­tric acute car­diopul­mo­nary by­pass-in­duced lung in­jury, Ph 3 trial is on­go­ing. Af­ter re­ceiv­ing FDA and EMA reg­is­tra­tion, the com­pany sold Chol­bam, for bile acid syn­th­e­sis de­fects and per­ox­i­so­mal di­s­or­ders, to Retrophin (RTRX) in 2015, pro­vid­ing an on­go­ing, quar­ter­ly rev­enue stream. [more in­for­ma­tion]
Assembly Biosciences, Inc. [ASMB] $47 MM MCap
A pi­oneer in the de­vel­op­ment of a new class of po­tent, oral core in­hibi­tor drug can­di­dates, As­sem­b­ly Bio’s ap­proach aims to break the com­plex vi­ral re­pli­ca­tion cy­cle of HBV to free pa­tients from a life­time of ther­a­py. As­sem­b­ly Bio’s strat­e­gy to­ward cure in­cludes a lead­ing port­fo­lio of more po­tent, next-gen­er­a­tion core in­hibi­tors, proof-of-con­cept com­bi­na­tion studies and a re­search pro­gram fo­cused on the dis­cov­ery of nov­el HBV tar­gets, in­clud­ing cc­cD­NA dis­rup­tors and two other undis­closed mech­anisms. In 202 [more in­for­ma­tion]
AxoGen [AXGN] $215 MM MCap
Lead­ing com­pany fo­cused on de­vel­op­ment and com­mer­cial­iza­tion of tech­nolo­gies for pe­ripher­al nerve re­gen­er­a­tion and re­pair. Lead prod­uct Avance Nerve graft is bi­o­log­i­cal­ly-ac­tive OTS hu­man al­lo­graft. FY 2022 rev­enue gui­dance of $135-$142M rep­re­sents 10%-15% YOY growth ex. $4.1M from dis­cont­in­ued Avive rev­enue. [more in­for­ma­tion]
Axsome Therapeutics [AXSM] $3,298 MM MCap
AXS-05 in Ph 3 trial in treat­ment re­sis­tant de­pres­sion (TRD); Ph 2/3 trial in ag­i­ta­tion in Alzheimer’s dis­ease (AD) planned. [more in­for­ma­tion]
Bavarian Nordic A/S [BAVA]
Glob­al lead­er in vaccines for in­fec­tious dis­eas­es. Com­mer­cial prod­ucts in­clude the world’s on­ly ap­proved mon­key­pox vaccine and mar­ket-lead­ing vaccines against ra­bies, tick-borne en­cephali­tis, in ad­di­tion to an Ebo­la vaccine li­censed to Janssen. Com­pany de­liv­ered more than 4 mil­lion dos­es of mon­key­pox vaccines in 2022 cov­er­ing more than 70 coun­tries and ex­pects to sup­p­ly ~10 mil­lion dos­es through 2023. En­roll­ment is on­go­ing in glob­al Ph 3 COVID boost­er trial and glob­al Ph 3 RSV trial with ac­cess to ac­cel­er­at [more in­for­ma­tion]
Bellicum Pharmaceuticals Inc. [BLCM] $3 MM MCap
BPX-601: Go­CAR-T fea­tur­ing iMC (in­du­ci­ble My­D88/CD40) ac­ti­va­tion switch tar­get­ing PS­CA ex­press­ing solid tu­mors; BPX-701: TCR tar­get­ing PRAME. [more in­for­ma­tion]
BeyondSpring Pharmaceuticals
Two Phase 3 clin­i­cal trials for two in­di­ca­tions with in­ter­im read-outs due in 2H 2017 and 1Q 2018, another in a Phase 1/2 trial. Unique US/Chi­na clin­i­cal de­vel­op­ment strat­e­gy to ex­pe­dite en­roll­ment and glob­al clin­i­cal trials. Com­pany has been fund­ed to date pri­mar­i­ly by Chi­nese-based in­ves­tors and has an R&D col­lab­o­ra­tion with the Fred Hutchin­son Can­cer Re­search Cen­ter. [more in­for­ma­tion]
BioPharmX [BPMX]
De­vel­op­ing BPX-04 for rosacea, us­ing pro­pri­e­ta­ty hy­drophil­ic top­i­cal for­mu­la­tion tech­nol­o­gy. En­rolling Ph2b w da­ta exp in 2019. Com­plet­ed Ph 2b with BPX-01 in acne. [more in­for­ma­tion]
Cascadian Therapeutics [CASC] $554 MM MCap
Pre­lim­i­nary re­sults from two on­go­ing Phase 1b studies of tu­ca­tinib in com­bi­na­tion showed promis­ing sys­temic ac­tiv­i­ty, a fa­vor­able safe­ty pro­file and en­cour­ag­ing ac­tiv­i­ty against brain me­tas­tas­es. Con­duct­ing a ran­domized, dou­ble-blind, place­bo-con­trolled Phase 2 study called HER2­CLIMB to eval­u­ate tu­ca­tinib ver­sus place­bo in com­bi­na­tion with capec­itabine and tras­tuzumab in late stage HER2+ breast can­cer pa­tients, with and with­out brain me­tas­tas­es, who have pre­vi­ous­ly been treat­ed with a tax­ane, tras­tuzumab, per­tuzumab and T-DM1. This study is ex­pect­ed to en­roll 180 pa­tients across ap­prox­i­mate­ly 100 clin­i­cal sites in the U.S., Ca­na­da, and West­ern Eu­rope. [more in­for­ma­tion]
Catalyst Pharmaceuticals [CPRX] $1,246 MM MCap
Ca­t­alyst's New Drug Ap­pli­ca­tion for FIR­DAPSE® (am­i­fam­pri­dine) Tablets 10 mg for the treat­ment of adults with Lam­bert-Ea­ton myas­thenic syn­drome ("LEMS") was ap­proved in 2018 by the U.S. Food & Drug Ad­min­is­tra­tion ("FDA"), and FIR­DAPSE is com­mer­cial­ly avai­l­able in the Unit­ed States as a treat­ment for adults with LEMS. Fur­ther, Ca­na­da's na­tio­n­al health­care reg­u­la­to­ry agen­cy, Health Ca­na­da, has ap­proved the use of FIR­DAPSE for the treat­ment of adult pa­tients in Ca­na­da with LEMS. [more in­for­ma­tion]
Cellectis SA [CLLS] $72 MM MCap
By cap­i­tal­iz­ing on its 19 years of ex­per­tise in gene edit­ing – built on its flag­ship TAL­EN® tech­nol­o­gy and pi­oneer­ing elec­tro­po­ra­tion sys­tem PulseAg­ile – Cel­lec­tis us­es the pow­er of the im­mune sys­tem to tar­get and erad­i­cate can­cer cells. Us­ing its life-sci­ence-fo­cused, pi­oneer­ing genome en­gi­neer­ing tech­nolo­gies, Cel­lec­tis’ goal is to cre­ate in­no­va­tive prod­ucts in mul­ti­ple fields and with vari­ous tar­get mar­kets. Cel­lec­tis is list­ed on the Nas­daq mar­ket (tick­er: CLLS) and on Eu­ronext Growth (tick­er: AL­CLS). [more in­for­ma­tion]
The com­pany's lead com­pound, tr­i­caprilin, is be­ing de­vel­oped for a range of dis­eas­es in­clud­ing Alzheimer’s dis­ease (en­ter­ing Phase 3), mi­graine pre­ven­tion (in Phase 2) and a rare form of pae­di­a­tric of epilep­sy (in­fan­tile spasms - plan­n­ing Phase 1b/2). Cerecin's Alzheimer's dis­ease pro­gram is one of the few pre­ci­sion medicines in de­vel­op­ment, ad­dress­ing a spe­cif­ic ge­net­ic sub-group, the non-car­ri­ers of the APOE4 al­lele (ac­counts for 45-85% of AD pa­tients). [more in­for­ma­tion]
Cernostics, Inc.
