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The Weekly Pill

03/11/24-03/17/24

Mar 16, 2024
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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.

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!

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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

  • Reminders

  • Biotech Valuation

  • Most Shorted Biotech Stock as of This Week

  • Market

  • Notes from analysts

  • Venture Capital Market

  • M&A

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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

  • Sumitomo Pharma Co., Ltd.

    • Symbol: 4506

    • Event Phase: III

    • Drug: SEP-363856

    • Indication: Schizophrenia

    • Lead Indication: Y

    • Target: Trace Amine Associated Receptor 1 (TAAR1)

    • LOA: 52%

    • Partner Companies: Otsuka Pharmaceutical Co. Ltd., PsychoGenics Inc.

    • Source Link: Otsuka Pharmaceutical Co. Ltd. Press Release

  • Theratechnologies Inc.

    • Symbol: THTX

    • Event Phase: Approved

    • Drug: Trogarzo

    • Indication: HIV / AIDS Treatment

    • Lead Indication: Y

    • Target: Cluster of Differentiation 4 (CD4)

    • LOA: 100%

    • Partner Companies: AcedrA Pharmaceutical Company, Biogen, Inc. (BIIB), Roche Holding AG (RHHBY), TaiMed Biologics, Inc. (4147)

    • Source Link: TaiMed Biologics Inc. Press Release

  • First Wave BioPharma, Inc.

    • Symbol: FWBI

    • Event Phase: II

    • Drug: IMGX003

    • Indication: Celiac Disease

    • Lead Indication: Y

    • Target: Gluten

    • LOA: 19%

    • Partner Companies: AbbVie Inc. (ABBV)

    • Source Link: GlobeNewswire Press Release

  • Eloxx Pharmaceuticals, Inc.

    • Symbol: ELOX

    • Event Phase: IND

    • Drug: ZKN-013

    • Indication: Epidermolysis Bullosa

    • Lead Indication: N

    • Target: Adenomatous Polyposis Coli (APC), Collagen, Laminin Receptor

    • Partner Companies: Almirall SA (ALM)

    • Source Link: Almirall SA Press Release

  • NovaBay Pharmaceuticals, Inc.

    • Symbol: NBY

    • Event Phase: Approved

    • Drug: APP13007

    • Indication: Ocular Pain and/or Inflammation (Ophthalmology)

    • Lead Indication: Y

    • Target: Glucocorticoid Receptor (GR)

    • LOA: 100%

    • Partner Companies: Cristalia Produtos Quimicos Farmaceuticos Ltda., Eyenovia, Inc. (EYEN), Formosa Pharmaceuticals Inc., Grand Pharmaceutical Group Limited (512), Sosei Heptares (4565)

    • Source Link: GlobeNewswire Press Release

  • Allogene Therapeutics Inc.

    • Symbol: ALLO

    • Event Phase: Preclinical

    • Drug: ALLO-329

    • Indication: Autoimmune Disorders

    • Lead Indication: N

    • Target: Cluster of Differentiation 19 (CD19), Cluster of Differentiation 70 (CD70)

    • Partner Companies: N/A

    • Source Link: GlobeNewswire Press Release

  • Sosei Heptares

    • Symbol: 4565

    • Event Phase: Development Outside U.S.

    • Drug: HTL0048149

    • Indication: Schizophrenia

    • Lead Indication: N

    • Target: Unknown

    • Partner Companies: Boehringer Ingelheim GmbH

    • Source Link: GlobeNewswire Press Release

  • Merck & Co., Inc.

    • Symbol: MRK

    • Event Phase: II

    • Drug: HPN328

    • Indication: Small Cell Lung Cancer (SCLC)

    • Lead Indication: Y

    • Target: Delta-like 3 (DLL3), Human Serum Albumin (HSA), T lymphocytes, Tumor Cells

    • LOA: 11%

    • Partner Companies: N/A

    • Source Link: BusinessWire Press Release

  • Merck & Co., Inc.

    • Symbol: MRK

    • Event Phase: II

    • Drug: HPN536

    • Indication: Solid Tumors

    • Lead Indication: Y

    • Target: Human Serum Albumin (HSA), Mesothelin, T lymphocytes, Tumor Cells

    • LOA: 11%

    • Partner Companies: N/A

    • Source Link: BusinessWire Press Release

  • Merck & Co., Inc.

    • Symbol: MRK

    • Event Phase: I

    • Drug: HPN217

    • Indication: Multiple Myeloma (MM)

    • Lead Indication: Y

    • Target: B-cell maturation antigen (BCMA), Human Serum Albumin (HSA), Immune System, T lymphocytes, Tumor Cells

    • LOA: 5%

    • Partner Companies: N/A

    • Source Link: BusinessWire Press Release

  • Merck & Co., Inc.

    • Symbol: MRK

    • Event Phase: Preclinical

    • Drug: HPN601

    • Indication: Solid Tumors

    • Lead Indication: Y

    • Target: Immune System, T lymphocytes, Tumor Cells

    • Partner Companies: N/A

    • Source Link: BusinessWire Press Release

  • Merck & Co., Inc.

