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Vote for the Buzz of BIO at the 2019 CEO & Investor Conference

The voting period has ended. Check back December 11 to meet the winners!

Read the official contest rules here.

If you are interested in giving a company presentation, but missed the nominations window, you can apply to present here.

Nominees

Public Therapeutic Biotech

Company

Why You Should Vote

Allogene Therapeutics

  1. Leading the Development of AlloCAR T Therapy: Developing a pipeline of "off-the-shelf" CAR T cell therapy candidates with the goal of delivering readily available cell therapy faster, more reliably and at greater scale to more patients.
  2. We were co-founded by a team that includes Dr. Arie Belldegrun and Dr. David Chang, who developed one of the first CAR T cell therapies approved by the FDA. 
  3. The Allogene portfolio includes rights to 16 pre-clinical CAR T cell therapy targets and UCART19, the first AlloCAR T in Clinical Development.
Anavex logo
  1. Anavex has identified a gene "signature" from ANAVEX®2-73 patients which allows for a precision medicine treatment approach in future trials. This approach has been used in oncology, yet it is the first time to the best of our knowledge that it has been utilized in neurological clinical studies. It is also of value that the biomarker is drug specific not indication specific, so can be applied to all indications involving ANAVEX®2-73. ANAVEX®2-73 has confirmed safety and dose-dependent target engagement with SIGMAR1 using PET imaging study with Stanford University which was presented at AAIC 2018. 
  2. Three-year, longitudinal clinical efficacy data for ANAVEX®2-73 in Alzheimer's disease demonstrates significant association between higher ANAVEX®2-73 concentration and maintained activities of daily living and reduced cognitive decline in Alzheimer’s disease patients. Data also confirms SIGMAR1 and COMT as genetic biomarkers of response to ANAVEX®2-73. This was presented recently at the 2018 Clinical Trials on Alzheimer's Disease (CTAD) meeting.
  3. Anavex Life Sciences initiated three clinical trials this year. A Phase 2/3 Alzheimer's trial, a Phase 2 Parkinson's disease dementia trial, and a Phase 2 Rett syndrome study. We are applying what knowledge we have to de-risk the clinical trials by applying the latest methods in trial design in hopes to bring relief to patients who have been affected by these devastating neurological conditions.
Arcturus Therapeutics
  1. Arcturus' LUNAR delivery technology has the potential to deliver multiple types of RNA to different cell types throughout the human body. The biodegradable properties of its components minimize toxicity, making it potentially more versatile than other RNA delivery approaches. ARCT is the only company currently with a known biodegradable LNP platform to overcome lipid accumulation toxicity of previous LNPs for chronic repeat dosing (According to one of the analysts).
  2. Arcturus' UNA  oligomer chemistry can be incorporated into multiple types of nucleic acid medicines and is designed to improve the in vivo efficiency of mRNA-mediated protein replacement and siRNA-mediated gene silencing. These wholly-owned technologies are supported by 140 patents and patent applications. Arcturus is one of the only few companies with both a proprietary RNA delivery and RNA chemistry platform.
  3. External validation of these platforms is validated by the multiple partnerships with leading pharmaceutical partners that Arcturus has entered into date. Some notable collaborations include: Janssen Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc., Millennium Pharmaceuticals, Inc., a wholly-owned subsidiary of Takeda Pharmaceutical Company Limited, Cystic Fibrosis Foundation Therapeutics, Inc., Synthetic Genomics, Inc. and CureVac AG.