De­vel­op­er of tis­sue-based di­ag­nos­tic test de­signed to quan­ti­fy the com­plex­i­ty of the tis­sue sys­tem. The com­pany's di­ag­nos­tic test unique­ly an­a­lyzes whole slide dig­i­tal im­ages with mul­ti­plexed flu­o­res­cence, pro­vid­ing physi­cians and pa­tients with in­di­vi­d­u­al­ized, ac­tion­able in­for­ma­tion to im­prove out­comes and re­duce the in­ci­dence and mor­tal­i­ty of can­cer. [more in­for­ma­tion]
A com­mer­cial stage, pre­ci­sion gene edit­ing com­pany. Cibus' tech­nol­o­gy has ap­pli­ca­tions in non-GMO crop de­vel­op­ment, in­dus­trial biotech and hu­man health. The com­pany uti­l­izes Gene Re­pair Oli­gonu­cleotides (GRONs) that pro­duce pre­cise, sta­ble, pre­dictable changes in plants and other or­ganisms with­out in­te­grat­ing for­eign ge­net­ic ma­te­rial. [more in­for­ma­tion]
Cipher Pharmaceuticals Inc. [CPH:CA] Can$107 MM MCap
Spe­cial­ty phar­ma­ceu­ti­cal der­ma­tol­o­gy com­pany with a di­ver­si­fied port­fo­lio of com­mer­cial-stage prod­ucts with the goal of be­com­ing the most cus­tomer-cen­tric der­ma­tol­o­gy com­pany in North Amer­i­ca. Com­plet­ed sev­en tran­s­ac­tions in 2015, in­clud­ing the ac­qui­si­tion of In­no­cutis and its nine brand­ed der­ma­tol­o­gy prod­ucts, to build its U.S. com­mer­cial pres­ence, ex­pand its Ca­na­dian der­ma­tol­o­gy franchise and broa­d­en its pipe­line. [more in­for­ma­tion]
Clearside Biomedical [CLSD] $54 MM MCap
Lead in­di­ca­tion mac­u­lar ede­ma as­so­ci­at­ed with uvei­tis now in Phase 3 with a po­ten­tial NDA fil­ing in 2017. RVO, with com­plete Ph2 da­ta pre­sent­ed at AAO in Oc­to­ber, ex­pect­ed to ini­ti­ate Ph3 trial in 1H 2017. Clear­side an­nounced a new in­di­ca­tion, DME, and dosed first pa­tient in a Phase 1/2 trial in Novem­ber. [more in­for­ma­tion]
Cocrystal Pharma, Inc. [COCP] $19 MM MCap
Pro­pri­e­tary struc­tu­ral in­for­ma­tion, en­zy­mol­o­gy and nu­cle­o­side ex­per­tise to de­vel­op nov­el, pan-geno­typ­ic an­tivi­ral agents for se­ri­ous and chron­ic vi­ral dis­eas­es. Pipe­line cur­rent­ly in­cludes as­sets in de­vel­op­ment to treat in­fluen­za, norovirus and HCV. Re­cent­ly an­nounced da­ta from a Ph 1a/1b clin­i­cal study of CC-31244 for the treat­ment of HCV. [more in­for­ma­tion]
CytomX Therapeutics, Inc. [CTMX] $86 MM MCap
Lead pro­gram CX-072 is a whol­ly owned PD-L1 Pro­body cur­rent­ly be­ing studied in a Phase 1/2 study as monother­a­py and in com­bi­na­tion with ip­il­i­mumab and with ve­mu­rafenib. CX-2009 is a CD166-tar­get­ed PDC cur­rent­ly be­ing studied in a Phase 1/2 study in solid tu­mors. BMS-part­nered Pro­body of ip­il­i­mumab be­ing studied in on­go­ing Phase 1/2 trial in monother­a­py and in com­bi­na­tion with nivolumab. [more in­for­ma­tion]
DBV Technologies [DBVT] $262 MM MCap
An in­vesti­ga­tio­n­al non-in­va­sive, once-dai­ly, epi­cu­ta­neous patch, Vi­askin Peanut seeks to de­liv­er mi­cro­gram quan­ti­ties of peanut anti­gen to ac­ti­vate the im­mune sys­tem. [more in­for­ma­tion]
EnGeneIC Limited
Com­plet­ed Phase 1 trial in re­cur­rent glio­ma, re­sult­ing in in­creased over­all sur­vi­val (3/8 still alive); IND ac­cept­ed by FDA for Phase 1/2a trial, cur­rent­ly re­cruit­ing at ma­jor US in­sti­tu­tion. On­go­ing Phase 1 in end-stage me­sothe­lio­ma pro­duced sta­tis­ti­cal­ly sig­ni­f­i­cant over­all sur­vi­val. Re­cent re­search col­lab­o­ra­tion deal signed with Take­da. [more in­for­ma­tion]
