The eyes have it: Adcom slam-dunk vote favors Horizon thyroid candidate
As expected, Horizon Pharma plc sailed through the meeting of the FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee regarding the BLA for teprotumumab in thyroid eye disease (TED) with few surprises but much discussion.

The eyes have it: Adcom slam-dunk vote favors Horizon thyroid candidate
As expected, Horizon Pharma plc sailed through the meeting of the FDA’s Dermatologic and Ophthalmic Drugs Advisory Committee regarding the BLA for teprotumumab in thyroid eye disease (TED) with few surprises but much discussion.
Transgene shows TG-4010 the guillotine after NSCLC trial miss
Transgene SA is quitting development of one of its lead therapeutic vaccines after a combination of the candidate, TG-4010, with chemotherapy and Opdivo (nivolumab, Bristol-Myers Squibb Co.) missed its primary endpoint in a phase II trial evaluating the combination as a first-line treatment for certain advanced non-squamous non-small-cell lung cancers.
Two positive CHMP opinions in December take 2019 tally to 41
DUBLIN – The European Medicines Agency’s Committee for Medicinal Products for Human Use closed out its year’s work with positive opinions on two applications, Novartis AG’s Beovu (brolucizumab) in wet age-related macular degeneration and Merck & Co. Inc.’s Recarbrio for treating gram negative infections in adults.
Sarepta’s Vyondys 53 wins a surprise accelerated approval
In August the FDA was skeptical about Sarepta Therapeutics Inc.’s injectable Vyondys 53 (golodirsen), but that changed swiftly Friday with the agency’s accelerated approval for the Duchenne muscular dystrophy (DMD) follow-on therapy, the first treatment specifically for this subtype.
EMA takes pharmacovigilance to the next level: ‘Nearly’ real-time decision-making
LONDON – Over the next decade pharmacovigilance will move on from a sole focus on adverse drug reactions (ADRs) to encompass monitoring of the in-market efficacy of medicines, according to Guido Rasi, executive director of EMA.
Cautious optimism for Biolinerx’s CXCR4 inhibitor in pancreatic cancer
HONG KONG – Israel-based Biolinerx Ltd. has unveiled the latest data from its ongoing phase IIa COMBAT/KEYNOTE-202 study at the European Society of Medical Oncology Immuno-Oncology Congress 2019 which evaluates Biolinerx’s BL-8040, an inhibitor of the chemokine receptor CXCR4, in combination with pembrolizumab and chemotherapy in second-line pancreatic cancer patients.

Analysis and data insight

Dec. 9-13, 2019 week in review: Biopharmas impress investors at ASH
A quick look back at top stories.
Phase I clinical trials: November 2019
Therapeutic area data reported by biopharma companies on phase I trials in November 2019.
Phase II clinical trials: November 2019
Therapeutic area data reported by biopharma companies on phase II trials in October 2019.
Phase III clinical trials: November 2019
Therapeutic area data reported by biopharma companies on phase III trials in November 2019.

Financings

Hummingbird Bioscience scores $19M in series B financing
Cancer
HONG KONG – Singapore and U.S.-based Hummingbird Bioscience Pte Ltd. has raised $19 million in a series B financing round. Proceeds will be used to support the discovery of new disease targets and to fuel the work of co-discovery projects that are part of a multitarget collaboration agreement valued at more than $1 billion signed with Amgen Inc. in September.

Science

Bench Press for Dec. 13, 2019
BioWorld looks at translational medicine.

Malaria drug hopeful also controls intestinal, other parasites
Infection
Cryptosporidium is a parasite that affects both the very poor and the very rich. It can cause severe diarrheal infections in malnourished children and in people whose immune systems are compromised, for example, by HIV.
Scientists create artificial neurons capable of mimicking biological counterparts
Europe
LONDON – Scientists in the U.K. are claiming a world first, after successfully reproducing the electrophysiology of biological neurons in silicon chips. It is said that artificial neurons respond to non-linear physiological feedback in real time, in exactly the same way as their biological...

Newco news

With another $26M, Japan’s Heartseed aims to advance iPSC-derived regenerative medicine for heart failure
Cardiovascular
BEIJING – Tokyo-based biotech firm Heartseed Inc., which focuses on regenerative medicine, has closed a series B financing round to pocket $26 million that will go to its lead drug candidate, HS-001, for treating heart failure.
Dyno to scale the heights of AAV vector engineering
Gene therapy
Startup firm Dyno Therapeutics Inc. is attempting to engineer a new generation of adeno-associated virus (AAV) capsids by navigating its way across what it calls the “capsid fitness landscape,” in order to optimize the key parameters that affect capsid performance: production, delivery efficiency, biodistribution, immunogenicity and thermostability.
South Korea’s Avixgen secures ₩10.6 billion in pre-IPO investment
HONG KONG – South Korea’s Avixgen Inc., which recently secured ₩10.6 billion (US$9 million) in pre-IPO investment and has raised total funding of ₩27 billion since its series A in 2015, plans to use funds to further develop drug platform technologies and therapeutics for virus-causing diseases such as HIV-1/AIDS, using inhibitors based on small molecules and cancer drugs based on peptides.
Human nature: Single-cell genomics Immunitas launches with a series A
The research challenge facing the scientific founders of newly launched Immunitas Therapeutics Inc. was getting human samples to deeply analyze in their quest to understand autoimmunity. While the research continued slowly, the science raced ahead.
SL Bigen secures ₩3B from Yuhan for CAR-NK therapy
HONG KONG – South Korean pharmaceutical giant Yuhan Corp. has invested ₩3 billion (US$2.6 million) in SL Bigen Co. Ltd., a Korean biotech based in Seongnam, Gyeonggi-do, that is focused on chimeric antigen receptor-directed natural killer (CAR-NK) therapy.

