Looking at the pipelines of recent biotech IPOs is a great way to get a snapshot of new technologies and approaches to drug discovery or to see what’s coming in smaller indications. Complementing our recent highlight of large molecule-focused companies, below is a summary of 2020’s class of biotech IPOs focused on small molecule drug discovery.
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| Company | Symbol | Market cap. (Jul. 2021) | Lead molecule | Lead mechanisms | Stage | Other programs | Headquarters | Founded | |
|---|---|---|---|---|---|---|---|---|---|
| Schrodinger | https://www.schrodinger.com/ | SDGR | $5,474 | undisclosed | MALT1-inhibitor for RR-NHL | IND-enabling | CDC7, WEE1, HIF2a, SOS1/KRAS | New York, NY | 1995 |
| Relay Therapeutics | https://relaytx.com/ | RLAY | $3,317 | RLY-1971 | allosteric inhibitor of tyrosine phosphatase SHP2, the latter of which promotes cancer cell survival and growth | phase 1 | RLY-4008: inhibitor of a receptor tyrosine kinase FGFR2 to treat solid tumours, phase 1 | Cambridge, MA | 2015 |
| Revolution Medicines | https://www.revmed.com/ | RVMD | $2,449 | RMC-4630 | SHP2 inhibitor as the treatment for RAS-addicted cancers | phase 2 | RMC-5552: inhibitor of mTORC1 to preserve the tumour suppressive activity of 4EBP1 without the undesired inhibition of mTORC2, phase 1; RMC-4550: allosteric inhibitor of SHP2 | Redwood City, CA | 2014 |
| Zentalis Pharmaceuticals | https://zentalis.com/ | ZNTL | $2,126 | ZN-c5 | estrogen receptor degrader for the treatment of ER+/HER2- advanced or metastatic breast cancer | phase 2 | ZN-c3: inhibitor of WEE1, a DNA damage response protein, for the treatment of advanced solid tumors, phase 1/2 | New York, NY | 2014 |
| Kymera Therapeutics | https://www.kymeratx.com/ | KYMR | $1,998 | KT-474 | orally bioavailable IRAK4 degrader for the treatment of autoimmune and inflamatory diseases | phase 1 | STAT3 for tumors and autoimmune diseases, various undisclosed | Cambridge, MA | 2015 |
| Atea Pharmaceuticals | https://ateapharma.com/ | AVIR | $1,970 | AT-527 | oral purine nucleotide antiviral prodrug for COVID-19 | phase 3 | antiviral drugs for ssRNA viruses: dengue, HCV NS5B, NS5A, RSV | Boston, MA | 2012 |
| C4 Therapeutics | https://c4therapeutics.com/ | CCCC | $1,787 | CFT7455 | orally bioavailable monofunctional degrader targeting IKZF1/3 for treatment of hematologic malignancies | IND-enabling | CFT8634: oral bifunctional degradation activating compound targeting BRD9 in some solid tumors, preclinical | Watertown, MA | 2015 |
| Harmony Biosciences Holdings | https://www.harmonybiosciences.com/ | HRMY | $1,683 | pitolisant | approved H3 antagonist (pitolisant) in neurological disorders | approved | label expansion in pediatric narcolepsy, Prader-Willi, myotonic dystrophy | Plymouth Meeting, PA | 2017 |
| PMV Pharmaceuticals | https://www.pmvpharma.com/ | PMVP | $1,517 | PC14586 | oral small molecule p53 reactivator for the treatment of solid tumors that have a p53 Y220C mutation | phase 1 | undisclosed: p53 R273H | Cranbury, NJ | 2013 |
