Report
Ashiq Mubarack ...
  • David Nierengarten
  • Jeffrey La Rosa
  • Liana Moussatos
  • Robert Driscoll
  • Shveta Dighe

The Week Ahead in Life Sciences

Underlyings
Aimmune Therapeutics

Aimmune Therapeutics is a clinical-stage biopharmaceutical company developing a therapeutic approach, including the development of product candidates, for the treatment of peanut and other food allergies. The company's Characterized Oral Desensitization ImmunoTherapy (CODIT?), is designed to desensitize patients to food allergens. The company's primary CODIT product candidate, AR101 is an investigational biologic for the treatment of patients with peanut allergy. The company is also exploring a product candidate designed to treat multi-nut allergy, including walnut allergy. The company is also conducting research and development activities for CODIT targeting other food allergies, including cow's milk allergy.

Allena Pharmaceuticals

Allena Pharmaceuticals is a late-stage, clinical biopharmaceutical company focused on developing and commercializing oral enzyme therapeutics to treat patients with rare and severe metabolic and kidney disorders. The company's main product candidate, reloxaliase, is an oral enzyme therapeutic that it is developing for the treatment of hyperoxaluria, a metabolic disorder characterized by markedly elevated urinary oxalate levels and commonly associated with kidney stones, chronic kidney disease (CKD) and other serious kidney disease. The company's second product candidate, ALLN-346, is being developed for patients with hyperuricemia and moderate to severe CKD.

Alpine Select AG

Alpine Select AG. Alpine Select AG (Alpine) is a Switzerland-based investment company. The Company's emphasis lies on securities, quoted or otherwise, of Swiss and foreign corporations, which are subject to particular corporate events such as spin-offs, acquisitions, recapitalizations and reorganizations, or circumstances including valuation anomalies or technical market situations. Alpine Select AG's investment portfolio is primarily comprised of quoted securities. As of December 31, 2011, the Company's investments included Absolute Invest AG, Absolute Private Equity AG, AIRE GmbH & Co KGaA, Atrium V Ltd, BB Biotech AG and HBM Bioventures AG, among others. The Company operates through its wholly owned subsidiary, Sumara AG.

Anaptys Bio

AnaptysBio is a clinical stage biotechnology company developing antibody product candidates. Etokimab, the company's anti-IL-33 antibody drug candidate, inhibits the activity of the interleukin-33 cytokine that is applicable to the treatment of atopic inflammatory disorders, such as moderate-to-severe atopic dermatitis, eosinophilic asthma, chronic rhinosinusitis with nasal polyps, and potentially other allergic conditions. ANB019 inhibits the interleukin-36 receptor, for the treatment of rare inflammatory diseases including generalized pustular psoriasis and palmoplantar pustulosis. The company's wholly-owned pipeline includes anti-inflammatory checkpoint receptor modulator antibodies.

APPLIED GENETIC TECHNOLOGIES

Applied Genetic Technologies is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. The company focuses on the field of ophthalmology, where it has clinical programs in X-linked retinitis pigmentosa and achromatopsia. The company also has preclinical programs in optogenetics and adrenoleukodystrophy, which is a disease of the central nervous system (CNS), and several other ophthalmology, CNS and otology indications. The company has developed technological capabilities in the design, construction and manufacture of viral vectors using adeno-associated virus technology.

Aquestive Therapeutics, Inc.

Aravive

Aravive is clinical-stage biotechnology company focused on developing therapies that target survival pathways for both advanced solid tumors as well as hematologic malignancies. The company's primary therapeutic focus is the GAS6-AXL pathway, where AXL receptor signaling plays a role in multiple types of malignancies by promoting metastasis, cancer cell survival, resistance to treatments, and immune suppression. The U.S. Food and Drug Administration, designated as a Fast Track development program the investigation of the company's primary development candidate, Aravive-S6-500, for platinum-resistant recurrent ovarian cancer.

Arbutus Biopharma Corp.

