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
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. [more in­for­ma­tion]
Abide Tx
Serine hy­dro­lase-tar­get­ed ther­a­peu­tics har­ness­ing the en­do­cann­abi­noid sys­tem to ad­dress neu­ro­log­i­cal and pain di­s­or­ders w. limit­ed or no treat­ment op­tions. Ph 2: Tourette Syn­drome on­go­ing with da­ta ex­pect­ed in 1Q20; Ph 1b: neu­ro­path­ic pain com­plete, with plans to ini­ti­ate Ph 2 in 1H19. [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]
Abpro [ABP]
Lead prod­uct, ABP-100, is a HER2/CD3 bis­pe­cif­ic anti­body, tar­get­ing HER2-high and in­ter­me­di­ate breast, ovarian, gas­tric and en­dome­trial can­cers. Se­cond prod­uct, ABP-200, is a VEGF/ANG-2 bis­pe­cif­ic anti­body for wet age-re­lat­ed mac­u­lar de­gen­er­a­tion, DME, and on­col­o­gy with an IND ex­pect­ed H1 2019. [more in­for­ma­tion]
Acceleron Pharma Inc [XLRN] US$2,230 MM MCap
Lead ther­a­peu­tic can­di­date in he­ma­tol­o­gy, lus­pa­ter­cept, un­der a glob­al part­n­er­ship with Cel­gene, re­port­ed pos­i­tive Phase 3 ME­DAL­IST and BE­LIEVE trial re­sults, and cont­in­ues to ad­vance mul­ti­ple Ph2 and Ph3 trials for the treat­ment of chron­ic ane­mia in myelodys­plas­tic syn­dromes, be­ta-tha­lassemia, and myelo­fi­bro­sis. [more in­for­ma­tion]
AcelRx Pharmaceuticals, Inc. [ACRX] US$215 MM MCap
Re­cent­ly launched DSU­VIA; rev-gen­er­at­ing in Q1. Plans to re­sub­mit Za­lvi­so NDA in US by YE [more in­for­ma­tion]
Achillion Pharmaceuticals, Inc. [ACHN] US$336 MM MCap
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] US$165 MM MCap
Launched GO­COV­RI™ for LID in pa­tients w/ Parkin­son’s dis­ease. 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 PLC [ADAP] US$342 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]
Affimed N.V. [AFMD] US$197 MM MCap
Mo­d­u­lar ROCK™plat­form en­abling mul­ti-spe­cif­ic NK / T-cell bi-and tri-spe­cif­ic Abs. Aug 2018 deal w Ge­nen­tech ($96M up­front / $5B po­ten­tial). – AFM13 in P1b (com­bo) in HL and P2a(mono) in CD30+lym­pho­ma [more in­for­ma­tion]
Allecra Therapeutics
En­me­ta­zobac­tam is an ex­tend­ed-spec­trum ß-lac­ta­mase in­hibi­tor be­long­ing to the peni­cil­lan­ic acid sul­fone class that ex­hibits po­tent in vit­ro and in vi­vo ac­tiv­i­ty against many re­sis­tant Gram-neg­a­tive patho­gens. [more in­for­ma­tion]
Amarin Corporation [AMRN] US$5,979 MM MCap
Vas­ce­pa: 1st 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 [more in­for­ma­tion]
Amphivena Therapeutics, Inc.
MPM Cap­i­tal, Am­phive­na’s ma­jor share­hold­er, led a $19.5 M Se­ries A fi­nanc­ing, with par­ti­ci­pa­tion by Aeris Cap­i­tal and Af­fimed GmbH. More re­cent­ly, funds ma­n­aged by Tek­la Cap­i­tal Ma­n­age­ment LLC joined the found­ing in­ves­tors in a Se­ries B fi­nanc­ing. [more in­for­ma­tion]
Anavex Life Sciences Corp. [AVXL] US$155 MM MCap
The anal­y­sis of RNA and whole ex­ome DNA genome se­quenc­ing da­ta has char­ac­ter­ized sev­er­al ge­nom­ic al­ter­a­tions in well-char­ac­ter­ized tar­gets that can be used as bio­mark­ers to iden­ti­fy op­ti­mal pa­tients in forth­com­ing clin­i­cal trials (Rett, Alzheimer’s, and Parkin­son’s). [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] US$57 MM MCap
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]
Ascentage Pharma
Glob­al­ly-fo­cused clin­i­cal-stage bio­phar­ma­ceu­ti­cal com­pany de­vel­op­ing first- and best-in-class tar­get­ed ther­a­peu­tics for can­cers, he­p­ati­tis B & age-re­lat­ed dis­eas­es. As­cen­t­age is a lead­er in the de­sign of small molecules ther­a­peu­tics for tar­get­ing pro­tein-pro­tein in­ter­ac­tions in­clud­ing Bcl-2, IAP and MD­M2-p53. The com­pany has 6 prod­uct can­di­dates in clin­i­cal de­vel­op­ment. [more in­for­ma­tion]
Asklepion Pharmaceuticals
