Alexion Pharmaceuticals Inc. is looking to the second half of 2018 as the target for regulatory filings in the U.S., EU and Japan after ALXN-1210, its long-acting C5 complement inhibitor, hit noninferiority to Soliris (eculizumab) across co-primary and the four key secondary endpoints in the pivotal phase III Study 301 in complement inhibitor treatment-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH).

In the two years since Ideaya Biosciences Inc. closed its $40 million series A, the South San Francisco-based company has grown from two to 42 employees, identified lead programs in synthetic lethality and immunotherapy, prepared to file three INDs in the next 12 to 18 months and positioned itself to move multiple candidates into the clinic by the end of 2019. That trajectory was more than sufficient to attract $94 million in a series B financing that will see the company into the second half of 2021, according to CEO Yujiro S. Hata.

Trogarzo (ibalizumab-uiyk) became the first HIV treatment with a new mechanism of action in more than a decade to gain FDA approval. The CD4-directed post-attachment HIV-1 inhibitor, developed by Theratechnologies Inc., of Montreal, and partner Taimed Biologics Inc., of Taiwan, China, gained the nod in combination with other antiretroviral treatments (ARTs) to treat HIV-1 infection in adults with multidrug-resistant HIV-1 infection who failed an existing ART regimen.

With regulatory support from the EMA and financial backing from the European Investment Bank (EIB), Biondvax Pharmaceuticals Ltd. moved a step closer to initiating the pivotal phase III trial of its universal flu vaccine candidate, Multimeric-001 (M-001), by putting a master service agreement in place with a contract research organization (CRO) for the trial, expected to begin in the third quarter prior to the 2018-19 Northern Hemisphere flu season.

Anthera Pharmaceuticals Inc. reported disastrous results from phase III RESULT study of Sollpura (liprotamase), which failed to reach the noninferiority margin of the coefficient of fat absorption (CFA) primary endpoint in individuals with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). Sollpura did achieve the secondary endpoint of coefficient of nitrogen absorption (CNA), which measures protein absorption.

Multiple therapeutic candidates directly targeting migraine are preparing to rush to market. For patients and prescribers, they couldn't come soon enough.

Nightstar Therapeutics plc opened the phase III registration trial of lead candidate NSR-REP1 to evaluate the gene therapy in individuals with the rare degenerative, X-linked disorder choroideremia (CHM), which presents in childhood as night blindness and ultimately leads to complete blindness.

Stanley Crooke, chairman and CEO of Ionis Pharmaceuticals Inc., was buoyant on a conference call Friday as the company reported details from the phase I/II study of IONIS-HTTRx (RG-6042) in individuals with Huntington's disease (HD).

Novartis AG planted another stake in the fertile digital therapeutics terrain through a collaboration with Pear Therapeutics Inc. The Basel, Switzerland-based pharma will work with Pear, of Boston, to advance development of Pear's Thrive digital therapeutic for individuals with schizophrenia.