Rec­og­niz­ing that the Phar­ma in­dus­try is on­ly be­gin­n­ing to un­der­s­tand how to po­ten­tial­ly op­ti­mize the ther­a­peu­tic ben­e­fits of T cell ther­a­pies (CART & TCRT) and with a great deal still to learn, En­terome is tak­ing a de­lib­er­ate­ly new and dif­fer­ent ap­proach with its tech­nol­o­gy plat­form en­abling each pa­tient to ben­e­fit from a spe­cif­ic and ma­jor ex­pan­sion of the best part of its own T cell reper­toire (Ef­fec­tor Me­m­o­ry T cells), in a high­ly so­phis­ti­cat­ed and un­prece­dent­ed way. Our On­coMim­icTM tech­nol­o­gy is en­abling the over­com­ing of im­mune tol­er­ance against can­cer cells with broad ap­pli­ca­bil­i­ty in the high un­met need solid tu­mor space via an off-the-shelf, easy to man­u­fac­ture tech­nol­o­gy. Our lead as­set EO2401 is cur­rent­ly in phase 2 in re­cur­rent Glioblas­to­ma (rGBM) and piv­o­tal trial in Adre­nal car­ci­no­ma (ACC). [more in­for­ma­tion]
Enzo Biochem, Inc. [ENZ] $70 MM MCap
$100m revs in F18. Pi­oneer in molec­u­lar di­ag­nos­tics (Dx), lead­ing con­ver­gence of clin­i­cal lab­o­ra­to­ries, life sci­ences and IP through de­vt of unique Dx plat­form tech­nolo­gies pro­vid­ing nu­mer­ous ad­van­tages over pre­vi­ous stan­dards. [more in­for­ma­tion]
Epigenomics AG [EPGNY] $8 MM MCap
Blood-based can­cer de­tec­tion com­pany with DNA methy­la­tion bio­mark­er tech­nol­o­gy. De­vel­ops/com­mer­cial­izes Dx prod­ucts across mul­ti­ple can­cer in­di­ca­tions w/high med­i­cal need. Lead, Epi pro­Colon: blood-based screen­ing test de­tect­ing col­orec­tal can­cer. FDA ap­proved, mar­ket­ed in US, EU, Chi­na et al. [more in­for­ma­tion]
Evotec AG [EVTA:FF] $3,221 MM MCap
Evotec is a drug dis­cov­ery al­liance and de­vel­op­ment part­n­er­ship com­pany fo­cused on rapid­ly pro­gress­ing in­no­va­tive prod­uct ap­proach­es with lead­ing phar­ma­ceu­ti­cal and biotech­nol­o­gy com­pa­nies, aca­demics, pa­tient ad­vo­ca­cy groups and ven­ture cap­i­tal­ists. The EVT In­no­vate seg­ment is an in­ter­nal drug dis­cov­ery plat­form with over 70 prod­uct op­por­tu­ni­ties and ex­per­tise across dis­eas­es ar­eas. The EVT Ex­e­cute ser­vice busi­ness al­lows the firm to build its pipe­line with­out bear­ing the ex­ten­sive fi­nan­cial risk and costs typ­i­cal­ly in­volved in drug de­vel­op­ment. [more in­for­ma­tion]
De­vel­op­ment pro­grams ad­dress­ing rare and se­vere liv­er dis­eas­es. Ph3 da­ta for ela­fi­branor in pri­mary biliary cholan­gi­tis (PBC) EL­A­TIVE study, ex­pect­ed 2Q23. Ac­qui­si­tion of Ver­san­tis brings VS-01-ACLF, a Ph2 ready pro­gram for se­vere liv­er dis­ease. Li­cens­ing and de­vel­op­ment agree­ment with IPSEN be­gan De­cem­ber 2021. [more in­for­ma­tion]
GenSight Biologics [SIGHT]
Op­to­ge­net­ic pro­gram and tar­get­ed dis­ease im­me­di­ate­ly trans­fer­able to dry AMD. Plat­form com­bines mono-ge­net­ic ther­a­py-based ap­proach w core tech­nol­o­gy plat­form and pro­pri­e­tary Mi­to­chon­drial Tar­get­ing Se­quence. [more in­for­ma­tion]
Geron [GERN] $1,110 MM MCap
Late-stage bio­phar­ma­ceu­ti­cal com­pany pur­su­ing ther­a­pies for pa­tients with he­ma­to­log­ic ma­lig­nan­cies. First-in-class telom­erase in­hibi­tor, ime­tel­s­tat, in two Ph3 piv­o­tal clin­i­cal trials in low­er risk myelodys­plas­tic syn­dromes (LR MDS; top-line da­ta ear­ly Jan 2023), and in re­lapsed/re­frac­to­ry myelo­fi­bro­sis (MF). [more in­for­ma­tion]
Gly­co­tope is a clin­i­cal stage biotech­nol­o­gy com­pany ap­p­ly­ing world-lead­ing gly­co-bi­ol­o­gy ex­per­tise to de­vel­op high­ly in­no­va­tive mAb-based ther­a­peu­tics in im­muno-on­col­o­gy and on­col­o­gy. The Com­pany’s Gly­co­bodies tar­get spe­cif­ic gly­co-epi­topes to gen­er­ate first-in-class prod­ucts in­clud­ing naked anti­bodies and bis­pe­cifics. The lead can­di­date, Gatipo­tozumab, tar­gets TA-MUC1 and is in the clin­ic for the treat­ment of solid tu­mors in com­bi­na­tion with an an­ti-EGFR anti­body. [more in­for­ma­tion]
On­col­o­gy- fo­cused com­pany with two reg­is­tra­tio­n­al studies on­go­ing in Ew­ing's Sar­co­ma and Ovarian Can­cer with po­ten­tial fil­ings in 2019 and 2021, re­spec­tive­ly. In­ter­im re­sults of 4 com­bo studies ex­pect­ed in next 12 months. > 160 pa­tients treat­ed to-date in 19 tu­mor types. [more in­for­ma­tion]