Pri­vate, fo­cused on the de­vel­op­ment of IV Cit­rul­line for rare pe­di­a­tric dis­eas­es. First in­di­ca­tion, the pre­ven­tion of sig­ni­f­i­cant se­que­lae of pe­di­a­tric acute car­diopul­mo­nary by­pass-in­duced lung in­jury, Ph 3 trial is on­go­ing. [more in­for­ma­tion]
Assembly Biosciences, Inc. [ASMB] US$333 MM MCap
The mi­cro­biome pro­gram is de­vel­op­ing nov­el oral live mi­cro­bial bio­ther­a­peu­tic can­di­dates with As­sem­b­ly’s ful­ly in­te­grat­ed plat­form, in­clud­ing a ro­bust pro­cess for strain iden­ti­fi­ca­tion and se­lec­tion, GMP bank­ing and pro­duc­tion, and tar­get­ed de­liv­ery to the low­er gas­troin­testi­nal tract with the GEMI­CEL® tech­nol­o­gy. [more in­for­ma­tion]
Aurinia Pharmaceuticals, Inc. [AUPH] US$578 MM MCap
De­vel­op­ing vo­clos­porin ad­dress­ing lu­pus nephri­tis (LN), fo­cal seg­men­tal glomeru­los­cle­ro­sis (FS­GS) & dry eye syn­drome (DES). - Dry eye top-line da­ta: ear­ly Q1 19 - LN top line da­ta: late Q4 19 - FS­GS da­ta: open la­bel da­ta: 2H 19 [more in­for­ma­tion]
AxoGen [AXGN] US$761 MM MCap
Da­m­age or tran­sec­tion to pe­ripher­al nerves. Q1 revs $23.3M, 35% growth YoY; exp 2019 rev bt $109-$114M [more in­for­ma­tion]
Bavarian Nordic A/S [BVNRY] US$854 MM MCap
Vaccines for in­fec­tious dis­eas­es and on­col­o­gy. Can­cer vaccines in com­bo with mul­ti­ple check­point in­hibi­tors (Roche/Mer­ck/BMS). Uni­ver­sal RSV vaccine with pos­i­tive phase 2 da­ta. Re­cent con­tract from US Gov. for small­pox vaccine val­ued at over $500M. Part­n­er­ship with JNJ for vaccines against HIV/HBV/HPV with over $1B in po­ten­tial mile­s­tones. [more in­for­ma­tion]
Bellicum Pharmaceuticals Inc. [BLCM] US$74 MM MCap
Molec­u­lar switch tech­nol­o­gy and co-stim do­mains en­ables phar­ma­co­log­ic con­trol over cells for en­hanced ef­fi­ca­cy and safe­ty. Lead (reg­is­tra­tio­n­al EU) pro­gram: Ph2 for ri­vo-cel (al­pha/be­ta ad­d­back T-cell ther­a­py for post al­lo­gene­ic HSTC in non-ma­lig­nant and ma­lig­nant dis­eas­es). Pos­i­tive late in­ter­im anal­y­sis on ri­vo-cel exp at ASH 2018; [more in­for­ma­tion]
Biohaven Pharmaceuticals, Inc [BHVN] US$2,284 MM MCap
Lead de­vel­op­ment pro­grams in­clude mul­ti­ple com­pounds across its CGRP re­cep­tor an­ta­g­on­ist and glu­ta­mate mo­d­u­la­tion plat­forms. In­ter­nal de­vel­op­ment and re­search with in­tel­lec­tu­al prop­er­ty li­censed from com­pa­nies and in­sti­tu­tions in­clud­ing BMS, As­traZene­ca, Yale Uni­ver­si­ty, Ca­ta­l­ent, ALS Bio­phar­ma and Mas­sachusetts Gen­er­al Hos­pi­tal. [more in­for­ma­tion]
BioPharmX [BPMX] US$5 MM MCap
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]
Camurus AB [CAMX:ST] SKr3,502 MM MCap
De­vel­op­ing/com­mer­cial­iz­ing in­no­va­tive/long-act­ing medicines for se­vere & chron­ic con­di­tions (e.g. opi­oid de­pen­dence, pain, can­cer & en­docrine di­s­or­ders). Fluid­Crys­tal® drug de­liv­ery tech­nolo­gies for im­proved Q of life, treat­ment out­comes and re­source uti­l­iza­tion. [more in­for­ma­tion]
Catabasis Pharmaceuticals Inc [CATB] US$82 MM MCap
Rare dis­ease pipe­line, with lead prod­uct Edasa­lonex­ent pre­par­ing for Ph 3 trial in Duchenne mus­cu­lar dys­tro­phy re­gard­less of mu­ta­tion type. Start Ph 3 trial in H1 2018 with North Star Am­bu­la­to­ry As­sess­ment as the pri­mary end­point based on Phase 2 re­sults and FDA in­put. Top-line re­sults ex­pect­ed in 2020. CAT-5571 ad­vanc­ing for cys­tic fi­bro­sis; Ph 1 to start 2H 2018. [more in­for­ma­tion]
Catalyst Pharmaceuticals, Inc. [CPRX] US$417 MM MCap
Fir­dapse ap­proved 11/18, Q1 sales of $12m: 380 ac­tive LEMS pa­tients on ther­a­py (a/o May 9) [more in­for­ma­tion]
Cellectis SA [CLLS] US$648 MM MCap