| Prelude Therapeutics | https://preludetx.com/ | PRLD | $1,498 | PRT543 | inhibitor of PRMT5 for advanced solid tumors and hematologic maliganncies | phase 1 | PRT811: brain penetrant PRMT5 inhibitor for treatment of GBM and CNS metastatic cancers, phase 1; PRT1419: inhibitor of the anti-apoptotic protein MCL1 for selected hematological malignancies | Wilmington, DE | 2016 |
| Kronos Bio | https://kronosbio.com/ | KRON | $1,400 | entospletinib | inhibitor of spleen tyrosine kinase to treat acute myeloid leukemia | phase 2 | KB-0742: oral CDK9 inhibitor for treatment of MYC-amplified solid tumors, IND-enabling | San Mateo, CA | 2017 |
| Arcutis Biotherapeutics | https://arcutis.com/ | ARQT | $1,375 | roflumilast | topical roflumilast cream; roflumilast is an inhibitor of phosphodiesterase type 4 (PDE4) for chronic plaque psoriasis and atopic dermatitis | approved/phase 3 | ARQ-154: foam formulation of roflumilast, phase 2; ARQ-252: topical JAK1 inhibitor to treat inflammatory dermatologic diseases, phase 2 | Westlake Village, CA | 2016 |
| Nurix Therapeutics | https://www.nurixtx.com/ | NRIX | $1,309 | NX-2127 | chimeric degrader of BTK for B-cell malignancies | phase 1 | NX-1607: oral Cbl-B inhibitor, various undisclosed | San Francisco, CA | 2009 |
| Repare Therapeutics | https://www.reparerx.com/ | RPTX | $1,240 | RP-3500 | oral ATR inhibitor in solid tumors | phase 1/2 | RP-6306: CCNE1-SL inhibitor to treat cancers, IND-enabling, RP-6306: PKMYT1 inhibitor for cancers; various synthetic lethal targets | St-Laurent, QC | 2016 |
| Forma Therapeutics Holdings | https://www.formatherapeutics.com/ | FMTX | $1,235 | olutasidenib | oral, brain-penetrant mIDH1 inhibitor for AML | phase 2 | etavopivat: allosteric PKR kinase inhibitor for the treatment of hemolytic anemias, phase 1; FT-7051: CBP/p300 inhibitor for mCRPC; FT-8225: FASN inhibitor for NASH; CC-95775: BETi for NHL, BI-1701963: KRASi for solid tumors | Watertown, MA | 2007 |
| Cullinan Oncology | https://www.cullinanoncology.com/ | CGEM | $1,197 | CLN-081 (TAS6417) | oral irreversible EGFR(ex20) inhibitor | Phase 1/2a | FLT3/CD3 mAb, MICA/B mAb, IL2/12 fusion, CD19/CD3 mAb | Cambridge, MA | 2017 |
| Olema Pharmaceuticals | https://olema.com/ | OLMA | $1,128 | OP-1250 | ER antagonist to treat metastatic breast cancer | phase 1/2 | n/a | San Francisco, CA | 2006 |
| Kinnate BioPharma | https://www.kinnate.com/ | KNTE | $1,076 | KIN002787 | RAF inhibitors for BRAF mutants to treat lung cancer, melanoma and other solide tumours | IND-enabling | KIN003: FGFR2/3 inhibitor, lead optimisation stage; KIN004: CDK12 inhibitor, lead optimisation stage | San Diego, CA | 2018 |
| Pliant Therapeutics | https://pliantrx.com/ | PLRX | $1,052 | PLN-74809 | dual selective inhibitor of aVb1/6 integrins to treat idiopathic pulmonary fibrosis and primary sclerosing cholangitis | phase 2 | PLN-1474: inhibitor of aVb1/6 in F3/F4 NASH-associated liver fibrosis, phase 1 | South San Francisco, CA | 2015 |
| Keros Therapeutics | https://www.kerostx.com/ | KROS | $1,046 | KER-047 | inhibitor of ALK2 kinase, a TGF-beta receptor for treatment of anemia | phase 1 | KER-050: fusion protein inhibitor of TGF-beta superfamily signling, for treatment of myelodysplastic syndrome, phase 2 | Lexington, MA | 2015 |