    • Symbol: MRK

    • Event Phase: Preclinical

    • Drug: TriTAC-XR

    • Indication: Cancer

    • Lead Indication: N

    • Target: Immune System, T lymphocytes, Tumor Cells

    • Partner Companies: N/A

    • Source Link: BusinessWire Press Release

  • Merck & Co., Inc.

    • Symbol: MRK

    • Event Phase: Preclinical

    • Drug: TROP2 ProTriTAC

    • Indication: Solid Tumors

    • Lead Indication: N

    • Target: Trop-2

    • Partner Companies: N/A

    • Source Link: BusinessWire Press Release

  • Merck & Co., Inc.

    • Symbol: MRK

    • Event Phase: Preclinical

    • Drug: ITGB6 ProTriTAC

    • Indication: Solid Tumors

    • Lead Indication: N

    • Target: Integrins

    • Partner Companies: N/A

    • Source Link: BusinessWire Press Release

  • Astellas Pharma, Inc.

    • Symbol: 4503:JP

    • Event Phase: Approved

    • Drug: Cresemba

    • Indication: Aspergillosis

    • Lead Indication: N

    • Target: Cytochrome p450

    • LOA: 100%

    • Partner Companies: Asahi Kasei Corporation (AHKSY), Avir Pharma, Inc., Basilea Pharmaceutica Ltd. (BSLN), Hikma Pharmaceuticals plc (HIK), Knight Therapeutics, Inc. (GUD), Pfizer Inc. (PFE)

    • Source Link: GlobeNewswire Press Release

Clinical trials (LOA=likelihood of approval)

  • BioMarin Pharmaceutical Inc. (BMRN)

    • Approved Voxzogo for Achondroplasia

    • Molecule: Peptide

    • Target: Natriuretic Peptide Receptors

    • LOA: 100%

    • Partner Companies: Chugai Pharmaceutical Co., Ltd. (4519)

    • Source Link: ACMG

  • Immuneering Corporation (IMRX)

    • Event Phase: II

    • Drug: IMM-1-104

    • Indication: Solid Tumors

    • Molecule: Small Molecule

    • Target: Mitogen-activated ERK kinase (MEK), Relaxin Family Peptide Receptors 1-4 (RXFP1-4)

    • LOA: 11%

    • Source Link: GlobeNewswire

  • Nuvectis Pharma, Inc. (NVCT)

    • Event Phase: I

    • Drug: NXP800

    • Indication: Ovarian Cancer

    • Molecule: Small Molecule

    • Target: General Control Nonderepressible 2 (GCN2), Heat shock transcription factor 1 (HSF-1)

    • LOA: 5%

    • Source Link: GlobeNewswire

  • Madrigal Pharmaceuticals, Inc. (MDGL)

    • Approved Rezdiffra for Non-Alcoholic Steatohepatitis (NASH)

    • Molecule: Small Molecule

    • Target: Thyroid hormone receptors (TRs)

    • LOA: 100%

    • Partner Companies: Roche Holding AG (RHHBY)

    • Source Link: GlobeNewswire

  • Aquestive Therapeutics, Inc. (AQST)

    • Event Phase: III

    • Drug: Anaphylm for Anaphylaxis

    • Molecule: Small Molecule

    • Target: Alpha Adrenergic Receptors, Beta Adrenergic Receptors

    • LOA: 65%

    • Source Link: Aquestive

  • Cantargia AB (CANTA)

    • Event Phase: IND

    • Drug: CAN10 for Systemic Sclerosis

    • Molecule: Monoclonal Antibody

    • Target: IL-1 (Interleukin-1)

    • Source Link: Accesswire

  • Bristol Myers Squibb Company (BMY)

    • Approved Breyanzi for Chronic Lymphocytic Leukemia (CLL)/Small Cell Lymphocytic Lymphoma (SLL) - NHL

    • Molecule: Cellular

    • Target: Autologous Chimeric Antigen Receptor T-cells (CAR-T), Cluster of Differentiation 19 (CD19), T lymphocytes

    • LOA: 100%

    • Source Link: BusinessWire

  • BioNxt Solutions Inc. (BNXT)

    • Event Phase: Preclinical

    • Drug: Cladribine (BioNxt) for Multiple Sclerosis (MS)

    • Molecule: Small Molecule

    • Target: Ribonucleotide Reductase (RNR)

    • Source Link: Accesswire

  • Revelation Biosciences Inc. (REVB)

    • Event Phase: Preclinical

    • Drug: REVTx-300 for Acute Kidney Injury (AKI)/Acute Renal Failure (ARF)

    • Molecule: Small Molecule

    • Target: Immune System

    • Source Link: BusinessWire

  • Ionis Pharmaceuticals, Inc. (IONS)

    • Event Phase: II

    • Drug: ION224 for Non-Alcoholic Steatohepatitis (NASH)

    • Molecule: Antisense

    • Target: Diglycerol Acyltransferase (DGAT)

