Hi everyone,
Welcome to all our new subscribers who joined us this week! We're thrilled to have you as part of The Weekly Pill community. Your readership means the world to us. If you could kindly share this newsletter with your friends or coworkers, we would greatly appreciate your support! This resource holds value for a wide range of individuals, including venture capitalists, students, professionals at all levels within pharmaceutical companies, and anyone with a curious mindset eager to learn. Your assistance in this endeavor is greatly appreciated.
I sincerely hope you enjoy reading our newsletter and that it adds value to your understanding of the biotech world. Wishing each of you a blessed week ahead, and I look forward to connecting with you all again next week for another update. This week is pretty heavy. Big week, big news, we are so back. Grab a coffee, sit tight and enjoy your reading! Psst..Paid section is way more fun!
If you really enjoy what we're doing here, consider becoming a paid subscriber to encourage us and gain access to the paid section, which includes insights on upcoming catalysts next week, the most shorted biotech stocks, earnings, market comments, notes and M&A comments. Now let’s dive into last week recap!
Table of contents
How the market performed this week
Licenses / Partnerships
Clinical trials
Financing
RIF
Disease of the week
What I’ve read this week
Paid Content Section
Upcoming Catalysts Next Week
Reminder: October-December Catalysts
Conference for the Rest of the Year
PDUFA Dates
Most Shorted Biotech Stock
Market Analysis
Option Market
Biotech EV
Notes: When it Rains, it Pours
Biotech Bankruptcy
Venture Capital Market
Biotech and Interest Rates
M&A
How the market performed this week
ETFs
Gene Therapy
RNA Therapy
Big Pharma
Gainers / Decliners this week in biotech
News of the week
Licenses/Partnerships
Ayala Pharmaceuticals, Inc. (ADXS)
Event Phase: II
Drug: Aspacytarabine
Indication: Acute Myelogenous Leukemia (AML)
Lead Indication: Yes
Molecule: Small Molecule
Target: DNA polymerase, DNA synthesis
LOA (Likelihood of Approval): 12%
Partner Companies: N/A
Source Link: Ayala Pharmaceuticals Announces Closing of Merger with Biosight
Ayala Pharmaceuticals, Inc. (ADXS)
Event Phase: II
Drug: Aspacytarabine
Indication: Myelodysplastic Syndrome (MDS)
Lead Indication: No
Molecule: Small Molecule
Target: DNA polymerase, DNA synthesis
LOA (Likelihood of Approval): 11%
Partner Companies: N/A
Source Link: Ayala Pharmaceuticals Announces Closing of Merger with Biosight
Merck & Co., Inc. (MRK)
Event Phase: III
Drug: Patritumab Deruxtecan
Indication: Non-Small Cell Lung Cancer (NSCLC)
Lead Indication: No
Molecule: Monoclonal Antibody
Target: ErbB3/HER3
LOA (Likelihood of Approval): 49%
Partner Companies: Daiichi Sankyo Co., Ltd. (4568)
Tourmaline Bio, Inc. (TRML)
Event Phase: II
Drug: TOUR006
Indication: Thyroid Eye Disease (TED)
Lead Indication: No
Molecule: Monoclonal Antibody
Target: IL-6 (Interleukin-6)
LOA (Likelihood of Approval): 24%
Partner Companies: Pfizer Inc. (PFE)
Source Link: Tourmaline Bio Announces Closing of Merger with Talaris Therapeutics and Concurrent Private Placement of $75 Million
Basilea Pharmaceutica Ltd. (BSLN)
Event Phase: I
Drug: BAL2062
Indication: Aspergillosis
Lead Indication: N
Molecule: Small Molecule
Target: Unknown
LOA: 13%
Partner Companies: Gravitas Therapeutics, Inc.
Source Link: Basilea announces acquisition of novel clinical stage antifungal for treatment of Aspergillus mold infections
Advicenne (ADVIC)
Event Phase: III
Drug: Sibnayal
Indication: Renal Disease / Renal Failure
Lead Indication: N
Molecule: Small Molecule
Target: Hydrogen, Potassium
LOA: 43%
Partner Companies: Avanzanite Bioscience B.V., FrostPharma AB, SPA - Antibiotic Products Co SpA, Taiba Healthcare
Source Link: Advicenne's Sibnayal Achieves Phase III Milestone in Renal Disease / Renal Failure
Takeda Pharmaceutical Co. Ltd. (TAK)
Event Phase: III
Drug: Soticlestat
Indication: Dravet Syndrome (Epilepsy)
Lead Indication: N
Molecule: Small Molecule
Target: Cytochrome p450
LOA: 50%
Partner Companies: Ligand Pharmaceuticals, Inc. (LGND)
Takeda Pharmaceutical Co. Ltd. (TAK)
Event Phase: III
Drug: Soticlestat
Indication: Lennox-Gastaut Syndrome (LGS; Epilepsy)
Lead Indication: Y
Molecule: Small Molecule
Target: Cytochrome p450
LOA: 50%
Partner Companies: Ligand Pharmaceuticals, Inc. (LGND)
AN2 Therapeutics, Inc. (ANTX)
Event Phase: Preclinical
Drug: AN2-502998
Indication: Anti-Parasitic and Anti-Protozoal
Lead Indication: N
Molecule: Small Molecule
Target: Unknown
Source Link: AN2 Therapeutics Advances Preclinical Drug Candidate for Anti-Parasitic and Anti-Protozoal
Glaukos Corporation (GKOS)
Event Phase: Preclinical
Drug: ST-113
Indication: Glaucoma / Ocular Hypertension (Ophthalmology)
Lead Indication: N
Molecule: Peptide
Target: Collagen
Partner Companies: Stuart Therapeutics, Inc.
