BIO 2024

Meet our Amazing Clients

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4D Molecular Therapeutics

Unlocking the full potential of genetic medicine to treat large market diseases

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4D Molecular Therapeutics

Unlocking the full potential of genetic medicine to treat large market diseases

Key Highlights

Sought after leader in genetic medicines, with proprietary Nobel Prize-winning technology: 4DMT is applying the principles of directed evolution to invent novel, next generation AAV vectors to combat the stigma and shortcomings of conventional vectors within the genetic medicine space. Their work is attracting top pharmaceutical companies seeking to partner with the company and to license 4DMT’s vectors, including Astellas and Arbor Biotechnologies.
Quality synthetic vectors fit for purpose:Phase 2 clinical data show encouraging safety and efficacy results and supports the therapeutic potential of 4DMT’s genetic medicines in retinal diseases and cystic fibrosis lung disease. Each 4DMT candidate has been designed with optimized capsids that are delivered by the routine route of administration for each disease – including intravitreal for eye diseases and aerosol for cystic fibrosis.
Key Events in 2024:
- Recent upsized public offering of $300 million supplemented cash position of $299 million, extending company operational runway into H1 2027
- Positive interim results from Phase 2 trial for wet AMD enable rapid advancement towards pivotal development, with additional FDA and EMA regulatory interactions planned in Q2 2024, updates expected in Q3 2024, and clinical trial initiation expected in Q1 2025
- Initial interim data of Phase 2 study in DME expected in H2 2024
- Dose selection and initiation of Phase 2 expansion cohort in cystic fibrosis expected in Q2 2024, with pivotal trial initiation anticipated in H2 2025
- Expected IND filing of 4D-175 for Geographic atrophy (GA) in Q2 2024; Phase 1 initiation expected in H2 2024

Interview

Uneek Mehra, Chief Financial & Business Officer

NASDAQ

$FDMT

Website

4dmoleculartherapeutics.com

Disease Area

Ophthalmology
Pulmonology

Technology

Gene Therapy

Biocom California

The Leader and Advocate for California’s Life Science Sector

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Biocom California

The Leader and Advocate for California’s Life Science Sector

Key Highlights

Where is Biotech Headed? As one of the most seasoned veterans advocating for the life science industry, Joe has navigated the ups and downs for nearly 25 years. He can speak to current trends in policy and biotech and what they might mean for the life science industry in the coming 12-18 months.
Opening the Door to Opportunities. Building the workforce of tomorrow is one of Biocom’s key initiatives for the life sciences industry. Through programs like Generation STEAM, Biocom works with students of all ages and backgrounds, teachers, women, and groups who are traditionally underrepresented in the life sciences to provide them with the tools and resources they need to pursue a career in the STEAM field.
BIO Panel: Diversifying the Talent Pipeline: Industry-Recognized Curriculum for a Skilled Workforce

Interview

Joe Panetta, President and CEO & Vicki Brannock, Sr. Director of Workforce Strategy and Innovation

Website

www.biocom.org

CinRx Pharma

Hub & spoke biotech featuring a novel, combination approach to obesity

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CinRx Pharma

Hub & spoke biotech featuring a novel, combination approach to obesity

Key Highlights

Combination approach to obesity treatment:CinRx portfolio company CinFina is advancing four first-in-class obesity medicines with potential to achieve improved tolerability and efficacy than currently available GLP-1s. The combination of its two gut peptide monotherapies work centrally to reduce appetite and require less frequent dosing, and the two dual-agonists aim to alter metabolism and improve standard of care when combined with GLP-1 therapies.
A “hub and spoke” model: CinRx’s portfolio of CinCos are centrally funded and managed by a team of scientific, technical, and drug development experts with decades of experience. This enables nimble decision-making and resource allocation, with value at each individual company, which de-risks buy-in from investors. The model’s success was proven in January 2023 when former portfolio company, CinCor, was acquired by AstraZeneca for $1.8 billion. Among CinRx’s leading CinCos are CinFina, CinDome and CinPhloro.
Recent milestones: In March 2024, CinFina announced the dosing of the first patients in its Phase 1 trial of CIN-110. Upcoming milestones include anticipated Phase 2 data evaluating CIN-109 with and without semaglutide in 2024 and Investigational New Drug (IND) application submissions for CIN-209 and CIN-210. In February 2024, CinPhloro dosed the first patients in the Phase 2 enviva study to evaluate the safety, efficacy and tolerability of CIN-103 for IBS-D, and initial data is expected later this year.
CinRx Chief Business Officer Gavin Samuels is participating in a panel titled A Licensing Deal for All Seasons on Monday, June 3 at 1:45 PM.

