“All along our guidance has been that, look, we are doing multiple open-label studies with the intent of picking a winner,” said Protagonist Therapeutics Inc. CEO Dinesh Patel during a conference call with investors. “Today, we have picked a winner by a huge margin” in the shape of PTG-300, an injectable hepcidin mimetic for the rare blood cancer polycythemia vera (PV).
CEO Carsten Brunn said Selecta Biosciences Inc. has “not seen a material impact” from the COVID-19 pandemic and remains on track to report in the third quarter phase IIb data from a head-to-head trial comparing its refractory gout candidate, SEL-212, with Krystexxa (pegloticase), from Horizon Therapeutics plc, of Dublin.
Novartis AG won accelerated FDA clearance for Tabrecta (capmatinib, formerly INC-280), an oral MET inhibitor, for adult patients with metastatic non-small-cell lung cancer (NSCLC) whose tumors have a mutation that leads to MET exon 14 skipping (METex14) as detected by a companion diagnostic, also given the green light.
Praxis Precision Medicines Inc. CEO Marcio Souza told BioWorld that the line figuratively walked by PRAX-114, a GABAA positive allosteric modulator in major depressive disorder (MDD), means upside relief without the downside of adverse effects.
With regard to agitation in Alzheimer’s disease (AD), doctors “are in a very difficult position right now,” said Axsome Therapeutics Inc. CEO Herriot Tabuteau. That’s because the products currently used off-label are antipsychotics – all of which carry an FDA black box warning “specifically against their use in elderly patients with dementia, including AD,” since they double the risk of stroke and of mortality.
CEO and co-founder Rosana Kapeller told BioWorld that Rome Therapeutics Inc.’s $50 million series A will fund early work in an “exploding” new area of biology: the repeatome – “the dark genome” or “junk DNA,” as many previously deemed the roughly 60% of the human genome that consists of repetitive sequences of nucleic acids.
Seattle-based Neoleukin Therapeutics Inc. is targeting the end of 2020 for an IND submission related to prospective cancer immunotherapy NL-201, described as an engineered, hyperstable agonist of interleukin-2 (IL-2) and IL-15. It’s meant to eliminate alpha receptor binding and thereby overcome the problems with native IL-2.
A pair of good-news items from Chimerix Inc. pushed the Durham, N.C.-based company’s stock (NASDAQ:CMRX) to $2.15, closing up 64 cents, or 42%, higher as backers reacted to near-term NDA plans for smallpox countermeasure brincidofovir (BCV) and the start of a phase II/III trial with dociparstat sodium (DSTAT) in COVID-19 patients with acute lung injury (ALI).
With accelerated approval in hand for Trodelvy (sacituzumab govitecan-hziy) to treat metastatic triple-negative breast cancer (mTNBC), Immunomedics Inc. is looking ahead to data related to the next indication for the antibody-drug conjugate (ADC) – urothelial tumors – “in the near future,” Chairman Behzad Aghazadeh told investors during a conference call.
Immunomedics Inc. gained accelerated FDA clearance for Trodelvy (sacituzumab govitecan-hziy) to treat patients with metastatic triple-negative breast cancer (TNBC) who have undergone at least two prior therapies. It’s the first antibody-drug conjugate (ADC) given the go-ahead specifically in relapsed/refractory TNBC and the first anti-Trop-2 ADC bound for the market. Trodelvy, which was granted breakthrough therapy designation and priority review, moved along faster thanks to the objective response rate (ORR) and duration of response (DoR) turned up by Morris Plains, N.J.-based Immunomedics in a single-arm, multicenter phase II study. Continued approval may be contingent on verifying clinical benefit in the confirmatory phase III experiment called Ascent, recently halted by the independent data safety monitoring committee due to compelling evidence of efficacy across multiple endpoints.