Lead in­ter­nal pro­gram UCART123, an off-the-shelf CAR-T ther­a­peu­tic, in Ph 1 for the treat­ment of AML and BPD­CN. Al­lo­gene / Servi­er-part­nered UCART19 off-the-shelf CAR-T is cur­rent­ly be­ing studied in Ph 1 studies for the treat­ment of pe­di­a­tric and adult ALL. [more in­for­ma­tion]
Cibus [CBUS]
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]
CTI BioPharma [CTIC] US$45 MM MCap
Lead can­di­date pa­cri­tinib, oral ki­nase in­hibi­tor w spe­ci­fic­i­ty for JAK2, FLT3, IRAK1, and CS­F1R in Ph 2 for treat­ment of myelo­fi­bro­sis. Up­com­ing ca­t­alysts: pa­cri­tinib PAC203 Ph 2 da­ta - mid'19, ini­ti­a­tion of ph3 - Q3. [more in­for­ma­tion]
CureVac AG
Found­ing Co of mR­NA space (last round $1bn+). In­tra­tu­mo­ral ther­a­py Ph1 (mul­ti­ple onc in­di­ca­tions) ini­ti­at­ed Q4 17, ear­ly da­ta rep at SITC. First pt en­rolled Ph 1 pro­phy­lac­tic mR­NA ra­bies vax Oct 2018. Pro­phy­lac­tic in­fluen­za vax ini­ti­a­tion Ph 1 2020, OTC de­fi­cien­cy Ph 1 ini­ti­a­tion H219. In­ter­im da­ta /all trials: 2019. [more in­for­ma­tion]
CytomX Therapeutics, Inc. [CTMX] US$555 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. BMS-part­nered Pro­body of ip­il­i­mumab be­ing studied in on­go­ing Phase 1/2 trial in monother­a­py and in com­bi­na­tion with nivolumab. [more in­for­ma­tion]
Enzo Biochem, Inc. [ENZ] US$177 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] US$71 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]
FivePrime Therapeutics [FPRX] US$202 MM MCap
Five Prime's com­pre­hen­sive dis­cov­ery plat­form, which en­com­pass­es vir­tu­al­ly ev­ery med­i­cal­ly rel­e­vant ex­tra­cel­lu­lar pro­tein, po­si­tions it to ex­plore path­ways in can­cer, in­flam­ma­tion and their in­ter­sec­tion in im­muno-on­col­o­gy, an area with sig­ni­f­i­cant ther­a­peu­tic po­ten­tial and a grow­ing fo­cus of the com­pany's R&D ac­tiv­i­ties. [more in­for­ma­tion]
Flex Pharma, Inc. [FLKS] US$0 MM MCap
Pos­i­tive da­ta in Ph2 ex­plo­ra­to­ry study in MS pa­tients - sta­tis­ti­cal­ly sig­ni­f­i­cant 27.3% re­duc­tion in the fre­quen­cy of cramps/spasms com­pared with con­trol. Ph 2 trials on­go­ing in ALS and Char­cot-Marie-Tooth with da­ta ex­pect­ed in ear­ly 2019. MoA is based up­on nov­el in­sights in neu­ro­mus­cu­lar phy­si­ol­o­gy (ion and TRP bi­ol­o­gy) from No­bel Prize win­n­ers. [more in­for­ma­tion]
Forward Pharma A/S [FWP] US$45 MM MCap
The Com­pany grant­ed to Bio­gen an ir­re­vo­ca­ble li­cense to all of its IP through the Settle­ment and Li­cense Agree­ment and re­ceived from Bio­gen a non-re­fund­able cash fee of $1.25 bil­lion in Fe­bruary 2017, with the re­turn of EUR 917.7 mil­lion to share­hold­ers through a cap­i­tal re­duc­tion in Septem­ber 2017. [more in­for­ma­tion]
Genfit SA [GNFTF] US$758 MM MCap
$155m Mar 2019 US IPO; FDA BTD for Ela­fi­branor (4/19); (+) 36-mo DSMB rec for cont­in­u­a­tion of Ph 3 study in NASH; da­ta by YE [more in­for­ma­tion]
GenSight Biologics [SIGHT:PA] €48 MM MCap
Gene ther­a­py: reti­nal & CNS de­gen­er­a­tive patholo­gies and mi­to­chon­drial dis­ease. Fil­ing for MAA exp Q1 2020 in EU and Q4 2020 in US. 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 Corporation [GERN] US$229 MM MCap
-Ime­tel­s­tat is be­ing eval­u­at­ed in an on­go­ing Phase II trial (IM­bark) in re­lapsed/re­frac­to­ry MF, and a Phase II/III trial (IMerge) in low­er risk MDS. -GERN has part­nered with Janssen Biotech, Inc. to de­vel­op and com­mer­cial­ize ime­tel­s­tat. [more in­for­ma­tion]
Glycotope
The com­bi­na­tion of Gatipo­tuzumab (former­ly known as PankoMab-GEX™, an­ti TA-MUC1 anti­body) with To­mu­zo­tux­imab (former­ly known as Ce­tuGEX™, an­ti EGFR antin­ody) or a mar­ket­ed an­ti EGFR anti­bodies is based on a strong pre­clin­i­cal ra­tio­nale and pre­lim­i­nary clin­i­cal evi­dence from the first 20 pa­tients en­rolled in the study Next to the clin­i­cal pro­gram the Com­pany’s pipe­line in­cludes pre­clin­i­cal anti­body bio­phar­ma­ceu­ti­cals for vari­ous on­co­log­i­cal in­di­ca­tions in­clud­ing the bis­pe­cif­ic PankoMab PM-CD3 and PankoMab PM-IL 15. [more in­for­ma­tion]
