Participating Companies

AcelRx Pharmaceuticals, Inc. [ACRX] $156 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]
Adrenomed AG
Pre­ci­sion medicine, car­dio­vas­cu­lar and Sep­sis, Adre­cizumab Ph 2, bio­mark­er-guid­ed, mon­o­clo­n­al anti­body for sep­tic shock; fi­nal da­ta re­leased late Novem­ber: Ear­ly ap­pli­ca­tion of Adre­cizumab leads to a rapid and sta­tis­ti­cal­ly sig­ni­f­i­cant im­prove­ment of fluid bal­ance, sys­temic or­gan func­tion and > 50% rel­a­tive re­duc­tion of all cause mor­tal­i­ty af­ter 28 days and 90 days com­pared to place­bo on top of SoC. [more in­for­ma­tion]
Affibody
Pro­pri­e­tary Af­fi­body® molecules and Al­bu­mod plat­forms. Lead can­di­date is an IL-17 with pos­i­tive Ph2 52wk da­ta in pso­ri­a­sis and Ph2 IND for lead in­di­ca­tion uvei­tis will be filed in Q1 2021. In ad­di­tion, the com­pany has an on­go­ing re­la­tion­ship with In­ma­gene in Chi­na. Backed by In­ves­tor AB. [more in­for­ma­tion]
Affimed N.V. [AFMD] $1,186 MM MCap
NK cell en­gager AFM13 (CD30/CD16A) to en­ter a Phase 2 reg­is­tra­tion study in CD30-pos­i­tive pe­ripher­al T cell lym­pho­ma and an in­vesti­ga­tor-spon­sored study by MDACC of AFM13 com­bined with al­lo­gene­ic NK cells in re­lapsed/re­frac­to­ry CD30-pos­i­tive lym­pho­ma. [more in­for­ma­tion]
AM-Pharma B.V.
Raised $195M in equi­ty and debt in 2019/2020 af­ter re­sults from Ph 2 in 300+ pts de­mon­s­trat­ed ro­bust sig­ni­f­i­cant im­prove­ment of 40%+ OS in re­cAP group. TL safe­ty & fu­til­i­ty anal­y­sis on first 400 pts Q1 2022. Ex­pects to com­plete tgt en­roll­ment & an­nounce pri­mary end­point da­ta of 28-day all-cause mor­tal­i­ty in 2023, or as ear­ly as 2022 based on in­ter­im anal­y­s­es. [more in­for­ma­tion]
Antev
Phase 3 ready, Tevere­lix, a long act­ing in­jectable pep­tide GnRH an­ta­g­on­ist. Lead in­di­ca­tion for pros­tate can­cer, fol­lowed by be­nign pro­stat­ic hy­per­pla­sia (BPH), acute uri­nary re­ten­tion (AUR), uterine fi­broids and en­dometrio­sis. Over 450 sub­jects have been dosed over 850 times to date with no se­ri­ous ad­verse re­ac­tions and good lo­cal tol­er­ance. [more in­for­ma­tion]
Apexigen
CD40 ag­on­ist mAb; 170+ pts dosed (mono & com­bi sett­ings). 7 on­go­ing Ph 2s, undis­closed oc­u­lar dis­ease pro­gram in Ph 3. AACR: Ph 1b da­ta (w/Park­er Inst); col­labs w/BMY, MDACC, Yale, et al [more in­for­ma­tion]
Aptorum Group [APM] $89 MM MCap
Ap­to­rum is Hong Kong based, US list­ed com­pany (Mcap $100m) fo­cused on gen­er­at­ing late stage clin­i­cal can­di­dates by de­vel­op­ing al­ready ap­proved drugs for new or­phan in­di­ca­tions, as well as in­fec­tious dis­eas­es. Pipe­line – • SACT-1 for Neu­roblas­to­ma – ph2/3 trial planned for 2H’20 • SACT-COV­19 – COVID-19 treat­ment - pre­clin­i­cal [more in­for­ma­tion]
AsclepiX Therapeutics
AX­T107 de­mon­s­trat­ed ef­fi­ca­cy and dura­bil­i­ty in rab­bit and mouse mod­els that is su­pe­ri­or to stan­dard of care Eylea. [more in­for­ma­tion]
Bellus Health [BLU] $352 MM MCap
BEL­LUS' lead prod­uct can­di­date, BLU-5937, is be­ing de­vel­oped for the treat­ment of chron­ic cough (Phase II an­nounced Ju­ly 2020) and chron­ic pru­ri­tus (Phase II planned to be­gin 1H20). [more in­for­ma­tion]