Source Link: Stuart Therapeutics Announces Exclusive Worldwide License Agreement with Glaukos Corporation
Assembly Biosciences, Inc. (ASMB)
Event Phase: I
Drug: ABI-4334
Indication: Hepatitis B (HBV) Treatment (Antiviral)
Lead Indication: N
Molecule: Not Specified
Target: Hepatitis B/HBV
LOA: 13%
Partner Companies: Gilead Sciences, Inc. (GILD)
Source Link: Assembly Biosciences Initiates Phase I Clinical Development of ABI-4334 for Hepatitis B (HBV) Treatment
Assembly Biosciences, Inc. (ASMB)
Event Phase: I
Drug: ABI-5366
Indication: Herpes Simplex Virus (HSV) (Antiviral)
Lead Indication: N
Molecule: Small Molecule
Target: Helicase-primase enzyme complex - viral
LOA: 13%
Partner Companies: Gilead Sciences, Inc. (GILD)
Source Link: Assembly Biosciences Initiates Phase I Clinical Development of ABI-5366 for Herpes Simplex Virus (HSV) Treatment
Assembly Biosciences, Inc. (ASMB)
Event Phase: I
Drug: ABI-6250
Indication: Hepatitis D (HDV) (Antiviral)
Lead Indication: N
Molecule: Small Molecule
Target: Hepatitis D/HDV, Na+-taurocholate cotransporting polypeptide (NTCP)
LOA: 13%
Partner Companies: Gilead Sciences, Inc. (GILD)
Source Link: Assembly Biosciences Initiates Phase I Clinical Development of ABI-6250 for Hepatitis D (HDV) Treatment
Novo Nordisk A/S (NVO)
Event Phase: III
Drug: KBP-5074
Indication: Hypertension (Systemic)
Lead Indication: N
Molecule: Small Molecule
Target: Mineralocorticoid Receptor
LOA: 48%
Source Link: Novo Nordisk to Acquire Ocedurenone for Uncontrolled Hypertension from KBP Biosciences
Novo Nordisk A/S (NVO)
Event Phase: I
Drug: KBP-5074
Indication: Chronic Heart Failure and Cardiomyopathies - Unspecified
Lead Indication: N
Molecule: Small Molecule
Target: Mineralocorticoid Receptor
LOA: 5%
Source Link: Novo Nordisk to Acquire Ocedurenone for Uncontrolled Hypertension from KBP Biosciences
Novo Nordisk A/S (NVO)
Event Phase: II
Drug: KBP-5074
Indication: Chronic Kidney Disease (CKD)
Lead Indication: N
Molecule: Small Molecule
Target: Mineralocorticoid Receptor
LOA: 15%
Source Link: Novo Nordisk to Acquire Ocedurenone for Uncontrolled Hypertension from KBP Biosciences
Fortress Biotech Inc. (FBIO)
Event Phase: II
Drug: Triplex
Indication: Cytomegalovirus (CMV) Infection (Antiviral)
Lead Indication: Y
Molecule: Vaccine
Target: Human Cytomegalovirus (HCMV), Immune System
LOA: 23%
Partner Companies: City of Hope
Fortress Biotech Inc. (FBIO)
Event Phase: Preclinical
Drug: Triplex
Indication: HIV / AIDS
Lead Indication: N
Molecule: Vaccine
Target: Human Cytomegalovirus (HCMV), Immune System
Partner Companies: City of Hope
Daré Bioscience, Inc. (DARE)
Event Phase: Approved
Drug: Xaciato
Indication: Urinary Tract and Reproductive Tract Infections (Antibacterial)
Lead Indication: Y
Molecule: Small Molecule
Target: Bacterial ribosome, Protein synthesis
LOA: 100%
Partner Companies: Hammock Pharmaceuticals, Inc., Organon (OGN)
Clinical trials (LOA=likelihood of approval)
Johnson & Johnson (JNJ)
Event Phase: II
Drug: JNJ-1802
Indication: Dengue Fever - Vaccines and Treatments
Lead Indication: Yes
Molecule: Small Molecule
Target: Dengue virus
LOA: 23%
Partner Companies: N/A
Source Link: Janssen Announces Promising Antiviral Activity Against Dengue in a Phase 2a Human Challenge Model
Diamyd Medical AB (DMYDB)
Event Phase: II
Drug: Diamyd
Indication: Diabetes Mellitus, Type I
Lead Indication: Yes
Molecule: Vaccine
Target: Glutamic Acid Decarboxylase (GAD), Immune System
LOA: 5%
Partner Companies: N/A
Source Link: Link
Gilead Sciences, Inc. (GILD)
Event Phase: Approved
Drug: Sunlenca
Indication: HIV / AIDS
Lead Indication: Yes
Molecule: Small Molecule
Target: Viral Capsid
LOA: 100%
Partner Companies: N/A
TME Pharma N.V. (ALTME)
Event Phase: II
Drug: NOX-A12
Indication: Brain Cancer (Malignant Glioma; AA and glioblastoma (GBM))
Lead Indication: No
Molecule: Cellular
Target: Chemokine (C-X-C motif) Ligand 12 (CXCL12)/Stromal Cell-Derived Factor 1 (SDF-1)
LOA: 11%
Partner Companies: Merck & Co., Inc. (MRK)
Source Link: TME GLORIA: 18-Month Overall Survival
Merck & Co., Inc. (MRK)
Event Phase: Approved
Drug: Keytruda
Indication: Gastric Cancer
Lead Indication: No
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-1 receptor (PD-1)
LOA: 100%
Partner Companies: DRI Capital Inc.
Source Link: Link
Merck & Co., Inc. (MRK)
Event Phase: Approved
Drug: Keytruda
Indication: Non-Small Cell Lung Cancer (NSCLC)
Lead Indication: No
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-1 receptor (PD-1)
LOA: 100%
Partner Companies: DRI Capital Inc.
Source Link: Link
Eli Lilly and Company (LLY)
Event Phase: Approved
Drug: Verzenio
Indication: HR+/HER2- Breast Cancer
Lead Indication: Yes
Molecule: Small Molecule
Target: Cyclin Dependent Kinase 4 (CDK-4), Cyclin Dependent Kinase 6 (CDK-6)
LOA: 100%
Partner Companies: N/A
Source Link: Link
Arcutis Biotherapeutics, Inc. (ARQT)
Event Phase: Approved
Drug: Zoryve
Indication: Psoriasis
Lead Indication: Yes
Molecule: Small Molecule
Target: Phosphodiesterase 4 (PDE4)
LOA: 100%
Partner Companies: AstraZeneca PLC (AZN), Huadong Medicine Co., Ltd. (000963)
Mirati Therapeutics, Inc. (MRTX)
Event Phase: III
Drug: Sitravatinib
Indication: Non-Small Cell Lung Cancer (NSCLC)
Lead Indication: Yes
Molecule: Small Molecule
Target: EPH receptor B4 (EPHB4), Hepatocyte growth factor receptor (c-Met, HGFR), RET, VEGF Receptor (VEGFR)
LOA: 39%
Partner Companies: BeiGene, Ltd. (BGNE)
Source Link: Link
Merck & Co., Inc. (MRK)
Event Phase: III
Drug: Keytruda
Indication: HR+/HER2- Breast Cancer
Lead Indication: No
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-1 receptor (PD-1)
LOA: 47%
Partner Companies: DRI Capital Inc.