GW Pharmaceuticals PLC [GWPH] $82,884 MM MCap
GW has estab­lished a world lead­ing po­si­tion in the de­vel­op­ment of plant-de­rived cann­abi­noid ther­a­peu­tics through its proven drug dis­cov­ery and de­vel­op­ment pro­cess­es, in­tel­lec­tu­al prop­er­ty port­fo­lio and reg­u­la­to­ry and man­u­fac­tur­ing ex­per­tise. GW has de­vel­oped an oral for­mu­la­tion of pu­ri­fied cann­abi­di­ol (CBD), ap­proved as Epi­di­olex® in the U.S. by the U.S. [more in­for­ma­tion]
NAS­DAQ, AIM and Hong Kong-list­ed glob­al com­mer­cial-stage on­col­o­gy com­pany with 3 ap­proved and mar­ket­ed tar­get­ed can­cer drugs in Chi­na and plans for reg­u­la­to­ry sub­mis­sions in the US, EU and Ja­pan in 2023. Key part­n­er­ships with Eli Lil­ly, As­traZene­ca and Bei­Gene. [more in­for­ma­tion]
Immutep (formerly Prima BioMed) [IMMP] $165 MM MCap
Cur­rent trials: IM­P321 Ph2b (AI­PAC), po­ten­tial­ly EU-reg­is­tra­tio­n­al, for me­tastat­ic breast can­cer, top line da­ta 1H 2019; and IM­P321 Ph1 (TAC­TI-mel) for me­tastat­ic me­lano­ma, safe­ty and dose es­ca­la­tion da­ta due 1H 2018. De­vel­op­ment part­n­er­ships with GSK and No­var­tis. CMO/CSO Fr&ea­cute;d&ea­cute;ric Trie­bel dis­cov­ered the LAG-3 gene in 1990 and iden­ti­fied the func­tions and med­i­cal ap­pli­ca­tions of the tar­get. [more in­for­ma­tion]
Innovative Med Concepts
Lead com­pound, IMC-1 is a nov­el pro­pri­e­tary oral fixed-dose-com­bi­na­tion de­signed to treat fi­bromyal­gia, IBS and other re­lat­ed con­di­tions us­ing a po­ten­tial break­through mech­anism of ac­tion. Re­sults from the ran­domized, dou­ble blind, place­bo con­trolled mul­ti-cen­ter Phase 2 trial dif­fer­en­ti­ates IMC-1 from the cur­rent stan­dard of care. FDA grant­ed Fast Track sta­tus based on un­met med­i­cal need in a se­ri­ous dis­ease and be­cause IMC-1 has the po­ten­tial to meet that need. [more in­for­ma­tion]
Intellia Therapeutics [NTLA] $2,798 MM MCap
Com­pany’s in-vi­vo pro­grams fo­cus on the use of Lipid Nano­par­ti­cle for de­liv­ery of the CRIS­PR/Cas9 com­plex to the liv­er and the Com­pany’s ex-vi­vo pro­grams in­clude both pro­pri­e­tary and part­nered pro­grams fo­cused on chimer­ic anti­gen re­cep­tor T cells, or CAR T cells, and he­ma­topoi­et­ic stem cells. [more in­for­ma­tion]
Kiadis Pharma N.V. [KDS-AMS]
Launched FIH POC trial in r/r AML pa­tients (Feb 2020); set to launch ad­di­tio­n­al HSCT trial lat­er in 2020 w/in­ter­im da­ta exp 2021. Pre­sent­ing new da­ta on ex-vi­vo FC21 ex­pand­ed NK-cell ther­a­py in 13 pa­tients at EBMT in 2020. Estab­lished POC in 37 pa­tients to­tal. [more in­for­ma­tion]
Loxo Oncology, Inc. [LOXO] $7,184 MM MCap
The com­pany's pro­cess­es are ded­i­cat­ed to the de­vel­op­ment of high­ly se­lec­tive medicines for pa­tients with ge­net­i­cal­ly defined can­cers and its ap­proach is spe­cial­ized on two sci­en­tif­ic trends that are in­creas­ing use of ge­net­ic test­ing in can­cer clin­i­cal medicine and im­prov­ing chem­istry ap­proach­es to build­ing high­ly se­lec­tive in­hibi­tors against sin­gle tar­gets in the can­cer cell. [more in­for­ma­tion]
MedinCell [MEDCL]
Pro­f­itable clin­i­cal stage plat­form tech­nol­o­gy drug de­liv­ery com­pany fo­cused on long-act­ing in­jectable for­mu­la­tions for sub­cu­ta­neous de­liv­ery in­jec­tion to pro­vide sys­temic ef­fect while min­i­miz­ing in­jec­tion pain. Mul­ti-prod­uct agree­ments with top 10 phar­ma com­pa­nies in CNS and on­col­o­gy. [more in­for­ma­tion]
Medivir AB [MVIR-B]
Nu­cleotide-pro­drug plat­form: MIV-818, an oral pro­drug of the clin­i­cal­ly ef­fec­tive troxac­itabine, for HCC and MIV-828 for AML. Ad­vanced clin­i­cal pro­grams for part­ner­ing: Birini­pant, a bi­va­lent SMAC mimet­ic; Remeti­no­s­tat for CT­CL and Ph 3 ready MIV-711 for OA. [more in­for­ma­tion]
