All Companies

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]
Lead can­di­date Izok­ibep, sup­port­ed by part­n­er­ship with Ace­lyrin, is an IL-17 in­hibi­tor with mul­ti­ple Ph2 pro­grams in progress (PSO, axS­pA, uvei­tis, HS, PsA). ACR50 pri­mary end­point met for 16 wk da­ta in PsA. First pa­tient dosed in piv­o­tal Phase 2b trial for uvei­tis filed in 1H2022 with 24 wk da­ta ex­pect­ed in 2023. [more in­for­ma­tion]
Affimed [AFMD] $73 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]
Agiliti Health [AGTI] $877 MM MCap
health­care in­dus­try with so­lu­tions that help sup­port a more ef­fi­cient, safe and sus­tain­able health­care de­liv­ery sys­tem. Agil­i­ti serves more than 9,000 na­tio­n­al, re­gio­n­al and lo­cal acute care and al­ter­nate site providers across the U.S. For more than eight de­cades, Agil­i­ti has de­liv­ered med­i­cal equip­ment ma­n­age­ment and ser­vice so­lu­tions that help health­care providers re­duce costs, in­crease op­er­at­ing ef­fi­cien­cies and sup­port op­ti­mal pa­tient out­comes. [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]
De­vel­op­ing a po­ten­tial­ly best-in-class oral im­munother­a­py that ad­dress­es a broad range of food al­ler­gies. AD­P101, which is in a Ph 1/2 study and an OLE study, is de­signed to de­sen­si­tize pa­tients al­ler­g­ic to one or to mul­ti­ple foods si­mul­ta­ne­ous­ly to miti­gate the risk of se­vere, life-threat­en­ing al­ler­g­ic re­ac­tions. The Com­pany was co-found­ed by Dr. Kari Nadeau, a renowned food al­ler­gist, and former 23andMe ex­ec, Ash­ley Dom­bkows­ki who serves is CEO. Da­ta from Ph 1/2 study ex­pect­ed 1Q23. [more in­for­ma­tion]
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]
Arbor Biotechnologies
With the most ex­ten­sive li­brary of CRIS­PR nu­cleas­es in the in­dus­try, a deep bench of world-class foun­ders, sci­en­tif­ic ad­vi­sors, and a lead­er­ship team with a proven track re­cord of de­vel­op­ing nov­el plat­forms and ther­a­peu­tics, Ar­bor is well-po­si­tioned to de­vel­op cu­ra­tive ge­net­ic medicines for all pa­tients with ge­net­ic dis­eas­es. Ar­bor’s pipe­line is pow­ered by its pro­pri­e­tary biodis­cov­ery en­gine, which blends com­pu­ta­tio­n­al search and sci­en­tif­ic ex­per­tise to dis­cov­er, screen, and en­gi­neer pro­teins at un­prece­dent­ed scale and speed. Ar­bor has raised more than $300 mil­lion to date and top in­ves­tors in­clude ARCH Ven­ture Part­n­ers, Te­masek, Al­ly Bridge Group, and TCG Crossover. [more in­for­ma­tion]
Arbutus Biopharma [ABUS] $322 MM MCap
To ad­dress HBV, de­vel­op­ing a RNAi ther­a­peu­tic, an oral PD-L1 in­hibi­tor, and oral RNA desta­bi­l­iz­er and in­tend to com­bine with the aim of pro­vid­ing a func­tio­n­al cure for pa­tients with chron­ic HBV by sup­press­ing vi­ral re­pli­ca­tion, re­duc­ing sur­face anti­gen and reawak­en­ing the im­mune sys­tem. AB-729, is po­ten­tial­ly the on­ly RNAi ther­a­peu­tic with evi­dence of im­mune re-awak­en­ing. It is cur­rent­ly be­ing eval­u­at­ed in mul­ti­ple phase 2 clin­i­cal trials. [more in­for­ma­tion]