Source Link: Link
AstraZeneca PLC (AZN)
Event Phase: III
Drug: Imfinzi
Indication: Gastric Cancer
Lead Indication: No
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-ligand 1 (PD-L1)
LOA: 44%
Partner Companies: Bristol Myers Squibb Company (BMY), Eli Lilly and Company (LLY)
Source Link: Link
Bristol Myers Squibb Company (BMY)
Event Phase: Approved
Drug: Opdivo
Indication: Renal Cell Cancer (RCC)
Lead Indication: No
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-1 receptor (PD-1)
LOA: 100%
Partner Companies: Ono Pharmaceutical Company, Ltd. (4528:JP)
Source Link: Link
Cytokinetics, Inc. (CYTK)
Event Phase: III
Drug: Aficamten
Indication: Cardiomyopathy - Hypertrophic
Lead Indication: Yes
Molecule: Small Molecule
Target: Myosin
LOA: 52%
Partner Companies: Ji Xing Pharmaceuticals, Royalty Pharma plc (RPRX)
Cytokinetics, Inc. (CYTK)
Event Phase: I
Drug: CK-4021586
Indication: Chronic Heart Failure - Preserved Ejection Fraction (Chronic HFpEF)
Lead Indication: No
Molecule: Small Molecule
Target: Myosin
LOA: 5%
Partner Companies: N/A
Source Link: Link
Acurx Pharmaceuticals, Inc. (ACXP)
Event Phase: II
Drug: ACX-362E
Indication: Clostridium difficile-Associated Diarrhea/Infection (CDAD/CDI)
Lead Indication: Yes
Molecule: Small Molecule
Target: DNA polymerase
LOA: 23%
Partner Companies: N/A
MyMD Pharmaceuticals, Inc. (MYMD)
Event Phase: Preclinical
Drug: Supera-CBD
Indication: Inflammatory Pain
Lead Indication: Yes
Molecule: Small Molecule
Target: Cannabinoid reuptake/Endocannabinoid system, Cannabinoid-1 (CB1) receptor, Cannabinoid-2 (CB2) receptor
LOA: N/A
Partner Companies: N/A
Source Link: Link
Bristol Myers Squibb Company (BMY)
Event Phase: III
Drug: Opdivo (Subcutaneous)
Indication: Renal Cell Cancer (RCC)
Lead Indication: No
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-1 receptor (PD-1)
LOA: 44%
Partner Companies: Halozyme Therapeutics, Inc. (HALO)
Source Link: Link
AEON Biopharma, Inc. (AEON)
Event Phase: II
Drug: ABP-450
Indication: Migraine and Other Headaches
Lead Indication: No
Molecule: Protein
Target: Botulinum toxin, SNARE Proteins (e.g. synaptobrevin, syntaxin, SNAP-25)
LOA: 12%
Partner Companies: N/A
Tonix Pharmaceuticals Holding Corp. (TNXP)
Event Phase: Preclinical
Drug: TNX-801
Indication: Monkeypox (Mpox)
Lead Indication: No
Molecule: Vaccine
Target: Smallpox virus
LOA: N/A
Partner Companies: N/A
Source Link: Tonix Pharmaceuticals Presents Data from Its Horsepox-Based Vaccines Program at IDWeek 2023
AstraZeneca PLC (AZN)
Event Phase: I
Drug: AZD1390
Indication: Brain Cancer (Malignant Glioma; AA and glioblastoma (GBM))
Lead Indication: Y
Molecule: Small Molecule
Target: Ataxia Telangiectasia and Rad3-related Protein (ATR)/Frap-related Protein 1 (FRP1)
LOA: 5%
Partner Companies: None mentioned
Source Link: Link
Candel Therapeutics, Inc. (CADL)
Event Phase: I
Drug: CAN-3110
Indication: Brain Cancer (Malignant Glioma; AA and glioblastoma (GBM))
Lead Indication: Y
Molecule: Viral
Target: Herpes Simplex Virus (HSV)
LOA: 5%
Partner Companies: Brigham and Women's Hospital
Source Link: Link
Supernus Pharmaceuticals, Inc. (SUPN)
Event Phase: II
Drug: SPN-817
Indication: Partial / Focal Seizures (Epilepsy)
Lead Indication: Y
Molecule: Small Molecule
Target: Cholinesterases
LOA: 12%
Partner Companies: Global Drug Development Centre (GDCC) China
Source Link: Link
CytomX Therapeutics, Inc. (CTMX)
Event Phase: Preclinical
Drug: CX-2051
Indication: Solid Tumors
Lead Indication: N
Molecule: Monoclonal Antibody
Target: Epithelial cell surface antigen (Ep-CAM)/ CD326
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
Novartis AG (NVS)
Event Phase: Preclinical
Drug: Lamprene
Indication: Infectious Disease
Lead Indication: N
Molecule: Small Molecule
Target: Mycobacterial DNA, Phospholipases, various
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
AbbVie Inc. (ABBV)
Event Phase: Approved
Drug: Vraylar
Indication: Schizophrenia
Lead Indication: Y
Molecule: Small Molecule
Target: Dopamine 2 (D2) Receptor, Dopamine 3 (D3) Receptor, Serotonin 5-HT2B receptor
LOA: 100%
Partner Companies: Gedeon Richter Plc. (RICHT), Hikma Pharmaceuticals plc (HIK), Mitsubishi Tanabe Pharma Corporation, Recordati SpA (REC:IM)
Source Link: Link
Sanofi (SNY)
Event Phase: Approved
Drug: Tzield
Indication: Diabetes Mellitus, Type I
Lead Indication: Y
Molecule: Monoclonal Antibody
Target: Cluster of Differentiation 3 (CD3)
LOA: 100%
Partner Companies: MacroGenics, Inc. (MGNX), PDL BioPharma, Inc. (PDLI)
Source Link: Link
Roche Holding AG (RHHBY)
Event Phase: Approved
Drug: Alecensa
Indication: Non-Small Cell Lung Cancer (NSCLC)
Lead Indication: Y
Molecule: Small Molecule
Target: Anaplastic lymphoma kinase (ALK)
LOA: 100%
Partner Companies: Chugai Pharmaceutical Co., Ltd. (4519)
Source Link: Link
UCB S.A. (UCB)
Event Phase: Approved
Drug: Bimzelx
Indication: Psoriasis
Lead Indication: Y
Molecule: Monoclonal Antibody
Target: IL-17 (Interleukin 17)
LOA: 100%
Partner Companies: None mentioned
Source Link: Link
Hyloris Pharmaceuticals SA (HYL)
Event Phase: Approved
Drug: Combogesic IV
Indication: Postsurgical Pain
Lead Indication: Y
Molecule: Small Molecule
Target: Cyclooxygenase 1 (COX1) / Prostaglandin-Endoperoxide Synthase 1 (PTGS1), Cyclooxygenase 2 (COX2) / Prostaglandin-Endoperoxide Synthase 2 (PTGS2)
LOA: 100%
Partner Companies: AFT Pharmaceuticals Limited (AFT), Hikma Pharmaceuticals plc (HIK), Laboratoire Aguettant
Source Link: Link
Ultimovacs ASA (ULTIMO)
Event Phase: Development Outside U.S.