| Aligos Therapeutics | https://www.aligos.com/ | ALGS | $1,022 | ALG-000184 | capsid assembly modulator to treat chronic hepatitis B | phase 1 | HBsAg (oligos, ASOs; siRNA); THR-β SM agonist | South San Francisco, CA | 2018 |
| iTeos Therapeutics | https://www.iteostherapeutics.com/ | ITOS | $894 | inupadenant | ATP-adenosine pathway via A2AR inhibitor for immuno-oncology | phase 1b/2a | EOS-448: anti-TIGIT human IgG1 antibody to treat cancers, phase 1 | Cambridge, MA | 2011 |
| Praxis Precision Medicines | https://praxismedicines.com/ | PRAX | $877 | PRAX114 | GABAA receptor positive allosteric modulators to treat major depressive disoder and permenopausal depression | phase 2 | PRAX944: small-molecule inhibitor of T-type calcium channels to treat essential tremor, phase 2; RAX-562: persistent sodium current blocker to treat rare CNS disorders phase 1 | Cambridge, MA | 2015 |
| Oric Pharmaceuticals | https://oricpharma.com/ | ORIC | $806 | ORIC-101 | glucocorticoid receptor antagonist to overcome resistance mechanisms in multiple cancers | phase 1 | ORIC-533: CD73 inhibitor to enhance chemo. or immunotherapy; ORIC-944: PRC2/EED allosteric inhibitor | South San Francisco, CA | 2014 |
| Calliditas Therapeutics AB | https://www.calliditas.se/en/ | CALT | $722 | Nefecon | novel oral formulation of approved budesonide for IgA nephropathy | phase 3 | n/a | Stockholm, SE | 2004 |
| Annovis Bio | https://www.annovisbio.com/ | ANVS | $649 | ANVS401 | oral phenserine for neurological diseases | phase 2 | ANVS301: ibisnorcymcerine | Berwyn, PA | 2008 |
| Tarsus Pharmaceuticals | https://tarsusrx.com/ | TARS | $605 | TP-03 | eye-drop, parasite-specific GABA-Cl inh. to treat Demodex blepharitis | phase 3 | TP-05: preventive treatment for Lyme disease, phase 1; TP-04: treat rosacea, preclinical | Irvine, CA | 2016 |
| Black Diamond Therapeutics | https://www.blackdiamondtherapeutics.com/ | BDTX | $465 | BDTX-189 | oral irreversible HER2/EGFR(ex20) inhibitor to treat advanced solid tumors | phase 1 | BDTX-1535: targets allosteric EGFR mutations and brain penetrant, IND anticipated in 1H 2022 | Cambridge, MA | 2014 |
| Inventiva S.A | https://inventivapharma.com/ | IVA | $442 | lanifibranor | oral pan-PPAR agonist to induce anti-fibrotic, anti-inflammatory and beneficial metabolic changes in NASH | phase 3 | Odiparcil: decrease the lysosomal accumulation of chondroitin sulfate and dermatan sulfate in patients with certain mucopolysaccharidoses subtypes, phase 3; ABBV157: orally available retinoic acid-related orphan receptor gamma inverse-agonist to treat psoriasis, phase 2 | Bourgogne-Franche-Comte, FR | 2012 |
| Athira Pharma | https://www.athira.com/ | ATHA | $403 | ATH-1017 | brain-penetrant small molecule that activates signaling via HGF/MET receptor system to treat dementias | phase 2/3 | n/a | Bothell, Wa, WA | 2011 |
| Foghorn Therapeutics | https://foghorntx.com/ | FHTX | $379 | FHD-286 | allosteric BRG1/BRM ATPase inhibitor for AML and uveal melanoma | phase 1 | FHD-609: protein degrader of BRD9 to treat synovial sarcoma, phase 1 | Cambridge, MA | 2015 |