    • LOA: 17%

    • Source Link: PRNewswire

  • Silence Therapeutics plc (SLN)

    • Event Phase: I

    • Drug: Zerlasiran for Dyslipidemia / Hypercholesterolemia

    • Molecule: siRNA/RNAi

    • Target: Lipoprotein (a) [Lp(a)]

    • LOA: 6%

    • Source Link: BusinessWire

  • Spruce Biosciences, Inc. (SPRB)

    • Event Phase: II

    • Drug: Tildacerfont for Congenital Adrenal Hyperplasia (CAH)

    • Molecule: Small Molecule

    • Target: Corticotropin Releasing Factor - Receptor1 (CRF-R1)

    • LOA: 17%

    • Partner Companies: Kaken Pharmaceutical Company, Ltd (4521)

    • Source Link: BusinessWire

  • Xencor, Inc. (XNCR)

    • Event Phase: II

    • Drug: XmAb20717 for Solid Tumors

    • Molecule: Monoclonal Antibody

    • Target: Cluster of Differentiation 3 (CD3), Cytotoxic T-Lymphocyte Antigen 4 (CTLA4), Programmed death-1 receptor (PD-1)

    • LOA: 12%

    • Source Link: Xencor Investors

  • Cybin Inc. (CYBN)

    • Event Phase: II

    • Drug: CYB-003 for Major Depressive Disorder (MDD)

    • Molecule: Small Molecule

    • Target: Serotonin Receptors - Unspecified

    • LOA: 14%

    • Source Link: BusinessWire

  • Pfizer Inc. (PFE)

    • Event Phase: III

    • Drug: Adcetris for Diffuse Large B-Cell Lymphoma (DLBCL) - NHL

    • Molecule: Monoclonal Antibody

    • Target: Cluster of Differentiation 30 (CD30) / TNFRSF8, Microtubules (Tubulin)

    • LOA: 46%

    • Partner Companies: Takeda Pharmaceutical Co. Ltd. (TAK)

    • Source Link: BusinessWire

  • Arcutis Biotherapeutics, Inc. (ARQT)

    • Approved ZORYVE for Dermatitis of the Scalp

    • Molecule: Small Molecule

    • Target: Phosphodiesterase 4 (PDE4)

    • LOA: 100%

    • Partner Companies: AstraZeneca PLC (AZN), Huadong Medicine Co., Ltd. (000963), Sato Pharmaceutical Co., Ltd.

    • Source Link: ePosters

  • Regulus Therapeutics Inc. (RGLS)

    • Event Phase: I

    • Drug: RGLS8429 for Polycystic Kidney Disease

    • Molecule: microRNA

    • Target: microRNA (miRNA), miR-17

    • LOA: 10%

    • Source Link: PRNewswire

  • Crinetics Pharmaceuticals, Inc. (CRNX)

    • Event Phase: II

    • Drug: Paltusotine for Carcinoid Syndrome

    • Molecule: Small Molecule

    • Target: Somatostatin Receptors

    • LOA: 11%

    • Partner Companies: Sanwa Kagaku Kenkyusho Co., Ltd.

    • Source Link: Crinetics

  • Fractyl Health (GUTS)

    • Event Phase: Preclinical

    • Drug: RJVA-001 for Diabetes Mellitus, Type II

    • Molecule: Viral Gene Therapy

    • Target: Unknown

    • Source Link: GlobeNewswire

  • Kineta, Inc. (KA)

    • Event Phase: II

    • Drug: KVA12123 for Solid Tumors

    • Molecule: Monoclonal Antibody

    • Target: VISTA (V-domain immunoglobulin-containing suppressor of T-cell activation)

    • LOA: 11%

    • Source Link: GlobeNewswire

  • Marvel Biosciences Corp. (MRVL)

    • Development Outside U.S.: MB-204 for Autism Spectrum Disorders (Autism)

    • Molecule: Not Specified

    • Target: Adenosine A2a Receptor

    • Source Link: Newsfilecorp

  • Tempest Therapeutics, Inc. (TPST)

    • Event Phase: Preclinical

    • Drug: TPST-1120 for Solid Tumors

    • Molecule: Small Molecule

    • Target: PPAR alpha

    • Source Link: GlobeNewswire

  • AB Science S.A. (AB)

    • Event Phase: III

    • Drug: Masitinib

    • Indication: Amyotrophic Lateral Sclerosis (ALS)

    • Molecule: Small Molecule

    • Target: Fibroblast Growth Factor Receptor (FGFR), KIT/c-KIT, Platelet-derived growth factor receptor (PDGFR)

    • LOA: 46%

    • Partner Companies: University of Chicago

    • Source Link: GlobeNewswire

  • Unicycive Therapeutics, Inc. (UNCY)

    • Event Phase: Preclinical

    • Drug: UNI-494

    • Indication: Acute Kidney Injury (AKI)/Acute Renal Failure (ARF)

    • Molecule: Small Molecule

    • Target: Potassium channels

    • LOA: Not specified

    • Partner Companies: Sphaera Pharma Pte. Ltd.