Drug: UV1
Indication: Mesothelioma
Lead Indication: N
Molecule: Vaccine
Target: Cluster of Differentiation 4 (CD4), Cluster of Differentiation 8 (CD8), Telomerase
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
IDEAYA Biosciences, Inc. (IDYA)
Event Phase: II
Drug: IDE196
Indication: Uveal Melanoma
Lead Indication: Y
Molecule: Small Molecule
Target: Protein Kinase C (PKC)
LOA: 13%
Partner Companies: Novartis AG (NVS), Roche Holding AG (RHHBY)
Source Link: Link
INmune Bio, Inc. (INMB)
Event Phase: II
Drug: XPro-1595
Indication: Alzheimer's Disease (AD)
Lead Indication: Y
Molecule: Protein
Target: Tumor Necrosis Factor-alpha (TNF-alpha)
LOA: 12%
Partner Companies: FPRT Bio, Xencor, Inc. (XNCR)
Source Link: Link
Checkpoint Therapeutics, Inc. (CKPT)
Event Phase: NDA/BLA
Drug: Cosibelimab
Indication: Skin Cancer - Squamous Cell Carcinoma (SCC)
Lead Indication: Y
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-ligand 1 (PD-L1)
LOA: 92%
Partner Companies: Fortress Biotech Inc. (FBIO), TG Therapeutics, Inc. (TGTX)
Source Link: Link
Zealand Pharma A/S (ZEAL)
Event Phase: Development Outside U.S.
Drug: ZP8396
Indication: Obesity
Lead Indication: Y
Molecule: Peptide
Target: Amylin Receptor
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
Medigene AG (MDG1)
Event Phase: Preclinical
Drug: MDG1015
Indication: Solid Tumors
Lead Indication: N
Molecule: Cellular
Target: NY-ESO-1 (Cancer-testis antigen)
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
AbbVie Inc. (ABBV)
Event Phase: Approved
Drug: Rinvoq
Indication: Ulcerative Colitis (UC)
Lead Indication: N
Molecule: Small Molecule
Target: JAK/STAT
LOA: 100%
Partner Companies: None mentioned
Source Link: Link
Apellis Pharmaceuticals, Inc. (APLS)
Event Phase: III
Drug: Syfovre
Indication: C3 Glomerulopathy (C3G), including Dense Deposit Disease (DDD) and C3 Glomerulonephritis (C3GN)
Lead Indication: N
Molecule: Peptide
Target: Complement component 3 (C3), Complement Pathway
LOA: 44%
Partner Companies: SFJ Pharmaceuticals, Inc, Swedish Orphan Biovitrum AB (SOBI)
Source Link: Link
Mirati Therapeutics, Inc. (MRTX)
Event Phase: Approved
Drug: Krazati
Indication: Non-Small Cell Lung Cancer (NSCLC)
Lead Indication: N
Molecule: Small Molecule
Target: KRas
LOA: 100%
Partner Companies: Zai Lab Ltd. (ZLAB)
Source Link: Link
Bristol Myers Squibb Company (BMY)
Defence Therapeutics Inc. (DTC)
Event Phase: Preclinical
Drug: AccuTOX-Chitosan
Indication: Hematologic Cancer
Lead Indication: N
Molecule: Small Molecule
Target: Cluster of Differentiation 47 (CD47), Cytotoxic T-Lymphocyte Antigen 4 (CTLA4), Programmed death-1 receptor (PD-1)
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
UCB S.A.