MEI Pharma, Inc. [MEIP] $47 MM MCap
Broa­d­en­ing zan­delis­ib de­vel­op­ment ac­tiv­i­ty in­cludes ad­di­tion of margi­nal zone lym­pho­ma arm to TI­DAL to sup­port ex­pan­sion of an ac­cel­er­at­ed ap­pro­val strat­e­gy if suc­cess­ful, the ini­ti­a­tion of a Phase 3 study in se­cond line fol­lic­u­lar and margi­nal zone lym­pho­mas in mid-2021, and plans to sup­port se­lect in­vesti­ga­tor-ini­ti­at­ed trials, ini­tial­ly in first-line DL­B­CL. Mile­s­tones al­so in­clude Ini­tial phase 1b da­ta from study eval­u­at­ing zan­delis­ib with Brukin­sa™ in col­lab­o­ra­tion with Bei­Gene. An­ti­ci­pat­ed up­dates in other pro­grams in­clude: voru­ci­clib Ini­tial da­ta from the Phase 1 monother­a­py and +BCL2i Phase 1b; pra­ci­no­s­tat phase 2 MDS up­date from part­n­er Helsinn; and ME-344 plan to lev­er­age clin­i­cal­ly de­mon­s­trat­ed an­ti-tu­mor ac­tiv­i­ty in com­bi­na­tion with an­ti-VEGF. [more in­for­ma­tion]
MorphoSys AG [MOR] $920 MM MCap
In its pro­pri­e­tary de­vel­op­ment seg­ment, the com­pany de­vel­ops anti­bodies that con­cen­trate on in­flam­ma­to­ry and au­toim­mune dis­eas­es, as well as can­cer and in­fec­tious dis­eas­es. [more in­for­ma­tion]
Naia Pharma
De­vel­op­ing a long-act­ing GLP-1 ag­on­ist ( XTEN™-GLP-1) for Short Bow­el Syn­drome, which has or­phan drug sta­tus. Ex­pects to ini­ti­ate re­cruit­ment the P1b study in 1H17, and file an NDA by the fourth quar­ter of 2018. [more in­for­ma­tion]
Nanobiotix SA [NBTX] $308 MM MCap
NBTXR3 (Hen­si­fy®), which re­cent­ly re­ceived Eu­ro­pean Mar­ket Ap­pro­val, is a first-in-class prod­uct de­signed to de­stroy, when ac­ti­vat­ed by ra­dio­ther­a­py tu­mors through phys­i­cal cell death and me­tas­ta­sis due to im­muno­genic cell death lead­ing to ac­ti­va­tion of the im­mune sys­tem. Nano­bi­otix’s Im­muno-On­col­o­gy pro­gram has the po­ten­tial to bring a new di­men­sion to can­cer im­munother­a­pies. [more in­for­ma­tion]
Neuralstem, Inc. [CUR]
In ad­di­tion, NSI-566 was eval­u­at­ed in a Phase 1 safe­ty study to treat paral­y­sis due to chron­ic spi­nal cord in­jury as well as a Phase 1 and Phase 2a risk es­ca­la­tion, safe­ty trials for ALS. Pa­tients from all three in­di­ca­tions are cur­rent­ly in long-term ob­ser­va­tio­n­al fol­low-up pe­ri­ods to cont­in­ue to mon­i­tor safe­ty and pos­si­ble ther­a­peu­tic ben­e­ ner­vous sys­tem. [more in­for­ma­tion]
Nivalis Therapeutics, Inc. [NVLS]
Re­cent­ly com­plet­ed en­roll­ment in a se­cond Phase 2 study of N91115 eval­u­at­ing the ef­fect of N91115 as add-on ther­a­py to Ka­ly­de­co in adult pa­tients who have one copy of the F508del mu­ta­tion and a se­cond mu­ta­tion that re­sults in a gat­ing de­fect in the CFTR pro­tein. [more in­for­ma­tion]
Nordic Nanovector ASA [NANO.OL]
Lead as­set Be­ta­lutin, a nov­el an­ti-CD37 ARC (Luteti­um-177) ra­dioim­munother­a­peu­tic for the treat­ment of Non-Hodgkin Lym­pho­ma, en­ter­ing piv­o­tal Ph 2 PARADIGME trial in 4Q17. In­ter­im NHL da­ta showed 64% ORR, 28% CR, sus­tained du­ra­tion of re­sponse and fa­vor­able side ef­fect pro­file. Con­ve­nient ready-to-use sin­gle in­jec­tion for­mu­la­tion, can be ad­min­is­tered by the nurse with pa­tient go­ing home 15 min lat­er. [more in­for­ma­tion]
OncoMed Pharmaceuticals [OMED] $34 MM MCap
- TIG­IT: ini­tial clin­i­cal da­ta in 2H18 - whol­ly-owned GITRL-Fc trimer with ini­tial da­ta in 2019 - navi­cix­izumab up­dat­ed Ph 1/2 in 2H18 [more in­for­ma­tion]
Oryzon Genomics [ORY]
Tar­get­ing ly­sine spe­cif­ic demethy­lase 1 (LS­D1, aka KD­M1A). Lead CNS com­pound in 3 Phase 2a trials in MS, AD and bas­ket trial. Onc com­pound in 2 com­bo Phase 2a trials in AML and SCLC. Da­ta exp mid-2019 [more in­for­ma­tion]
OticPharma, Inc.