    • Source Link: GlobeNewswire

  • Poseida Therapeutics, Inc. (PSTX)

    • Event Phase: I

    • Drug: P-BCMA-ALLO1

    • Indication: Multiple Myeloma (MM)

    • Molecule: Cellular

    • Target: Allogeneic Chimeric Antigen Receptor T-cells (CAR-T), B-cell maturation antigen (BCMA), Immune System, Stem Cells/Other Cell Therapies

    • LOA: 5%

    • Partner Companies: Amgen, Inc. (AMGN), Johnson & Johnson (JNJ), Roche Holding AG (RHHBY)

    • Source Link: PRNewswire

  • Tonix Pharmaceuticals Holding Corp. (TNXP)

    • Event Phase: III

    • Drug: Tonmya

    • Indication: Fibromyalgia

    • Molecule: Small Molecule

    • Target: Alpha 2 Adrenergic Receptor, Serotonin 5-HT2A receptor

    • LOA: 39%

    • Partner Companies: Not specified

    • Source Link: GlobeNewswire

  • ACADIA Pharmaceuticals Inc. (ACAD)

    • Status: Suspended

    • Drug: Nuplazid

    • Indication: Schizophrenia

    • Molecule: Small Molecule

    • Target: Serotonin 5-HT2A receptor

    • Source Link: BusinessWire

  • Avenue Therapeutics Inc. (ATXI)

    • Event Phase: Preclinical

    • Drug: BAER-101

    • Indication: Seizure Disorders (Epilepsy)

    • Molecule: Small Molecule

    • Target: GABA Receptors

    • LOA: Not specified

    • Partner Companies: AstraZeneca PLC (AZN), Cincinnati Children's Hospital Medical Center

    • Source Link: GlobeNewswire

  • Ventyx Biosciences, Inc. (VTYX)

    • VTX-3232:

      • Event Phase: I

      • Indication: Parkinson's Disease (PD)

      • Molecule: Small Molecule

      • Target: NLRP3/Inflammasome

      • LOA: 6%

      • Partner Companies: Not specified

      • Source Link: GlobeNewswire

    • ZMG-2735:

      • Event Phase: II

      • Indication: Cryopyrin (CIAS1)-Associated Periodic Syndromes (CAPS)

      • Molecule: Small Molecule

      • Target: NLRP3/Inflammasome

      • LOA: 19%

      • Partner Companies: Not specified

      • Source Link: GlobeNewswire

  • BioSenic SA (BIOS)

    • Status: Development Outside U.S.

    • Drug: Arscimed

    • Indication: Graft vs. Host Disease (GVHD) - Treatment

    • Molecule: Small Molecule

    • Target: Reactive Oxygen Species/Free Radicals, T lymphocytes

    • Partner Companies: Phebra Pty Ltd

    • Source Link: GlobeNewswire

Financing events

Amolyt Pharma

  • Description: Operator of a clinical-stage biotechnology company intended for the treatment of endocrine and metabolic disorders. The company offers severe insulin resistance to control the symptoms of hypoparathyroidism by safely producing sustained levels of calcium in the blood, enabling healthcare professionals to combat critical and rare metabolic conditions.

  • Verticals: HealthTech, Life Sciences, Oncology

  • Deal Date: 14-mars-2024

  • Deal Type: Merger/Acquisition

  • Deal Synopsis: The company reached a definitive agreement to be acquired by AstraZeneca (LON: AZN) for $1.05 billion on March 14, 2024.

  • Investors: AstraZeneca (LON: AZN)(Marc Dunoyer)

  • Deal Size: $1,050.00

  • Apogee Therapeutics (NAS: APGE)

    • Description: Apogee Therapeutics Inc is a biotechnology company seeking to develop differentiated biologics for the treatment of atopic dermatitis (AD), chronic obstructive pulmonary disease (COPD) and related inflammatory and immunology (I&I) indications with high unmet need.

    • Verticals: Life Sciences

    • Deal Date: 11-mars-2024

    • Deal Type: Public Investment 2nd Offering

    • Deal Synopsis: The company raised $420 million in its second public offering on the Nasdaq stock exchange under the ticker symbol of APGE on March 11, 2024.

    • Deal Size: $420.00

  • Aprea Therapeutics (NAS: APRE)

    • Description: Aprea Therapeutics Inc is a clinical-stage biopharmaceutical company focused on developing and commercializing cancer therapeutics that reactivate mutant p53 tumor suppressor protein.

    • Verticals: Life Sciences, Oncology

    • Deal Date: 11-mars-2024

    • Deal Type: PIPE

    • Deal Synopsis: The company (NAS:APRE) is in talks to receive $16 million of development capital from various investors on March 11, 2024, over 2 tranches through a private placement.

    • Investors: DAFNA Capital Management, Exome Asset Management, Nantahala Capital Management, Sphera Healthcare, Stonepine Capital Management

    • Deal Size: $16.00

  • Asgard Therapeutics

    • Description: Operator of a private biotech company intended to explore the application of direct cell reprogramming technologies for cancer immunotherapies.

