The world’s biggest hematology gathering, the 61st American Society of Hematology (ASH) conference, ended in Orlando Tuesday, having brought > 30,000 people from 25 countries to the event. The amount of research covered was staggering, with 5,978 abstracts available for review. Key themes included work aimed at overcoming obstacles to CAR T therapy, new progress in preventing and treating venous thromboembolism, moves to address health care disparities and new developments in the care of sickle cell diseases. Late-breakers highlighted new data on Blincyto (blinatumomab, Amgen Inc.), Sanofi SA's sutimlimab, azacitidine and Darzalex (daratumumab, Janssen Biotech Inc.).

Overall, investors and analysts alike were impressed with what they heard. The BioWorld Cancer index pushed 17.6% higher in November, and, by market close Tuesday, it was tracking up 4% in December, helped by the performance of those index members presenting at the ASH meeting.

Public performance

The BioWorld Neurological Diseases index, a price-weighted index of public biopharmaceutical companies that are focused on developing therapies to treat neurological diseases, have been on a roll climbing 22% during the past two months. However, a surprising trial failure reported by Sage Therapeutics Inc. last week served to eat into those positive index gains.

Alphamab Oncology Co. Ltd. (HK:9966), which aims to bring the first PD-L1/CTLA4 bispecific antibody to China, saw its share prices jump almost 30% on its debut on the Hong Kong Stock Exchange (HKEX), ending the year on a positive note for the pre-revenue biotech. Shares of Equillium Inc. (NASDAQ:EQ) jumped 17% as Wall Street hailed the FDA’s granting of fast track status to itolizumab – the first clinical-stage anti-CD6 therapy – for the treatment of lupus nephritis (LN).

Cash flows

No shortage of public offerings this week as companies took advantage of accommodating markets with leading deals by money raised including: Constellation Pharmaceuticals Inc. raised gross proceeds of $224.3 million; Aurinia Pharmaceuticals Inc. hauled in approximately $191.7 million; Relmada Therapeutics Inc. generated $115 million; and Rocket Pharmaceuticals Inc. closed on gross proceeds of $84.99 million.

Three new funds rolled out, with Omega Funds closing on $438 million, Perceptive Advisors announcing a new series A-focused $210 million Perceptive Xontogeny Venture Fund, and a med-tech-focused $115 million fund from Israel's Peregrine Ventures was reported.

Pharmas pre-holiday shopping spree

Big pharma companies went on a pre-holiday shopping spree this week shelling out almost $7 billion to acquire companies and assets.

Sanofi SA is paying a 172% premium to acquire Synthorx Inc. for $68 per share, or $2.5 billion in all, to add a clinical-stage immuno-oncology asset to its pipeline and a platform technology that will enable it to optimize cytokine-based drugs for cancer and autoimmune disease. Merck & Co. Inc. is paying about $2.7 billion in cash for Arqule Inc. that has a phase II dose-expansion study ongoing with Bruton tyrosine kinase (BTK) inhibitor ARQ-531 for B-cell malignancies. Janssen Biotech Inc. is buying Xbiotech Inc.’s interleukin-1a (IL-1a) inhibitor bermekimab for $750 million up front and will pay a further $600 million in potential milestones as the product advances in development for the treatment of atopic dermatitis and the chronic immune-mediated skin disease, hidradenitis suppurativa. In addition, the Johnson & Johnson company will pay Xbiotech for clinical trial services and to manufacture bermekimab to complete two ongoing phase II studies.

Also, this week, Abbvie Inc. and Scripps Research said they will collaborate to develop therapies for a range of diseases, including indications in oncology, immunology, neurology and fibrosis. Under terms of the license agreement, Scripps Research will conduct preclinical R&D and, in some cases, phase I trials, with Abbvie holding an exclusive option to conduct additional development and commercialization. Engitix Ltd., which is developing a portfolio of programs in fibrosis and solid tumors using its human extracellular matrix (ECM) platform, and Morphic Therapeutic Inc. said they entered a research collaboration to identify ECM-related drug targets in fibrostenotic inflammatory bowel disease, an intestinal fibrosis complication that affects approximately 30% of Crohn’s disease patients.

Regulatory news

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.

Correvio Pharma Corp. said it is seeking strategic alternatives that include an acquisition, merger, business combination or other transaction regarding the company or its assets. In a Dec. 10 advisory committee meeting, the FDA’s Cardiovascular and Renal Drugs Advisory Committee, having reviewed the company’s NDA, decided the benefit-risk profile of Brinavess (vernakalant I.V.) for atrial fibrillation conversion, was inadequate to support approval.


Word on the Street: BioWorld's favorite quotes of the week

“The Wright brothers showed that you could fly a plane, but it wasn’t very far, and it wasn’t very safe. That’s where cell therapy is now.”
At a scientific session at the American Society of Hematology meeting on gene editing, Wendell Lim, professor and chair of cellular and molecular pharmacology at the University of California, San Francisco, and the co-founder of Cell Design Labs, discussed his laboratory’s work to use gene editing to improve cell therapies, in particular CAR T cells

[This is] "the most exciting time to be investing in life sciences in a generation."
Otello Stampacchia, managing director, Omega Funds that raised $438 million to deploy into new and existing life sciences companies

“We made a semiconductor chip that mimics the behavior of ion channels of hippocampal and respiratory neurons. The chip itself is scaled to a few tens of microns in size. Our work is paradigm changing because it provides a robust method to reproduce the electrical properties of real neurons in minute detail.”
Alain Nogaret, professor of physics at Bath University, who led the research

“If you look around, the antibiotic space is not flavor of the month, which is interesting given all the press coverage of a looming AMR [antimicrobial resistance] apocalypse. The stock prices of most listed antibiotics companies in the U.S. have been hammered.”
Glyn Edwards, CEO in Summit Therapeutics plc, that intends to cancel trading on the Alternative Investment Market (AIM) in London, but it will still retain its Nasdaq listing

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