GW Pharmaceuticals PLC [GWPH] US$5,100 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]
Hutchison China MediTech Ltd ("Chi-Med") [HCM] US$2,777 MM MCap
It has a port­fo­lio of eight can­cer drug can­di­dates cur­rent­ly in late stage clin­i­cal studies around the world. In Novem­ber 2018, fruquin­tinib (Elu­nate) was com­mer­cial­ly launched in Chi­na for the treat­ment of pa­tients with me­tastat­ic col­orec­tal can­cer (CRC). [more in­for­ma­tion]
Idera Pharmaceuticals, Inc. [IDRA] US$71 MM MCap
Lead can­di­date Til­so­toli­mod (TLR 9 ag­on­ist) used in comb w Ipi in PD1 re­frac­to­ry me­lano­ma pts. En­rolling ad­dl pts in Ph 2 ex­pan­sion and Phase 3 en­roll­ment ex­pect­ed to com­plete Q4 2019. Com­bo of ipi and Til­so­toli­mod ap­pears to ac­ti­vate im­mune re­sponse in pts who have ex­haust­ed all op­tions, e.g. PD1/CPIs. Fast Track des for Til­so­toli­mod (Nov’17). [more in­for­ma­tion]
Immunomedics, Inc. [IMMU] US$2,860 MM MCap
Im­munomedics’ most ad­vanced prod­uct can­di­date is IM­MU-132 (sac­i­tuzumab govite­can), an anti­body-drug con­ju­gate that has re­ceived Break­through Ther­a­py Desig­na­tion from the FDA for the treat­ment of pa­tients with me­tastat­ic tri­ple-neg­a­tive breast can­cer who have failed at least two pri­or ther­a­pies for me­tastat­ic dis­ease. [more in­for­ma­tion]
Immutep (formerly Prima BioMed) [IMMP] US$58 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. 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]
Inspirion Delivery Sciences LLC
Mor­phaBond ER™ US com­mer­cial launch Nov 2017 (FDA-ap­proved, first sin­gle-en­ti­ty abuse de­ter­rent ER mor­phine prod­uct) and Rox­y­Bond™ (FDA ap­proved, first and on­ly IR abuse-de­ter­rent opi­oid prod­uct) both li­censed to Daiichi Sankyo, Inc. Nov­el ex­tend­ed and im­me­di­ate re­lease tech­nolo­gies en­able pain med­i­ca­tions with abuse de­ter­rent fea­tures, ad­dress­ing the Opi­oid epi­dem­ic. [more in­for­ma­tion]
Loxo Oncology, Inc. [LOXO] US$7,217 MM MCap
SOLD De­vel­ops tar­get­ed drugs for the treat­ment of can­cer in ge­net­i­cal­ly defined pa­tient pop­u­la­tions. Lead pro­gram: pur­pose built pan TRK in­hibi­tor in a Ph 2 solid tu­mor study with FDA break­through desig­na­tion. Se­cond pro­gram: RET in­hibi­tor, en­tered the clin­ic in Q2 2017. Loxo ac­quired Redx Phar­ma Plc's BTK in­hibi­tor pro­gram in Q3 2017 and ex­pects to en­ter the clin­ic in 2018. [more in­for­ma­tion]
Medigene AG [MDG1:DB] €161 MM MCap
blue­bird bio col­lab; BLUE ini­ti­at­ing trial w/MDG MAGEA-4 can­di­date in 2020. TCRs tar­get­ing HA-1 & MAGE-A1 in de­vt. . DC vax in on­go­ing Ph 1/2; fi­nal read­out in 2019. [more in­for­ma­tion]
MedinCell
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:ST] SKr936 MM MCap
Me­di­vir is a phar­ma­ceu­ti­cal com­pany fo­cus­ing on the de­vel­op­ment and com­mer­cial­iza­tion of in­no­va­tive treat­ments for can­cer. To do this ef­fi­cient­ly and with a high suc­cess rate, Me­di­vir brings to­gether a unique com­bi­na­tion of clin­i­cal de­vel­op­ment skills, a col­lab­o­ra­tive cul­ture and an ex­ten­sive in­dus­try ex­pe­ri­ence, with a bal­anced pro­ject port­fo­lio. [more in­for­ma­tion]
MEI Pharma, Inc. [MEIP] US$138 MM MCap
4 clin-stage can­di­dates, in­cl 1 in on­go­ing glob­al reg­is­tra­tion trial (pra­ci­no­s­tat), another (ME-401) en­ter­ing Ph 2; in­tends to sub­mit to FDA to sup­port ac­cel­er­at­ed ap­pro­val mar­ket­ing ap­pli­ca­tion. Can­di­dates tar­get dif­fer­ent mech­anisms crit­i­cal to over­com­ing can­cer pro­gres­sion and drug re­sis­tance. [more in­for­ma­tion]
Meves Pharmaceuticals, Inc.
