BioMarin Pharmaceutical has reported that the multi-centre Phase III PEGASUS trial of Palynziq (pegvaliase-pqpz) has met its primary efficacy endpoint, showcasing a reduction in blood phenylalanine (Phe) levels in adolescents with phenylketonuria (PKU) against only diet.

Shares in Synlogic have halved in value after the company reported that a phase 3 trial of its main pipeline drug for rare metabolic disorder phenylketonuria (PKU) failed to show efficacy, putting its future in doubt.

The company will lay off 87% of its workforce and halt its research, including for a gene editing therapy to treat the rare disease PKU.

PTC Therapeutics’ phase 3 clinical trial in the rare inherited disorder phenylketonuria (PKU) has met its primary endpoint. And yet, the design of the study means analysts remain unsure about the strength of PTC’s hand and whether it can rival BioMarin’s Kuvan and live up to its $400 million peak sales forecast.

The phenylketonuria pipeline already had one project on clinical hold, and now it has another: Homology Medicines’ HMI-102 on Friday joined Biomarin’s BMN 307 in attracting scrutiny from the FDA. It is not yet clear exactly what the problem is with HMI-102, but Homology cited “elevated liver function tests” in the phase 1/2 Phenix trial.

BioMarin Pharmaceutical faces a long clinical hold on its phenylketonuria (PKU) gene therapy trial. After stopping the trial over tumors in mice, the FDA has asked BioMarin to run additional nonclinical studies that are expected to take “several quarters” to assess the risk of BMN 307.

Pfizer is making a $60 million equity investment in the gene therapy company Homology Medicines, betting on what one of its rare disease executives termed a “potentially transformational” therapeutic option for patients with a rare metabolism disorder known as phenylketonuria, or PKU.

While industry-shaking M&A deals are still nowhere in sight, smaller deals by bargain shoppers continue to trickle in. PTC Therapeutics has joined the club, bagging Censa Pharmaceuticals and its rare metabolic disease drug.

Those waiting for the first look at human data from Rubius will have to be patient for a few more months. On Thursday, the Cambridge, Massachusetts-based biotech delayed the timeline for its first clinical readout to early 2020, admitting the trial has yet to dose its first patient.

Synlogic, Inc., (Nasdaq: SYBX) a clinical stage company applying synthetic biology to beneficial microbes to develop novel, living medicines, announced on 4/30/19 that data demonstrating its development of a robust and reproducible process to generate a solid oral formulation of its Synthetic Bioticmedicine, SYNB1618, are being presented today at the 22nd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT). Synlogic is developing SYNB1618 for the treatment of phenylketonuria (PKU).