Arcellx [ACLX] $1,735 MM MCap
A clin­i­cal-stage biotech­nol­o­gy com­pany rei­magin­ing cell ther­a­py by en­gi­neer­ing in­no­va­tive im­munother­a­pies for pa­tients with can­cer and other in­cur­able dis­eas­es. Ar­cel­lx's lead prod­uct can­di­date, CART-dd­BC­MA, is be­ing de­vel­oped for the treat­ment of re­lapsed or re­frac­to­ry mul­ti­ple myelo­ma (r/r MM) in an on­go­ing Phase 1 study. Ar­cel­lx is al­so ad­vanc­ing its dos­able and con­trol­lable CAR-T ther­a­py, ARC-SparX, in­to the clin­ic through two pro­grams: ACLX-001 in r/r MM and ACLX-002 in re­lapsed or re­frac­to­ry acute mye [more in­for­ma­tion]
[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]
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]
Be Biopharma
Be Bio­phar­ma is a lead­er in de­vel­op­ing B cells as medicines, treat­ing dis­ease with the hu­man body’s na­tive pro­tein fac­to­ries. We pre­cise­ly en­gi­neer B cells to har­ness their in­trin­sic drug-like prop­er­ties – re­mark­able pro­tein pro­duc­tion, se­lec­tive tis­sue tar­get­ing, and fine con­trol of their cel­lu­lar en­vi­ron­ment – to forge a new cat­e­go­ry of cell ther­a­py. Be Bio was found­ed in Oc­to­ber 2020 by Long­wood Fund with a $52 mil­lion Se­ries A in­vest­ment led by At­las Ven­tures and RA Cap­i­tal, joined by Al­ta Part­n­ers and [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]
Bellus Health [BLU] $1,866 MM MCap
BEL­LUS Health is de­vel­op­ing nov­el ther­a­peu­tics for the treat­ment of chron­ic cough and other hy­persen­si­ti­za­tion-re­lat­ed di­s­or­ders. The com­pany's lead can­di­date, BLU-5937, is cur­rent­ly in the CALM phase 3 pro­gram with ini­tial to­p­line da­ta ex­pect­ed in 2H 2024 and 2H 2025. [more in­for­ma­tion]
BenevolentAI [BAI]
Com­bines ad­vanced AI and ma­chine learn­ing with cutt­ing edge sci­ence to de­ci­pher com­plex dis­ease bi­ol­o­gy, gen­er­ate nov­el in­sights and dis­cov­er more ef­fec­tive medicines. The unique com­pu­ta­tio­n­al R&D plat­form spans ev­ery step of the drug dis­cov­ery pro­cess, pow­er­ing a grow­ing in-house pipe­line of 13 named drug pro­grammes and over 10 ex­plo­ra­to­ry pro­grammes, main­tain­ing suc­cess­ful col­lab­o­ra­tions. [more in­for­ma­tion]
Benitec Biopharma [BNTC] $5 MM MCap
Their lead can­di­date BB-301 in OP­MD has achieved mech­anis­tic and ther­a­peu­tic proof of con­cept in two pre clin­i­cal an­i­mal mod­els. A na­t­u­ral his­to­ry study to col­lect base­line da­ta in OP­MD pa­tients ne­ces­sary for a clin­i­cal trial is on­go­ing, with the IND clear­ance ex­pect­ed in 2Q23. [more in­for­ma­tion]
Bexion Pharmaceuticals