De­vel­op­ing prod­ucts for ear, nose, and throat (ENT) di­s­or­ders with two core tech­nolo­gies that both have broad ap­pli­ca­tion. First tech­nol­o­gy is a Sur­fac­tant-based nasal prod­uct en­ter­ing Ph1 for acute oti­tis me­dia, which has po­ten­tial to re­place tym­panos­to­my tubes. Se­cond tech­nol­o­gy is a foam-based steroid-free antibi­ot­ic for acute oti­tis ex­ter­na (“swim­mers ear”). [more in­for­ma­tion]
Remi­ma­zo­lam: short-act­ing anes­thet­ic. Ph 3 study in pro­ce­du­ral se­da­tion in US. Safe­ty da­ta in ASA III/IV pa­tients in Ph 3 colonos­copy study and Ph 3 bron­chos­copy study. US ap­pro­val filed by part­n­er Cos­mo Phar­ma­ceu­ti­cals in June 2019. Ini­ti­at­ed Ph 3 in gen­er­al anes­th­e­sia in EU in Ju­ly 2018, exp com­ple­tion 2020. [more in­for­ma­tion]
Perrigo Company [PRGO] $4,326 MM MCap
Per­ri­go Com­pany is ded­i­cat­ed to mak­ing lives bet­ter by bring­ing high Qual­i­ty, Af­ford­able Self-Care Prod­ucts™ that con­sumers trust ev­ery­where they are sold. The Com­pany is a lead­ing provider of over-the-coun­ter health and well­ness so­lu­tions that en­hance in­di­vi­d­u­al well-be­ing by em­pow­er­ing con­sumers to proac­tive­ly pre­vent or treat con­di­tions that can be self-ma­n­aged. Vis­it Per­ri­go on­line at (http://www.per­ri­ [more in­for­ma­tion]
PharmaMar SA [PHM:ES]
Zepzel­ca (lur­binecte­din), op­ti­mized ana­log of Yon­delis, for re­lapsed small cell lung can­cer. Signed li­cens­ing agree­ment with Jazz Phar­ma for lur­binecte­din in the US - up­front pay­ment of $200M, $100mm on ap­pro­val and up to $550M sales mile­s­tones. Zepzel­ca was launched by Jazz Ju­ly 2020. [more in­for­ma­tion]
Pharming Group [PHAR] $748 MM MCap
Build­ing a sus­tain­able rare dis­ease busi­ness, pre­par­ing for ap­pro­val/launch of le­ni­olis­ib for APDS. Rev­enues >$200M (TTM) from Ru­con­est (mar­ket­ed) for the treat­ment of acute hered­i­tary an­gioede­ma (HAE) at­tacks in­clud­ing break­through at­tacks. Le­ni­olis­ib, a tar­get­ed PI3K&del­ta; in­hibi­tor ini­tial­ly be­ing de­vel­oped for PI3K del­ta syn­drome (APDS), is on track to be the first ap­proved dis­ease-mod­i­fy­ing treat­ment for APDS, a rare ge­net­ic im­mun­od­e­fi­cien­cy, in Q1 2023 (FDA) and H1 2023 (EMA). Du­al list­ed on Nas­daq. [more in­for­ma­tion]
PhaseBio Pharmaceuticals, Inc. [PHAS]
PB1046 lev­er­ages pro­pri­e­tary elastin-like po­lypep­tide (ELP) biopo­ly­mer tech­nol­o­gy plat­form to de­vel­op ther­a­pies with po­ten­tial for less-fre­quent dos­ing and bet­ter pa­tient com­pliance. [more in­for­ma­tion]
Photocure [PHCUF] $136 MM MCap
Lead prod­uct Hexvix®/Cysview® im­proves de­tec­tion of blad­der can­cer un­der blue light cys­tos­copy (BLC). Ap­proved in over 30 coun­tries, Hexvix®/Cysview® is di­rect­ly sold in the US and EU, and through world­wide part­n­er­ships. Pho­to­cure ex­pects up to $250M from li­cense of non-core as­set CE­VI­RA® to Asieris. Head­quar­tered in Os­lo, Nor­way, Pho­to­cure is 20% owned by US in­ves­tors (sig­ni­f­i­cant in­crease over past few years) and com­mer­cial­ly fo­cused on US mar­ket. [more in­for­ma­tion]
Pieris Pharmaceuticals [PIRS] $29 MM MCap
Lead pro­gram (AZN-part­nered) PRS-060, is an in­haled IL4Ra in­hibi­tor for mod­er­ate-to-se­vere asth­ma, with Phase 2 to­p­line da­ta ex­pect­ed by 3Q23; Phase 1/2 en­roll­ment cont­in­ues for Servi­er-part­nered PRS-344 (4-1BB/PD-L1 bis­pe­cif­ic) for solid tu­mors. [more in­for­ma­tion]
Pixium Vision SA [PIX:PA]
De­vel­op­ing reti­nal im­plant sys­tems to re­s­tore vi­sion us­ing sen­so­ry neu­ro­mo­d­u­la­tion. IRIS II, an epi-reti­nal im­plant tar­get­ing re­tini­tis pig­men­tosa, pro­vides po­ten­tial­ly su­pe­ri­or vi­sion and surgery ben­e­fits; CE Mark ap­proved Ju­ly 2016 and EU com­mer­cial launch un­der­way. Clin­i­cal trials for the sub-reti­nal PRI­MA sys­tem for dry age-re­lat­ed mac­u­lar ede­ma (dry AMD) ex­pect­ed in mid 2017. [more in­for­ma­tion]
Poxel SA [POXEL:PA] €14 MM MCap
PXL770: di­rect AMPK ac­ti­va­tor en­ter­ing Phase 2a for NASH in Q418. PXL065 (deu­teri­um-sta­bi­l­ized R-pi­ogl­i­ta­zone), mi­to­chon­drial pyru­vate car­ri­er (MPC), in Ph 1 for treat­ment of NASH. [more in­for­ma­tion]
ProQR Therapeutics NV [PRQR] $105 MM MCap
Dys­troph­ic epi­der­mol­y­sis bul­losa (DEB) Ph 1 / 2 trial to start in 2018; in­ter­im da­ta in 2018, full re­sults in ear­ly 2019. QR-421 for Ush­er syn­drome: in­ter­im and full da­ta in 2019. Re­search col­lab. [more in­for­ma­tion]
Protagonist Therapeutics [PTGX] $960 MM MCap