Bioasis

  1. Bioasis has the best-in-class blood-brain barrier platform technology called the xB3 platform with advantages over other technologies 
  2. Bioasis is advancing focused, internal programs to treat brain cancers and rare diseases and is actively pursuing licensing, business development opportunities for the advancement of external neuroscience and oncology programs.
  3. Bioasis has an experienced, biotech-savvy board of directors, a world-class scientific advisory board and a management team with proven expertise in neuroscience/CNS diseases, biotech and pharma.
Exicure
  1. Technological breakthrough of spherical nucleic acids (SNAs) is unlocking the potential of nucleic acid therapeutics with localized delivery to the skin, eye, GI tract, liver, lung and brain
  2. 3 drugs in the clinic and a proprietary discovery platform with applications in oncology, inflammatory diseases and genetic disorders
  3. Diverse, credentialed and high-energy team focused on translational science
Gamida_Cell
  1. GMDA completed its IPO in October 2018.
  2. In 2H19, the company will finish enrollment in its Phase 3 study of NiCord for bone marrow transplant, which has FDA Breakthrough Designation. NiCord has the potential to be the first FDA-approved cell therapy for bone marrow transplant.
  3. The company is also using its cell expansion technology for natural killer (NK) cells and expects additional data from its Phase 1 study of NAM-NK in 1H19.
Geovax
  1. A novel approach to vaccine development using our proprietary MVA-VLP platform, which generates non-infectious virus-like particles (VLPs) in vivo. This approach mimics a natural viral infection, triggering a robust and durable immune response may require just a single-dose for protection.
  2. GeoVax has rapidly expanded its pipeline over the past several years... moving from a singular focus on HIV to now >10 indications (including immuno-oncology) demonstrating the broad utility of our vaccine platform. We have a growing list of US & international collaborators including corporate, government, academia and NFP.  US govt sponsors include both NIH and Dept of Defense.
  3. Attractive market opportunities in HIV, immuno-oncology, chronic Hepatitis B (therapeutic), HPV (therapeutic), Zika, Ebola, Lassa Fever + others.  Clinical trials ongoing in HIV and poised to enter for Zika and Ebola.
INmune Bio
  1. INMB is focused on reprogramming the innate immune system to reverse chronic inflammation, an unrecognized but pervasive disease, with clinical programs in cancer and beyond. INMB is planning an IPO NASDAQ IPO in December 2018.
  2. The lead oncology asset, INB03 targets myeloid derived suppressor cells, the most important cell of the tumor microenvironment that is responsible for resistance to immunotherapy including checkpoint inhibitors. The goal is to eliminate resistance to CPI in patients with biomarkers of chronic inflammation including elevated MDSC.  INB03, also known as XPro1595, has been studied in other therapeutic areas with more than 50 publications.  The company’s development activities will expand beyond oncology as additional resources become available. 
  3. INKmune, a novel biologic that activates the patient’s NK cells to attack their cancer is  being developed for the treatment of relapse refractory ovarian cancer and high-risk MDS; both trials are expected to begin during 2019.  
MeiraGTx
  1. Broad clinical pipeline with 3 ocular trials and 1 Xerostomia trial ongoing.  Also, programs in neurodegenerative disease including ALS, and a completed Ph2 Parkinson's Disease study.
  2. In-house wholly-owned Manufacturing facility capable of supplying all pipeline targets from pre-clinical through potential commercial manufacturing supply.
  3. Next-generation gene therapy Riboswitch based gene regulation.  Proprietary technology that may allow for innovative gene therapy treatments whose expression can be turned on and off with an easily administered small molecule.
UroGen Pharma UroGen is a clinical-stage bio-pharmaceutical company developing advanced non-surgical treatments to address unmet needs in the field of urology.   
  1. Developed RTGTM, a sustained release, hydrogel-based platform technology with the potential for use in a range of body cavities where increased dwell time may improve therapeutic profiles of existing drugs. 
  2. Management team with a proven track record in oncology & urology 
  3. Potential for UGN-101 to be the first ever FDA drug approved for the treatment of LG UTUC:
    • FDA Orphan Drug, Breakthrough Therapy & Fast Track Designation 
    • Potential Approval in 2019

Private Therapeutic Biotech

Company

Why You Should Vote

Amplyx Pharmaceuticals, Inc

Resistance to current treatments for invasive fungal infections represents a growing threat to global health.

  1. Amplyx's lead product candidate has an innovative mechanism of action and has the potential to be the first new class of antifungal agents approved in the last two decades.
  2. Not only is Amplyx developing first-in class drugs, but the company also plays an active role in government policy issues that aim to prevent and treat infectious diseases with safe and effective medicines.
  3. Most importantly, Amplyx's drug development efforts are focused on saving the lives of vulnerable immunocompromised patients.
Azitra

Azitra, Inc. is an emerging biotechnology company that leverages its extensive knowledge of proteomics, genetic engineering and the skin microbiome to discover and develop novel products for the treatment of skin conditions and diseases. Azitra has several engineered microbes as live biotherapeutic product (LBP) candidates for skin diseases. 