Event Phase: Approved
Drug: Zilbrysq
Indication: Myasthenia Gravis (MG)
Lead Indication: Y
Molecule: Peptide
Target: Complement component 5 (C5), Complement Pathway
LOA: 100%
Partner Companies: None mentioned
Source Link: Link
Evelo Biosciences, Inc. (EVLO)
Event Phase: Suspended
Drug: EDP2939
Indication: Psoriasis
Lead Indication: Y
Molecule: Bacterial Product
Target: Immune System, Microbiota (flora, microbiome) - intestinal
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
Medigene AG (MDG1)
Event Phase: Preclinical
Drug: MDG2011
Indication: Solid Tumors
Lead Indication: Y
Molecule: Cellular
Target: KRAS G12V, Stem Cells/Other Cell Therapies, T-Cell Receptor (TCR)
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
Viking Therapeutics, Inc. (VKTX)
Event Phase: II
Drug: VK2735
Indication: Obesity
Lead Indication: N
Molecule: Not Specified
Target: GIP Receptor (GIPR)/Glucose-Dependent Insulinotropic Polypeptide Receptor, GLP-1 Receptor
LOA: 25%
Partner Companies: None mentioned
Source Link: Link
Bristol Myers Squibb Company (BMY)
Event Phase: Approved
Drug: Opdivo
Indication: Bladder Cancer
Lead Indication: N
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-1 receptor (PD-1)
LOA: 100%
Partner Companies: Ono Pharmaceutical Company, Ltd. (4528:JP)
Source Link: Link
Kura Oncology, Inc. (KURA)
Event Phase: II
Drug: Tipifarnib (Oncology)
Indication: Head and Neck Cancer
Lead Indication: N
Molecule: Small Molecule
Target: Farnesyl transferase
LOA: 14%
Partner Companies: Johnson & Johnson (JNJ)
Source Link: Link
Morphic Therapeutic, Inc. (MORF)
Event Phase: II
Drug: MORF-057
Indication: Ulcerative Colitis (UC)
Lead Indication: N
Molecule: Not Specified
Target: Integrin Alpha-4 beta-7/LPAM
LOA: 20%
Partner Companies: None mentioned
Source Link: Link
Rhythm Pharmaceuticals, Inc. (RYTM)
Event Phase: Approved
Drug: Imcivree
Indication: Metabolic - General
Lead Indication: N
Molecule: Peptide
Target: Insulin Receptor, Melanocortin (MC) receptors
LOA: 100%
Partner Companies: Camurus AB (CAMX), Genpharm Services, Ipsen SA (IPSEY), RareStone Ltd.
Source Link: Link
Monte Rosa Therapeutics, Inc. (GLUE)
Event Phase: II
Drug: MRT-2359
Indication: Non-Small Cell Lung Cancer (NSCLC)
Lead Indication: N
Molecule: Small Molecule
Target: GSPT1
LOA: 11%
Partner Companies: None mentioned
Source Link: Link
Monte Rosa Therapeutics, Inc. (GLUE)
Event Phase: II
Drug: MRT-2359
Indication: Small Cell Lung Cancer (SCLC)
Lead Indication: N
Molecule: Small Molecule
Target: GSPT1
LOA: 11%
Partner Companies: None mentioned
Source Link: Link
NKGen Biotech, Inc.
Event Phase: I
Drug: SNK01
Indication: Alzheimer's Disease (AD)
Lead Indication: N
Molecule: Cellular
Target: Immune System, Natural Killer Cells (NK Cells)
LOA: 6%
Partner Companies: None mentioned
Source Link: Link
GSK plc
Event Phase: II
Drug: Jemperli
Indication: Non-Small Cell Lung Cancer (NSCLC)
Lead Indication: N
Molecule: Monoclonal Antibody
Target: Immune System, Programmed death-1 receptor (PD-1)
LOA: 11%
Partner Companies: AnaptysBio, Inc. (ANAB)
Source Link: Link
Palatin Technologies, Inc. (PTN)
Event Phase: Preclinical
Drug: Vyleesi
Indication: Obesity
Lead Indication: N
Molecule: Small Molecule
Target: Melanocortin (MC) receptors
LOA: Not specified
Partner Companies: Kwangdong Pharmaceutical, Shanghai Fosun Pharmaceutical (Group) Co., Ltd. (600196)
Source Link: Link
NextCure, Inc. (NXTC)
Event Phase: Preclinical
Drug: NC605
Indication: Osteogenesis Imperfecta (OI)
Lead Indication: N
Molecule: Monoclonal Antibody
Target: Siglec-15 (S15)
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
Sage Therapeutics, Inc. (SAGE)
Event Phase: III
Drug: SAGE-718
Indication: Huntington's Disease
Lead Indication: Y
Molecule: Small Molecule
Target: NMDA Glutamate Receptor
LOA: 46%
Partner Companies: None mentioned
Source Link: Link
Corbus Pharmaceuticals Holdings, Inc. (CRBP)
Event Phase: Preclinical
Drug: CRB-913
Indication: Obesity
Lead Indication: N
Molecule: Small Molecule
Target: Cannabinoid-1 (CB1) receptor
LOA: Not specified
Partner Companies: None mentioned
Source Link: Link
Cantargia AB (CANTA)
Event Phase: II
Drug: CAN04
Indication: Triple-Negative Breast Cancer (TNBC)
Lead Indication: N
Molecule: Monoclonal Antibody
Target: IL-1 Receptor (IL-1R)
LOA: 11%
Partner Companies: Cellerant Therapeutics, Inc.
Source Link: Link
Ambrx Biopharma, Inc. (AMAM)
Event Phase: II
Drug: ARX517
Indication: Prostate Cancer
Lead Indication: No
Molecule: Monoclonal Antibody
Target: Prostate-specific Membrane Antigen (PSMA)
Lack of Achievement (LOA): 11%
Partner Companies: None mentioned
Source Link: Ambrx ARX517 Phase 1/2 Trial Data