BerGenBio [BGBIO:OS] NKr2,735 MM MCap
De­vel­op­ing first-in-class se­lec­tive Axl ki­nase in­hibi­tors for liquid and solid can­cers, lead can­di­date is bem­cen­tinib (BG­B324) in four com­pany-spon­sored trials. [more in­for­ma­tion]
Biohaven Pharmaceuticals, Inc [BHVN] $4,900 MM MCap
NURTEC® ODT ap­proved in 1Q20. To­p­line re­sults ex­pect­ed 1Q20 in Ph 3 study of tro­r­ilu­zole for mi­graine pre­ven­tion. To­p­line re­sults ex­pect­ed 2Q20 in Ph 2/3 study of tro­r­ilu­zole for OCD. ~$600M to­tal in cash on hand, in­clud­ing re­cent $282.3M raise to sup­port NURTEC ODT launch. [more in­for­ma­tion]
BioXcel Therapeutics, Inc. [BTAI] $859 MM MCap
BX­CL501 (sublin­gual dexmede­to­mi­dine): Met pri­mary, key se­condary and ex­plo­ra­to­ry end­points in two piv­o­tal Phase 3 trials for the for the acute treat­ment of ag­i­ta­tion in pa­tients with schi­zophre­nia (SERENI­TY I) and bipo­lar di­s­or­der (SERENI­TY II). Phase 1b/2 trial (TRAN­QUIL­I­TY) on­go­ing for the acute treat­ment of ag­i­ta­tion as­so­ci­at­ed with ge­ri­a­tric de­men­tia. [more in­for­ma­tion]
CASI Pharmaceuticals Inc. [CASI] $263 MM MCap
Co-com­mer­cial as­set CNCT-19/CD19 CAR-T (part­nered w/ Ju­ven­tas): on­go­ing Ph 1 trials in B-NHL and B-ALL, exp to ini­ti­ate reg­is­tra­tion trials by YE 2020. Exp to ini­ti­ate Ph 1 study for CID-103 (an­ti-CD38 mAb) in EU in Q1 2021. [more in­for­ma­tion]
Catabasis Pharmaceuticals Inc [CATB] $59 MM MCap
Rare dis­ease pipe­line, with lead prod­uct Edasa­lonex­ent in Ph 3 trial in Duchenne mus­cu­lar dys­tro­phy re­gard­less of mu­ta­tion type. North Star Am­bu­la­to­ry As­sess­ment as the pri­mary end­point based on Phase 2 re­sults and FDA in­put. Top-line re­sults ex­pect­ed in 2020. [more in­for­ma­tion]
Catalyst Pharmaceuticals, Inc. [CPRX] $463 MM MCap
Fir­dapse ap­proved for Lam­bert-Ea­ton Myas­thenic Syn­drome (LEMS) with 3Q20 rev­enue of $29.2M. Top SMA Type 3 proof of con­cept trial ex­pect­ed to read­out by YE 2020. Fir­dapse long act­ing for­mu­la­tion de­vel­op­ment cont­in­ues. Studies for 2 ad­di­tio­n­al neu­ro­mus­cu­lar in­di­ca­tions are ex­pect­ed to com­mence by YE 2020. [more in­for­ma­tion]
Celldex Therapeutics, Inc. [CLDX] $882 MM MCap
I-O pipe­line of Abs, ADCs, im­mune sys­tem mo­d­u­la­tors and vaccines tar­get­ed to spe­cif­ic pa­tient pop­u­la­tions. STIC da­ta on an­ti-CD40; col­lab with Mer­ck. [more in­for­ma­tion]
CellMax Life
The com­pany is cur­rent­ly fo­cused on col­orec­tal can­cer (CRC) screen­ing us­ing the clin­i­cal­ly vali­dat­ed CMx plat­form, a blood biop­sy test plat­form that de­tects pre-can­cer­ous le­sions (ade­no­mas) in as lit­tle as two tea­spoons of blood (less than 10mL). [more in­for­ma­tion]
CerSci Therapeutics
De­vel­op­ing a new gen­er­a­tion of non-opi­oid medicines to treat acute post-op­er­a­tive and chron­ic neu­ro­path­ic pain. Lead pro­gram in Phase 1 (MAD/SAD/Food Ef­fect), CT-044, tar­get­ing re­ac­tive spe­cies that cause neu­ro­n­al ion chan­nel hy­per-sen­si­ti­za­tion, known to trig­ger pain. [more in­for­ma­tion]
Enterin
