Author Archives: ac_internal

Biogen and Skyhawk Therapeutics Announce Agreement to Develop Novel Small Molecule RNA Splicing Modifiers for Neurological Disease Targets

January 4, 2019 at 7:30 AM EST

  • Biogen made a one-time upfront $74 million payment to Skyhawk for research services and an exclusive license to therapeutic candidates for multiple sclerosis, spinal muscular atrophy and other neurological diseases to be developed using Skyhawk’s SkySTAR™ platform
  • Biogen to pay Skyhawk potential future milestone payments and royalties

CAMBRIDGE, Mass. and WALTHAM, Mass., Jan. 04, 2019 (GLOBE NEWSWIRE) — Biogen Inc (Nasdaq: BIIB) and Skyhawk Therapeutics, Inc. (Skyhawk) today announced a strategic collaboration in which the companies will leverage Skyhawk’s SkySTAR™ technology platform with the goal of discovering innovative small molecule treatments for patients with neurological diseases. Biogen will have the option to license therapies resulting from the collaboration and will be responsible for their development and potential commercialization.

The agreement grants Biogen an exclusive license to worldwide intellectual property rights on research-stage therapeutic candidates for the treatment of multiple sclerosis (MS), spinal muscular atrophy (SMA) and additional neurological disorders. As part of the agreement, Skyhawk received an upfront payment of $74 million from Biogen and may receive potential future milestone payments and royalties. A portion of the upfront payment will be allocated to future research services, with the remainder expensed in the first quarter of 2019 as research and development.

Read full press release here.

Pliant Therapeutics Initiates Phase 1 Clinical Study of PLN-74809

Development in Orphan Lung Disease Idiopathic Pulmonary Fibrosis Planned

SOUTH SAN FRANCISCO, CA — January 3, 2019 — Pliant Therapeutics, Inc., a biotechnology company focused on discovering, developing and commercializing treatments for fibrotic diseases, today announced that it has dosed the first cohort of subjects in a Phase 1 clinical study evaluating the safety, tolerability, pharmacodynamics and pharmacokinetics of anti- fibrotic agent PLN-74809.

“Based on positive preclinical studies, and shortly after submitting our first investigational new drug application, Pliant’s first-in-human trial for PLN-74809 is underway and will inform further development of our novel, proprietary compound for the potential treatment of patients with idiopathic pulmonary fibrosis, a disease with very poor prognosis and few impactful treatment options,” said Éric Lefebvre, M.D., chief medical officer of Pliant Therapeutics. “This study’s results will also guide our development plans to evaluate PLN-74809 in other serious diseases where integrins are key drivers of fibrosis by activating TGF-β, including primary sclerosing cholangitis.”

This randomized, double-blind, placebo-controlled, single and multiple ascending oral dose study will enroll approximately 90 healthy participants. The primary objective is the assessment of the safety and tolerability to PLN-74809 after a single dose and following 14 days of dosing. The study also includes evaluation of pharmacokinetics and exploratory assessments of biomarkers related to the activity of the αVβ6 and αVβ1 integrins, the target of PLN-74809 treatment.

Read full press release here.

Dice Molecules

Preclinical company bringing directed chemical evolution to small molecule drug discovery.

Palvella Therapeutics Announces $10 Million in Funding from Ligand Pharmaceuticals to Accelerate PTX-022 through Phase 2/3 Clinical Study in Pachyonychia Congenita

– Fast Track-Designated Program Commencing Phase 2/3 Study in Q1 2019 –

Wayne, PA – December 18, 2018 – Palvella Therapeutics, Inc., a rare disease biopharmaceutical company focused on developing and commercializing pathogenetically targeted therapies for debilitating genetic diseases with no approved treatments, today announced that the company has entered into a development funding and royalties agreement with Ligand Pharmaceuticals, Inc. (NASDAQ: LGND) to advance Palvella’s lead product candidate PTX-022 (QTORIN™ rapamycin formulation) for the treatment of pachyonychia congenita (PC). Under the terms of the agreement, Palvella will receive $10 million from Ligand in exchange for milestones and a tiered single-digit royalty on future sales of PTX-022. As part of the agreement, Palvella will continue to have responsibility for all clinical development, regulatory, manufacturing, marketing and other commercialization activities on a worldwide basis. 

Read full press release here.

Entrada Therapeutics

Preclinical company developing novel treatments for rare disease.

Entrada Therapeutics Raises $59 Million Series A to Advance Intracellular Biologics Platform


Series A co-led by 5AM Ventures and MPM Capital, along with other top investors

Dipal Doshi appointed as President and Chief Executive Officer


BOSTON, Mass., Dec. 18, 2018 – Entrada Therapeutics, a biotechnology company dedicated to transforming the treatment of devastating diseases through the intracellular delivery of biologics, today announced it has raised $59 million in a Series A financing. The financing was co-led by 5AM Ventures and MPM Capital, along with Roche Venture Fund, MRL Ventures Fund (MRLV) and Agent Capital.