| Spruce Biosciences | https://sprucebiosciences.com/ | SPRB | $257 | tildacerfont | oral, brain penetrant CRF1 antagonist to treat congenital adrenal hyperplasia | phase 2b | n/a | Daly City, CA | 2014 |
| Orphazyme A/S | https://www.orphazyme.com/ | ORPH | $188 | arimoclimol | heat shock protein amplifier for lysosomal storage diseases | NDA rej. | n/a | Copenhagen, DK | 2009 |
| Lantern Pharma | https://www.lanternpharma.com/ | LTRN | $166 | LP-100 | oral DNA damaging agent (repaired by TC-NER) for cancer | phase 2 | LP-300: disulfide chemosensitizer, phase 2 | Dallas, TX | 2013 |
| Ayala Pharmaceuticals | https://www.ayalapharma.com/ | AYLA | $143 | AL-101 (formerly BMS-906024) | IV gamma secretase inhibitor for cancer treatment, phase 2, granted Orphan drug designation and fast track designation | phase 2 | AL-102: oral gamma secretase inhibitor, phase 1 | Wilmington, DE | 2017 |
| Imara | https://imaratx.com/ | IMRA | $138 | IMR-687 | oral PDE9 inhibitor for sickle cell disease and beta-thalassemia | phase 2 | n/a | Boston, MA | 2016 |
| Galecto | https://galecto.com/ | GLTO | $133 | GB0139 | inhaled Galectin-3 inhibitor for idiopathic pulmonary fibrosis | phase 2b | GB2064: oral LOXL2 inhibitor to treat myelofibrosis, phase 1/2a, GB1211: Galectin-3 inhibitor to treat liver fibrosis associated with non-alcoholic steatohepatitis (NASH) phase 1/2a | Copenhagen, DK | 2011 |
| Lyra Therapeutics | https://lyratherapeutics.com/ | LYRA | $110 | LYR-210 | anti-inflammatory implantable drug matrix containing mometasone furoate (approved) for chronic sinusitis | phase 2 | n/a | Watertown, MA | 2005 |
| Graybug Vision | https://www.graybug.vision/ | GRAY | $109 | GB-102 | intravitreally delivered microparticle depot formulation of sunitinib (pan-VEGF inhibitor) for treatment of wet age-related degeneration | phase 2b | GB-103: pan-VEGF inhibitor to treat wet age-related degeneration, IND-enabling; GB-401: injectable depot formulation of a beta-adrenergic antagonist prodrug to treat primary open angle glaucoma, IND-enabling | Redwood City, CA | 2015 |
| Metacrine | https://www.metacrine.com/ | MTCR | $103 | MET409 | oral farnesoid X receptor (FXR) agonist for NASH | phase 2 | MET642 : farnesoid X receptor agonist to treat non-alcoholic steatohepatitis, phase 1 (granted FDA Fast Track designation) | San Diego, CA | 2014 |
| Inhibikase Therapeutics | https://www.inhibikase.com/ | IKT | $73 | IkT-148009 | oral, brain-penetrant Abl kinase inhibitor for PD | phase 1 | iKT-001Pro: safer imatinib formulation, iKT-01427: oral, brain-penetrant JC virus entry inhibitor | Atlanta, GA | 2010 |
| Virios Therapeutics | https://www.virios.com/ | VIRI | $55 | IMC-1 (famciclovir + celecoxib) | combo. of approved drugs to provide antiviral activity against herpes simplex virus 1 to alleviate fibromyalgia symptoms | phase 2a, FDA Fast Track Review designation | n/a | Alpharetta, GA | 2012 |
| Lixte Biotechnology Holdings | https://lixte.com/ | LIXT | $40 | LB-100 | IV inhibitor of protein phosphatase PP2A for cancer treatment | phase 1 | LB-200: histone deacetlase inhibitor, pre-clinical | East Setauket, NY | 2005 |