Arbutus Biopharma is a therapeutic solutions company focused on discovering, developing and commercializing a cure for patients suffering from chronic Hepatitis B virus (HBV) infection. Co.'s HBV pipeline consists of multiple drug candidates, with differing and complementary mechanisms of action. The candidates include: RNAi 1.0 (ARB-1467), which is Co.'s main RNA Interference (RNAi) HBV candidate designed to eliminate HBV surface antigen expression in patients chronically infected with HBV; RNAi 2.0 (ARB-1740), Co.'s follow-on RNAi HBV candidate; and Core Protein/ Capsid Assembly Inhibitors (AB-423), which is being developed as oral therapeutics for the treatment of chronic HBV infection.

Arcus Biosciences

Arcus Biosciences is a clinical-stage biopharmaceutical company focused on creating cancer immunotherapies. The company has four clinical-stage product candidates: AB928, which is an orally bioavailable, reversible antagonist of the adenosine 2a and adenosine 2b receptors; AB680, which is a reversible and selective inhibitor of the CD73 enzyme; AB122, which is a human antibody with similar binding affinity and other characteristics to the marketed anti-PD-1 antibodies pembrolizumab and nivolumab; and AB154, which is a humanized antibody that inhibits an immune checkpoint target involved in a pathway that plays both inhibitory and stimulatory roles in the immune system.

Argenx S.E

Argenx N.V. is a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer. Utilizing its suite of differentiated technologies, Co. is focused on developing product candidates with the potential to be either first-in-class against novel targets or best-in-class against known, but complex, targets in order to treat diseases with a significant unmet medical need.

Ascendis Pharma ADR

Audentes Therapeutics

Audentes Therapeutics is a clinical stage biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects. The company has a portfolio of product candidates, including AT132 for the treatment of X-Linked Myotubular Myopathy, AT845 for the treatment of Pompe disease, AT342 for the treatment of Crigler-Najjar Syndrome, and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia. The company focuses on the treatment of rare diseases caused by single gene, or monogenic, defects in DNA that can be addressed using gene therapy.

AVROBIO

AVROBIO is a clinical stage gene therapy company focused on developing potentially curative ex vivo lentiviral-based gene therapies to treat patients with rare diseases following a single dose treatment regimen. The company's gene therapies employ hematopoietic stem cells that are harvested from the patient and then modified with lentiviral vectors to insert a functional copy of the gene that is defective in the target disease. The company's pipeline is comprised of four lentiviral-based gene therapy programs, including AVR-RD-01 for the treatment of Fabry disease, AVR-RD-02 for the treatment of Gaucher disease, AVR-RD-04 for the treatment of cystinosis and AVR-RD-03 for the treatment of Pompe disease.

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical is a biotechnology company that develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. The company's commercial products are Aldurazyme (laronidase) for Mucopolysaccharidosis I; Brineura (cerliponase alfa) for the treatment of late infantile neuronal ceroid lipofuscinosis type 2; Kuvan (sapropterin dihydrochloride) for the treatment of phenylketonuria; Naglazyme (galsulfase) the treatment of Mucopolysaccharidosis VI ; Palynziq (pegvaliase-pqpz) for adult patients with phenylketonuria; and Vimizim (elosulfase alpha) for the treatment of Mucopolysaccharidosis IV Type A.

Bluebird Bio

bluebird bio is a clinical-stage biotechnology company focused on developing potentially transformative gene therapies for severe genetic diseases and cancer. The company's clinical programs in severe genetic diseases include its LentiGlobin? product candidate as a treatment for transfusion-dependent ?-thalassemia and sickle cell disease and its Lenti-D? product candidate as a treatment for cerebral adrenoleukodystrophy. bb2121 and bb21217, the company's clinical-stage product candidates in oncology, are CAR T cell product candidates for the treatment of multiple myeloma.

Blueprint Medicines

Blueprint Medicines is a therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy. The company's drug candidates are: avapritinib, which targets KIT and PDGFRa mutations; BLU-667, which targets RET, a receptor tyrosine kinase that is abnormally activated by mutations or fusions; BLU-554, which targets FGFR4, a kinase that is aberrantly activated in a defined subset of patients with hepatocellular carcinoma, the common type of liver cancer; and BLU-782, which is for the treatment of fibrodysplasia ossificans progressiva, a rare genetic disease caused by mutations in the activin-like kinase 2 gene, ACVR1.