Philogen S.p.A. (PHIL)
Event Phase: III
Drug: Nidlegy
Indication: Melanoma
Lead Indication: Yes
Molecule: Monoclonal Antibody
Target: IL-2 (Interleukin-2), Tumor Necrosis Factor-alpha (TNF-alpha)
Lack of Achievement (LOA): 47%
Partner Companies: Sun Pharmaceutical Industries Ltd. (SUNP)
Source Link: Nidlegy Phase III Trial Data
Immunic Inc (IMUX)
Event Phase: Suspended
Drug: IMU-838
Indication: Ulcerative Colitis (UC)
Lead Indication: No
Molecule: Small Molecule
Target: Dihydroorotate Dehydrogenase (DHODH), DNA synthesis
Partner Companies: 4SC AG (VSC)
Source Link: IMU-838 Phase 2 Trial Data
Silo Pharma, Inc. (SILO)
Event Phase: Preclinical
Drug: SPU-21
Indication: Rheumatoid Arthritis (RA)
Lead Indication: No
Molecule: Peptide
Target: Unknown
Partner Companies: None mentioned
Source Link: SPU-21 Peptide Preclinical Data
NuCana plc (NCNA)
Event Phase: II
Drug: NUC-7738
Indication: Solid Tumors
Lead Indication: Yes
Molecule: Small Molecule
Target: Nucleoside/Nucleotide
Lack of Achievement (LOA): 11%
Partner Companies: None mentioned
Source Link: NUC-7738 Phase 2 Trial Data
SELLAS Life Sciences Group, Inc. (SLS)
Event Phase: II
Drug: SLS009
Indication: Acute Myelogenous Leukemia (AML)
Lead Indication: No
Molecule: Small Molecule
Target: Cyclin Dependent Kinase 9 (CDK-9)
Lack of Achievement (LOA): 11%
Partner Companies: GenFleet Therapeutics
Source Link: SLS009 Phase 2a Trial Data
Johnson & Johnson (JNJ)
Event Phase: III
Drug: Tremfya
Indication: Crohn's Disease
Lead Indication: No
Molecule: Monoclonal Antibody
Target: IL-23 (Interleukin-23)
Lack of Achievement (LOA): 63%
Partner Companies: MorphoSys AG (MOR), Otsuka Holdings Co., Ltd. (4578)
Source Link: Tremfya Phase 2 Study Data
Palatin Technologies, Inc. (PTN)
Event Phase: II
Drug: PL-8177
Indication: Ulcerative Colitis (UC)
Lead Indication: No
Molecule: Peptide
Target: Melanocortin (MC) receptors
Lack of Achievement (LOA): 19%
Partner Companies: None mentioned
Source Link: PL-8177 Ulcerative Colitis Data
Theriva Biologics, Inc. (TOVX)
Event Phase: I
Drug: VCN-01
Indication: Head and Neck Cancer
Lead Indication: No
Molecule: Viral
Target: Hyaluronic acid, Oncolytic Virus Therapy
Lack of Achievement (LOA): 5%
Partner Companies: Grifols, S.A. (GRFS)
Source Link: VCN-01 Phase 1 Study Data
Landos Biopharma, Inc. (LABP)
Event Phase: II
Drug: NX-13
Indication: Ulcerative Colitis (UC)
Lead Indication: No
Molecule: Small Molecule
Target: Mitochondria
Lack of Achievement (LOA): 19%
Partner Companies: LianBio (LIAN)
Source Link: NX-13 Phase 2 Trial Data
Oncolytics Biotech, Inc. (ONC)
Event Phase: III
Drug: Pelareorep
Indication: Pancreatic Cancer
Lead Indication: No
Molecule: Viral
Target: Immune System, Oncolytic Virus Therapy, Ras
Lack of Achievement (LOA): 43%
Partner Companies: Adlai Nortye Biopharma Co., Ltd. (ANL)
Source Link: Pelareorep in Pancreatic Cancer
Oncolytics Biotech, Inc. (ONC)
Event Phase: II
Drug: Pelareorep
Indication: Colorectal Cancer (CRC)
Lead Indication: No
Molecule: Viral
Target: Immune System, Oncolytic Virus Therapy, Ras
Lack of Achievement (LOA): 11%
Partner Companies: Adlai Nortye Biopharma Co., Ltd. (ANL)
Source Link: Pelareorep in Colorectal Cancer
Inhibikase Therapeutics, Inc. (IKT)
Event Phase: II
Drug: IkT-148009
Indication: Parkinson's Disease (PD)
Lead Indication: Yes
Molecule: Small Molecule
Target: ABL1
Lack of Achievement (LOA): 12%
Partner Companies: None mentioned
Source Link: Risvodetinib Phase 201 Trial Data
Tonix Pharmaceuticals Holding Corp. (TNXP)
Event Phase: II
Drug: TNX-601 ER
Indication: Major Depressive Disorder (MDD)
Lead Indication: No
Molecule: Small Molecule
Target: Serotonin Reuptake
Lack of Achievement (LOA): 14%
Partner Companies: None mentioned
Source Link: TNX-601 ER Phase 2 UPLIFT Study Data
Iovance Biotherapeutics, Inc. (IOVA)
Event Phase: NDA/BLA
Drug: LN-144
Indication: Melanoma
Lead Indication: Yes
Molecule: Cellular
Target: Immune System, Stem Cells/Other Cell Therapies, T lymphocytes
Lack of Achievement (LOA): 97%
Partner Companies: None mentioned
Source Link: Clinical Data for Lifileucel (LN-144)
Trevena Inc. (TRVN)
Event Phase: I
Drug: TRV045
Indication: Neuropathic Pain
Lead Indication: Yes
Molecule: Small Molecule
Target: Sphingosine 1-Phosphate Receptor (S1P-R)
Lack of Achievement (LOA): 6%
Partner Companies: None mentioned
Source Link: TRV045 Proof of Concept Studies
Trevena Inc. (TRVN)
Event Phase: Development Outside U.S.
Drug: TRV045
Indication: Seizure Disorders (Epilepsy)
Lead Indication: No
Molecule: Small Molecule
Target: Sphingosine 1-Phosphate Receptor (S1P-R)
Partner Companies: None mentioned
Source Link: TRV045 Development Outside U.S.