“With this financing, Entrada is well-positioned to advance multiple programs through the application of our proprietary intracellular biologics platform,” said Dipal Doshi, President and Chief Executive Officer of Entrada Therapeutics. “Entrada has an opportunity to create the next generation of therapeutics for previously undruggable intracellular targets. Leveraging the exciting science from the lab of our Co-Founder, Dehua Pei, at The Ohio State University, we look forward to building a world-class team that will translate this platform intomeaningful therapies.”

Entrada was funded and incubated within 5AM’s 4:59 Initiative via a seed investment by 5AM Ventures, RocheVenture Fund and MRLV. Entrada’s proprietary intracellular biologics platform has the demonstrated ability to deliver a variety of modalities into the cytosol of the cell, allowing for the development of programs across several intracellular target classes. Entrada’s delivery vehicles boast improved bioavailability, biodistribution,metabolic stability and enhanced efficiency compared to previous gold standards. The Company is applying its platform to the delivery of intracellular enzyme replacement therapies, protein-protein interaction inhibitors and nucleic acids.

The first application of Entrada’s platform is focused on treating a rare and fatal mitochondrial disease with no approved treatments that is caused by mutations in a gene that codes for an intracellular enzyme. The Series A financing will allow Entrada to advance its lead program into human clinical trials. Additionally, the Company plans on broadening its rare, monogenic disease pipeline by identifying and progressing additional intracellular enzyme replacement therapies.

“Patients with severe, debilitating diseases still suffer without suitable treatment options,” said Kush Parmar, M.D., Ph.D., Managing Partner at 5AM Ventures. “Entrada has an executive team that is capable of tackling the challenges posed by the delivery of biologics into the cell. Entrada is focused on leveraging a highly validated biological mechanism to fundamentally change the standard of care for several of these diseases via multipleintracellular targets.”

“Entrada’s unique vision to transform the treatment paradigm of certain diseases via intracellular biologics delivery could result in innovative treatments for thousands of patients,” said Todd Foley, Managing Director at MPM Capital. “While there has been extensive development of biologics that address extracellular targets, there is a significant opportunity for novel biologics that address intracellular targets. Entrada’s strategy andtechnology address this profound unmet need.”

Read full press release here.

Orchard Therapeutics Announces the Build-out of New Gene Therapy Manufacturing Facility in California

Boston, USA and London, UK, December 13, 2018 / B3C newswire / —Orchard Therapeutics (NASDAQ: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that it has signed a long-term lease agreement to build-out a gene therapy manufacturing facility in Fremont, California. The new 150,000-square-foot facility significantly increases Orchard’s California footprint and adds to the Foster City and Menlo Park, California sites, which oversee the ongoing development and validation of the manufacture of Orchard’s ex vivo gene therapy product candidates.

Full press release here.

Precision BioSciences and Durham STEM Students Lead Launch of First Genome Editing Experiment Into Space

CAPE CANAVERAL, Fla.–(BUSINESS WIRE)–Precision BioSciences today announced that the world’s first genome editing experiment in space has been launched aboard a SpaceX Dragon cargo spacecraft on a Falcon 9 rocket. The mission was launched yesterday from Cape Canaveral Air Force Station and is anticipated to deliver its cargo to the International Space Station (ISS). Astronauts aboard the ISS are expected to perform the first genome editing experiment in space using Precision’s proprietary, next-generation genome editing technology, ARCUS.

Read full press release here.

Precision BioSciences Receives FDA Authorization to Initiate Clinical Study of Gene Edited Cancer Immunotherapy

DURHAM, North Carolina, USA, November 27, 2018 Precision BioSciences and Servier today announced that the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for PBCAR0191, an allogeneic anti-CD19 CAR T therapy for B-cell acute lymphoblastic leukemia (B-ALL) and non-Hodgkin lymphoma (NHL). Upon trial initiation, PBCAR0191 will be Precision’s first clinical-stage product candidate.

“Our allogeneic CAR T program has exceeded all expectations, moving from concept to IND acceptance in under three years,” said Matt Kane, Chief Executive Officer of Precision. “We are thrilled to have received authorization to initiate clinical studies for a therapy that we believe could be transformative for patients suffering with NHL and B-ALL.”

Precision’s PBCAR0191 is positioned to be the first gene-edited allogeneic CAR T product candidate in human clinical trials for NHL. Data provided to the FDA in connection with the IND submission included the results of in depth off target cutting analyses and evidence to support the elimination of graft versus host interactions. Manufacturing data were provided to the FDA in support of the scalability and reproducibility of the T-cell genome editing production process. Precision is currently producing clinical trial material at full scale with its manufacturing partner.

Full press release here.

Pliant Therapeutics Receives FDA Orphan Drug Designation for Lead Program PLN-74809 in Primary Sclerosing Cholangitis

SOUTH SAN FRANCISCO, Calif., Nov. 20, 2018 /PRNewswire/ — Pliant Therapeutics, Inc., a biotechnology company focused on discovering, developing and commercializing treatments for fibrotic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the company’s anti-fibrotic lead compound, PLN-74809, for the treatment of primary sclerosing cholangitis (PSC). In addition, Bertus Eksteen, Ph.D., MBChB, FRCP, a leading clinical researcher in PSC, has joined Pliant’s scientific advisory board.

Full press release here.