Interview

Gavin Samuels, Chief Business Officer

Website

cinrx.com

Disease Area

Obesity
Metabolic Disorders
Gastroenterology
Neurology
Oncology

Technology

Small Molecule
Biologic
Peptide

Clavyst Bio

Accelerating Life Sciences Innovations with Ties to Singapore

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Clavyst Bio

Accelerating Life Sciences Innovations with Ties to Singapore

Key Highlights

Empowering Singapore’s Rise as a Global Life Sciences Powerhouse: Singapore is emerging as a global biotech hub, attracting major players and investments. Under the guidance of Khoo Shih, a seasoned executive and investor, ClavystBio aims to fund and build biotech companies with ties to Singapore, and realize the country’s potential to bring new medicines to patients in need across the globe.
Pioneering Biotech Investments in the US and Asia: With 20+ years of experience investing in life science companies globally, Khoo Shih was previously responsible for growing the life science portfolio at Temasek – Singapore’s state-owned investment company with a net portfolio value of $290 billion. After spinning out the work into ClavystBio, Khoo Shih led investments into multiple companies with operations in Singapore and the U.S., including Engine Biosciences, Allay Therapeutics, Paratus Sciences, Sunbird Bio and more.
What’s Next for Biotech Investing? As leader in life science venture building, ClavystBio can weigh in on the future of biotech investing, including promising trends, the potential of cross-border collaborations and the benefits of tapping into emerging markets.

Interview

Khoo Shih, PhD, CEO

Website

www.clavystbio.com

Kelonia Therapeutics

Pioneering in vivo CAR-T Therapies to bring genetic medicines to the masses

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Kelonia Therapeutics

Pioneering in vivo CAR-T Therapies to bring genetic medicines to the masses

Key Highlights

Democratizing CAR-T Therapy: Kelonia’s best-in-class in vivo gene placement system (iGPS) enables the efficient delivery of genetic medicine with high tissue specificity and avoids costly and lengthy ex vivo manufacturing, burdensome lymphodepleting chemotherapy and reliance on large research hospitals
$800M Collaboration with Astellas aims to develop universal in vivo CAR-T therapies and has potential applications in cancer and autoimmune conditions
Key Events in 2024: IND for lead candidate KLN-1010 in multiple myeloma

Interview

Bharat Reddy, Vice President, Strategy & Business Development

Website

keloniatx.com

Disease Area

Oncology

Technology

Cell Therapy

LEXEO Therapeutics

Harnessing Genetic Medicine’s Potential in Cardiology

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LEXEO Therapeutics

Harnessing Genetic Medicine’s Potential in Cardiology

Key Highlights

Fresh Off IPO: Following Lexeo’s IPO in November, the company showed ability to breakthrough during difficult market with investors and analysts identifying potential of technology followed up with a PIPE investment in March.
Varied Pipeline: Using AAV vectors, Lexeo is targeting the genetic causes of cardiac conditions like cardiac Friedreich ataxia and arrhythmogenic cardiomyopathy caused by mutations in the PKP2 gene (PKP2-ACM). This is coupled with a pipeline of genetic CNS conditions, including APOE4-associated Alzheimer’s, for a well-rounded pipeline of genetic medicine programs.
Interim data from Lexeo’s lead SUNRISE-FA Phase 1/2 program of LX2006 in cardiac Friedreich ataxia anticipated in mid-2024.
Additional milestones in the second half of 2024:
- Interim data readout of Cohort 1 of the Phase 1/2 clinical trial of LX2020 in PKP2-ACM
- Interim data readout of the Phase 1/2 clinical trial of LX1001 in APOE4-associated Alzheimer’s
- Initiation of IND-enabling studies of LX2021 in Desmoplakin (DSP) cardiomyopathy

Disease Area

Cardiology
Rare Disease

Technology

Gene Therapy

Mythic Therapeutics

Leave less to chance: sending ADCs to their targets with FateControl™

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Mythic Therapeutics

Leave less to chance: sending ADCs to their targets with FateControl™

Key Highlights

Helping ADCs Find Their Fate: Mythic manipulates the trafficking of antibody drug conjugates in cells with FateControl to increase uptake in the desired tissue while avoiding payload release in undesired tissues – potentially increasing therapeutic potency and improving tolerability.
Well-Known Target, Common in NSCLC? It’s KisMET: Therapeutic pipeline is led by KisMET-01 clinical study of MYTX-011, a cMET targeted ADC, for treatment of NSCLC where 70% of tumors have cMET over-expression. Notably, Mythic is pursuing a left behind patient population – those with low-to-moderate cMET expression
Key Events in 2024: Progress of KisMET-01 and presentation of preliminary data