    • Verticals: Life Sciences, Oncology

    • Deal Date: 14-mars-2024

    • Deal Type: Later Stage VC

    • Deal Synopsis: The company raised EUR 30 million of Series A venture funding in a deal led by RV Invest and Johnson & Johnson Innovation - JJDC on March 14, 2024.

    • Investors: Boehringer Ingelheim Venture Fund(Philipp Mueller), Industrifonden(Jonas Jendi), Johnson & Johnson Innovation - JJDC(Fiona MacLaughlin), Novo Holdings(João Ribas), RV Invest(Aleksei Zeifman)

    • Deal Size: EUR 32.51

  • Cognition Therapeutics (NAS: CGTX)

    • Description: Cognition Therapeutics Inc is a clinical-stage biopharmaceutical company engaged in the discovery and development of innovative, small-molecule therapeutics targeting age-related degenerative diseases and disorders of the central nervous system and retina.

    • Verticals: Life Sciences

    • Deal Date: 13-mars-2024

    • Deal Type: Public Investment 2nd Offering

    • Deal Synopsis: The company raised $11.5 million in its second public offering on the Nasdaq stock exchange under the ticker symbol of CGTX on March 13, 2024.

    • Deal Size: $11.50

      • Cybin (Drug Discovery) (NEOE: CYBN)

        • Description: Cybin Inc is a biopharmaceutical company involved in creating safe and effective therapeutics for patients to address a multitude of mental health issues.

        • Verticals: Life Sciences

        • Deal Date: 13-mars-2024

        • Deal Type: PIPE

        • Deal Synopsis: The company (NEOE:CYBN) is in talks to receive $150 million of development capital from various investors through a private placement as of March 13, 2024.

        • Investors: Acorn Bioventures, Altium Capital, Avidity Partners, Deep Track Capital, Logos Capital, Octagon Capital, RA Capital Management, Rosalind Advisors, Sphera Healthcare

        • Deal Size: $150.00

      • Deep Origin

        • Description: Developer of biology research and development accelerator technology intended for accelerating basic science, drug development, and bioengineering.

        • Verticals: Life Sciences

        • Deal Date: 13-mars-2024

        • Deal Type: Early Stage VC

        • Deal Synopsis: The company raised $25.5 million of venture funding from undisclosed investors on March 13, 2024.

        • Deal Size: $25.50

      • DepYmed

        • Description: Developer of potent inhibitors of the enzyme PTP1B intended to offer therapeutics for cancer and rare diseases.

        • Verticals: Life Sciences, Oncology

        • Deal Date: 11-mars-2024

        • Deal Type: Later Stage VC

        • Deal Synopsis: The company raised $500,000 of venture funding from undisclosed investors on March 11, 2024.

        • Deal Size: $0.50

      • Intelligent Bio Solutions (NAS: INBS)

        • Description: Intelligent Bio Solutions Inc is a life sciences company developing non-invasive, real-time monitoring and diagnostic tests for patients and their primary health practitioners.

        • Verticals: Life Sciences

        • Deal Date: 12-mars-2024

        • Deal Type: PIPE

        • Deal Synopsis: The company (NAS:INBS) received $10.1 million of development capital from undisclosed investors on March 12, 2024 through a private placement.

        • Deal Size: $10.10

      • Lexeo Therapeutics (NAS: LXEO)

        • Description: Lexeo Therapeutics Inc is a clinical-stage genetic medicines company dedicated to transforming healthcare by applying pioneering science to fundamentally change how disease is treated.

        • Verticals: Life Sciences, LOHAS & Wellness, Oncology

        • Deal Date: 13-mars-2024

        • Deal Type: PIPE

        • Deal Synopsis: The company (NAS: LXEO) received $95 million of development capital from various investors through a private placement on March 13, 2024.

        • Investors: Eventide Asset Management, Invus Opportunities, Laurion Capital Management, Longitude Capital, Novo Holdings, Omega Funds(Bernard Davitian), RA Capital Management, Surveyor Capital, Woodline Partners

        • Deal Size: $95.00

        • exicon Pharmaceuticals (NAS: LXRX)

          • Description: Lexicon Pharmaceuticals Inc is a biopharmaceutical company engaged in the discovery, development, and commercialization of pharmaceutical products for the treatment of human diseases.

          • Verticals: Life Sciences

          • Deal Date: 11-mars-2024

          • Deal Type: PIPE

          • Deal Synopsis: The company (NAS:LXRX) is in talks to receive $250 million of development capital from various investors through a private placement as of March 11, 2024.

          • Investors: Braidwell, Great Point Partners, OrbiMed, The Invus Group(Philippe Amouyal)

          • Deal Size: $250.00

        • Locate Bio

          • Description: Developer of a regenerative medicine platform designed to help surgeons restore patients' lives through research, innovation, and performance.

          • Verticals: Life Sciences

          • Deal Date: 14-mars-2024

          • Deal Type: Later Stage VC

          • Deal Synopsis: The company raised GBP 8.4 million of venture funding from Mercia Asset Management and other undisclosed investors on March 14, 2024.