2019 will be a transformational year for Azitra with multiple inflections points expected: 

  1. Azitra's science of engineering the microbiome is innovative but supported by substantial data, forthcoming publications, collaborations with Jackson Laboratory and Yale, and grant funding from the NIH, NSF, and DoD. 
  2. Azitra is transitioning into a clinical stage company and will announce clinical results of its first program in healthy volunteers in February 2019. 
  3. The company is aggressively working on both large indications (e.g., eczema) and rare diseases with no or few approved treatment options (e.g., Netherton syndrome and EGFR inhibitor-associated skin rashes). Azitra is addressing these indications using its innovative platform with clinical studies planned for 2019.
BlueRock Therapeutics
  1. Cutting edge regenerative cell therapy
  2. Record setting Series A
  3. About to enter clinic in first major clinical area (Parkinson's disease)
Cantex logo
  1. Older adults with acute myeloid leukemia are rarely cured with available chemotherapeutic or targeted agents; development of better treatment options remains a significant unmet need.
  2. Cantex's CX-01 targets the bone marrow microenvironment by disrupting cytokine and signaling pathways within the bone marrow, and making it less hospitable to, and protective of, leukemia stem cells 
  3. Based on phase II clinical trials, CX-01 received FDA Orphan Drug and Fast Track Designations for the initial induction treatment of older patients with AML and plans to initiate a phase 3 trial in 2019.
E-Scape Bio
  1. Focus on genetic forms of neurodegenerative diseases
  2. Have the only APOE4 structure modulator for APOE4 Alzheimer's, an incredibly promising and elusive target
  3. Highest calibar management team (CMO and CSO hires to be announced very soon)
InCarda Therapeutics 
  1. InCarda is pioneering a novel inhaled therapeutic candidate to rapidly treat acute episodes of paroxysmal atrial fibrillation. Over 30 million people suffer from atrial fibrillation globally (6 million in the US), and the condition results in over $25B in healthcare costs annually in the US.
  2. If approved, InRhythm will become the only therapy available to quickly (within a few minutes) treat and resolve acute episodes of atrial fibrillation, wherever and whenever they occur, resulting in rapid relief and greatly improved quality of life for millions of patients.
  3. InCarda has raised $50M from top investors, including Sofinnova, Deerfield, HealthCap and Morningside, and is led by a highly experienced team of leaders in the biotech, cardiovascular and inhalation fields.
Kleo Pharmaceuticals
  1. Pioneering new classes of small molecules and synthetic peptides that are designed to emulate or enhance biologics in Immuno-oncology.
  2. Poised to transform proof-of-concept studies into a lead clinical candidate in Multiple Myeloma and research collaborations with major Pharma in 2019.
  3. An exciting collaboration with Series B lead investor Peptidream Inc. to discover and transform high quality, synthetic binders into next-generation therapeutics.
Landos
  1. Landos Biopharma aims to disrupt the standard of care IBD treatment paradigm with lead product, BT-11, an oral, locally-acting, small molecule targeting the novel LANCL2 pathway and in development for treatment of ulcerative colitis and Crohn's disease. 
  2. Their Phase 1 study of BT-11 in IBD began in July 2018 and is expected to have topline results by year end. 
  3. The company is raising a Series B round to fund their Phase 2 trial in early 2019 and continues to advance their strong pipeline.
QUE Oncology, Inc.
  1. ATTRACTIVE PARTNERING STAGE: QUE's lead asset has commenced Phase 2 trials and thus represents an attractive partnering stage.
  2. HIGH UNMET NEED: QUE's lead asset is in an area of high inlet need - the treatment of hot flashes and night sweats in breast cancer patients on endocrine therapy. 
  3. APPROPRIATE FINANCING STAGE: At BIO QUE will be in a position to raise new funds to take the drug into more extensive Phase 2b trials and to expand into new indications, such as the broader menopausal market and the treatment of prostate cancer patients on androgen deprivation therapy.
Rafael Pharmaceuticals logo 1. Only Cancer metabolism company in late stage clinal development - Two Phase III Pivotal trials ( Stage IV Pancreatic cancer and R/R AML)
2. Only oncology company with 5 Orphan designations in the uS and two in EMA
3.This is one of the most powerful approaches to targeting cancer metabolism currently in development.

Diagnostics and Beyond

Company

Why You Should Vote

A2A Pharma
  1. A2A's mission is to help other companies accelerate very high quality new drug candidates into clinical trials at dramatically lower cost with proprietary computational approaches including deep learning.
  2. We have been able to design and generate highly inhibitory and novel molecules vs difficult to drug targets like Mll-Menin, Yap-Tead & LpxC.
  3. The time and money spent getting novel molecules into Pre-IND scale up and toxicology is much lower than that of competitive programs with much better potency and ADMET properties.