Cidara Therapeutics

Cidara Therapeutics is a biotechnology company focused on the discovery, development and commercialization of anti-infectives for the treatment of diseases. The company is developing a pipeline of product and development candidates, with an initial focus on fungal infections. The company's primary product candidate is rezafungin acetate, an intravenous formulation of an echinocandin. Rezafungin is being developed as a once-weekly, high-exposure therapy for the treatment and prevention of invasive fungal infections. In addition, the company's proprietary Cloudbreak? platform is designed to discover compounds that directly kill pathogens and also direct a patient's immune system to attack and eliminate pathogens.

Clearside Biomedical Inc

Clearside Biomedical is a clinical biopharmaceutical company developing pharmacological therapies to restore and preserve vision for people with serious eye diseases. The company's suprachoroidal injection platform is a patented approach for delivering pharmacotherapy to the back of the eye in the anatomic structure known as the suprachoroidal space. The company's main product candidate, XIPERE, is a proprietary, preservative-free suspension of the corticosteroid triamcinolone acetonide formulated for administration via suprachoroidal injection, for the treatment of macular edema associated with uveitis. The company is also developing XIPERE for the treatment of diabetic macular edema.

CytomX Therapeutics

CytomX Therapeutics is clinical-stage, oncology-focused biopharmaceutical company with a class of investigational antibody therapeutics based on its Probody? technology platform the treatment of cancer. The company uses its platform to create proprietary cancer immunotherapies against clinically-validated targets. The company's two programs, CX-072, a wholly owned PD-L1-targeting Probody therapeutic and CX-2009, wholly owned CD166-targeting Probody drug conjugate. Both CX-072 and CX-2009 are part of PROCLAIM (Probody Clinical Assessment in Man), an international umbrella clinical trial program that provides clinical trial sites with access to the company's novel therapies under one central protocol.

Eiger Biopharmaceuticals

Eiger BioPharmaceuticals is a clinical stage biopharmaceutical company focused on bringing to market product candidates for the treatment of rare diseases. The company's product candidate pipeline includes four programs: Lonafarnib (LNF) in Hepatitis Delta Virus (HDV), which is an orally bioavailable, farnesylation inhibitor in Phase 3 clinical trials for HDV infection; Peginterferon Lambda in HDV, which is a late-stage, type III interferon that stimulates immune responses; LNF in Progeria and Progeroid Laminopathies (PL), which the company develops LNF for treatment of PL; and Avexitide in Post-Bariatric Hypoglycemia (PBH), which is a peptide that the company is developing as a treatment for PBH.

Entasis Therapeutics Holdings

Entasis Therapeutics is a holding company. Through its subsidiary, the company is clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel antibacterial products to treat serious infections caused by multi-drug resistant Gram-negative bacteria.

Epizyme Inc

Epizyme is a late-stage biopharmaceutical company that is focused on the treatment for people with cancer and other diseases through the discovery, development, and commercialization of epigenetic medicines. The company's lead product candidate, tazemetostat, is an oral, selective small molecule inhibitor of the EZH2 histone methyltransferase for the treatment of a range of cancer types in multiple treatment settings, and its G9a program, EZM8266, is used for the treatment of sickle cell disease. The company owns the global development and commercialization rights to tazemetostat outside of Japan. Eisai Co. Ltd holds the rights to develop and commercialize tazemetostat in Japan.

Fate Therapeutics Inc

Fate Therapeutics is a clinical-stage biopharmaceutical company. The company's immuno-oncology product candidates include: FATE-NK100, which is a natural killer (NK) cell cancer immunotherapy comprised of adaptive memory NK cells; and FT500, which is an off-the-shelf NK cell cancer immunotherapy for the treatment of solid tumors; and FT516, which is an off-the-shelf immunotherapy for the treatment of cancer. The company's immuno-regulation product candidates include: ProTmune?, which is an investigational programmed cellular immunotherapy for use as an allogeneic hematopoietic cell transplantation cell graft; and FT301, which is an off-the-shelf, immuno-regulatory cell product candidate.