          • Investors: Mercia Asset Management (LON: MERC)(Mark Payton)

          • Deal Size: GBP 8.4 million ($10.65 million)

        • Lytica Therapeutics

          • Description: Operator of a biotechnology company intended to develop novel peptide conjugates for next-generation ADCs.

          • Verticals: Life Sciences

          • Deal Date: 11-mars-2024

          • Deal Type: Later Stage VC

          • Deal Synopsis: The company raised $4.07 million of venture funding in the form of convertible debt from undisclosed investors on March 11, 2024.

          • Deal Size: $4.07 million

        • Melt Pharmaceuticals

          • Description: Developer of non-intravenous and non-opioid sedation and analgesia medicines intended for short-duration medical procedures in outpatient and in-office settings.

          • Verticals: HealthTech, Life Sciences

          • Deal Date: 11-mars-2024

          • Deal Type: Later Stage VC

          • Deal Synopsis: The company raised $41.55 million of venture funding from undisclosed investors on March 11, 2024.

          • Deal Size: $41.55 million

        • Mission Therapeutics

          • Description: Operator of a pharmaceutical research and drug discovery company intended to facilitate the development of advanced medical treatments.

          • Verticals: Life Sciences, Oncology

          • Deal Date: 14-mars-2024

          • Deal Type: Later Stage VC

          • Deal Synopsis: The company raised GBP 25.2 million of venture funding in a deal led by Roche Venture Fund, Pfizer Ventures, and IP Group on March 14, 2024.

          • Investors: IP Group (LON: IPO), Pfizer Ventures(Denis Patrick), Roche Venture Fund(Carole Nuechterlein), Rosetta Capital (UK)(Jonathan Hepple), Sofinnova Partners(Graziano Seghezzi), SR One Capital Management(Matthew Foy)

          • Deal Size: GBP 25.2 million ($31.96 million)

        • Noa Therapeutics

          • Description: Developer of tailored multimodal therapies designed to revolutionize the treatment of immune diseases through a systems biology approach. The company offers singular, non-steroidal small molecules targeting three discrete therapeutic drivers to address complex immune diseases.

          • Verticals: Life Sciences

          • Deal Date: 13-mars-2024

          • Deal Type: Seed Round

          • Deal Synopsis: The company raised CAD 2.2 million of pre-seed funding in a deal led by UCeed on March 13, 2024. Phoenix Fires, NorthSpring Capital Partners, Ontario Centre of Innovation, Archangel Network of Funds, Golden Triangle Angelnet, and Angel One Investor Network also participated in the round. The funds will be used to advance its lead drug candidate for atopic dermatitis and to fuel the hit-to-lead phase which optimizes promising molecules into potential drugs for treating barrier dysfunction-related inflammatory diseases, such as ulcerative colitis and inflammatory bowel disease.

          • Investors: Angel One Investor Network, Archangel Network of Funds, Golden Triangle Angelnet, NorthSpring Capital Partners, Ontario Centre of Innovation (Maura Campbell), Phoenix Fires, UCeed

          • Deal Size: CAD 2.2 million ($1.74 million)

        • Nocion Therapeutics

          • Description: Developer of small molecule charged sodium channels designed to treat conditions arising from neurogenic inflammation. The company develops notions that target activated sensory neurons and enter activated nociceptors via large-pore channels on these neurons.

          • Verticals: HealthTech, Life Sciences

          • Deal Date: 14-mars-2024

          • Deal Type: Later Stage VC

          • Deal Synopsis: The company raised $62 million through a combination of Series B and Series B-1 venture funding in a deal led by Arkin Bio Ventures and Monograph Capital on March 14, 2024, putting the company's pre-money valuation at $32 million. Mission BioCapital, F-Prime Capital, Canaan Partners, Mass General Brigham Ventures, and Osage University Partners also participated in the round. The funds will be used to support a global multi-center Phase 2b trial in Refractory Chronic Cough and activities for Phase 3 Readiness.

          • Investors: Arkin Bio Ventures (Pini Orbach), Canaan Partners (Julie Grant), F-Prime Capital (Jessica Alston), Mass General Brigham Ventures (Meredith Fisher), Mission BioCapital (Steven Tregay), Monograph Capital (Freddie Dear), Osage University Partners

          • Deal Size: $62 million

        • ONL Therapeutics

          • Description: Operator of a biopharmaceutical company intended to protect and improve the vision of patients with a range of retinal diseases and conditions. The company develops innovative therapeutics treatments for serious, vision-threatening retinal diseases and conditions with small-molecule peptides for the protection of photoreceptors in retinal detachment.

          • Verticals: Life Sciences

          • Deal Date: 12-mars-2024

          • Deal Type: Later Stage VC

          • Deal Synopsis: The company raised $10.00 million of venture funding from undisclosed investors on March 12, 2024.

          • Deal Size: $10.00 million

        • Oxford Biodynamics (LON: OBD)

          • Description: Oxford BioDynamics PLC is a biotechnology company focused on the discovery and development of biomarkers for use within the pharmaceutical and biotechnology industry.