U.S.

Senate confirms Hahn to take over as FDA commissioner
FDA
The U.S. Senate voted 72-18 to confirm Stephen Hahn as the commissioner of the FDA, providing the agency with another commissioner with a deep background in oncology. Hahn succeeds Scott Gottlieb, who stepped down from the post in April and returned to the American Enterprise Institute.

Europe

U.K. startups, early stage investment on the rise
LONDON - The number of biotech startups in the U.K. increased by almost 50% between 2014 and 2018, compared to 2012 – 2016, according to the latest data from the consultancy Biocity, which has been tracking the sector since 2005.

Celltrion’s Remsima SC wins marketing approval in EU
Immune
HONG KONG – Celltrion Healthcare Co. Ltd., a South Korean biopharmaceutical corporation, won European Union (EU) marketing approval for Remsima SC for the treatment of rheumatoid arthritis (RA), as a subcutaneous version of Celltrion’s infliximab biosimilar, CT-P13.
EMA advancing strategy for future regulatory science, with new tech, real-world data
Regulatory
LONDON – “Innovation only matters if patients benefit.” So said Guido Rasi, executive director of the EMA, as the agency embarks on the next phase of updating its regulatory science, both to tap into a torrent of new technologies and to ensure drug development generates evidence to demonstrate cost...

Clinical

Abdominal snowman melts for Iterum but proposition solid with sulopenem in UTI
Infection
Shares of Iterum Therapeutics plc (NASDAQ:ITRM) slid 36%, or $1.73, to close at $3.12 after the Dublin-based firm disclosed the much-anticipated but less-than-stellar results from the phase III trial called Sulopenem for Resistant Enterobacteriaceae, or SURE 3, testing oral and I.V. versions of the drug in complicated intra-abdominal infections (cIAI).
In all Candor, Darzalex has positive data
Conferences
ORLANDO, Fla. – As the enormous American Society of Hematology annual meeting wound down Tuesday, the Janssen Pharmaceutical Cos. of Johnson & Johnson released phase III data showing that adding Darzalex (daratumumab) to carfilzomib (Kyprolis from Amgen Inc.) and dexamethasone (DKd), compared to carfilzomib and dexamethasone (Kd) alone, significantly improved progression-free survival in patients with relapsed/refractory multiple myeloma.
‘Multiple ways’ of fighting once-incurable sickle cell disease
Conferences
ORLANDO, Fla. – At the 61st American Society of Hematology (ASH) annual meeting late-breaking abstracts session on Tuesday, researchers from Boston Children’s Hospital reported that three adult patients who had received an autologous transplant of gene-edited hematopoietic stem cells lacking BCL11A produced high levels of functional hemoglobin and had reduced disease symptoms for at least eight months after transplantation.
Sanofi's sutimlimab delivers benefit for CAD patients in phase III study
Conferences
ORLANDO, Fla. – New research on sutimlimab, an investigational complement pathway inhibitor under development by Sanofi SA's Bioverativ unit, showed substantial benefits for people with the rare autoimmune disorder cold agglutinin disease (CAD).
Forty Seven’s data eat up the market, with positive results in MDS and AML
Conferences
ORLANDO, Fla. – Data from two studies from Forty Seven Inc. announced at the American Society of Hematology (ASH) annual meeting moved the market in a major way Monday as the company stock (NASDAQ:FTSV) soared 111% to close at $30.43, up $15.99.
Constellation shares fall after myelofibrosis update, despite progress
Conferences
ORLANDO, Fla. – Preliminary data from a phase II test of Constellation Pharmaceuticals Inc.'s CPI-0610 in patients with myelofibrosis (MF), previewed to market adulation in November, got a full airing Monday at the American Society of Hematology (ASH) annual meeting.

BioWorld Perspectives

A decade of incentives to promote antibiotic development and still no viable route to commercial success
It should be motoring to profitability, but nine months after the U.S. launch of its new antibiotic, Zemdri (plazomicin), Achaogen Inc. has filed for...
BioWorld's Top 10: Biggest newsmakers and trending stories of 2018
BioWorld
In many ways, 2018 resembled the previous year in fast motion. Political turmoil accelerated on both sides of the Atlantic, with Asia swept into the fold,...
Deck the halls, or get decked out: BioWorld’s 12th annual Holiday Gift Guide
BioWorld
The holidays are (already) here again. Much as we try to push back the calendar, those Thanksgiving doorbuster, Black Friday blowout, Small Business...
Price increases - tone deaf or defiant?
BioWorld
Defying the laws of nature, many drug companies seem intent on proving that what goes up doesn’t have to come down. For them, the sky’s the limit when it...
PABNAB – A disappointing distraction at BIO, not associated with BIO
BioWorld
On what planet are the PABNAB (Party at BIO not associated with BIO) organizers living to think it was acceptable to spotlight topless dancers with company...
Shoes, ‘Stones’ and surfing: BioWorld’s 12th annual Summer Reading List
BioWorld
Summer arrives with a bang for biopharma in the dueling agendas of the American Society of Clinical Oncology (ASCO) annual meeting, the BIO International...
If no one reads it, what’s the purpose of a drug label?
BioWorld
Robert Califf said it when he was serving as FDA commissioner. Patient advocates have said it when testifying before Congress or commenting at public...

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