Fennec Pharma

Five Prime Therapeutics Inc

Five Prime Therapeutics is a clinical-stage biotechnology company focused on discovering and developing protein therapeutics. The company's key product candidates are: FPA150, which is a CD8 T cell checkpoint inhibitor antibody that targets B7-H4 that it is studying in a clinical trial in multiple cancers; FPT155, which is a soluble CD80 fusion protein that enhances co-stimulation of T cells through CD28 that the company is studying in a clinical trial in multiple cancers; and Cabiralizumab (FPA008) is an antibody that the company and its partner Bristol-Myers Squibb Company, or BMS, are studying in clinical trials in multiple cancers in combination with BMS's PD-1 immune checkpoint inhibitor, Opdivo? (nivolumab).

G1 Therapeutics

G1 Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of small molecule therapeutics for the treatment of patients with cancer. The company's product pipeline includes three clinical candidates: trilaciclib, a short-acting intravenous cyclin-dependent kinases (CDK) 4/6 inhibitor that preserves hematopoietic stem and progenitor cells and immune system function; lerociclib, an oral CDK4/6 inhibitor that inhibits tumor proliferation and growth; and G1T48, an oral selective estrogen receptor degrader that inhibits estrogen receptor driven tumor proliferation.

Global Blood Therapeutics

Global Blood Therapeutics is a clinical-stage biopharmaceutical company determined to discover, develop and deliver treatments that provide hope to underserved patient communities. The company's primary product candidate is voxelotor, an oral, once-daily therapy that modulates hemoglobin's affinity for oxygen, which inhibits hemoglobin polymerization in sickle cell disease (SCD). The company is evaluating voxelotor in adult and adolescent patients with SCD in a Phase 3 clinical trial. The company is also evaluating the safety and pharmacokinetics of single and multiple doses of voxelotor in adolescent and pediatric patients with SCD in a Phase 2a clinical trial.

Harpoon Therapeutics

Idera Pharmaceuticals

Idera Pharmaceuticals is a biopharmaceutical company. The company's focus is on its Toll-like receptor, or TLR, agonist, tilsotolimod (IMO-2125), for oncology. The company's TLR-targeted clinical-stage drug candidate, tilsotolimod, is an agonist of TLR9. The company is developing tilsotolimod, via intratumoral injection, for the treatment of anti-PD1 refractory metastatic melanoma in combination with ipilimumab, an anti-CTLA4 antibody marketed as Yervoy? by Bristol-Myers Squibb Company (BMS). The company is also evaluating intratumoral tilsotolimod in combination with nivolumab, an anti-PD1 antibody marketed as Opdivo? by BMS, and ipilimumab for the treatment of multiple solid tumors in a Phase 2 trial.

Intellia Therapeutics

Intellia Therapeutics is a genome editing company focused on developing therapeutics utilizing a biological tool known as Clustered, Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR associated 9 (Cas9). This is a technology for genome editing, the process of altering selected sequences of genomic deoxyribonucleic acid. The company utilizes its CRISPR/Cas9 platform across two areas: in vivo applications, in which CRISPR/Cas9 is therapy, delivered to target cells within the body; and ex vivo applications, in which CRISPR/Cas9 creates therapy of engineered human cells.

Ironwood Pharmaceuticals

Ironwood Pharmaceuticals is a biotechnology company utilizing its development and commercial capabilities as it brings various medicines to patients. The company and its U.S. linaclotide partner Allergan plc (together with its affiliates), or Allergan, are exploring ways to enhance the clinical profile of LINZESS by studying linaclotide in additional indications, populations and formulations to assess its potential to treat various conditions. The company is developing another GI development program, IW-3718, a gastric retentive formulation of a bile acid sequestrant for the potential treatment of persistent gastroesophageal reflux disease.

Kala Pharmaceuticals

Kala Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of therapeutics using the company's proprietary nanoparticle-based Mucus Penetrating Particles, or MPP, technology, with an initial focus on the treatment of eye diseases. The company's MPPs are selectively-sized nanoparticles and have proprietary coatings enhancing the mobility of drug particles through mucus and preventing drug particles from becoming trapped and eliminated by mucus. The company's product candidates include treatments for post-operative inflammation and pain following ocular surgery, temporary relief of the signs and symptoms of dry eye disease and retinal diseases.