A clin­i­cal-stage bio­phar­ma­ceu­ti­cal com­pany de­vel­op­ing a new gen­er­a­tion of bi­o­log­ic im­munother­a­py to treat Solid Tu­mor Can­cers and Che­mother­a­py In­duced Pe­ripher­al Neu­ro­pa­thy (CIPN). Bexion's lead as­set BXQ-350 ac­ti­vates Sphin­goloid Metabolism and has de­mon­s­trat­ed ex­cel­lent safe­ty and monother­a­py ben­e­fits across mul­ti­ple tu­mor types in its com­plet­ed adult and pe­di­a­tric Ph 1 study. Ph 2 study in CRC ex­pect­ed 4Q22. The Com­pany has $80M raised to date. [more in­for­ma­tion]
Brooklyn ImmunoTherapeutics [BTX]
Brook­lyn has mul­ti­ple next-gen­er­a­tion cell and gene-edit­ing ther­a­pies in pre­clin­i­cal de­vel­op­ment for vari­ous in­di­ca­tions in­clud­ing acute re­s­pi­ra­to­ry dis­tress syn­drome, solid tu­mor in­di­ca­tions, and in vi­vo gene-edit­ing ther­a­pies for rare ge­net­ic dis­eas­es in ad­di­tion to its clin­i­cal-stage IRX-2 cy­tokine ther­a­py pro­gram for pa­tients with head and neck can­cer. [more in­for­ma­tion]
CASI Pharmaceuticals Inc. [CASI] $34 MM MCap
Launched EVOMELA® (mel­pha­lan for in­jec­tion, mul­ti­ple myelo­ma) in Au­gust '19, on­ly mel­pha­lan com­mer­cial­ly avai­l­able in Chi­na: $7.1M 2Q21 rev­enues/guid­ing FY 2020 of over 80% growth. Part­n­er­ship w BioIn­vent: acq'd Greater Chi­na comm/dev rights to an­ti-FcyRI­IB mAb w po­ten­tial ap­pli­ca­tions across mul­ti­ple tu­mor types in 1L & re­frac­to­ry sett­ings. Co-com­mer­cial as­set CNCT-19/CD19 CAR-T (part­nered w/ Ju­ven­tas): on­go­ing Ph 2 trials in B-NHL and B-ALL. [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]
Clerio Vision, Inc.
Nov­el oph­thalmic tech­nol­o­gy to treat re­frac­tive er­ror ad­dress­ing mul­ti-bil­lion mar­kets in con­tact lens­es (pres­by­opia, astig­ma­tism, my­opia, my­opia pro­gres­sion) and corneal vi­sion cor­rec­tion (non sur­g­i­cal fla­p­less las­er so­lu­tion). Om­ni­fo­cal con­tact lens pres­by­opia 510k sub­mis­sion and my­opia pro­gres­sion clin­i­cal da­ta in 2H2022. Based on No­bel Prize win­n­ing fem­to-se­cond las­er tech­nol­o­gy de­vel­oped by Bausch & Lomb and the Uni­ver­si­ty of Roch­ester. [more in­for­ma­tion]
Compass Pathways [CMPS] $313 MM MCap
Men­tal health com­pany fo­cused on psilo­cy­bin ther­a­py in con­junc­tion with psy­cho­log­i­cal sup­port, with FDA Break­through Ther­a­py desig­na­tion for lead as­set COM­P360 in treat­ment-re­sis­tant de­pres­sion. Top-line Ph 2b da­ta showed sig­ni­f­i­cant im­prove­ments in de­pres­sive symp­toms, Ph 3 to com­mence in Q4 '22. Add'l Ph 2 trials in PTSD and anorexia ner­vosa on­go­ing. [more in­for­ma­tion]
Com­mer­cial-stage neu­ro­s­cience com­pany lead­ing de­vel­op­ment and com­mer­cial­iza­tion of neu­ro­s­cience ther­a­pies. Two FDA-ap­proved drugs AZS­TARYS (AD­HD) and AD­LAR­I­TY (Alzheimer's Dis­ease) rep­re­sent com­bined peak sales po­ten­tial in ex­cess of >$1B. [more in­for­ma­tion]
CureVac [CVAC] $1,529 MM MCap
