On its second-quarter earnings call, Affimed NV promised much-anticipated clinical and abstract data updates in the fourth quarter on several pipeline programs, including AFM-11, its CD19/CD3-targeting T-cell engager.

"A good RNAi therapeutic against hepatitis B just makes intuitive sense and scientific sense," Christopher Anzalone, president and CEO of Arrowhead Pharmaceuticals Inc., told BioWorld to explain the attraction behind its third-generation HBV candidate, ARO-HBV. The agent is designed to silence the entire HBV transcriptome, intervening upstream of the reverse transcription process that standard-of-care nucleotide and nucleoside analogues affect. "If you can accomplish that, you disrupt the life cycle of that virus," Anzalone said.

Paratek Pharmaceuticals Inc. waited less than 24 hours for word late Tuesday of its second and bigger win with FDA approval of lead candidate, omadacycline, branded Nuzyra. Earlier in the day, the Boston-based company received word that Seysara (sarecycline) was greenlighted by the FDA to treat inflammatory moderate to severe acne in patients 9 and older. (See BioWorld, Oct. 3, 2018.)

Nearly two weeks ahead of its presumed PDUFA date, sarecycline, branded Seysara, was greenlighted by the FDA to treat inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients 9 and older. The once-daily, oral, narrow spectrum tetracycline-derived antibiotic was discovered by Paratek Pharmaceuticals Inc., which originally licensed U.S. development and commercialization rights to Warner Chilcott Ltd., which went to Actavis plc, now Allergan plc. In August, Almirall SA gained those rights in conjunction with its acquisition of Allergan's U.S. medical dermatology unit for $550 million in cash and a potential $100 million earn-out based on business performance. The transaction closed last week.

Although analyst forecasts ranged from exuberant to temperate, all agreed that approval of Libtayo (cemiplimab-rwlc, previously REGN-2810) marked a new chapter for longtime partners Regeneron Pharmaceuticals Inc. and Sanofi SA. The anti-PD-1 sneaked under the third-quarter wire, a month ahead of its PDUFA date, as the first FDA-approved therapy to treat advanced cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC in individuals who are not candidates for curative surgery or radiation.

"Encouraged," apparently, is not a word investors relish when it comes to trial results – even early phase I data – as Avrobio Inc. learned after disclosing findings on the first participants in an investigator-sponsored phase I study and company-sponsored phase II of ex vivo lentiviral (LV) gene therapy AVR-RD-01 in Fabry disease. The Cambridge, Mass.-based company, which priced its June IPO at $19 to raise $114.7 million, including overallotments, and hit a high of $53.70 last week, saw shares (NASDAQ:AVRO) give back more than half their value Monday, falling to $21.58 before closing at $25.13 for a loss of $26.74, or 51.6 percent. 

The FDA handed Pfizer Inc. an on-time approval for the oral kinase inhibitor Vizimpro (dacomitinib) to treat individuals with metastatic non-small-cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletion or exon 21 L858R substitution mutations as detected by an FDA-approved companion diagnostic. The nod followed a priority review of the application, which the agency granted earlier this year.

Arvinas Inc., Sutro Biopharma Inc. and Urovant Sciences Ltd. became the latest biopharmas to head to Nasdaq, pricing their IPOs to raise a combined $345 million, but – like Entasis Therapeutics Inc. a day earlier – not all enjoyed a warm welcome on their public debut.

Alexion Pharmaceuticals Inc. plucked another rare disease pearl with a deal to acquire Syntimmune Inc. and its humanized monoclonal antibody, SYNT-001, which is in phase I/II studies in the skin diseases pemphigus vulgaris (PV) and pemphigus foliaceus and the hematology indication warm autoimmune hemolytic anemia (WAIHA). SYNT-001, which inhibits interaction of the neonatal Fc receptor (FcRn) with immunoglobulin G (IgG) and IgG immune complexes, offers Boston-based Alexion development prospects in other rare IgG-mediated diseases, including neurology indications, with proof of mechanism showing rapid IgG reduction.

Months after inking an agreement with GC Labcell (GCLC), Canadian startup Feldan Therapeutics Inc. pocketed $12.5 million in a series A financing led by parent company GC Holdings Corp., the recently rebranded Korean conglomerate that also houses GC Pharma, formerly Green Cross Corp. Participants in the round included South Korea's Stonebridge Ventures and Anges Québec Capital and members – a previous seed investor – along with undisclosed existing investors. Feldan, based in Québec City, is developing therapies based on its peptide-based intracellular delivery technology, dubbed the Feldan Shuttle.