Karyopharm Therapeutics

Karyopharm Therapeutics is a clinical-stage pharmaceutical company. The company is focused on the discovery, development, and commercialization of drugs directed against nuclear transport and related targets for the treatment of cancer and other diseases. The company has discovered and is developing small molecule Selective Inhibitors of Nuclear Export (SINE), compounds that inhibit the nuclear export protein (XPO1). The company's SINE compounds were the first oral XPO1 inhibitors in clinical development. The company's focus is on seeking the commercialization of its primary drug candidate, selinexor (KPT-330), as an oral agent in cancer indications with clinical need, initially for hematologic malignancies.

Kiniksa Pharmaceuticals

KURA ONCOLOGY

Kura Oncology is a clinical-stage biopharmaceutical company engaged in developing medicines for the treatment of cancer. The company's key product candidate, tipifarnib, is a selective and orally bioavailable inhibitor of farnesyl transferase. The company's second product candidate is KO-947, a selective small molecule inhibitor of extracellular signal related kinase, which the company is developing as a potential treatment for patients with tumors that have dysregulated activity due to mutations or other mechanisms in the mitogen-activated protein kinase pathway. The company's product candidate is KO-539, a selective small molecule inhibitor of the menin-mixed lineage leukemia, or menin-MLL, protein-protein interaction.

Lexicon Pharmaceuticals Inc

Lexicon Pharmaceuticals is a biopharmaceutical company. The company discovers the functions and pharmaceutical utility of genes and uses those gene function discoveries in the discovery and development of pharmaceutical products for the treatment of human disease. The company is commercializing XERMELO? (telotristat ethyl), an orally-delivered small molecule drug for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy. The company is also developing sotagliflozin as a treatment for type 1 and type 2 diabetes; LX9211 as a treatment for neuropathic pain; and LX2761 as a treatment for diabetes.

Liquidia Technologies

Liquidia Technologies is a late-stage clinical biopharmaceutical company focused on the development and commercialization of human therapeutics using the company's PRINT? technology to transform the lives of patients. The company is focused on the development of its product candidates for which it holds commercial rights: LIQ861, which is being evaluated for the treatment of pulmonary arterial hypertension, a disease caused by the hardening and narrowing of the pulmonary arteries that can lead to right heart failure and eventually death; and LIQ865, which is an injectable, sustained-release formulation of bupivacaine for the management of local post-operative pain for three to five days after a procedure.

MacroGenics

MacroGenics discovers and develops antibody-based therapeutics to modulate the human immune response for the treatment of cancer. The company has a pipeline of product candidates in human clinical testing, including immuno-oncology programs, that have been created using its proprietary antibody-based technology platforms. The company is developing product candidates that target various tumor-associated antigens, including HER2. The company is also developing a franchise of molecules that target programmed cell death protein 1 (PD-1). The company is developing two bispecific DART product candidates that engage PD-1 and lymphocyte-activation gene 3, as well as PD-1 and cytotoxic T-lymphocyte-associated protein 4.

Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to extend the curative capability of stem cell transplant, gene therapy, genome editing and cell therapy to more patients. The first conditioning program in the company's portfolio is C100, under which it is developing antibody-drug conjugates (ADC) that specifically deplete host HSCs and immune cells. The company's key conditioning program, C200, is designed to specifically deplete HSCs and disease-causing cells. The company's lead ADC product candidate targets CD117, also known as c-Kit, which is highly expressed on HSCs and leukemia cells, making it an ideal target for conditioning across sets of diseases.

Mersana Therapeutics

Mersana Therapeutics is a clinical stage biopharmaceutical company focused on developing antibody drug conjugates (ADCs) for cancer patients. The company's primary product candidate, XMT-1536, is an ADC targeting NaPi2b, an antigen broadly expressed in ovarian cancer and non small cell lung cancer. In addition, the company has established research and development partnerships with Merck KGaA and Asana Biosciences for the development and commercialization of additional ADC product candidates against a limited number of targets selected by the company's partners based on its Dolaflexin platform.