Meves Phar­ma­ceu­ti­cals, Inc. is de­vel­op­ing nu­cle­o­side based ther­a­pies for mi­to­chon­drial DNA (mtD­NA) de­ple­tion syn­dromes (MDS or MDDS), a cat­e­go­ry of se­vere ge­net­ic dis­eas­es. The ini­tial fo­cus is on ther­a­py tailored spe­cif­i­cal­ly to treat pa­tients with Thymi­dine Ki­nase 2 (TK2) de­fi­cien­cy, a dis­crete mono­genic di­s­or­der as­so­ci­at­ed with pro­found skele­tal my­o­pa­thy. [more in­for­ma­tion]
MorphoSys AG [MOR] US$3,528 MM MCap
Based on its lead­ing ex­per­tise in anti­body, pro­tein and pep­tide tech­nolo­gies, Mor­phoSys, to­gether with its part­n­ers, has de­vel­oped and con­tribut­ed to the de­vel­op­ment of more than 100 prod­uct can­di­dates, of which 29 are cur­rent­ly in clin­i­cal de­vel­op­ment. [more in­for­ma­tion]
Motif Bio plc [MTFB] US$12 MM MCap
PD­U­FA date: Feb 13, 2019 for Antibi­ot­ic Iclaprim. Two Phase 3 trials in pts w acute bac­te­rial skin and skin struc­ture in­fec­tions (ABSS­SI) achieved pri­mary and se­condary end­points. Com­pared to SoC (van­comycin), no ob­served nephro­tox and a fixed dose. Po­ten­tial other in­di­ca­tions in­clude HABP/VABP and S. au­reus lung in­fec­tions in CF (or­phan). [more in­for­ma­tion]
Nanobiotix SA [NANO:PA] €211 MM MCap
Nano­bi­otix is a lead­ing, clin­i­cal-stage nanomedicine com­pany pi­oneer­ing new ap­proach­es to sig­ni­f­i­cant­ly change pa­tient out­comes by bring­ing nano­physics to the heart of the cell. Nano­bi­otix’s Im­muno-On­col­o­gy pro­gram has the po­ten­tial to bring a new di­men­sion to can­cer im­munother­a­pies. [more in­for­ma­tion]
NeuroRx
De­vel­op­ing first Glx tar­get­ed an­tide­pres­sant to ad­dress sui­ci­dal­i­ty in bipo­lar de­pres­sion. Lead can­di­date, NRX-101 in Ph 2b/3 de­vt (Break­thru Desig­na­tion, SPA and Fast Track Desig­na­tion by FDA) for Se­vere Bipo­lar De­pres­sion in pts w Acute Sui­ci­dal Idea­tion and Be­havior. Plans to pre­sent Ph 2 POC da­ta lat­er this year. [more in­for­ma­tion]
NexImmune, Inc.
Anti­gen-spe­cif­ic T cell ther­a­py. Syn­thet­ic nano­par­ti­cles de­c­o­rat­ed w a com­bo of mul­ti­ple tu­mor rel­e­vant anti­gens and co-sti­m­u­la­to­ry sig­nals en­gag­ing di­rect­ly w tar­get­ed T cells. Mul­ti­ple IND fil­ings exp H1'19. [more in­for­ma­tion]
Nordic Nanovector ASA [NANO.OL]
Fo­cused on de­vel­op­ment of Anti­body-Ra­dionu­clide-Con­ju­gates (ARC). 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. Deep pipe­line of ad­di­tio­n­al tar­get­ed ther­a­pies for hae­ma­to­log­i­cal can­cers. [more in­for­ma­tion]
Novus Therapeutics [NVUS] US$11 MM MCap
Novus Ther­a­peu­tics is a de­vel­op­ment stage phar­ma­ceu­ti­cal com­pany fo­cused on the de­vel­op­ment of prod­ucts for di­s­or­ders of the ear, nose, and throat (ENT). Novus has two tech­nolo­gies, each of which has the po­ten­tial to be de­vel­oped for mul­ti­ple ENT in­di­ca­tions. [more in­for­ma­tion]
OncoMed Pharmaceuticals [OMED] US$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 - ad­di­tio­n­al nov­el undis­closed dis­cov­ery pro­grams [more in­for­ma­tion]
Oncopeptides AB [ONCO:ST] SKr8,154 MM MCap
Melflufen (Yga­lo): alky­lat­ing pep­tide se­lec­tive­ly tar­gets MM; su­pe­ri­or ef­fi­ca­cy vs. SOC. In­ter­im da­ta at EHA (2018) from on­go­ing Ph2 study in late stage r/r MM showed ORR of ~32%. [more in­for­ma­tion]
Oryzon Genomics [ORY:MA]
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]
PAION AG [PAIOF] US$163 MM MCap
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 2019. [more in­for­ma­tion]
Perrigo Company [PRGO] US$7,023 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. [more in­for­ma­tion]
PharmaMar SA [PHM:ES]
