It may be a shortened week for news, but it was headlined with Novartis AG’s $9.7 billion bet that the economics of an siRNA-based drug can better those of monoclonal antibodies and thus provide it with a dominant position in the cardiovascular drug market. The pharma is offering $85 per share for The Medicines Co., which has taken inclisiran, an siRNA-based inhibitor of proprotein convertase subtilisin/kexin type 9 (PCSK9) to the brink of an approval in reducing the risk of a cardiovascular event – heart attack or stroke – in high-risk patients with cardiovascular disease or high levels of low-density lipoprotein cholesterol who are inadequately controlled on current therapies. 

In other M&As of note, Asahi Kasei Corp. said it is putting up $1.3 billion to acquire Veloxis Pharmaceuticals A/S and bring on board Envarsus XR, an improved formulation of tacrolimus for prophylaxis of organ rejection in kidney transplant patients converted from tacrolimus and for use in de novo kidney transplant patients. Envarsus XR boasts better bioavailability and controlled, smooth delivery, which means once-daily dosing, a lower total daily dose requirement, and lower peak concentrations with less fluctuation. 

Alkermes plc completed the acquisition of Rodin Therapeutics Inc. for $100 million up front and up to $850 million in clinical, regulatory and commercial milestones. Rodin has been working to develop first-in-class, orally available, brain-permeable therapeutics for synaptopathies by designing molecules that target specific HDAC complexes. 

In other transactions:  

Asuragen Inc. is joining forces with Wave Life Sciences USA Inc. to develop companion diagnostics for Wave’s investigative allele-selective therapeutic programs targeting the underlying genetic cause of Huntington’s disease. 

Enochian Biosciences Inc. agreed in principle to acquire an exclusive license for a hepatitis B virus infections treatment being developed by G-Tech Bio LLC. No financial terms were announced.  

Ferring Pharmaceuticals and Blackstone Life Sciences will jointly invest more than $570 million in nadofaragene firadenovec (rAd-IFN/Syn3), an investigational gene therapy in late-stage development for patients with high-grade, Bacillus Calmette-Guérin unresponsive, non-muscle invasive bladder cancer.  

Companies slide on disappointing trial results 

Shares of Adamis Pharmaceuticals Corp. (NASDAQ:ADMP) tumbled 52% Monday after the FDA issued a complete response letter regarding its high-dose naloxone injection candidate, Zimhi, for the treatment of opioid overdose. The agency raised questions about chemistry, manufacturing and controls (CMC), but not the candidate's safety or efficacy, the company said. 

Cymabay Therapeutics Inc.’s shares (NASDAQ:CBAY) took a 77% hit Monday after the company announced that it was terminating its phase IIb study of PPAR-delta agonist seladelpar in subjects with non-alcoholic steatohepatitis and its recently initiated phase II study of seladelpar in subjects with primary sclerosing cholangitis.  In addition, the company is putting on hold all studies of seladelpar in subjects with primary biliary cholangitis. 

La Jolla Pharmaceutical Co. discontinued its study LJ401-BT01 due to lack of efficacy and will reassess further development of LJPC-401 (synthetic human hepcidin) based on recent mixed clinical results. The fallout concluded a rough day for the company, which saw its stock (NASDAQ:LJPC) lose more than half its value, and the departure of its president and CEO.  

Better news for Chemocentryx  

Investors rewarded Chemocentryx Inc. big time with its shares (NASDAQ:CCXI) rocketing 281% on Tuesday on positive phase III data from the Advocate trial announced by the company and Vifor Fresenius Medical Care Renal Pharma. In the global study of avacopan, an oral, selective complement 5a receptor inhibitor, in patients with anti-neutrophil cytoplasmic antibody-associated vasculitis, it hit both of its primary endpoints: disease remission at 26 weeks and sustained remission at 52 weeks as assessed by the Birmingham Vasculitis Activity Score.  

Cash flow 

Several public companies completed transactions this week: 

Sierra Oncology Inc. closed its previously announced underwritten public offering, with gross proceeds of $103 million. The company is developing momelotinib, a JAK1, JAK2 and ACVR1 inhibitor with a potentially differentiated therapeutic profile for the treatment of myelofibrosis. Onconova Therapeutics Inc. closed its public offering for gross proceeds of $11 million. Sonoma Pharmaceuticals Inc. launched a best efforts public offering. Surface Oncology Inc. secured a debt financing facility for up to $25 million from K2 Healthventures. X4 Pharmaceuticals Inc. priced a public offering with gross proceeds expected to be about $65 million. 