MERUS B V

miRagen Therapeutics

Miragen Therapeutics is a clinical-stage biopharmaceutical company discovering and developing proprietary RNA-targeted therapeutics with a specific focus on microRNAs and their role in certain diseases. The company has three product candidates, cobomarsen, remlarsen and MRG-110, in clinical development. Cobomarsen is an inhibitor of miR-155, which is a microRNA that is found at abnormally high levels in malignant cells of several blood cancers. Remlarsen is a replacement for miR-29, a microRNA that is found at abnormally low levels in a number of pathological fibrotic conditions. MRG-110 is an inhibitor of miR-92, a microRNA expressed in endothelial cells.

MyoKardia

MyoKardia is a clinical stage biopharmaceutical company engaged in a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. The company's initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. The company's main product candidate, mavacamten, is initially being developed for the treatment of hypertrophic cardiomyopathy. The company's second candidate, MYK-491, is being studied in a Phase 2a multiple-ascending dose clinical trial in patients with stable heart failure.

Nabriva Therapeutics plc

NovoCure

ObsEva

Omeros

Omeros is a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. The company's drug product OMIDRIA? is marketed in the U.S. for use during cataract surgery or intraocular lens replacement. In its pipeline the company has clinical-stage development programs focused on: complement-associated thrombotic microangiopathies; complement-mediated glomerulonephropathies; and addictive and compulsive disorders.

Orchard Therapeutics

Pacira Pharmaceuticals

Pacira BioSciences is a holding company. Through its subsidiaries, the company is a pharmaceutical company focused on delivering non-opioid pain management and regenerative health solutions to surgeons and anesthesiologist. The company's marketed product, EXPAREL (bupivacaine liposome injectable suspension) is indicated for single-dose infiltration in adults to produce postsurgical local analgesia and as an interscalene brachial plexus nerve block to produce postsurgical regional analgesia. EXPAREL consists of bupivacaine, an amide-type local anesthetic, encapsulated in DepoFoam, the company's extended release drug delivery technology, that delivers bupivacaine over time for extended analgesia.

Paratek Pharmaceuticals

Paratek Pharmaceuticals is a commercial-stage biopharmaceutical company focused on the development and commercialization of therapeutics. The company has created small molecules derived from the minocycline core structure. The company's NUZYRA? (omadacycline) is a once-daily oral and intravenous antibiotic for the treatment of adult patients with community-acquired bacterial pneumonia, and acute skin and skin structure infections, caused by susceptible pathogens. In addition, SEYSARA? (sarecycline) is a once-daily oral therapy for the treatment of moderate to severe acne vulgaris, with respect to which the company has licensed in the United States certain rights to Almirall, LLC.

Replimune Group

Replimune Group is a clinical-stage biotechnology company focused on the development of oncolytic immunotherapies to treat cancer. The company's primary product candidate, RP1, is a selectively replicating version of herpes simplex virus 1 that expresses GALV-GP R(-) and human GM-CSF. The company has designed its RP2 product candidate to express an anti-CTLA-4 antibody-like protein in order to block the inhibition of the immune response otherwise caused by CTLA-4, a protein that inhibits the immune response to tumors. The company has designed its RP3 product candidate to express immune-activating proteins that stimulate T cells, in addition to anti-CTLA-4 and GALV-GP R(-).

resTORbio Inc

resTORbio is a clinical-stage biopharmaceutical company focused on the development and commercialization of therapeutics for the treatment of aging-related diseases. The company's initial focus is on the development of RTB101, an orally administered, small molecule, target of rapamycin complex 1 inhibitor, alone and in combination with other mechanistic target of rapamycin inhibitors such as everolimus, as an immunotherapy program designed to improve immune function and thereby reduce the incidence of respiratory tract infections in the elderly regardless of the causative pathogen.

Sangamo Therapeutics

Sangamo BioSciences is a clinical stage biotechnology company focused on translating science into genomic medicines using its platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The company is focused on these therapeutic areas: inherited metabolic diseases, central nervous system diseases and inflammatory and autoimmune diseases. The company has an ongoing clinical trial evaluating SB-318 for the treatment of Mucopolysaccharidosis Type I. The company is also evaluating SB-FIX in a clinical trial which is designed to assess the SB-FIX in adults with severe hemophilia B. The company is evaluating ST-400 in a clinical trial to evaluate the adult subjects with beta-thalassemia.