2018 to­tal rev­enues of &eu­ro;162M. Yon­delis (soft tis­sue sar­co­ma and ovarian can­cer) com­mer­cial­ized w di­rect sales-force in EU and part­n­er­ship in US w Janssen. Zep­syre in Ph 3 trials for SCLC - da­ta due 2H19. MOA: Op­ti­mized ana­log of Yon­delis. [more in­for­ma­tion]
Pharming Group NV [PHARM:AS] €497 MM MCap
On­ly prod­uct with fu­ture po­ten­tial to be ap­proved for both pro­phy­laxis and treat­ment of at­tacks of HAE. An­nounced pos­i­tive re­sults from a ph2 study of RU­CON­EST in Con­tract-in­duced Nephro­pa­thy for pa­tients re­ceiv­ing elec­tive coro­nary an­giog­ra­phy with or with­out a per­cu­ta­neous coro­nary in­ter­ven­tion (PCI). [more in­for­ma­tion]
PhaseBio Pharmaceuticals, Inc. [PHAS] US$287 MM MCap
De­vel­op­ing ther­a­pies for treat­ment of or­phan dis­eas­es; Al­so de­vel­op­ing PB1046, a week­ly va­soac­tive in­testi­nal pep­tide re­cep­tor ag­on­ist for treat­ment of PAH. 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 ASA [PHO:OS] NKr1,250 MM MCap
Pho­to­cure is a rev­enue-gen­er­at­ing com­mer­cial stage com­pany fo­cused on urol­o­gy. The com­pany re­cent­ly an­nounced FDA ap­pro­val of sN­DA to ex­tend in­di­ca­tion to in­clude Flex­i­ble Cy­to­s­copes for on­go­ing surveil­lance of blad­der can­cer. The com­pany is fo­cused on grow­ing Cysview rev­enues in the US. [more in­for­ma­tion]
Pieris Pharmaceuticals, Inc. [PIRS] US$226 MM MCap
An­ti­calins (en­gi­neered lipo­calin pro­teins) for re­sp & on­co dis­eas­es. Im­mi­nent da­ta for AZN-part­nered PRS-060 in­haled IL4 for mod-to-se­vere asth­ma. Cash of $128MM in the bank as of March 2019. [more in­for­ma­tion]
Pliant Therapeutics, Inc.
Lead pro­gram is a small molecule du­al se­lec­tive in­hibi­tor of avb6/avb1 in­te­grins for IPF, pri­mary scle­ros­ing cholan­gi­tis (PSC), and re­nal fi­bro­sis - re­ceived or­phan drug desig­na­tion and will en­ter Ph1 in De­cem­ber 2018. Se­cond pro­gram is a se­lec­tive avb1 in­hibi­tor for the treat­ment of end stage liv­er fi­bro­sis and has en­tered in­to de­vel­op­ment can­di­date se­lec­tion. [more in­for­ma­tion]
Poxel SA [POXEL:PA] €167 MM MCap
Imeglimin (mi­to­chon­dria-based MOA) for T2D be­gan Ph 3 in Ja­pan with Asian part­n­er Su­m­i­t­o­mo Dainip­pon. Com­ple­tion of Phase 3 on track for 2019 and JN­DA sub­mis­sion in 2020. In 1Q18, an­nounced agree­ment w Roi­vant for Imeglimin (Ph 3 ready) in US, EU, and ad­dl coun­tries WW. [more in­for­ma­tion]
ProQR Therapeutics NV [PRQR] US$333 MM MCap
Ph 2 in­ter­im read­out in 2018. 2018, full 12-mo re­sults in 2019. 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. [more in­for­ma­tion]
Protagonist Therapeutics [PTGX] US$311 MM MCap
PTG-300, in­jectable hep­cidin mimet­ic in Ph 2 Be­ta-Tha­lassemia study with ini­tial re­sults ex­pect­ed 2H19. PTG-200, part­nered w Janssen, oral GI-re­strict­ed IL-23R an­ta­g­on­ist for Crohn’s dis­ease com­plet­ed Ph 1 in Novem­ber 2018. PN-943, oral GI-re­strict­ed al­pha4­be­ta7 in­te­grin-spe­cif­ic an­ta­g­on­ist in UC in Ph 1, with mul­ti­ple as­cend­ing dose re­sults ex­pect­ed 3Q19. [more in­for­ma­tion]
Protalix BioTherapeutics [PLX] US$57 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]
RedHill Biopharma [RDHL] US$230 MM MCap
Three com­pounds in Ph3 de­vel­op­ment, RHB-105 for H.Py­lori, RHB-104 for Crohn’s and BEKIN­DA for gas­troen­teri­tis and gas­tri­tis. Ad­di­tio­n­al as­sets in­clude YE­LI­VA, a SK2 se­lec­tive in­hibi­tor with a planned Ph2a study for cholan­gio­car­ci­no­ma and ME­SUPRON, a Ph2 stage pro­tease in­hibi­tor, tar­get­ing pan­cre­at­ic can­cer and in­flam­ma­to­ry gas­troin­testi­nal dis­eas­es. [more in­for­ma­tion]
ReNeuron Group plc [RENE:LN] £78 MM MCap