Pro­pri­e­tary pep­tide tech­nol­o­gy plat­form to de­vel­op oral tar­get­ed ther­a­py-based drugs and in­jecta­bles for rare dis­eas­es. PTG-300 in­jectable hep­cidin mimet­ic Ph 2 trials on­go­ing in po­ly­cythemia ve­ra and hered­i­tary he­mochro­ma­to­sis; pos­i­tive up­dat­ed Ph 2 re­sults in PV pre­sent­ed at ASH 2020. PN-943 oral GI-re­strict­ed al­pha-4-be­ta-7 an­ta­g­on­ist Ph 2 in ul­cer­a­tive coli­tis study sites ac­ti­vat­ed; PTG-200, part­nered w/ Janssen, oral GI-re­strict­ed IL-23R an­ta­g­on­ist Ph 2 Crohn’s dis­ease study on­go­ing. [more in­for­ma­tion]
Protalix BioTherapeutics [PLX] $119 MM MCap
Lead as­set in mul­ti Ph 3 trials for Fab­ry dis­ease, part­nered glob­al­ly w Chie­si - In­ter­im Ph 3 (PRX-102) da­ta: im­prove­ment in kid­ney func­tion in pts switched from Re­pla­gal®. Ad­dl Ph 2 (in Cys­tic Fi­bro­sis) [more in­for­ma­tion]
Reata Pharmaceuticals, Inc. [RETA] $6,576 MM MCap
De­vel­op­ing an­tioxi­dant in­flam­ma­tion mo­d­u­la­tors [AIMs] that tar­get cel­lu­lar en­er­get­ics & in­flam­ma­tion reg­u­la­tion via mi­to­chon­dria. Bar­dox­olone methyl in Ph3 for CTD-PAH and Ph2 for PH-ILD and Al­ports Syn­drome. Omav in Ph2 for Frei­drich's Ataxia and Mi­to­chon­drial My­o­pa­thy. [more in­for­ma­tion]
RedHill Biopharma [RDHL] $3 MM MCap
The com­pany pro­motes gas­troin­testi­nal drugs, in­clud­ing Mo­van­tik for opi­oid-in­duced con­sti­pa­tion in adults, Tali­cia for the treat­ment of He­li­cobac­ter py­lori in­fec­tion in adults, and Aem­co­lo for the treat­ment of trav­el­ers' diar­rhea in adults. Its key clin­i­cal late-stage de­vel­op­ment pro­grams in­clude RHB-104 for Crohn's dis­ease; RHB-204, for pul­mo­nary non­tu­ber­cu­lous my­cobac­te­ria in­fec­tions; RHB-102 (Bekin­da) with pos­i­tive re­sults from a first Phase 3 study for acute gas­troen­teri­tis and gas­tri­tis; Opa­ganib (Ye­li­va); RHB-106, an en­cap­su­lat­ed bow­el pre­pa­ra­tion and RHB-107, a Phase 2-stage serine pro­tease in­hibi­tor tar­get­ing can­cer and in­flam­ma­to­ry gas­troin­testi­nal dis­eas­es. [more in­for­ma­tion]
ReNeuron Group plc [RENE:LN]
Clin-stage, UK-based cell ther­a­py; al­lo­gene­ic, off-the-shelf can­di­dates: chron­ic stroke dis­a­bil­i­ty (Ph3 de­sign ap­proved by FDA); re­tini­tis pig­men­tosa (Ph1/2 on­go­ing), cone rod dys­tro­phy (Ph2 ini­ti­a­tion); PC ex­o­some nanomedicine plat­form pro­gram, first in­di­ca­tion GMBM. GMP com­mer­cial scale cell pro­duc­tion ca­pa­bil­i­ty. [more in­for­ma­tion]
Repros Therapeutics Inc. [RPRXW]
De­vel­op­ing Proellex, an oral de­liv­er­able, in Ph 2b to treat uterine fi­broids and en­dometrio­sis - ex­pect­ing to com­plete Ph 2b in Q2 2017; Re­pros al­so de­vel­op­ing An­drox­al in Ph3 to treat se­condary hy­pog­o­nadism which re­port­ed pos­i­tive top-line da­ta from all it’s cur­rent 4 Phase III trials ZA-301, 302, 304, and 305. The com­pany ini­ti­at­ed the for­mal ap­pro­val pro­cess for ob­tain­ing mar­ket­ing au­tho­riza­tion by the Eu­ro­pean Medicines Agen­cy (EMA). [more in­for­ma­tion]
Rigel Pharmaceuticals [RIGL] $188 MM MCap
Fo­cused on he­ma­to­log­ic di­s­or­ders, can­cer, and rare im­mune dis­eas­es. Rigel's first FDA ap­proved prod­uct is TA­VALISSE® (fos­ta­ma­tinib di­s­odi­um hex­ahy­drate) tablets, the on­ly oral spleen ty­ro­sine ki­nase in­hibi­tor for the treat­ment of chron­ic im­mune throm­bo­cy­tope­nia. The Com­pany is al­so tar­get­ing SYK in Warm Anti­body AI­HA and COVID-19, in­ter­leukin re­cep­tor-as­so­ci­at­ed ki­nas­es 1&4 in on­col­o­gy and im­mune dis­eas­es, and re­cep­tor-in­ter­act­ing serine/thre­o­nine-pro­tein ki­nase 1 in CNS dis­eas­es. [more in­for­ma­tion]
Stemline Therapeutics, Inc. [STML] $621 MM MCap
Stem­line Ther­a­peu­tics is a clin­i­cal stage bio­phar­ma­ceu­ti­cal com­pany de­vel­op­ing on­col­o­gy com­pounds that tar­get can­cer stem cells. [more in­for­ma­tion]
Au­to­ma­tion sys­tems and soft­ware for di­ag­nos­tics in­dus­try. In­s­talled base of >13,000 sys­tems across 20 fam­i­lies in­clud­ing PAN­THER (Ho­log­ic), Li­a­son XL (Di­a­Sorin), VI­DAS (BioMerieux) and AD­VIA (Sie­mens). Rev gen­er­at­ing and pro­f­itable; growth drivers in­cl prod­uct launch­es/ramp-ups, new tests; de­mo­graph­ics and out­sourc­ing in IVD in­dus­try. [more in­for­ma­tion]
Strongbridge Biopharma [SBBP] $136 MM MCap
Rare dis­eas­es; Keveyis for PPP revs ~21.7M for 2019 and $26 - $27M for 2020. Re­cor­lev for en­doge­nous Cush­ing's syn­drome Ph 3 pub­lished with to­p­line place­bo-con­trolled Ph 3 3Q20. [more in­for­ma­tion]
Sutro Biopharma [STRO] $210 MM MCap