De­vel­op­er of nov­el drugs in­tend­ed to re­pair the dys­func­tio­n­al gut-brain axis. The com­pany's drugs dis­places &al­pha;-synu­clein ag­gre­gates and re­s­tores nor­mal sig­nal­ing be­tween the gut and the brain, en­abling pa­tients with Parkin­son's dis­ease and other neu­rode­gen­er­a­tive con­di­tions to have a bet­ter the qual­i­ty of life. [more in­for­ma­tion]
ESSA Pharma Inc. [EPIX] $773 MM MCap
1Q20 IND sub­mis­sion for lead as­set, EPI-7386, a small molecule tar­get­ing n-ter­mi­nal do­main of an­dro­gen re­cep­tor for mCR­PC. First pa­tient dosed in EPI-7386 Phase 1 study in mCR­PC pa­tients fail­ing se­cond gen­er­a­tion an­tian­dro­gens by 2Q20. [more in­for­ma­tion]
Faraday Pharmaceuticals
Chron­ic and acute agents to pre­vent car­di­ac and skele­tal mus­cle loss by mo­d­u­lat­ing neu­ro­hu­mo­ral path­ways and res­tor­ing metabolic flex­i­bil­i­ty: lead can­di­date FDY-5301 Ph 3 ready for reper­fu­sion in­jury fol­low­ing heart at­tack (AMI). $57M raised to date from Arch Ven­ture Part­n­ers, Po­laris Part­n­ers, WRF, Os­age [more in­for­ma­tion]
Forbius
Se­lec­tive in­hibi­tors of TGF Be­ta & EGFR path­ways. AACR: (+) Ph 1 da­ta. 3 on­go­ing Ph 2 trials (SCCHN, NS­CLC, TN­BC); ad­dl Ph 1 trials in IO, Sys­temic Scle­ro­sis & Myelo­fi­bro­sis [more in­for­ma­tion]
Freenome Holdings, Inc.
Biotech­nol­o­gy di­ag­nos­tics com­pany uti­l­iz­ing its deep ex­per­tise in molec­u­lar bi­ol­o­gy with ad­vanced com­pu­ta­tio­n­al bi­ol­o­gy and ma­chine learn­ing tech­niques; the com­pany pi­oneered a com­pre­hen­sive mul­ti­omics plat­form for ear­ly can­cer de­tec­tion through a rou­tine blood draw. At AS­CO GI 2020, de­mon­s­trat­ed a sen­si­tiv­i­ty of 94% and spe­ci­fic­i­ty of 94% for ear­ly-stage (I/II) col­orec­tal ade­no­car­ci­no­ma. [more in­for­ma­tion]
Galera Therapeutics [GRTX] $188 MM MCap
Clin­i­cal stage bio­phar­ma­ceu­ti­cal com­pany fo­cus­ing on trans­form­ing ra­dio­ther­a­py in can­cer. Lead as­set GC4419 is a po­tent, high­ly se­lec­tive small molecule dis­mu­tase mimet­ic be­ing de­vel­oped for se­vere oral mu­cosi­tis, cur­rent­ly in Phase 3 with Break­through Ther­a­py Desig­na­tion. To­p­line re­sults ex­pect­ed in 1H21. Top line re­sults in Phase 1b/2a in se­cond as­set, GC4419 in pan­cre­at­ic can­cer ex­pect­ed in 2H20. GC4419 ex­pect­ed to ini­ti­ate in Phase 1b/2a in lung can­cer in 2H20. [more in­for­ma­tion]
Genfit SA [GNFT] $194 MM MCap
De­vel­op­ment of ela­fi­branor in PBC: on­go­ing en­roll­ment for Ph 3 clin­i­cal trial EL­A­TIVE™. Com­mer­cial­iza­tion of NIS4™ for NASH di­ag­no­sis. [more in­for­ma­tion]
HaemaLogiX
Pro­pri­e­tary, dif­fer­en­ti­at­ed B cell tar­gets en­gaged via anti­body and CAR-T pro­grams Kap­paMab & Lamb­daMab tar­get ac­ti­vat­ed plas­ma cell anti­gens (KMA/LMA); Kap­paMab plus RevDex vs. RevDex Ph 2b in MM, da­ta 1H20; Kap­pa CAR-T Ph 1 Q120 in MM; Ca­pac­i­ty for du­al-tar­get­ing via CD-19, BC­MA, etc; Lamb­daMab in pre­clin­i­cal for amy­loi­do­sis [more in­for­ma­tion]
Hutchison China MediTech Ltd ("Chi-Med") [HCM] $4,346 MM MCap