Admetsys

  1. ​Over 80% of patients in hospital acute care suffer from blood sugar disregulation due to Type 1, Type 2 or Stress Diabetes.
  2. Poor blood sugar control in-hospital results in significantly longer patient length-of-stay, higher risk of mortality and a range of complications.
  3. Clinicians lack better tools and manage these patients automatically. Admetsys has developed the first automated blood glucose control system (cruise control) for hospital care. The device enables automated sensing, modeling and treatment--increasing the standard of care at lower cost.

Cellics Therapeutics, Inc.

  1. Our products are based on the disruptive "Nanosponge" technology, which has won domestic and international recognition. We use endogenous substances from the human body to make RBC Nanosponges, which are biocompatible and biodegradable and are also very effective in absorbing toxins or hemolytic processes produced by bacteria or viruses.   
  2. Our products can treat diseases that endanger people's lives but have limited or no treatment options and thus can have high impact on many patients' well-being. Our products also circumvent the problem of drug resistance as with most antibiotics. 
  3. Our products have big markets but face limited competition and thus could potentially bring very high financial return to our investors.
iPill Dispenser
  1. The iPill can save lives. 135 people die a day from the overconsumption of opioids. The iPill enforces adherence to the opioid prescription schedule, prevents diversion, provides a safe storage solution, uses blockchain to allow EMR connectivity and physician remote access, provides cognitive behavioral therapy for non-opioid interventions, and detects respiratory depression and can call 911 if respiratory distress is detected
  2. The iPill can make money.  By being reusable, tamper-resistant, and hack-resistant, a small user fee is charged per use when the iPill is returned to be reused for the next prescription.  After 3-4 months, the user fee pays for the production cost of the iPill and the remaining 8-9 months of the year the user fee represents 100% profit.
  3. The iPill can save money for the healthcare system.  The opioid crisis causes the US healthcare system $635 billion per year (CDC).  By saving this amount of money we can actually afford to give cognitive behavioral therapy, psychosocial therapy and social support for addicts.
Longeveron LLC
  1. Conducting trials for chronic conditions of aging that have no current treatment
  2. In house proprietary cellular production facility with capacity to partner with other biotech companies
  3. Only company currently running multiple FDA trials to address Aging Frailty

Lummee

  1. Your vote will help us serve the most underserved population in our nation today- individuals battling addictions
  2. Our products are clinically validated & have demonstrated the highest success rates of any commercial product
  3. Our suite of products are revolutionizing the way we monitor our health & is paving the way to a future where lifestyle-related illnesses is a thing of the past
ProterixBio, Inc
  1. ProterixBio is developing molecular assessments to guide therapy and accelerate drug development for patients with chronic obstructive pulmonary disease (COPD), an underserved population with poor health outcomes and high levels of healthcare spending.
  2. ProterixBio's technology, which uses a combination of protein biomarkers measured in blood and combined using an algorithm, is a novel approach and represents a first-in-market product for COPD.
  3. ProterixBio's initial product - a COPD stratification test - is targeted towards value-based chronic care programs, a growing market segment that needs improved tools for identifying patients for escalation of therapy and closer management.
Trigemina
  1. Very attractive valuation because of fully correctable mistakes by previous management
  2. Unique and innovative disruptor in a highly unmet area with huge potential
  3. Very short development course (possible orphan indication) at low cost

Xcell Biosciences

  1. We enable easy, reproducible control of wide range of primary human cells, including expanding challenging therapeutically important immune populations, patient-derived tumor cells, and driving rapid, controlled differentiation in stem cells for allogenic universal cell therapy programs.
  2. We use precise control of cellular microenvironments to change cell phenotype, population mix, and function, resulting in the first platform for cell therapy screening, optimization, and tailored therapeutic expansion.  
  3. We enable researchers with rapid, predictive drug screening in large, high growth markets including both immunotherapy and regenerative medicine.
Zumutor
  1. Zumutor is working in the space of Immuno-Oncology with its lead compound(first-in-class) in pre-clinical trial
  2. The lead asset :ZM008 will be the first in class for MCRPC (multiple castration resistant prostate cancer)
  3. Presenting at Bio would help raise monies for Phase 1trials and this would be critical for progress