Clin­i­cal-stage on­col­o­gy com­pany pi­oneer­ing site-spe­cif­ic and nov­el-for­mat anti­body drug con­ju­gates (ADCs). Sutro has two whol­ly owned ADCs in the clin­ic—STRO-002, a fo­late re­cep­tor al­pha (FolR&al­pha;)-tar­get­ing ADC, in clin­i­cal studies for ovarian and en­dome­trial can­cers; and STRO-001, a CD74-tar­get­ing ADC, in clin­i­cal studies for B-cell ma­lig­nan­cies. STRO-003, an op­ti­mal­ly de­signed ROR1-tar­get­ing ADC, is an­ti­ci­pat­ed to be the next pro­pri­e­tary prod­uct can­di­date to move in­to clin­i­cal studies. [more in­for­ma­tion]
Symic Bio
De­vel­op­ing a new ther­a­peu­tic cat­e­go­ry of bio­con­ju­gates that di­rect­ly tar­get the ex­tra­cel­lu­lar ma­trix (ECM). Two clin­i­cal Phase 1/2a can­di­dates which in­clude pre­ven­tion of pe­ripher­al vein graft fail­ure (SB-030) and pain + dis­ease mod­i­fi­ca­tion in the treat­ment of os­teoarthri­tis (SB-061). [more in­for­ma­tion]
Targovax [TRVX]
Pre­lim­i­nary Ph1b da­ta of OV ON­COS-102+che­mo in me­sothe­lio­ma re­sults: com­bo mPFS=8.4 mos. vs. con­trol mPFS=6.8 mos.; 1L pts mPFS-8.9 mos. Up­com­ing mile­s­tones: up­dat­ed clin­i­cal & im­mune da­ta in Ph2 me­sothe­lio­ma (1H20); pre­clin­i­cal da­ta on next-gen OV w/ dou­ble trans­genes (1H20); and clin­i­cal da­ta from Ph1 me­lano­ma study, part 2 with ex­tend­ed dos­ing (2H20). [more in­for­ma­tion]
Theravance Biopharma [TBPH] $456 MM MCap
Reve­fe­nacin, a ne­b­u­l­ized QD LA­MA for COPD, re­port­ed pos­i­tive Ph3 da­ta in Oc­to­ber, and will re­port long-term safe­ty da­ta in 1H 2017. TD-0714, a ne­prolysin (NEP) in­hibi­tor, is ex­pect­ed to re­port Ph1 MAD da­ta by the end of 2016. TD-1473, a pan-JAK in­hibi­tor for ul­cer­a­tive coli­tis, ini­ti­at­ed its Ph1b trial in ear­ly Q4 and is ex­pect­ed to re­port top-line da­ta in 1H 2017. [more in­for­ma­tion]
Tocagen Inc. [TOCA]
Toca­gen is de­vel­op­ing prod­uct can­di­date, To­ca 511 and To­ca FC, for the treat­ment of re­cur­rent high grade glio­ma, or HGG, a brain can­cer with limit­ed treat­ment op­tions, low sur­vi­val rates and, there­fore, a sig­ni­f­i­cant un­met med­i­cal need. Ge­o­graph­i­cal­ly, the ac­tiv­i­ties are car­ried out through Unit­ed States. [more in­for­ma­tion]
Vericel Corporation [VCEL] $1,597 MM MCap
Ad­vanced cell ther­a­py; mar­ket­ing MA­CI (car­ti­lage re­pair) and Epi­cel (skin re­place­ment) in TAMs of $2b+. Q3 MA­CI and Epi­cel revs of $31.1m. Re­port­ed re­cord third quar­ter MA­CI rev­enue and to­tal rev­enues, and the se­cond high­est quar­ter­ly Epi­cel rev­enue in his­to­ry. Nexo­Brid PD­U­FA date of June 29, 2021. [more in­for­ma­tion]
Vidac Pharma
This tech­nol­o­gy holds a promise of de­liv­er­ing first-in-class drugs that are both ef­fi­ca­cious and well tol­er­at­ed. [more in­for­ma­tion]
Vital Therapies, Inc. [VTL]
The Com­pany's ELAD Sys­tem is an ex­tra­cor­po­re­al hu­man al­lo­gene­ic cel­lu­lar liv­er ther­a­py cur­rent­ly be­ing eval­u­at­ed in a phase 3 clin­i­cal trial for se­vere al­co­holic he­p­ati­tis called VTL-308. To­p­line re­sults from VTL-308 are ex­pect­ed around mid-2018, like­ly in the third quar­ter. [more in­for­ma­tion]
Vivoryon [VVY:AS] €293 MM MCap
De­vel­ops small molecule medicines that mo­d­u­late the ac­tiv­i­ty and sta­bil­i­ty of patho­log­i­cal­ly al­tered pro­teins. Lead can­di­date varog­lu­tam­s­tat, cur­rent­ly in VIVI­AD EU Ph2b and VI­VA-MIND US Ph2a/2b studies in Alzheimer’s dis­ease (AD), tar­gets all three ma­jor hall­marks of AD (Abe­ta pathol­o­gy, tau pathol­o­gy, neu­roin­flam­ma­tion) and sy­nap­tic im­pair­ment. [more in­for­ma­tion]
Bio­phar­ma­ceu­ti­cal com­pany de­vel­op­ing in­no­va­tive, next-gen­er­a­tion ther­a­pies to ad­dress un­met needs in obe­si­ty, di­a­betes, sleep ap­nea and sex­u­al health for US, Eu­ro­pean and other world mar­kets. Qsymia, for the treat­ment of obe­si­ty, is mar­ket­ed in the US through a di­rect sales­force. Sten­dra for ED is mar­ket­ed in both the US and EU through part­n­er­ships with En­do and Me­nari­ni. [more in­for­ma­tion]
vTv Therapeutics, Inc. [VTVT] $50 MM MCap
June 2019 pos­i­tive da­ta for TT­P399 as an add-on ther­a­py to in­sulin ther­a­py in a 12 week phase 1/2 trial in pa­tients with T1D. Re­sults from the se­cond part of the Ph2 study ex­pect­ed Q1 2020.&nb­sp;Re­cent­ly an­nounced the first pa­tient has been screened for the Ph2 proof of con­cept study eval­u­at­ing the safe­ty and ef­fi­ca­cy of azeli­ra­gon in pa­tients with mild Alzheimer's dis­ease and Type 2 Di­a­betes. [more in­for­ma­tion]
Wilson Therapeutics [WTX]
Fo­cused on rare dis­eas­es. Ex­pects to ad­vance WTX101, a nov­el first-in-class cop­per-pro­tein-bind­ing agent for treat­ment of Wil­son Dis­ease in­to piv­o­tal Phase 3 FO­CuS study in ear­ly 2018; grant­ed OD in US/EU and FDA fast track sta­tus. WTX101 al­so grant­ed OD for ALS in US; plan to in­vesti­gate in SOD-1 mu­tat­ed ALS. [more in­for­ma­tion]