Scholar Rock Holding Corporation

Scholar Rock is a biopharmaceutical company. The company's primary product candidate, SRK-015, is a selective, fully human, monoclonal antibody, with a mechanism of action that results in inhibition of the activation of the growth factor, myostatin, in skeletal muscle. The company's second product candidate is SRK-181 and is being developed for the treatment of cancers resistant to checkpoint blockade therapies, such as anti-PD1 antibodies. The company's third antibody program targets the signaling of bone morphogenetic protein 6, another member of the TGF? superfamily, which is involved in a set of biological processes in various parts of the body.

Sierra Oncology

Sierra Oncology is a clinical stage drug development company advancing targeted therapeutics for the treatment of patients with unmet medical needs in hematology and oncology. The company's primary drug candidate, momelotinib, which is for the treatment of myelofibrosis. The company is also advancing, SRA737, is a selective, orally bioavailable small molecule inhibitor of Checkpoint kinase 1, a main regulator of cell cycle progression and the DNA Damage Response replication stress response. The company's pipeline also includes SRA141, a selective, orally bioavailable small molecule inhibitor of cell division cycle 7 kinase.

Stemline Therapeutics Inc

Stemline Therapeutics is a biopharmaceutical company focused on discovering, acquiring, developing and commercializing oncology therapeutics. The company's product, ELZONRIS? (tagraxofusp-erzs; SL-401) has been approved by the U.S. Food and Drug Administration for the treatment of blastic plasmacytoid dendritic cell neoplasm in adult and pediatric patients two years and older. The company's pipeline of product candidates includes: SL-801 for patients with solid tumors; SL-701, an immunotherapeutic, which has completed a Phase 2 trial in patients with second-line glioblastoma; and SL-901, an oral, small molecule kinase inhibitor, assessed in Phase 1 trial of patients with solid tumors.

Sutro Biopharma

Sutro Biopharma is a clinical stage drug discovery, development and manufacturing company. The company focuses on creating protein therapeutics for cancer and autoimmune disorders through integrated cell-free protein synthesis platform, XpressCF. The company's product candidates include STRO-001, an antibody-drug conjugate (ADC) directed against the cancer target CD74 for multiple myeloma and non-Hodgkin lymphoma; and STRO-002, an ADC directed against folate receptor-alpha for patients with ovarian and endometrial cancers.

Synlogic

Synlogic is a biopharmaceutical company focused on improving its drug discovery and development platform for Synthetic Biotic? medicines, which are designed using synthetic biology to genetically reprogram beneficial microbes to treat metabolic and inflammatory diseases and cancer. The company's therapeutics programs include: SYNB1020, which is an oral therapy for the treatment of hyperammonemia, including patients with liver disease such as hepatic encephalopathy and patients with urea cycle disorders; and SYNB1618, which is an oral therapy for the treatment of phenylketonuria, a metabolic disease in which the amino acid phenylalanine accumulates in the body as a result of genetic defects.

SYROS PHARMACEUTICALS

Syros Pharmaceuticals is a biopharmaceutical company engaged in the non-coding regulatory region of the genome to develop a new wave of medicines that control the expression of genes. The company's primary product candidates are: SY-1425, a selective retinoic acid receptor alpha agonist that is used to treat acute myeloid leukemia patients; SY-1365, a selective inhibitor of cyclin-dependent kinase 7 (CDK7), that is being evaluated in a Phase 1 clinical trial in patients with advanced solid tumors; and SY-5609, a CDK7 inhibitor that can be administered orally, which is being evaluated in investigational new drug application, enabling preclinical studies.

TCR2 Therapeutics

Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical is a biopharmaceutical company focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. The company's product categories are: biologics, which include Crysvita? (burosumab) for the treatment of X-linked hypophosphatemia and Mepsevii? (vestronidase alfa) for the treatment of Mucopolysaccharidosis VII; small molecules, which includes UX007 for the treatment of long-chain fatty acid oxidation disorders; and gene therapy, which includes DTX301 for the treatment of patients with ornithine transcarbamylase deficiency and DTX401 for the treatment of patients with glycogen storage disease type Ia.