          • Verticals: Life Sciences, Oncology

          • Deal Date: 14-mars-2024

          • Deal Type: PIPE

          • Deal Synopsis: The company (LON:OBD) received GBP 9.41 million of development capital from undisclosed investors on March 14, 2024 through a private placement. The proceeds from the placement will be used for working capital to support the ongoing commercial development of the EpiSwitch® product line, through continued investment in sales and marketing activities to grow adoption of EpiSwitch® PSE and EpiSwitch® CiRT, operation of the company's clinical, research and reference laboratory facilities worldwide and pursuit of partnering and licensing opportunities for deployable pipeline assets.

          • Deal Size: GBP 9.41 million ($11.92 million)

        • Regulus Therapeutics (NAS: RGLS)

          • Description: Regulus Therapeutics Inc is a biotechnology company focused on the development of microRNA therapies targeting diseases such as cancer, metabolic diseases, fibrosis, and inflammatory diseases.

          • Verticals: Life Sciences, Oncology

          • Deal Date: 12-mars-2024

          • Deal Type: PIPE

          • Deal Synopsis: The company (NAS:RGLS) is in talks to receive $100 million of development capital from Deep Track Capital, Vivo Capital, RA Capital Management, New Enterprise Associates, Federated Kaufmann Fund, Adage Capital Management, and Octagon Capital through a private placement as of March 12, 2024.

          • Investors: Adage Capital Management, Deep Track Capital, Federated Kaufmann Fund (NAS: KAUAX), New Enterprise Associates, Octagon Capital, RA Capital Management, Vivo Capital

          • Deal Size: $100 million

        • Siolta Therapeutics

          • Description: Operator of a clinical-stage biotech company intended to develop biotherapeutic products to target the core drivers of disease. The company's platform offers safe pharmaceutical-grade anaerobic organisms and is in phase 2 development for the prevention of atopic diseases, including atopic dermatitis, food allergy, and asthma.

          • Verticals: Life Sciences

          • Deal Date: 12-mars-2024

          • Deal Type: Later Stage VC

          • Deal Synopsis: The company raised $12 million of Series C venture funding in a deal led by Khosla Ventures and SymBiosis Capital Management on March 12, 2024, putting the company's pre-money valuation at $30 million. Kirin Holdings Company, Kirin Holdings Company, and 8 other investors also participated in the round. The funds will be used by the company support further clinical development of their lead product STMC-103H, a defined-consortia Live Biotherapeutic Product (LBP) focused on preventing the onset of atopic diseases in newborns and also to explore the potential of microbiome-based treatments in maternal and infant health.

          • Investors: Global Brain, Johnson & Johnson Innovation - JLABS, Khosla Ventures (Samir Kaul), Kirin Health Innovation Fund, Kirin Holdings Company (TKS: 2503), Marc Benioff, MBC BioLabs, National Institutes of Health, Seventure Partners, Springboard Enterprises, SymBiosis Capital Management (Aleksei Cremo)

          • Deal Size: $12 million

        • Tubulis

          • Description: Developer of chemotherapeutic drugs designed to fight against cancer and chronic diseases. The company's chemotherapeutic drugs are uniquely matched protein-drug conjugates by combining proprietary novel technologies with disease-specific biology.

          • Verticals: Life Sciences, Oncology

          • Deal Date: 14-mars-2024

          • Deal Type: Later Stage VC

          • Deal Synopsis: The company raised EUR 128 million of Series B2 venture funding in a deal led by EQT Life Sciences and Nextech Invest on March 14, 2024. Evotec, Andera Partners, Deep Track Capital, Fund+, BioMedPartners, High-Tech Gründerfonds, Seventure Partners, Coparion, Bayern Kapital, Occident Group, Frazier Lifesciences Acquisition, and other undisclosed investors also participated in the round. The funds will be used to support the clinical evaluation of lead solid tumor ADC candidates, TUB-030 and TUB-040, further technology development, and expansion into the US.

          • Investors: Andera Partners (Jan Van den Bossche), Bayern Kapital, BioMedPartners, Coparion (Sebastian Pünzeler), Deep Track Capital, EQT Life Sciences (Christoph Broja), Evotec (ETR: EVT), Frazier Lifesciences Acquisition, Fund+, High-Tech Gründerfonds, Nextech Invest (Kanishka Pothula), Occident Group, Seventure Partners

          • Deal Size: EUR 128 million ($141.61 million)

        • Unicycive (NAS: UNCY)

          • Description: Unicycive Therapeutics Inc is a biotechnology company dedicated to developing treatments for certain medical conditions, focusing on kidney diseases. Its lead drug, Renazorb, is a novel phosphate-binding agent being developed for the treatment of hyperphosphatemia. Unicycive's other drug, UNI-494, is a new chemical entity with issued composition of matter patent protection in late preclinical development for the treatment of acute kidney injury.