Chi-Med has a port­fo­lio of 8 drugs in clin­i­cal de­vel­op­ment from own dis­cov­ery en­gine in its In­no­va­tion plat­form and a cash gen­er­at­ing Com­mer­cial plat­form with 2,500+ sales reps. Glob­al col­lab­o­ra­tion with As­traZene­ca in two reg­is­tra­tion en­abling studies with savol­i­tinib in Ta­gris­so re­frac­to­ry lung can­cer that is MET+ and EGFRm+ (glob­al) and MET Ex­on 14 dele­tion NS­CLC (Chi­na). [more in­for­ma­tion]
Impact Therapeutics Inc
Im­pact Ther­a­peu­tics is a pri­vate­ly held clin­i­cal-stage bio­phar­ma­ceu­ti­cal com­pany and ded­i­cates to the dis­cov­ery and de­vel­op­ment of tar­get­ed an­ti-can­cer ther­a­peu­tics based on syn­thet­ic lethal­i­ty. [more in­for­ma­tion]
Invetx
In­vetx is build­ing the world’s pre­mi­er biotech­nol­o­gy plat­form for pro­tein-based ther­a­peu­tics in an­i­mal health to trans­form stan­dards of care in ve­t­eri­nary medicine. The In­vetx team of high­ly ex­pe­ri­enced R&D lead­ers from both hu­man biotech and an­i­mal health is sup­port­ed by best-in-class biotech­nol­o­gy part­n­ers and an ex­ten­sive net­work of in­dus­try ex­perts, ve­t­eri­nary sci­en­tists and clini­cians. In­vetx is a pri­vate com­pany head­quar­tered in Bos­ton, Mas­sachusetts. [more in­for­ma­tion]
Iovance Biotherapeutics [IOVA] $4,408 MM MCap
Late-stage cell ther­a­py com­pany uti­l­iz­ing pro­pri­e­tary au­tol­o­gous tu­mor in­fil­trat­ing lym­pho­cytes (TILs) to at­tack solid tu­mors. The com­pany is con­duct­ing piv­o­tal stage trials in me­tastat­ic me­lano­ma and ad­vanced cer­vi­cal can­cer with BLAs ex­pect­ed 2020. [more in­for­ma­tion]
ISA Pharmaceuticals BV
is an im­munother­a­py com­pany de­vel­op­ing ra­tio­n­al­ly de­signed, ful­ly syn­thet­ic im­munother­a­peu­tics against can­cer and per­sis­tent vi­ral in­fec­tions. The Com­pany has built a pro­pri­e­tary im­munother­a­py plat­form based on the Syn­thet­ic Long Pep­tide (SLP®) con­cept and AM­PLI­VANT®tech­nol­o­gy. [more in­for­ma­tion]
Junshi Biosciences [1877:HK]
Has a di­ver­si­fied R&D pipe­line com­pris­ing 21 drug can­di­dates with ther­a­peu­tic ar­eas cov­er­ing can­cer, metabolic, au­toim­mune, neu­ro­log­i­cal, and in­fec­tious dis­eas­es. Prod­uct types in­clude mon­o­clo­n­al anti­bodies, fu­sion pro­teins, anti­body-drug con­ju­gates, and small molecule drugs. With a com­bined 33,000L fer­men­ta­tion ca­pac­i­ty in two GMP-fa­cil­i­ties at Shang­hai and Suzhou. JS001, or tori­pal­imab, is ap­proved by the NM­PA and the first mar­ket­ed an­ti-PD-1 mAb anti­body self-de­vel­oped by a PRC com­pany. [more in­for­ma­tion]
Kadmon Holdings, Inc. [KDMN] $622 MM MCap
Aug 30 PD­U­FA da­ta for belu­mo­sudil (cGVHD) fol­low­ing pos­i­tive pri­mary anal­y­sis re­sults (May 2020); ob­served ORRs of 73% and 74% in 200mg QD and 200mg BID arms, re­spec­tive­ly. Ini­ti­at­ing 12-15 pt open-la­bel Ph 2 in pa­tients w dif­fuse cu­ta­neous sys­temic scle­ro­sis in Q1 2021. First 3-pt dose co­hort suc­cess­ful­ly com­plet­ed in on­go­ing dose-es­ca­la­tion Ph 1 (an­ti-PD-L1/IL-15 fu­sion pro­tein) in pa­tients w me­tastat­ic or lo­cal­ly ad­vanced solid tu­mors; en­roll­ment on­go­ing in next dose lev­el. Cont­inu­ing IND-en­abling ac­tivi [more in­for­ma­tion]
Kineta Inc.