Interview

Brian Fiske, Ph.D., Chief Scientific Officer and Co-Founder

Website

mythictx.com

Disease Area

Oncology

Technology

Antibody Drug Conjugates

Spruce Biosciences

Developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need

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Spruce Biosciences

Developing and commercializing novel therapies for rare endocrine disorders with significant unmet medical need

Key Highlights

CAH Landscape Poised for Major 2024: Outdated congenital adrenal hyperplasia (CAH) treatments leave patients with toxic steroids to manage irregular hormone levels, causing debilitating, long-term side effects. After a 70-year treatment drought, game-changing medicines are (almost) finally here, with Spruce as one of the companies leading the way.
Less Steroids, More Control: Current treatments (e.g., steroids) make up for the lack of cortisol, but they’re often given in higher doses than what the body naturally makes. Spruce’s investigational drug, tildacerfont, could be the first new, once-daily oral non-steroidal therapeutic for improving disease control and reducing steroid burden in patients.
Momentum in Targeting CRF1: With multiple shots on goal investigating breakthrough treatments for CAH, Spruce and Neurocrine are advancing oral corticotropin-releasing factor type 1 (CRF1) receptor antagonists closer to the finish line. These block a receptor involved in controlling hormone and androgen levels. Validated by a growing body of positive data, Spruce’s tildacerfont reduces adrenal androgen production and can potentially impact additional family of androgens.
2024: Make or Break year for Spruce
- Topline results from the CAHmelia-204 clinical trial in adult classic CAH patients on supraphysiologic doses of GCs with normal or near normal levels of A4 in the Q3 2024
- Topline interim results from additional dose-ranging in the Phase 2 CAHptain clinical trial in Q3 2024
- End of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) in the Q1 2025

Interview

Samir Gharib, MBA, President & CFO

NASDAQ

SPRB

Website

sprucebiosciences.com

Disease Area

Endocrine Disorders
Rare Disease

Technology

CRF1 Receptor Antagonist

Vincerx Pharma

Transforming Cancer Care via a Next Generation Bioconjugation Platform

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Vincerx Pharma

Transforming Cancer Care via a Next Generation Bioconjugation Platform

Key Highlights

Cancer is on the rise, as is the bar for treatments: We have yet to realize the full promise of precision oncology and provide patients with broadly applicable, safe, well-tolerated, curative treatments. Antibody Drug Conjugates (ADCs) offered initial promise to fill this need but many on the market are accompanied by Black Box warnings due to severe dose-limiting side effects. Vincerx is on a mission to create optimized ADC treatments. Vincerx’s clinical-stage pipeline of small-molecule drug conjugates (SMDCs) and ADCs are designed to precisely target and accumulate in tumor cells to drive efficacy, while protecting surrounding healthy tissue, and ultimately delivery cancer patients the gold medal medicines they deserve.
Next generation bioconjugation platform: Vincerx’s VersAptx bioconjugation platform is revolutionizing cancer treatment by significantly improving the efficacy of marketed ADCs. With compelling data showcasing a 20-fold improvement in tumor toxicity over existing therapies such as TRODELVY and ENHERTU in in vitro tumor models, VersAptx holds the promise of unlocking new levels of efficacy and safety in cancer treatment.
Key events for 2024:
- Presenting additional Phase 1 data for first in class SMDC, VIP236, being evaluated for multiple solid tumors in Q3 2024
- Presenting additional Phase 1 data for best-in-class ADC, VIP943, in Q4 2024
- Presenting additional dose-escalation data with NIH evaluating enitociclib, a novel CDK9 inhibitor, in a Phase 1 combination study for peripheral T cell lymphoma and double-hit diffuse large B cell lymphoma in June 2024

Interview

Steven Bloom, Chief Business Officer

NASDAQ

VINC

Website

vincerx.com

Disease Area

Oncology

Technology

Antibody Drug Conjugates
Small Molecule

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Companies to interview*

4D Molecular Therapeutics

Biocom California

CinRx Pharma

Clavyst Bio

Kelonia Therapeutics

LEXEO Therapeutics

Mythic Therapeutics

Spruce Biosciences

Vincerx Pharma

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