UCB Parkinson’s treatment OKd in China

• NEUPRO now approved in China for the treatment of the signs and symptoms of early-stage idiopathic Parkinson’s Disease as monotherapy (i.e. without levodopa) or in combination with levodopa
• NEUPRO approval in China reinforces UCB’s ongoing commitment to supporting patient value across China and to offering Chinese patients additional treatment choice to help manage their disease

Belgium-based global bio-pharmaceutical company UCB today announced it has received an Import Drug License (IDL) from the China Food and Drug Administration (CFDA), creating a pathway to make the company’s 24-hour continuous delivery transdermal rotigotine patch NEUPRO^® available to patients in China.

Parkinson’s disease (PD) is a chronic, degenerative neurological disease with an estimated prevalence of 1.7% in China in people aged 65 years and older.¹ Estimates suggest that around 3 million people in China are affected by the condition.²

With this IDL, NEUPRO is now indicated in China for the treatment of the signs and symptoms of early-stage idiopathic Parkinson’s Disease as monotherapy (i.e. without levodopa) or in combination with levodopa, i.e. over the course of the disease, through to late stages when the effect of levodopa wears off or becomes inconsistent and fluctuations of the therapeutic effect occur (end of dose or ‘on-off’ fluctuations).

NEUPRO is designed to support and improve individual patient treatment experiences for people living with Parkinson’s Disease. The product’s unique transdermal patch formulation is administered once daily, providing a continuous release of rotigotine over a 24-hour period.

‘The availability of NEUPRO^® in China is testament to UCB fulfilling our commitment to provide additional value to patients around the world.’ said Jeff Wren, Executive Vice-President, Head of UCB’s Neurology Patient Value Unit. ‘We know the significant impact Parkinson’s can have on the lives of patients and their family members, and how important it is to effectively manage symptoms to allow patients to keep their independence and maintain their quality of life. With the approval of NEUPRO^® in China, we are very excited that we will be able to make this medicine available to the millions of people affected by Parkinson’s Disease in the country, providing them with a convenient treatment option to help them to manage their condition.’

As a result of the IDL, UCB will now progress with NEUPRO launch planning, to ensure patients in China with Parkinson’s are able to benefit from this approval as quickly as possible.

UCB has been present in China since 1996 and has a prominent presence in the country, having launched Keppra for the treatment of some forms of epilepsy in 2007. This important landmark helped established the company’s reputation as a leader in neurology. Additionally, in 2014, UCB inaugurated a new state of the art 13,000 m² manufacturing site in Zhuhai, which strengthened our footprint in the country.

Alongside approval for NEUPRO, these activities reinforce UCB’s ongoing commitment to supporting patients with severe diseases in China.

https://bit.ly/2KGluRV

Valneva Announces Significant Progress of its Lyme Disease Vaccine Candidate

Valneva SE (“Valneva” or “the Company”), a fully integrated, commercial stage biotech company focused on developing innovative lifesaving vaccines, announced today a further update for its Lyme disease vaccine candidate, VLA15. The Company has successfully concluded the end of Phase 1 process for this candidate with the Food and Drug Administration (FDA) and has obtained alignment with regard to its Phase 2 strategy.

Valneva is now finalizing the detailed Phase 2 protocol and, subject to requisite regulatory approvals, expects to enter Phase 2 clinical development by the end of 2018.

Wolfgang Bender, MD, PhD, Chief Medical Officer of Valneva commented “We appreciate the valuable and constructive guidance the FDA has provided and are looking forward to progressing into Phase 2. Lyme is a vaccine preventable disease. We are strongly committed to advancing our Lyme disease vaccine candidate as quickly as possible with the aim of preventing this severe and often debilitating infection.”

The Company will provide a full update on its Lyme program during the publication of its H1 results on August 2^nd, 2018.

https://bit.ly/2NmmKZ4

Merck, AstraZeneca wins speedy approvals for cancer drugs in Japan

AstraZeneca has won rapid regulatory approval for new uses of two of its important cancer drugs in Japan, less than six months after the first global approvals in Western markets.

The decisions by the Japanese Ministry of Health, Labour and Welfare reflect an increased urgency by officials in the country to access modern medicines, after many years of slow adoption.

AstraZeneca said on Monday that its Imfinzi immunotherapy drug had been approved for use in lung cancer patients with inoperable disease that had advanced locally but not spread widely around the body.

Imfinzi was only given a U.S. green light for treating such stage III lung cancer in February and it is still awaiting approval in the European Union.

Japan also approved Lynparza, which AstraZeneca markets with Merck & Co, for breast cancer.

https://bit.ly/2z03DAR

Old diabetes drug metformin may find yet another role: reversing lung fibrosis

The drug metformin has been used to control blood sugar in many patients with Type 2 diabetes for more than two decades. Now scientists at the University of Alabama, Birmingham, have amassed evidence that the drug’s ability to target cell metabolism could help repair the lung.