Kine­ta (Pri­vate) - whol­ly owned pre­clin­i­cal an­ti-VIS­TA (in­nate im­mune tar­get) anti­body re­pro­grams the tu­mor mi­croen­vi­ron­ment and is dif­fer­en­ti­at­ed from other im­munother­a­pies by block­ing sup­pres­sive cells in cold tu­mors. $360M+ deal with Ge­nen­tech for KCP506, Ph1 non-opi­oid for chron­ic pain. [more in­for­ma­tion]
Landos Biopharma [LABP] $407 MM MCap
Based on ex­ten­sive pre­clin­i­cal studies, Lan­dos has vali­dat­ed a unique mech­anism of ac­tion in­volv­ing LAN­CL2 that ex­erts po­tent an­ti-in­flam­ma­to­ry ef­fects with an out­s­tand­ing safe­ty pro­file. Lead as­set BT-11 Phase 2 in­ter­im read out in mid-2020 in UC. [more in­for­ma­tion]
Lineage Cell Therapeutics Inc. [LCTX] $369 MM MCap
Cell ther­a­py com­pany with 3 cl­ni­cal pro­grams 1) OpRe­gen, an RPE tran­s­plant ther­a­py in Ph1/2a de­vel­op­ment for the treat­ment of dry-AMD. 2) OPC1, an oli­go­den­dro­cyte pro­gen­i­tor cell ther­a­py for acute spi­nal cord in­juries with Or­phan and RMAT desig­na­tions. 3) VAC2, an al­lo­gene­ic can­cer im­munother­a­py of anti­gen-pre­sent­ing den­drit­ic cells cur­rent­ly in a Ph 1 for NS­CLC con­duct­ed by CRUK. [more in­for­ma­tion]
Marker Therapeutics [MRKR] $176 MM MCap
A clin­i­cal-stage im­muno-on­col­o­gy com­pany with the po­ten­tial to sig­ni­f­i­cant­ly dis­rupt the cur­rent cell ther­a­py land­s­cape. Mark­er's cur­rent Mul­ti­TAA cell ther­a­py is look­ing to ini­ti­ate a Phase II study in AML in 2020 with en­roll­ment com­ple­tion an­ti­ci­pat­ed in 2H20. The com­pany al­so has a T-cell vaccine pro­gram, cur­rent­ly be­ing studied in breast can­cer. [more in­for­ma­tion]
Maxcyte [MXCT:LN]
glob­al cell ther­a­py com­pany pro­vid­ing pro­pri­e­tary non-vi­ral cell en­gi­neer­ing tech­nol­o­gy for the next gen­er­a­tion of cell-based ther­a­pies in­clud­ing gene edit­ing and im­muno-on­col­o­gy. Max­Cyte may be seen as a play on the cell ther­a­py mar­ket with its blue chip client base. [more in­for­ma­tion]
Minoryx Therapeutics
Oral PPARg ag­on­ist, lerigl­i­ta­zone, de­mon­s­trat­ed clin­i­cal ben­e­fit of sig­ni­f­i­cant­ly re­duc­ing pro­gres­sion of cere­bral le­sions and myelo­pa­thy symp­toms in piv­o­tal Phase 2/3 study re­sults in X-ALD pts w Adreno­myeloneu­ro­pa­thy (AMN), 6MWT end­point was not met; on­go­ing dis­cus­sions with reg­u­la­tors re: ap­pro­val. Phase 2 da­ta in Frie­drich’s Ataxia showed mo­d­u­la­tion of the Fra­taxin path­way and res­to­ra­tion of the bioen­er­get­ics de­fic­its in FR­DA pa­tients. VC in­ves­tors in­clude Kur­ma, Ysios, Fund+, Roche, Id­in­vest, etc [more in­for­ma­tion]
Neoleukin Therapeutics [NLTX] $514 MM MCap
Lead pro­gram NL-201: is a du­al-act­ing IL-2 and IL-15 ag­on­ist im­munother­a­py for can­cer, de­signed to im­prove tol­er­a­bil­i­ty and ac­tiv­i­ty by elim­i­nat­ing the al­pha re­cep­tor bind­ing in­ter­face (IND com­plet­ed 4Q20). First de no­vo pro­tein ther­a­peu­tic ev­er cre­at­ed. [more in­for­ma­tion]
NeuroRx
Lead­ing Re­lief Ther­a­peu­tics Ph 2 trial of Avip­tadil to treat COVID-19 in­duced ARDS in the US. In Phase 3 with read­out ex­pect­ed mid-Jan­uary 2021. Al­so de­vel­op­ing first Glx tar­get­ed an­tide­pres­sant to ad­dress sui­ci­dal­i­ty in bipo­lar de­pres­sion. NRX-101 is in Ph 2b/3 de­vel­op­ment, (Break­through Desig­na­tion, SPA and Fast Track Desig­na­tion by FDA) for Se­vere Bipo­lar De­pres­sion in pts w Acute Sui­ci­dal Idea­tion and Be­havior. [more in­for­ma­tion]
NexImmune, Inc. [NEXI] $544 MM MCap
Mul­ti-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. Two Ph 1's on­go­ing. [more in­for­ma­tion]
NorthSea Therapeutics
First-in class, oral, struc­tu­ral­ly en­gi­neered fat­ty acids [SE­FAs] ad­dress­ing NASH (pos­i­tive Ph2b in­ter­im re­sults) and as­so­ci­at­ed metabolic di­s­or­ders via unique tar­get­ing of path­ways reg­u­lat­ing metabolism, oxida­tive stress, in­flam­ma­tion, and fi­bro­sis. $40m Se­ries B (Jan 2020) with ven­Bio Part­n­ers (lead) and Sofin­no­va In­vest­ments. [more in­for­ma­tion]
Oncopeptides AB [ONCO:ST] SKr9,191 MM MCap
Po­ten­tial ap­pro­val for Melflufen (Yga­lo) 1Q21. First in class pep­tide-drug con­ju­gate (PDC) that tar­gets aminopep­ti­das­es and rapid­ly re­leas­es alky­lat­ing agents in­to tu­mor cells. H2H ver­sus Po­m­alyst com­plet­ed en­roll­ment Sep '20. [more in­for­ma­tion]
OnKure Therapeutics, Inc.