In Ju­ly 2020, Cure­Vac en­tered in a col­lab­o­ra­tion with GSK to joint­ly de­vel­op new prod­ucts in pro­phy­lac­tic vaccines for in­fec­tious dis­eas­es based on Cure­Vac’s se­cond-gen­er­a­tion mR­NA tech­nol­o­gy. This col­lab­o­ra­tion was lat­er ex­tend­ed to the de­vel­op­ment of se­cond-gen­er­a­tion COVID-19 vaccine can­di­dates, and mod­i­fied mR­NA vaccine tech­nolo­gies. Based on its pro­pri­e­tary tech­nol­o­gy, Cure­Vac has built a deep clin­i­cal pipe­line across the ar­eas of pro­phy­lac­tic vaccines, can­cer ther­a­pies, anti­body ther­a­pies, and the treat­ment of rare dis­eas­es. [more in­for­ma­tion]
Lead com­pound, lar­su­cos­terol, grant­ed FDA Fast Track Desig­na­tion for al­co­hol-as­so­ci­at­ed he­p­ati­tis (AH), re­sult­ed in 100% 28-day sur­vi­val for AH pa­tients in a Phase IIa study, with Phase IIb trial en­roll­ment com­ple­tion an­ti­ci­pat­ed 2Q23. [more in­for­ma­tion]
ESSA Pharma [EPIX] $136 MM MCap
De­vel­op­ing nov­el ther­a­pies for the treat­ment of pros­tate can­cer tar­get­ing the N-ter­mi­nal do­main of the an­dro­gen re­cep­tor. ES­SA’s lead clin­i­cal can­di­date, EPI-7386 is a high­ly-se­lec­tive, oral, small molecule in­hibi­tor of the N-ter­mi­nal do­main of the an­dro­gen re­cep­tor in de­vel­op­ment as a monother­a­py (Ph 1) and in com­bi­na­tion with stan­dard-of-care an­tian­dro­gens in pa­tients with cas­tra­tion-re­sis­tant pros­tate can­cer (Ph 1/2). [more in­for­ma­tion]
Galera [GRTX] $8 MM MCap
Clin­i­cal stage bio­phar­ma­ceu­ti­cal com­pany trans­form­ing ra­dio­ther­a­py in can­cer. Lead as­set ava­so­pasem is a po­tent, high­ly se­lec­tive small molecule dis­mu­tase mimet­ic be­ing de­vel­oped for se­vere oral mu­cosi­tis (SOM). To­p­line Ph 3 da­ta an­nounced in 4Q21, with NDA sub­mis­sion ex­pect­ed by the end of 2022. Com­ple­tion of en­roll­ment for se­cond as­set ru­cos­a­pasem in two an­ti-can­cer studies is ex­pect­ed 2H23. [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]
Hangzhou Chance Pharmaceuticals
Chance fo­cus­es on in­ha­la­tion prod­ucts with pro­pri­e­tary tech­nolo­gies. [more in­for­ma­tion]
Heidelberg Pharma AG [HPHA:DB] €158 MM MCap
AT­AC plat­form to de­vel­op nov­el can­cer treat­ments with a unique mode of ac­tion killing both in­ac­tive and di­vid­ing can­cers cells and over­com­ing tu­mor re­sis­tance mech­anisms. Lead prod­uct HDP-101, for blood can­cer, is ex­pect­ed to start clin­i­cal dev' end of 2021. Out-li­cens­ing the AT­AC tech­nol­o­gy us­ing the part­n­ers’ anti­bodies to cre­ate new AT­ACs and ex­pect long term in­come streams and ear­ly tech­nol­o­gy vali­da­tion. [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]
Immunocore [IMCR] $2,334 MM MCap
A pi­oneer­ing, com­mer­cial-stage T cell re­cep­tor biotech­nol­o­gy com­pany work­ing to de­vel­op and com­mer­cial­ize a new gen­er­a­tion of trans­for­ma­tive medicines to ad­dress un­met needs in can­cer, in­fec­tion and au­toim­mune dis­ease, with the first TCR ther­a­peu­tic to re­ceive reg­u­la­to­ry ap­pro­val. [more in­for­ma­tion]