Ascendis Pharma A/S (ASND)
Event Phase: NDA/BLA
Drug: TransCon PTH
Indication: Hypoparathyroidism
Lead Indication: Yes
Molecule: Peptide
Target: Parathyroid Hormone Receptor (PTHR)
Lack of Achievement (LOA): 99%
Partner Companies: Visen Pharmaceuticals
Source Link: TransCon PTH Phase 3 52-Week Skeletal Dynamics Data
Equillium, Inc. (EQ)
Event Phase: Preclinical
Drug: EQ302
Indication: Celiac Disease
Lead Indication: No
Molecule: Peptide
Target: IL-15 (Interleukin-15)/IL-15 Receptor, IL-21 (Interleukin-21)
Partner Companies: None mentioned
Source Link: Equillium Preclinical Data
Aptose Biosciences Inc. (APS)
Event Phase: II
Drug: HM43239
Indication: Acute Myelogenous Leukemia (AML)
Lead Indication: Yes
Molecule: Small Molecule
Target: FMS-like tyrosine kinase 3 (FLT-3)
Lack of Achievement (LOA): 11%
Partner Companies: Hanmi Pharmaceutical Co., Ltd. (128940)
Source Link: Aptose Clinical and Preclinical Data
Kymera Therapeutics, Inc. (KYMR)
Event Phase: Preclinical
Drug: KTX-049
Indication: Merkel Cell Carcinoma
Lead Indication: No
Molecule: Small Molecule
Target: Mdm2, p53
Partner Companies: None mentioned
Source Link: Kymera Preclinical Data
AbbVie Inc. (ABBV)
Event Phase: Approved
Drug: Rinvoq
Indication: Crohn's Disease
Lead Indication: No
Molecule: Small Molecule
Target: JAK/STAT
Partner Companies: None mentioned
Source Link: Rinvoq Data
Abivax S.A. (ABVX)
Event Phase: III
Drug: Obefazimod
Indication: Ulcerative Colitis (UC)
Lead Indication: Yes
Molecule: Small Molecule
Target: Cytoplasmic CAP Binding Complex (CBC80/20), microRNA (miRNA), miR-124
Lack of Achievement (LOA): 67%
Partner Companies: None mentioned
Source Link: Obefazimod Clinical Data
Amgen, Inc. (AMGN)
Event Phase: I
Drug: Xaluritamig
Indication: Prostate Cancer
Lead Indication: Yes
Molecule: Monoclonal Antibody
Target: Cluster of Differentiation 3 (CD3), Immune System, STEAP1
Lack of Achievement (LOA): 5%
Partner Companies: Xencor, Inc. (XNCR)
Source Link: Amgen Oncology Research
Rhythm Pharmaceuticals, Inc. (RYTM)
Event Phase: Approved
Drug: Imcivree
Indication: Metabolic - General
Lead Indication: No
Molecule: Peptide
Target: Insulin Receptor, Melanocortin (MC) receptors
Partner Companies: Camurus AB (CAMX), Genpharm Services, Ipsen SA (IPSEY), RareStone Ltd.
Source Link: Imcivree Long-term Extension Study
Tiziana Life Sciences plc (TLSA)
Event Phase: II
Drug: Foralumab
Indication: Multiple Sclerosis (MS)
Lead Indication: No
Molecule: Monoclonal Antibody
Target: Cluster of Differentiation 3 (CD3)
Lack of Achievement (LOA): 12%
Partner Companies: AstraZeneca PLC (AZN)
Source Link: Foralumab Clinical Data
Financing events
Celcuity (NAS: CELC)
Description: Celcuity Inc is a clinical-stage biotechnology company focused on the development of targeted therapies for the treatment of multiple solid tumor indications. The company's therapeutic candidate is gedatolisib, a potent, well-tolerated, small molecule dual inhibitor, administered intravenously, that selectively targets all Class I isoforms of PI3K and the mammalian target of rapamycin (mTOR).
Verticals: Life Sciences, Oncology
Deal Date: 19-Oct-2023
Deal Type: PIPE
Deal Size: $50.00 million
Investors: Undisclosed investors
Deal Synopsis: The company (NAS: CELC) is in talks to receive $50 million of development capital from undisclosed investors through a private placement as of October 19, 2023. The company expects to use the net proceeds to advance the clinical development of gedatolisib and for general corporate purposes.
DermBiont
Description: Developer of microbial therapeutics designed to impact the root causes of skin diseases. The company's therapeutics offer mechanisms of action to repair an imbalance of the microbiome and leverage computational biology and a proprietary bioinformatics platform to identify clinically meaningful dysbiosis related to common skin diseases and mines a curated library of microbes to build a pipeline of products to treat identified dysbiosis across indications.
Verticals: Life Sciences
Deal Date: 19-Oct-2023
Deal Type: Later Stage VC
Deal Size: $26.61 million
Investors: Undisclosed investors
Entopsis
Description: Developer of novel diagnostic technologies designed to improve early detection and treatment of diseases. The company provides a universal platform to diagnose numerous diseases through urine samples, outperforming liquid biopsies and consisting of a series of time and cost-saving technologies for research and clinical testing.
Verticals: Life Sciences, Oncology
Deal Date: 19-Oct-2023
Deal Type: Later Stage VC
Deal Size: Not specified
Investors: In the process of raising venture funding on October 19, 2023.
Laronde
Description: Developer of eRNA-based therapeutics intended to make more predictable and impactful medicines for the world. The company's Endless RNA is a uniquely engineered RNA that can be programmed to express diverse proteins inside the body, also it is persistent, non-immunogenic, allows for repeat dosing, and offers flexibility in formulation and delivery.
Verticals: Life Sciences
Deal Date: 19-Oct-2023
Deal Type: Merger/Acquisition
Deal Size: Not specified
Investors: Sail Biomedicines
Deal Synopsis: The company was acquired by Sail Biomedicines for an undisclosed amount on October 19, 2023.
RS BioTherapeutics
Verticals: HealthTech
Deal Date: 19-Oct-2023
Deal Type: Seed Round
Deal Size: $1.21 million
Investors: Undisclosed investors
Biolexis Therapeutics
Description: Developer of an AI-enabled discovery platform for small molecule therapeutics.
Verticals: Artificial Intelligence & Machine Learning, Life Sciences, Oncology
Deal Date: October 17, 2023
Deal Type: Early Stage VC
Deal Size: $1.72 million
Investors: Undisclosed investors
Coriolis Pharma
Description: Operator of a biopharmaceutical research company specializing in formulation research and development.
Deal Date: October 16, 2023
Deal Type: Early Stage VC
Investors: Flerie, Frontier, Global Atlantic Financial Group
Dania Therapeutics
Description: Developer of medicinal products for cancer and related activities.
Verticals: Life Sciences, Oncology
Deal Date: October 18, 2023
Deal Type: Early Stage VC
Investors: Banque CPH, BioCapital Investments, Denmark's Export and Investment Fund, Eir Ventures
Nucleus RadioPharma
Description: Developer of novel treatments and technology in cancer research.
Verticals: Life Sciences, Oncology
Deal Date: October 17, 2023
Deal Type: Early Stage VC
Deal Size: $56 million
Investors: Mayo Clinic, GE Healthcare, Eclipse Ventures, University of Missouri, and others
VedaBio
Description: Developer of molecular diagnostics equipment.
Verticals: Life Sciences
Deal Date: October 17, 2023
Deal Type: Early Stage VC
Deal Size: $40 million
Investors: OMX Ventures, Kleinmuntz Associates, and others
Actym Therapeutics
Description: Developer of a therapeutic platform for cancer treatment.