De­vel­op­ing epi­ge­net­ic can­cer ther­a­pies in­clud­ing HDAC in­hibi­tors. Lead as­set OKI-179 is a class 1 HDAC in­hibi­tor in Ph 1. [more in­for­ma­tion]
PAION AG [PA8:DE]
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]
Paradigm BioPharma [PBIGF] $411 MM MCap
Launch­ing Ph 3 in 2020 in OA pts w/bone mar­row ede­ma le­sions (BML). Pos­i­tive Ph2b re­sults in sub­jects w/OA & BML - stat sig im­prove­ment on pain re­duc­tion & BML vol­ume/grade re­duc­tions on MRI. Q12020: ini­tial da­ta from com­pas­sio­nate use trial (ex-NFL sub­jects w/OA) and joint IND sub­mis­sion to FDA/EMA for piv­o­tal trial in Mu­copolysacchari­do­sis. Exp AUS rev-gen in Q3 20. [more in­for­ma­tion]
PharmaMar SA [PHM:ES]
Rev­enue gen­er­at­ing with marine-de­rived and nov­el MoA on­col­o­gy prod­ucts. Yon­delis (soft tis­sue sar­co­ma and ovarian can­cer) com­mer­cial­ized with di­rect sales-force in EU and part­n­er­ship in US with Janssen. Signed li­cens­ing agree­ment with Jazz Phar­ma for lur­binecte­din in the US - up­front pay­ment of $200M, $100mm on ap­pro­val and up to $550M sales mile­s­tones. [more in­for­ma­tion]
Pharming Group NV [PHARM:AS] €687 MM MCap
re­cent in-li­cense from No­var­tis of late stage drug Le­ni­olis­ib for APDS (ul­tra-rare im­mun­od­e­fi­cien­cy con­di­tion), rhC1INH for new larg­er in­di­ca­tions in­clud­ing COVID-19, pre-eclamp­sia, AKI and ad­di­tio­n­al pipe­line prod­uct from tech­nol­o­gy plat­form: re­com­bi­nant al­pha-Glu­cosi­dase for Pompe dis­ease. [more in­for­ma­tion]
Pharvaris [PHVS] $954 MM MCap
De­vel­op­ing PHA121, a high­ly po­tent, spe­cif­ic, and oral­ly bioa­vai­l­able com­pet­i­tive an­ta­g­on­ist of the bradykinin B2 re­cep­tor, cur­rent­ly in Ph 1 trial for the treat­ment of HAE. [more in­for­ma­tion]
Photocure ASA [PHO:OS] NKr3,549 MM MCap
Rev­enue-gen­er­at­ing urol­o­gy-fo­cused com­pany. Lead prod­uct, Hexvix®/Cysview® im­proves de­tec­tion of blad­der can­cer un­der blue light cys­tos­copy (BLC), which can re­duce dis­ease re­cur­rence and pro­gres­sion rates. Hexvix®/Cysview® is in­clud­ed in clin­i­cal consen­sus guide­lines and re­mains on a strong growth path. Rev­enues in 2020 to­taled $31M. Re­cent­ly suc­cess­ful­ly launched the Eu­ro­pean Hexvix op­er­a­tions, con­soli­dat­ing the Com­pany’s prod­uct franchise glob­al­ly. Pho­to­cure is head­quar­tered in Os­lo, Nor­way and list­ed on [more in­for­ma­tion]
Pieris Pharmaceuticals, Inc. [PIRS] $125 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]
Protagonist Therapeutics [PTGX] $1,139 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]
Ryvu Therapeutics [RVU:WAR]
Po­ten­tial first in class CD­K8 in­hibi­tor for leukemias, lym­pho­mas, and solid tu­mors in Ph1b in AML and high-risk MDS, PIM/FLT3 ki­nase in­hibi­tor in Ph 1/2 in AML (part­nered w Me­nari­ni). Da­ta in 2020. Ear­ly stage pipe­line ad­dress­ing emerg­ing solid tu­mor tar­gets. [more in­for­ma­tion]
Savara Inc. [SVRA] $193 MM MCap