Impact Therapeutics Inc
The JV is cur­rent­ly con­duct­ing a Phase II piv­o­tal study and a Phase III study of Se­na­parib in ovarian can­cer third-line treat­ment and first-line main­te­nance treat­ment in Chi­na. Out­side Chi­na, IM­PACT alone is con­duct­ing a Phase I/II study of Se­na­parib and te­mo­zolo­mide com­bi­na­tion ther­a­py in solid tu­mors and small cell lung can­cer treat­ment, as well as a Phase II study of Se­na­parib monother­a­py in pros­tate can­cer main­te­nance treat­ment. [more in­for­ma­tion]
IN8bio, Inc. [INAB] $33 MM MCap
First com­pany to ad­vance ge­net­i­cal­ly-mod­i­fied gam­ma-del­ta T cells in­to clin­i­cal trials with its Del­tEx Plat­form, based on ex-vi­vo ex­pand­ed and ac­ti­vat­ed gam­ma-del­ta T cells. Two in­vesti­ga­tor-ini­ti­at­ed Phase 1 clin­i­cal trials are un­der­way for lead prod­uct can­di­dates: INB-200 for new­ly di­ag­nosed glioblas­to­ma and INB-100 for acute leukemia. [more in­for­ma­tion]
Iovance Biotherapeutics [IOVA] $1,063 MM MCap
Lead­er in TIL cell ther­a­pies with a rolling BLA sub­mis­sion un­der­way for li­fileu­cel for me­tastat­ic me­lano­ma, which has the po­ten­tial to be­come the first ap­proved one-time cell ther­a­py for a solid tu­mor can­cer. [more in­for­ma­tion]
Junshi Biosciences [1877.HK]
Has a di­ver­si­fied R&D pipe­line com­pris­ing over 21 drug can­di­dates with ther­a­peu­tic ar­eas cov­er­ing can­cer, metabolic, au­toim­mune, neu­ro­log­i­cal, and in­fec­tious dis­eas­es. Lead can­di­date, Tori­pal­imab (JS001), reached over 1B RMB in sales, and ex­pand­ed in­di­ca­tions in­to na­sopha­ryn­geal car­ci­no­ma/urothe­lial car­ci­no­ma. Other 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 [more in­for­ma­tion]
Cre­at­ing the new gen­er­a­tion of bi­o­log­ics us­ing its plant-based plat­form that has de­mon­s­trat­ed an abil­i­ty to cre­ate po­ten­tial­ly bet­ter drug can­di­dates at a frac­tion of the time and cost of con­ven­tio­n­al plat­forms. Fo­cused on dis­cov­er­ing mon­o­clo­n­al anti­bodies tar­get­ing vali­dat­ed path­ways for rare and in­fec­tious dis­eas­es and, due to the speed and ac­cu­ra­cy of its anti­body pro­duc­tion sys­tem, is po­si­tioned well to de­vel­op fast fol­low­er ther­a­pies against vali­dat­ed drug tar­gets. [more in­for­ma­tion]
Keros Therapeutics [KROS] $915 MM MCap
A lead­er in un­der­s­tand­ing the role of the TGF-Be­ta fam­i­ly of pro­teins that is a mas­ter reg­u­la­tor of RBC and platelet pro­duc­tion as well as the growth, re­pair and main­te­nance of mus­cle and bone. Lead pro­tein ther­a­peu­tic prod­uct can­di­date, KER-050, be­ing de­vel­oped for the treat­ment of cy­tope­nias, in­clud­ing ane­mia and throm­bo­cy­tope­nia, in pa­tients with MDS and MF. KER-012 is be­ing de­vel­oped for the treat­ment of PAH and for the treat­ment of di­s­or­ders as­so­ci­at­ed with bone loss. [more in­for­ma­tion]