Venture funding: 

New Venture Funds raised its third fund, New Ventures III, with a total of $90 million. The fund has made 12 investments so far and follows in the footsteps of the two previous funds, with an investment strategy focusing on a diversified portfolio of early to midstage therapeutics, digital medicine opportunities and pharmaceutical royalties. 

Research of note 

Researchers at the Murdoch Children’s Research Institute in Melbourne are pushing the boundaries on creating kidney tissue from stem cells. For more than two decades, Melissa Little and her team at Murdoch have investigated the molecular and cell development basis of kidney disease and the potential for regeneration. The team has developed approaches for directing the differentiation of human pluripotent stem cells to human kidney organoids and is applying that knowledge to disease modeling, drug screening, cell therapy and tissue engineering. 

An estimated 6 million children have allergic asthma, making asthma one of the most common long-term diseases of childhood.  Progress in understanding the disease was reported in the Dec 17, 2019, issue of Immunity. Investigators at Massachusetts General Hospital discovered that the T-cell-specific dopamine receptor DRD4 controlled Th2 specification and associated inflammation that ultimately influenced susceptibility to allergic asthma in mouse models.   

Regulatory news 

The FDA cleared, well ahead of the scheduled Feb. 26, 2020, PDUFA date, its 41st new molecular entity - Oxbryta (voxelotor), from Global Blood Therapeutics Inc., to treat sickle cell disease in adults and pediatric patients ages 12 and up. The new medicine makes cells less likely to bind together and form the distinctive sickle shape, leading to low hemoglobin levels due to red blood cell destruction.   

An FDA draft guidance describes the implementation of revised provisions for requesting and issuing a certificate of confidentiality, which is intended to protect the privacy of human research participants. Historically, the certificate generally protected researchers from being compelled to disclose identifiable and sensitive information about research participants. The 21st Century Cures Act broadened the protections by prohibiting certificate holders from disclosing the information unless a specific exception applies. Comments on the draft should be submitted by Jan. 9.  

The Accelerating Medicines Partnership program for Parkinson’s disease (AMP PD) launched a data portal to provide de-identified information collected from more than 4,000 patients and healthy controls to researchers working to develop therapies for the disease. The portal enables researchers to study complex datasets and perform genome-wide analyses at a scale previously impossible, according to the NIH. AMP PD is a partnership between the NIH, FDA, The Michael J. Fox Foundation for Parkinson’s Research and industry participants, including Celgene Corp., Glaxosmithkline plc, Pfizer Inc., Sanofi SA and Verily Life Sciences. The goal is to transform and accelerate drug development in Parkinson’s by helping to determine which biomarkers show the greatest potential for predicting the disease and its progression. 

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

“Pardoning This Bird Ahead of Thanksgiving (and ASH): Upgrading to Outperform” 
Headline from an SVB Leerink analyst report on Bluebird Bio Inc. that explains why they are upgrading the company to outperform and reiterating a $119 PT 

“Cancer registries are urgently needed in developing countries because the cancer burden is usually poorly known.”  
Gouri Shankar Bhattacharyya, a medical oncologist at Salt Lake City Medical Centre, Kolkata, India, speaking at the ESMO Asia Congress 

“Wave is developing oligonucleotide therapies that specifically target only the expanded HTT gene by targeting nearby polymorphisms (SNPs) on the same chromosome as the expanded allele. Building on work done previously by Wave, Asuragen will be using our Amplidex technology to build a diagnostic test that can rapidly, efficiently and accurately measure HTT CAG repeat length and determine whether the expanded allele is on the same chromosome with the SNP targeted by the Wave therapeutics.” 
Bernard Andruss, senior vice president of operations and regulatory affairs at Asuragen, which is joining forces with Wave Life Sciences USA Inc. to develop companion diagnostics for Wave’s investigative allele-selective therapeutic programs targeting the underlying genetic cause of Huntington’s disease 

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