          • Verticals: Life Sciences

          • Deal Date: 14-mars-2024

          • Deal Type: PIPE

          • Deal Synopsis: The company (NAS:UNCY) is in talks to receive $50 million of development capital from Great Point Partners, Vivo Capital, Logos Capital, Nantahala Capital Management, Octagon Capital Advisors, SilverArc Capital Management, Velan Capital, and Walleye Capital through a private placement as of March 14, 2024. The proceeds from the private placement will be used to support the advancement of the Company's clinical development pipeline and general working capital.

          • Investors: Great Point Partners, Logos Capital, Nantahala Capital Management, Octagon Capital Advisors, SilverArc Capital Management, Velan Capital, Vivo Capital, Walleye Capital

          • Deal Size: $50 million

Reduction in force (RIF)

March 14 - Spruce Biosciences: In the wake of a phase 2 trial for the biotech's only drug candidate, Spruce is sending 21% of staffers packing.to extend its cash runway through the end of 2025. Story

March 13 - Coherus BioSciences: About a year after launching a prior round of layoffs, Coherus is again reducing its head count, this time by 30%. The latest round of cuts started March 7 and are expected to be complete by the end of the year and save more than $25 million in annual costs. Story

March 13 - Takeda: After an unsuccessful attempt to sell an Austrian manufacturing and development site, Takeda is calling it quits on much of the operations at the facility. The Japan-based company is shutting down production and R&D operations at its AAV gene therapy plant in Austria, with 190 employees set to lose their jobs. Story

Disease of the week

Wolman disease, also known as Lysosomal Acid Lipase (LAL) Deficiency, is an extremely rare genetic disorder characterized by the deficiency or absence of the enzyme lysosomal acid lipase (LAL). This enzyme is responsible for breaking down cholesterol esters and triglycerides within cells. Without sufficient LAL activity, these lipids accumulate in various organs, particularly the liver, spleen, and intestines. The disease is a lysosomal storage disease. Multiple organs such as adrenal glands, liver, spleen, bone marrow, small bowel loops, and abdominal lymph nodes are infiltrated by the deposition of lipids. Infants generally present with failure to thrive, abdominal distention, vomiting, steatorrhea, and hepatosplenomegaly. Authors’ present a 1 month-old male infant with abdominal distention and failure to thrive who was referred for abdomen CT scan. The CT scan revealed stippled calcifications of both enlarged adrenal glands, without the distortion of the adreniform shape, fatty liver, splenomegaly and thickened small bowel loops; characteristic imaging findings of Wolman disease. CT scan is the imaging modality of choice for the recognition of the disease. There is no definite cure explained yet. Further studies are required to find the definite treatment of the disease.

(A-F): Selected axial, coronal and sagittal contrast-enhanced abdomen CT scan soft tissue and bone window images demonstrating demonstrating enlarged adrenal glands with stippled cortical calcifications preserving their adreniform shape (yellow arrows), fatty liver (black asterisk), and enlarged spleen (red asterisk).

Here's a comprehensive overview of Wolman disease:

  1. Genetics: Wolman disease is inherited in an autosomal recessive pattern, meaning that a child must inherit two copies of the defective gene (one from each parent) to develop the condition. The gene associated with Wolman disease is called LIPA, which provides instructions for making the lysosomal acid lipase enzyme. Mutations in this gene lead to the production of non-functional or insufficient lysosomal acid lipase.

  2. Onset and Symptoms: Symptoms of Wolman disease typically appear within the first few months of life, often shortly after birth. Infants with Wolman disease may present with:

    • Enlargement of the liver and spleen (hepatosplenomegaly)

    • Failure to thrive

    • Poor weight gain

    • Abdominal distension due to the enlarged organs

    • Diarrhea

    • Malabsorption of nutrients

    • Jaundice

    • Vomiting

    • Development of fatty deposits under the skin (xanthomas)

    • Growth retardation

    • Severe lipid abnormalities

  3. Progression: Wolman disease is progressive and rapidly fatal if left untreated. The accumulation of lipids in organs can lead to organ failure, particularly affecting the liver, leading to liver dysfunction and potentially liver failure. Additionally, the accumulation of lipids in the intestines can cause malabsorption, leading to severe malnutrition and growth impairment.

  4. Diagnosis: Diagnosis of Wolman disease is typically based on clinical presentation, imaging studies (such as ultrasound or MRI to assess organ enlargement), and laboratory tests to evaluate lipid levels and confirm deficient LAL enzyme activity. Genetic testing can confirm the presence of mutations in the LIPA gene.

  5. Treatment: Currently, there is no cure for Wolman disease. Treatment focuses on managing symptoms and complications. Enzyme replacement therapy (ERT) with recombinant human lysosomal acid lipase has shown promise in reducing lipid accumulation and improving some symptoms, but it is not curative. Supportive care may include nutritional support, management of complications such as liver dysfunction, and supportive therapies to address symptoms.

  6. Prognosis: The prognosis for individuals with Wolman disease is poor. Without treatment, the disease is rapidly progressive and fatal, often within the first year of life due to complications such as liver failure and severe malnutrition. Even with treatment, the disease can significantly impact quality of life and lifespan.

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