Lineage Cell Therapeutics Inc. [LCTX] $198 MM MCap
Cell ther­a­py com­pany with 3 clin­i­cal 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]
Longboard Pharmaceuticals [LBPH] $113 MM MCap
Long­board is eval­u­at­ing LP352, an oral, cen­tral­ly act­ing 5-hy­drox­ytryp­tamine 2C (5-HT2C) re­cep­tor su­per­a­g­on­ist, with neg­li­gi­ble ob­served im­pact on 5-HT2B and 5-HT2A re­cep­tor sub­types, in de­vel­op­ment for the po­ten­tial treat­ment of seizures as­so­ci­at­ed with a broad range of de­vel­op­men­tal and epilep­tic en­cephalo­pathies. The com­pany is al­so eval­u­at­ing LP659, a cen­tral­ly act­ing, sphin­go­sine-1-phos­phate (S1P) re­cep­tor sub­types 1 and 5 mo­d­u­la­tor and LP143, a cen­tral­ly act­ing, full cann­abi­noid type 2 re­cep­tor (CB2). [more in­for­ma­tion]
Monte Rosa Therapeutics [GLUE] $224 MM MCap
Mul­ti-as­set next-gen AI-driv­en molec­u­lar glue-based TPD plat­form. Lead pro­gram in Ph 1 fo­cused on GSP­T1, a key reg­u­la­tor and vul­n­er­a­bil­i­ty of MYC-driv­en solid tu­mors, in­clud­ing lung can­cer. [more in­for­ma­tion]
NexImmune [NEXI] $5 MM MCap
De­vel­op­ing unique ap­proach­es to T cell im­munother­a­pies based on its pro­pri­e­tary, mul­ti-anti­gen Ar­ti­fi­cial Im­mune Mo­d­u­la­tion (AIM™) tech­nol­o­gy. AIM™ tech­nol­o­gy em­ploys na­t­u­ral bi­ol­o­gy to di­rect spe­cif­ic T cell-me­di­at­ed im­mune re­spons­es ca­pa­ble of treat­ing a va­ri­e­ty of can­cers and au­toim­mune dis­eas­es. On­go­ing Phase I/II in R/R AML with da­ta ex­pect­ed around YE22. Ini­ti­at­ing a Phase I/II in HPV-me­di­at­ed solid tu­mors. Au­toim­mune pro­grams ad­vanc­ing quick­ly. [more in­for­ma­tion]
NorthSea Therapeutics
Fo­cused on de­vel­op­ing SE­FAs for the treat­ment of NASH and other metabolic di­s­or­ders. Phase 2b ICO­NA NASH trial icos­abu­tate showed sig­ni­f­i­cant de­creas­es in NASH and fi­brot­ic bio­mark­ers and is sche­d­uled to read­out biop­sy da­ta in 1Q23. Two ad­di­tio­n­al SE­FAs (1024 and 6179) in Phase 1 clin­i­cal trials for pa­tients with dys­lipi­demia and the or­phan in­di­ca­tion IFALD, re­spec­tive­ly. [more in­for­ma­tion]
Novaestiq Corporation
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Nykode Therapeutics [NYKD]
Clin­i­cal stage im­munother­a­py com­pany fo­cused on can­cer and in­fec­tious dis­ease. Mo­d­u­lar vaccine tech­nol­o­gy tar­gets anti­gens to Anti­gen Pre­sent­ing Cells to achieve rapid, strong and long-last­ing im­mune re­spons­es. Key pro­grams in­clude Ph2 pro­pri­e­tary HPV 16+ cer­vi­cal can­cer ther­a­peu­tic vaccine, Ph1/2 in­di­vi­d­u­al­ized can­cer neoanti­gen vaccine (Ge­nen­tech). Sig­ni­f­i­cant col­lab­o­ra­tions with Re­generon and Ge­nen­tech. [more in­for­ma­tion]
OnKure Therapeutics, Inc.
