Author Archives: ac_internal

Carisma Therapeutics Names Dr. Robert Petit As Chief Scientific Officer

Carisma Therapeutics is excited to welcome Robert Petit, PhD as its Chief Scientific Officer. Dr. Petit is an accomplished immuno-oncology scientist, innovator and drug developer, and a C-level executive with 25 years of experience in building and leading scientific, translational, and clinical development teams in public and private companies.

Press release here.

Carisma Therapeutics information here.

Carisma Therapeutics Featured in Nature Biotechnology’s Top Academic Spin-outs 2018

Nature Biotechnology’s annual survey highlights startups taking on gene therapy, adoptive immune cell therapy, gene editing, and drugs targeting RNA modifications and the unfolded protein response.

Publication link.

Precision BioSciences Appoints Christopher R. Heery, M.D., as Chief Medical Officer

DURHAM, N.C., May 14, 2019 (GLOBE NEWSWIRE) — Precision BioSciences (Nasdaq: DTIL), a genome editing company dedicated to improving life through the application of its proprietary ARCUS® genome editing platform, announced today that Christopher R. Heery, M.D. has joined Precision BioSciences as its Chief Medical Officer.

“We are thrilled to welcome Chris Heery as our Chief Medical Officer. Chris has exceptional experience in the rapidly evolving field of cell therapies and has led many first-in-human clinical trials during his time at the National Cancer Institute (NCI). We believe his skill set and experience are a perfect fit to guide our off-the-shelf immunotherapy and in vivo gene correction programs through human clinical trials,” said Matt Kane, Chief Executive Officer and co-founder of Precision BioSciences.

Full press release here.

Skyhawk Therapeutics Announces Agreement with Takeda for Neurodegenerative Diseases

WALTHAM, Mass., May 6, 2019 /PRNewswire/ — Skyhawk Therapeutics, Inc. (Skyhawk) today announced a strategic collaboration with Takeda Pharmaceutical Company Limited (Takeda) in which Skyhawk will use its SkySTAR™technology platform to discover and pre-clinically develop innovative small molecule treatments directed to certain neurological disease targets. The agreement grants Takeda an exclusive worldwide license to develop and commercialize compounds and products directed to multiple targets while Skyhawk receives an upfront payment and is eligible to receive future milestone payments and royalties. As part of the agreement, Takeda will be responsible for clinical development and potential commercialization.

Skyhawk will use its SkySTAR™ platform to develop drug candidates directed to multiple targets for neurodegenerative diseases.  In exchange Takeda will pay Skyhawk an upfront payment, future milestone payments and royalties.

Fullress release here.

Pliant Therapeutics Reports Positive Results Of Phase 1 Clinical Study

SOUTH SAN FRANCISCO, CA — April 9, 2019 — Pliant Therapeutics, Inc., a biotechnology company focused on discovering, developing and commercializing treatments for fibrotic diseases, today announced the successful completion of its first-in-human clinical study of its lead product candidate, PLN-74809. The small molecule inhibitor of the αVβ6 and αVβ1 integrins was shown to be safe and well tolerated in healthy volunteers, and the results support further development in patients with idiopathic pulmonary fibrosis (IPF), a chronic and progressive fibrotic lung disease with few treatment options.

The randomized, double-blind, placebo-controlled study of PLN-74809 clearly demonstrated the small molecule’s good oral bioavailability in humans and its dose-proportional pharmacokinetics after a single dose and following 14 days of once-daily administration, at doses ranging from 10 to 75 mg. The compound was well-tolerated across all doses in 71 study participants and its long half-life supports once-daily dosing.

Full press release here.

Genome editing company Precision proposes $100M IPO

With plans to bring an allogeneic CAR T immunotherapy into the clinic, Precision BioSciences Inc. (Durham, N.C.) proposed Friday to raise up to $100 million in an IPO on NASDAQ.

With plans to bring an allogeneic CAR T immunotherapy into the clinic, Precision BioSciences Inc. (Durham, N.C.) proposed Friday to raise up to $100 million in an IPO on NASDAQ.

Precision plans to start this half a Phase I/IIa trial of lead program, PBCAR0191, in acute lymphoblastic leukemia (ALL) and non-Hodgkin lymphoma (NHL). The allogeneic CAR T cell therapy targeting CD19 has Orphan Drug designation in the U.S. to treat ALL.

The company also has CAR T therapies targeting CD20, BCMA and CLEC12A in preclinical development. Precision plans to submit an IND to FDA for the CD20-targeting therapy in 4Q19, and is aiming to submit INDs for the other two programs next year.

Read full news article here.

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.