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]
Rigel Pharmaceuticals [RIGL] US$416 MM MCap
The com­pany's first FDA ap­proved prod­uct is TA­VALISSE™ (fos­ta­ma­tinib di­s­odi­um hex­ahy­drate), an oral spleen ty­ro­sine ki­nase (SYK) in­hibi­tor, for the treat­ment of adult pa­tients with chron­ic im­mune throm­bo­cy­tope­nia who have had an in­suf­fi­cient re­sponse to a pre­vi­ous treat­ment. [more in­for­ma­tion]
Savara Inc. [SVRA] US$94 MM MCap
Or­phan lung dis­ease; Mol­gradex, Ph 3, in­haled GM-CSF: au­toim­mune pul­mo­nary alve­o­lar pro­tei­no­sis (aPAP), Ph 2a: non­tu­ber­cu­lous my­cobac­te­ria (NTM) lung in­fec­tion [more in­for­ma­tion]
Solebury Trout
With al­most two de­cades of ex­pe­ri­ence in the biotech­nol­o­gy, phar­ma­ceu­ti­cal, med­i­cal tech­nol­o­gy and life sci­ence tech­nol­o­gy sec­tors, the Trout Group of­fers its clients the knowl­edge base need­ed to clar­i­fy in­vest­ment themes and lev­er­age key re­la­tion­ships for in­creased ex­po­sure to the prop­er au­di­ence. [more in­for­ma­tion]
Stemline Therapeutics, Inc. [STML] US$604 MM MCap
EL­ZON­RIS, tar­get­ed ther­a­py di­rect­ed to IL-3 re­cep­tor (CD123, ap­proved for BPDC; SL-801 an oral small molecule re­v­ersi­ble in­hibi­tor of XPO1 in Ph 1 trial; and SL-701 an im­munother­a­peu­tic that com­plet­ed Ph 2 trial in pts w/2nd line GBM [more in­for­ma­tion]
STRATEC Biomedical AG [SBS:FF] €628 MM MCap
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] US$153 MM MCap
Rare dis­eas­es; Keveyis for PPP 2019 rev­enue gui­dance of $18-20M; Re­cor­lev 2nd Phase 3 top-line re­sults 1Q 2020; NDA sub­mis­sion exp 3Q 2020 [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]
Taiwan Liposome Company [TLC] US$162 MM MCap
TLC is a clin­i­cal-stage spe­cial­ty phar­ma­ceu­ti­cal com­pany ded­i­cat­ed to the de­vel­op­ment and com­mer­cial­iza­tion of nov­el nanomedicines that com­bine pro­pri­e­tary lipid-as­sem­bled drug de­liv­ery plat­forms with ap­proved ac­tive phar­ma­ceu­ti­cal in­gre­di­ents (APIs). All pro­grams ex­pect­ed to be in late or piv­o­tal stage by the end of 2020. [more in­for­ma­tion]
Targovax [TRVX:OS] NKr409 MM MCap
Tar­go­v­ax is a clin­i­cal stage biotech­nol­o­gy com­pany de­vel­op­ing im­mune ac­ti­va­tors to tar­get hard-to-treat solid tu­mors. Tar­go­v­ax’s lead prod­uct can­di­date, ON­COS-102, is a ge­net­i­cal­ly mod­i­fied on­co­lyt­ic ade­n­ovirus that has been en­gi­neered to se­lec­tive­ly in­fect and re­pli­cate in can­cer cells. It has been shown to ac­ti­vate the im­mune sys­tem to gen­er­ate tu­mor-spe­cif­ic im­mune re­spons­es. [more in­for­ma­tion]
TCR2 Therapeutics [TCRR] US$275 MM MCap
is a clin­i­cal-stage im­munother­a­py com­pany de­vel­op­ing the next gen­er­a­tion of nov­el T cell ther­a­pies for pa­tients suf­fer­ing from can­cer. TCR2's pro­pri­e­tary T cell re­cep­tor (TCR) Fu­sion Con­struct T cells (TRuC-T™ cells) spe­cif­i­cal­ly rec­og­nize and kill can­cer cells by har­ness­ing sig­nal­ing from the en­tire TCR, in­de­pen­dent of hu­man leuko­cyte anti­gens (HLA). [more in­for­ma­tion]
Tessa Therapeutics
On­go­ing Ph III – (na­sopha­ryn­geal can­cer), Ph I – (cer­vi­cal, mov­ing in­to Ph II); mul­ti­ple other as­sets en­ter­ing clin­ic. Key col­labs: Bay­lor, Park­er Inst, St. Jude Chil­dren’s [more in­for­ma­tion]
The Jackson Laboratory
Found­ed in 1929, The Jack­son Lab­o­ra­to­ry has over 9 de­cades of ex­per­tise in hu­man and mice ge­net­ics. With ev­er-in­creas­ing pre­ci­sion, The Jack­son Lab­o­ra­to­ry iden­ti­fies the ge­net­ic and molec­u­lar bas­es of dis­ease and mar­shals its strengths in ge­nomics and mod­el­ing to dis­cov­er in­di­vi­d­u­al­ized treat­ments and cures. [more in­for­ma­tion]