Fo­cused on the dis­cov­ery and de­vel­op­ment of best-in-class pre­ci­sion medicines that tar­get bi­o­log­i­cal­ly vali­dat­ed drivers of can­cer. Lead as­set OKI-179 is a class 1 HDAC in­hibi­tor in Ph 1b/2 com­bi­na­tion with MEK in­hibi­tor. [more in­for­ma­tion]
Pharming Group [PHAR] $759 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]
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] $28 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]
Redx Pharma [REDX]
Lead fi­bro­sis as­set, the se­lec­tive ROCK2 in­hibi­tor RX­C007, is in de­vel­op­ment for lung fi­bro­sis, with Ph2a to­p­line da­ta for idio­path­ic pul­mo­nary fi­bro­sis ex­pect­ed H223. Lead on­col­o­gy as­set, Por­cu­pine in­hibi­tor RX­C004, for Wnt de­pen­dent can­cers, Ph2 to­p­line da­ta ex­pect­ed start­ing in H123 and in­to H223. RX­C008, a GI-tar­get­ed ROCK in­hibi­tor for fi­brostenot­ic Crohn's dis­ease, is in pre-IND stage, com­menc­ing Ph1 clin­i­cal studies in late 2023. [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. For sub­se­quent pre-clin­i­cal de­vel­op­ment, Se­car­na's molecule de­sign and se­lec­tion work is com­ple­ment­ed by part­n­er­ships with in­ter­na­tio­n­al­ly renowned cen­ters of ex­cel­lence across academia and in­dus­try. [more in­for­ma­tion]
Sigilon Therapeutics, Inc. [SGTX] $57 MM MCap
Plat­form cell ther­a­py com­pany de­vel­op­ing po­ten­tial­ly func­tio­n­al cures for chron­ic dis­eas­es (rare blood, ly­so­so­mal dis­eas­es, and T1D); En­gi­neer­ing cells to pro­duce miss­ing pro­teins/en­zymes/etc; en­cap­su­late cells in pro­pri­e­tary sphere ma­trix (dev’d at Bob Langer's Lab at MIT) to pre­vent fi­bro­sis & im­mune re­jec­tion; 2 can­di­dates in clin­ic by year-end 2021 (SIG-001 in Hem A & SIG-005 in MPS-1); Eli Lil­ly col­lab­o­ra­tion in T1D ($75m ini­tial com­mit­ment, $410 mile­s­tones & sin­gle to dou­ble dig­it roy­al­ties) [more in­for­ma­tion]
Sonnet Biotherapeutics [SONN] $5 MM MCap
Clin­i­cal-stage mul­ti-as­set im­muno-on­col­o­gy ther­a­peu­tics plat­form com­pany fea­tur­ing pro­pri­e­tary tech­nol­o­gy im­prov­ing in vi­vo half-life and tu­mor tis­sue spe­ci­fic­i­ty of in­ter­leukins. SON-1010 (IL-12-FHAB) in phase I SAD and SON-080 (low dose IL-6) in phase 1b/2a. [more in­for­ma­tion]
Spruce Biosciences [SPRB] $92 MM MCap
Fo­cused on de­vel­op­ing and com­mer­cial­iz­ing nov­el ther­a­pies for rare en­docrine di­s­or­ders. The com­pany’s lead prod­uct can­di­date, Til­dac­er­font, is cur­rent­ly be­ing eval­u­at­ed for the treat­ment of clas­sic adult con­geni­tal adre­nal hy­per­pla­sia (CAH), and in up­com­ing studies in pe­di­a­tric clas­sic CAH and Po­ly­cys­tic Ovary Syn­drome (PCOS). [more in­for­ma­tion]
Sutro Biopharma [STRO] $185 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]
Treos Bio
De­vel­ops pre­ci­sion pep­tide can­cer im­munother­a­pies us­ing pa­tient HLA tar­get recog­ni­tion ge­net­ics based on pro­pri­e­tary com­pu­ta­tio­n­al da­ta sci­ence. Tre­os’ lead can­di­date is Po­lyPEPI-1018, an off-the-shelf im­munother­a­py for the treat­ment of me­tastat­ic col­orec­tal can­cer cur­rent­ly in a bio­mark­er-based Phase 1b clin­i­cal trial. Tre­os has raised $42 mil­lion to date. [more in­for­ma­tion]