Or­phan lung dis­ease Com­pany; Mol­gradex, is an in­haled gran­u­lo­cyte-macrophage colony-sti­m­u­lat­ing fac­tor (GM-CSF) in Phase 3 de­vel­op­ment for au­toim­mune pul­mo­nary alve­o­lar pro­tei­no­sis (aPAP); study ex­pect­ed to start Q1 2021. Al­so, Phase 3 trial, AVAIL, is eval­u­at­ing per­sis­tent me­thi­cillin-re­sis­tant Sta­phy­lo­coc­cus au­reus (MR­SA) lung in­fec­tion in peo­ple liv­ing with cys­tic fi­bro­sis. Top line da­ta are ex­pect­ed ear­ly in 2021 [more in­for­ma­tion]
Secarna Pharmaceuticals GmbH
Se­car­na's unique­ly ef­fi­cient LNA­plus™ dis­cov­ery pro­cess is fu­eled by their pro­pri­e­tary and stream­lined Oli­go­fy­er™ bioin­for­mat­ics sys­tems large in-house cell li­brary and own in vit­ro screen­ing as­says. [more in­for­ma­tion]
Selecta Biosciences, Inc. [SELB] $470 MM MCap
Im­mune tol­er­ance plat­form (Imm­TOR) de­vel­oped in com­bo with rare dis­ease ther­a­pies. SEL-212 (chron­ic re­frac­to­ry gout, out-li­censed to So­bi) DIS­SOLVE Ph 3 pro­gram on­go­ing. Gene tx pipe­line in pre­clin­i­cal de­vel­op­ment with Imm­TOR where re-dos­ing re­quire. Ask­Bio and Sarep­ta col­labs. [more in­for­ma­tion]
Sol-Gel Technologies Ltd. [SLGL] $308 MM MCap
Com­mer­cial­iz­ing brand­ed gener­ic top­i­cal drug prod­ucts (skin dis­eas­es). Two pos­i­tive Phase 3 pro­grams, both with PD­U­FA dates in 2021: Ep­so­lay has po­ten­tial to be first FDA-ap­proved sin­gle-agent ben­zoyl per­oxide pre­scrip­tion drug prod­uct for rosacea (PD­U­FA date on April 26th). Twy­neo has po­ten­tial to be the first acne treat­ment that con­tains fixed dose com­bi­na­tion of BPO and treti­noin (PD­U­FA date on Au­gust 1st). [more in­for­ma­tion]
Sonnet Biotherapeutics, Inc. [SONN] $37 MM MCap
Son­net's in­ter­leukin-based can­di­date has been shown to en­hance pK (up to 10 fold) and im­prove tu­mor de­liv­ery with an in­crease in in vi­vo ef­fi­ca­cy (30 fold) as de­mon­s­trat­ed in a mice tu­mor mod­el. The Son­net plat­form de-risks the use of in­ter­leukins by great­ly ex­tend­ing their in vi­vo half-life, while al­so im­prov­ing their spe­ci­fic­i­ty to tu­mor tis­sue. [more in­for­ma­tion]
SphingoTec GmbH
The tests are be­ing im­ple­ment­ed on the Com­pany’s own point-of-care im­munoas­say an­a­lyz­er plat­form ac­quired form Sam­sung in 2018 that is ful­ly de­vel­oped and was orig­i­nal­ly launched in 2012 and was sold more than 1,800 times since then. [more in­for­ma­tion]
sterna biologicals
By tar­get­ing tran­scrip­tion fac­tors that play a cen­tral role in reg­u­lat­ing Th1- and Th2-driv­en in­flam­ma­to­ry mech­anisms, the Com­pany’s pro­pri­e­tary DNAzyme-based drug can­di­dates can in­ter­vene with up­stream in­flam­ma­to­ry pro­cess­es to ad­dress re­lat­ed dis­eas­es more ef­fec­tive­ly. Ster­na cur­rent­ly has four pro­grams in phase 2 de­vel­op­ment. [more in­for­ma­tion]
Strongbridge Biopharma [SBBP] $168 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] $914 MM MCap
FolR&al­pha;-tar­get­ing ADC (STRO-002) Phase 1, ad­di­tio­n­al dose es­ca­la­tion da­ta in 4Q20. CD74-tar­get­ing ADC (STRO-001) Phase 1, ad­di­tio­n­al dose es­ca­la­tion da­ta in 4Q20. BMS col­lab­o­ra­tion Phase 1 clin­i­cal de­vel­op­ment up­date for CC-99712 (BC­MA-tar­get­ing ADC). Mer­ck col­lab­o­ra­tion up­date in 2020 on first cy­tokine deri­va­tive pro­gram. [more in­for­ma­tion]