Verticals: Life Sciences, Oncology
Deal Date: October 18, 2023
Deal Type: Later Stage VC
Deal Size: $59.5 million
Investors: Boehringer Ingelheim Venture Fund, Illumina Ventures, and others
Amplifier Therapeutics
Description: Operator of a biopharmaceutical company focused on AMPK compounds.
Verticals: HealthTech, Life Sciences, Oncology
Deal Date: October 17, 2023
Deal Type: Later Stage VC
Deal Size: $33.25 million
Investors: Cambrian Bio, Future Ventures, and others
Endogenex
Description: Developer of a novel endoscopic procedure for diabetes treatment.
Verticals: Digital Health, HealthTech, Life Sciences
Deal Date: October 17, 2023
Deal Type: Later Stage VC
Deal Size: $18.33 million
Investors: Undisclosed investors
Specific Biologics
Description: Developer of gene-editing technologies for genetic disease treatment.
Verticals: Life Sciences
Deal Date: October 16, 2023
Deal Type: Later Stage VC
Investors: adMare BioInnovations, Lumira Ventures
Variantyx
Description: Developer of whole genome analytical technology for rare inherited disorders.
Verticals: HealthTech, Life Sciences
Deal Date: October 18, 2023
Deal Type: Later Stage VC
Investors: Bosch Ventures, Peregrine Ventures, and others
Invivo Healthcare
Description: Developer of microbiome tests and supplements.
Verticals: Life Sciences
Deal Date: October 19, 2023
Deal Type: Merger/Acquisition
Deal Size: AUD 21.2 million
Acquirer: Microba (ASX: MAP)
Mablink
Description: Operator of a biotechnology company developing antibody-drug-conjugates drugs for cancer.
Verticals: Life Sciences, Oncology
Deal Date: October 18, 2023
Deal Type: Merger/Acquisition
Acquirer: Eli Lilly (NYS: LLY)
Olink Proteomics (NAS: OLK)
Description: Operator of a company with proprietary Proximity Extension Assay technology.
Verticals: HealthTech
Deal Date: October 17, 2023
Deal Type: Merger/Acquisition
Deal Size: $3.1 billion
Acquirer: Thermo Fisher Scientific (NYS: TMO)
Notable Labs
Description: Clinical-stage platform therapeutics company developing predictive precision medicines for cancer.
Verticals: Life Sciences, Oncology
Deal Date: October 16, 2023
Deal Type: Reverse Merger
Deal Size: $134.7 million
Investors: Builders VC, First Round Capital, and others
Flashpoint Therapeutics
Description: Developer of cancer immunotherapies with optimized nanoscale architecture.
Verticals: Life Sciences, Nanotechnology, Oncology
Deal Date: October 17, 2023
Deal Type: Seed Round
Deal Size: $10 million
Investor: Beta Lab
Tessellate Bio
Description: Operator of biotechnology company focusing on cancer treatments.
Verticals: Life Sciences, Oncology
Deal Date: October 17, 2023
Deal Type: Seed Round
Deal Size: EUR 8 million
Investors: Forbion, BioGeneration Ventures, and others
Reduction in force (RIF)
October 19 - Beam Therapeutics: The gene therapy biotech is laying off about 100 employees and rejigging its pipeline priorities to save cash, citing a “challenging market environment.” The workforce reduction equates to 20% of the entire workforce and is expected to be complete before the end of the year. Story
October 17 - Nkarta: The cell therapy company is set to lay off 18 employees, cuts that align with the company’s plan to zoom in on its later-stage programs. The 10% workforce reduction are an attempt to save cash and support operations through 2024, when Nkarta anticipates multiple clinical data readouts. Story
Disease of the week
Niemann-Pick Disease is a group of rare genetic disorders characterized by the abnormal accumulation of lipids, particularly sphingolipids, in various tissues and organs of the body. It is named after the two physicians who independently described the condition, Albert Niemann and Ludwig Pick. Niemann-Pick Disease affects both children and adults, and there are several different types and subtypes, each with its own distinct genetic cause and clinical features. The most common types of Niemann-Pick Disease are Type A, Type B, and Type C. Here is a cool video if you ever interested by it :
Genetics: Niemann-Pick Disease is an autosomal recessive genetic disorder, which means that a child must inherit a mutated copy of the responsible gene from both parents to develop the disease. The specific genes affected are responsible for producing proteins involved in lipid metabolism.
Subtypes: There are several subtypes of Niemann-Pick Disease, including:
a. Niemann-Pick Type A (NPA): This is the most severe form, typically diagnosed in infancy. It leads to rapid neurological decline, enlargement of the liver and spleen, and a shortened lifespan.
b. Niemann-Pick Type B (NPB): NPB primarily affects the liver and spleen, leading to hepatosplenomegaly. Unlike NPA, individuals with NPB usually do not experience the severe neurological symptoms seen in the Type A form.
c. Niemann-Pick Type C (NPC): NPC is more diverse in its presentation and can affect various organ systems, including the nervous system. Symptoms may include neurological problems, liver and spleen enlargement, and difficulties with coordination and balance.
Symptoms: The symptoms of Niemann-Pick Disease can include:
Enlargement of the liver and spleen (hepatosplenomegaly)
Jaundice
Difficulty swallowing and feeding problems
Progressive loss of motor and cognitive skills
Seizures
Respiratory issues
Visual disturbances
Difficulty with fine motor skills
In the case of NPC, some may develop psychiatric symptoms.
Diagnosis: Niemann-Pick Disease is typically diagnosed through genetic testing to identify the specific gene mutations. Additional tests such as blood tests, imaging studies, and tissue biopsies may be performed to assess the extent of organ involvement.
Treatment: Currently, there is no cure for Niemann-Pick Disease. Management focuses on alleviating symptoms and complications. In some cases, medications and therapies may be used to address specific symptoms. Research into potential therapies, including enzyme replacement therapy and substrate reduction therapy, is ongoing.
Prognosis: The prognosis for individuals with Niemann-Pick Disease varies depending on the subtype and the severity of their symptoms. Type A is often fatal in early childhood, while individuals with Type B and NPC can have more variable outcomes, with some living into adulthood. Supportive care and early intervention can improve the quality of life for affected individuals.
What I’ve read this week
*Click on the pic to read*
3PrimeInvest is must follow if you are in the Gene Editing/CRISPR space