Tryp Therapeutics [TRYPF] $4 MM MCap
Tryp Ther­a­peu­tics Inc is a phar­ma­ceu­ti­cal com­pany fo­cused on iden­ti­fy­ing and de­vel­op­ing clin­i­cal-stage com­pounds for or­phan dis­eas­es and other dis­eas­es with high un­met med­i­cal needs. The com­pany's main de­vel­op­ment pro­gram PFN pro­gram is de­signed to treat neu­ropsychi­a­tric di­s­or­ders through the dos­ing of for­mu­la­tions of syn­thet­ic psilo­cy­bin. [more in­for­ma­tion]
Vaccitech Plc [VACC] $119 MM MCap
Pro­pri­e­tary ChA­dOx (prime) and MVA (boost) tech­nol­o­gy plat­form in­duces CD8+ T cell and anti­body im­mune re­spons­es to treat and pre­vent in­fec­tious dis­eas­es and can­cer. Broad ther­a­peu­tic pipe­line. En­cour­ag­ing Ph1/2 in­ter­im ef­fi­ca­cy da­ta for VTP-300 (HBV func­tio­n­al cure) and VTP-200 (high-risk HPV ther­a­peu­tic) ef­fi­ca­cy da­ta ex­pect­ed 4Q2022. $15M roy­al­ty rev­enue dur­ing Q42021 from As­traZene­ca's com­mer­cial sales of Vaxzevria® (out-li­censed ChA­dOx COVID-19 vaccine) [more in­for­ma­tion]
Verrica Pharmaceuticals [VRCA] $169 MM MCap
A der­ma­tol­o­gy ther­a­peu­tics com­pany de­vel­op­ing med­i­ca­tions for skin dis­eas­es re­quir­ing med­i­cal in­ter­ven­tions. Ver­ri­ca's lead prod­uct can­di­date, VP-102, is a GMP-con­trolled for­mu­la­tion of can­tharidin de­liv­ered via a sin­gle-use ap­pli­ca­tor for the treat­ment of mol­lus­cum con­ta­gio­sum (mol­lus­cum), com­mon warts and ex­ter­nal geni­tal warts. The Com­pany is al­so de­vel­op­ing treat­ments for plan­tar warts (VP-103) and basal cell car­ci­no­ma (LTX-315). [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]
XOMA Corporation [XOMA] $162 MM MCap
Biotech roy­al­ty and mile­s­tone ag­gre­ga­tor with port­fo­lio of 70+ as­sets in >30 dis­closed in­di­ca­tions where R&D costs are borne by part­n­ers e.g. Roche's Faricimab (BLA filed for nAMD and DME 7/29/21) [more in­for­ma­tion]
Fo­cused on pro­vid­ing po­ten­tial first- and best-in-class medicines for pa­tients suf­fer­ing from de­bil­i­tat­ing neu­ro­log­i­cal dis­eas­es. XW10172 is a clin­i­cal-stage con­ju­gate of the GABAB ag­on­ist, oxy­bate, in de­vel­op­ment as an in­vesti­ga­tio­n­al once-night­ly ther­a­py in­tend­ed to reg­u­late the pa­tient’s sleep cy­cle in Parkin­son's dis­ease and nar­colep­sy. XW10508 is a glu­ta­mater­g­ic NM­DA an­ta­g­on­ist and AM­PA ac­ti­va­tor in de­vel­op­ment as an oral, once-dai­ly ther­a­py for ma­jor de­pres­sive di­s­or­der and chron­ic pain. [more in­for­ma­tion]
Zentalis [ZNTL] $1,402 MM MCap
De­vel­op­ing best-in-class small molecule ther­a­peu­tics tar­get­ing fun­da­men­tal bi­o­log­i­cal can­cer path­ways. Lead can­di­dates are ZN-c3, a Wee1 in­hibi­tor for ad­vanced solid tu­mors and ZN-d5, a BCL-2 in­hibi­tor for he­ma­to­log­ic ma­lig­nan­cies and re­lat­ed di­s­or­ders. Mul­ti­ple mono and com­bo trials are on­go­ing/planned across the pipe­line. [more in­for­ma­tion]