TiGenix NV [TIG]
Al­lo­gene­ic stem cells. Lead prod­uct, Cx601 for the treat­ment of com­plex pe­ria­nal fis­tu­las, has been filed for reg­u­la­to­ry ap­pro­val in Eu­rope and will com­mence a glob­al Phase III trial in the U.S. Ti­Genix has en­tered in­to a li­cens­ing agree­ment with Take­da for ex­clu­sive right to de­vel­op and com­mer­cial­ize Cx601 for com­plex pe­ria­nal fis­tu­las out­side the U.S. [more in­for­ma­tion]
Tocagen Inc. [TOCA] US$141 MM MCap
Can­cer-se­lec­tive gene ther­a­py com­pany. Lead pro­gram To­ca 511 and To­ca FC are be­ing studied in a reg­is­tra­tio­n­al Ph 2/3 trial for re­cur­rent high grade glio­ma (rHGG). A Ph 1 study of To­ca 511 and To­ca FC showed a near dou­bling of sur­vi­val ver­sus his­tor­i­cal bench­marks with durable PRs and CRs. [more in­for­ma­tion]
Trout New Business
[more in­for­ma­tion]
TXP Pharma
Lead pro­gram TXP-1214 is a Ph 2b/3 ready me­lano­cortin re­cep­tor (MCR) ag­on­ist for Acute Kid­ney In­jury (AKI) in pa­tients un­der­go­ing open ch­est car­di­ac surgery, plus MS. MOA: tar­get­ing the me­lano­cortin sys­tem. TXP-1214 ac­quired from a big phar­ma. End of Ph 2 meet­ing with the FDA by ear­ly 2018. [more in­for­ma­tion]
Vericel Corporation [VCEL] US$841 MM MCap
Ad­vanced cell ther­a­py; mar­ket­ing MA­CI (car­ti­lage re­pair) and Epi­cel (skin re­place­mentt) in TAMs of $2b+. Re­cent­ly ac­quired US com­mer­cial rights for Nexo­Brid. [more in­for­ma­tion]
Veru Inc. [VERU] US$138 MM MCap
Zu­clomiphene in Ph 2 treat­ing hot flash­es caused by hor­mone treat­ment for pros­tate can­cer. Ad­vanc­ing 4 new drug for­mu­la­tions in urol­o­gy: Ta­dala­fil/fi­nas­teride com­bo tablets for symp­toms of BPH and erec­tile dys­func­tion (NDA fil­ing exp 2H19). Tam­su­losin DRS gran­ules and XR cap­sules for be­nign pro­stat­ic hy­per­pla­sia (BPH) treat­ment w/o food ef­fect (NDA fil­ing exp 2019). [more in­for­ma­tion]
Vital Therapies, Inc. [VTL]
is a bio­ther­a­peu­tic com­pany de­vel­op­ing a cell-based ther­a­py tar­get­ing the treat­ment of acute forms of liv­er fail­ure. 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. [more in­for­ma­tion]
Vivoryon [PRBGF] US$59 MM MCap
Vivo­ry­on's pro­grams are based on ded­i­cat­ed re­search, top-lev­el dis­cov­ery and de­ter­mined de­vel­op­ment. The com­pany de­vel­ops first-in-class medicines that tar­get post-trans­la­tio­n­al mod­i­fy­ing en­zymes. The pipe­line con­tains sev­er­al prod­uct can­di­dates across vari­ous stages of de­vel­op­ment, fo­cused on Alzheimer’s Dis­ease and on­col­o­gy. [more in­for­ma­tion]
vTv Therapeutics, Inc. [VTVT] US$76 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 a sub­group anal­y­sis of the STEAD­FAST Alzheimer’s trial pre­sent­ed at CTAD - mov­ing Azeli­ra­gon for­ward in pa­tients with De­men­tia and Di­a­betes. [more in­for­ma­tion]
Wave Life Sciences Ltd. [WVE] US$794 MM MCap
Top-line da­ta from first 3 clin­i­cal pro­grams, 1 in DMD and 2 in Hunt­ing­ton’s dis­ease, exp 2019. Da­ta read­outs from ad­dl pro­grams in DMD, ALS, and fron­totem­po­ral de­men­tia exp in 2020. Lev­er­ag­ing plat­form to ad­vance pro­grams across oph­thal­mol­o­gy and he­p­a­tol­o­gy. [more in­for­ma­tion]
Wilson Therapeutics [WTX:ST] SKr6,493 MM MCap
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]
ZAI Laboratory [ZLAB] US$2,118 MM MCap
The Com­pany has a broad pipe­line of 4 late-stage as­sets with rights in greater Chi­na and 3 as­sets with glob­al rights. Lead Can­di­date, Ni­ra­parib, is in Ph 3 de­vel­op­ment for Ovarian can­cer and is ex­pect­ed to be the first lo­cal PARP in­hibi­tor in Chi­na with­out a bio­mark­er test. [more in­for­ma­tion]