Syneos Health [SYNH] $8,216 MM MCap
Sy­neos Health® is an end-to-end, ful­ly in­te­grat­ed bio­phar­ma­ceu­ti­cal so­lu­tions com­pany that works dif­fer­ent­ly. At Sy­neos Health, all the dis­ci­p­lines in­volved in bring­ing new ther­a­pies to mar­ket, from clin­i­cal to com­mer­cial, work to­gether to cre­ate cus­tomer suc­cess. Our unique Bio­phar­ma­ceu­ti­cal Ac­cel­er­a­tion Mod­el de­liv­ers val­ue across the small to mid-size to large cus­tomer cont­in­u­um. [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]
Teon Therapeutics
Pipe­line of next-gen­er­a­tion can­cer ther­a­peu­tics tar­get­ing GPCRs in­clud­ing a high­ly-spe­cif­ic A2B an­ta­g­on­ist and EP4 an­ta­g­on­ist, both slat­ed to en­ter Phase 1b trials in 2021. [more in­for­ma­tion]
Tessa Therapeutics
On­go­ing Ph III (na­sopha­ryn­geal can­cer), up­com­ing piv­o­tal Ph II (Hodgkin lym­pho­ma), and mul­ti­ple other clin­i­cal-stage pro­grams. GMP fa­cil­i­ty un­der con­struc­tion. Key col­lab­o­ra­tions: Bay­lor, Park­er Inst., St. Jude Chil­dren’s, and JV in Chi­na. [more in­for­ma­tion]
Vaccitech Limited
Pro­pri­e­tary ChA­dOx (prime) and MVA (boost) tech­nol­o­gy plat­form in­duces and main­tains CD8+ T-cells at the max­i­mum lev­els pub­lished to date. Ph1/2 da­ta for HPV and HBV im­munother­a­pies ex­pect­ed 2H20. En­cour­ag­ing pros­tate can­cer, 5T4 im­munother­a­peu­tic da­ta re­cent­ly re­leased. Ph1/2 NS­CLC MAGE-NYE­SO im­munother­a­peu­tic en­ters clin­ic in Q1 2021. ChA­dOx COVID-19 vaccine out-li­censed to Ox­ford Uni/As­traZene­ca with post-pan­dem­ic rev­enue stream. $150M fund­ing at Jen­n­er In­sti­tute and com­pany raised $48M since for­ma­tion [more in­for­ma­tion]
Vericel Corporation [VCEL] $2,630 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]
Verrica Pharmaceuticals [VRCA] $374 MM MCap
Ver­ri­ca Phar­ma­ceu­ti­cals is a der­ma­tol­o­gy ther­a­peu­tics com­pany de­vel­op­ing med­i­ca­tions for vi­ral skin dis­eas­es re­quir­ing med­i­cal at­ten­tion. Lead prod­uct, VP-102 for mol­lus­cum con­ta­gio­sum has a PD­U­FA date of Ju­ly 13, 2020. Phase 2 da­ta in ex­ter­nal geni­tal warts ex­pect­ed in 2H20. Ini­ti­a­tion of Phase 3 in com­mon warts in 1H20 and ini­ti­a­tion of Phase 2 in plan­tar warts in mid-2020. [more in­for­ma­tion]
Viela Bio [VIE] $2,913 MM MCap
UPLIN­ZA® (ine­bi­l­izumab, an­ti-CD19 mAb) on the mar­ket for the treat­ment of neu­romyeli­tis op­ti­ca spec­trum di­s­or­der (NMOSD). Ad­di­tio­n­al trials with ine­bi­l­izumab on­go­ing, in­clud­ing Ph 3 trials in myas­the­nia gravis and Ig­G4-re­lat­ed dis­ease. VIB7734, an­ti-ILT7 mAb, Ph 2 trial to ini­ti­ate in 1H21. Ph 2 trials on­go­ing for VIB4920 (An­ti-CD40L-Tn3 Fu­sion Pro­tein) in Sjo­gren's Syn­drome and kid­ney tran­s­plant re­jec­tion. [more in­for­ma­tion]
Vyome Therapeutics Inc.
$22M fund raise Jan '19. Ad­vanced lead as­set VB-1953 to Ph 2 for mod­er­ate to se­vere acne. Pipe­line DART - du­al ac­tion over­com­ing an­timi­cro­bial re­sis­tance - and MRT - pa­tent­ed an­ti­fun­gal plat­form. Treat drug-re­sis­tant skin patho­gens. [more in­for­ma­tion]