United Therapeutics Corp.

United Therapeutics is a biotechnology company. The company markets and sells four commercial therapies to treat pulmonary arterial hypertension (PAH): Remodulin? (treprostinil) Injection; Tyvaso? (treprostinil) Inhalation Solution; Orenitram? (treprostinil) Extended-Release Tablets; and Adcirca? (tadalafil) Tablets. The company also markets and sells an oncology product, Unituxin? (dinutuximab) Injection for treatment of high-risk neuroblastoma. The company also engages in research and development of new indications and formulations and delivery devices for its existing products, as well as new products to treat PAH and other conditions. The company also engages in organ transplantation-related technologies.

Unum Therapeutics

Unum Therapeutics is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel immunotherapy products designed to harness the power of a patient's immune system to cure cancer. The company's proprietary technology, called antibody-coupled T cell receptor (ACTR), is a universal, engineered cell therapy that is intended to be used in combination with a wide range of tumor-specific antibodies to target different tumor types. The company's product candidates are composed of ACTR T cells co-administered with approved and commercially available antibodies or antibodies in preclinical or clinical development.

Verona Pharma ADS

Voyager Therapeutics

oyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for patients suffering from severe neurological diseases. The company is focused on neurological diseases with adeno-associated virus, gene therapy approach that either increases or decreases the production of a specific protein. The company's pipeline includes: Parkinson's disease, a monogenic form of amyotrophic lateral sclerosis; Huntington's disease; Friedreich's ataxia; tau-related diseases including Alzheimer's disease, frontotemporal dementia, and progressive supranuclear palsy; Alpha-synuclein related diseases for Parkinson's disease and other synucleinopathies; and severe, chronic pain.

Xencor

Xencor is a clinical-stage biopharmaceutical company focused on discovering and developing engineered monoclonal antibody and other protein therapeutics to treat diseases with unmet medical needs. The company is developing a suite of clinical-stage drug candidates from its proprietary XmAb? technology platforms that are designed to treat cancer, autoimmune and allergic diseases, and other conditions. The company's protein engineering efforts and the XmAb technologies are focused on the portion of the antibody that interacts with multiple segments of the immune system and controls antibody structure. This portion, referred to as the Fc domain, is constant and interchangeable among antibodies.

XOMA

XOMA is a biotech company engaged in discovering and developing therapeutic candidates. The company has a pipeline of monoclonal antibodies and technologies: X213, an allosteric inhibitor of prolactin action; XMetA, an insulin receptor-activating antibody designed to provide long-acting reduction of hyperglycemia in Type 2 diabetic patients; IL-2, which targets interleukin 2 and has been recognized as a therapy for metastatic melanoma and renal cell carcinoma; and PTH1R, an anti-parathyroid receptor pipeline that includes several functional antibody antagonists targeting PTH1R, a G-protein-coupled receptor involved in the regulation of calcium metabolism.

Zafgen

Zafgen is a clinical-stage biopharmaceutical company utilizing its proprietary methionine aminopeptidase 2, or MetAP2, biology platform to develop therapies for patients affected by complex metabolic diseases. The company's primary product candidate is ZGN-1061, a fumagillin-class MetAP2 inhibitor administered by subcutaneous injection, which is being profiled for its utility in the treatment of type 2 diabetes. The company also has initiated development of a second MetAP2 development candidate, ZGN-1258, which is administered by subcutaneous injection.

Provider
Wedbush
Wedbush

Since our founding in 1955, Wedbush has been a leader in the financial industry providing our clients with a wide range of services; including institutional sales, correspondent clearing services, equity research, corporate and municipal finance, equity market making, fixed income trading, prime brokerage, and wealth management. Headquartered in Los Angeles, with 100 registered offices, the firm focuses on dedicated service, client financial safety, continuity, and advanced technology.

Analysts
Ashiq Mubarack

David Nierengarten

Jeffrey La Rosa

Liana Moussatos

Robert Driscoll

Shveta Dighe

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