The team is investigating therapies for pulmonary fibrosis, a condition that can occur after lung injuries from infections or treatments like chemotherapy. They are particularly interested in finding effective treatments for idiopathic pulmonary fibrosis (IPF), a progressive, fatal disease that can strike out of the blue and affects more than 5 million patients worldwide.

The scientists zeroed in on cells called myofibroblasts, which are responsible for depositing the damaging collagen that proliferates in lung fibrosis. They found that the activity of an enzyme called AMP-activated protein kinase (AMPK) was suppressed in fibrotic areas of tissue samples taken from IPF patients. The metabolisms of those cells went into overdrive, making them less likely to undergo apoptosis, or programmed cell death.

Metformin activates AMPK. In experiments with the tissue samples and lung fibroblasts from mice, the researchers found that metformin reversed lung fibrosis. So they decided to try the diabetes drug in mouse models of lung fibrosis. They started the treatment three weeks after the lungs were damaged—making the lung fibrosis well established—and continued it for the next 5 weeks. That accelerated lung repair.

To confirm their suspicion that AMPK was the key to metformin’s activity in myofibroblasts, they tried the same experiment in mice that lacked the enzyme. Lung fibrosis did not improve in those animals. They published their research in the journal Nature Medicine.

Metformin has long been of interest in the oncology community because observational studies have shown that diabetics who take the drug have a lower risk of cancer than those who don’t. The drug’s effect on AMPK seems to play a role there. Scientists at the University of California, San Diego, discovered that metformin activates a protein pathway that prompts AMPK to build barriers in the body against inflammation and other cancer-causing stressors, for example.

Scientists are also studying the potential of metformin in treating other conditions, including autismand disorders of aging like heart disease.

The University of Alabama researchers believe their study shows that AMPK is a “critical metabolic switch” that shifts the metabolism of lung cells so they can resolve fibrosis, they wrote in their study. “Additionally, we provide proof-of-concept that activation of AMPK by metformin or other pharmacologic agents that activate these pro-resolution pathways may be a useful therapeutic strategy for progressive fibrotic disorders.”

https://bit.ly/2KtnPjO

Henry Schein creates key software joint venture with Internet Brands

Henry Schein Inc. and partner Internet Brands have completed the creation of a joint venture to create and market software for the global dental market, the companies announced Monday.

Analysts have said that practice-management software is crucial in cementing Henry Schein’s supplier relationship with dentists as online providers such as Seattle-based Amazon.com enter the market.

The joint venture, named Henry Schein One, incorporates Henry Schein’s practice-management software for dentists, including Dentrix and Easy Dental, with Internet Brands’ web-based programs such as Demandforce and DentalPlans.com.

Melville-based Henry Schein, which distributes products to clinics and the offices of dentists, physicians and veterinarians, owns more than 70  percent of the joint venture, which is based in American Fork, Utah.

Stanley M. Bergman, chairman and chief executive of Henry Schein, said in an interview Friday that the company began offering dentists Henry Schein branded software in the early 1990s.

That business has grown in importance, he said, with the proliferation of electronic medical records and the need for “interoperability” on computers and mobile devices.

“We want to become the iPad for the dentist,” he said.

James A. Harding, who formerly served as the chief technology officer of Henry Schein, is the joint venture’s chief executive.

If the joint venture had existed in 2017, it would have had sales of about $400 million, with three quarters attributed to Henry Schein.

El Segundo, California-based Internet Brands runs consumer websites in the areas of health, automotive, legal and home and travel, including WebMD and Fodor’s Travel.

Internet Brands is owned by private equity firms KKR & Co., based in Manhattan, and Temasek, based in Singapore.

https://nwsdy.li/2KBT0J4

Novo Nordisk upped to buy by Bernstein

Novo Nordisk upgraded to Outperform from Market Perform at Bernstein

https://bit.ly/2lRfwzE

Record Year So Far for Massachusetts Biotech IPOs

It’s been a pretty good year for mergers and acquisitions in the biopharma industry, with Takeda Pharmaceutical buying Shire for $62.2 billion, and the year starting with Sanofi buying Bioverativfor $11.6 billion and then Ablynx for $4.8 billion. Celgene acquired Juno Therapeutics for $9 billion, shortly after buying Impact Biomedicines for $1.1 billion.

But the landscape for initial public offerings (IPOs) has been excellent as well, particularly in Massachusetts, which has recorded 13 IPOs since the beginning of the year, raising a combined $1.3 billion. This exceeds by money raised the record in 2014, when 17 Massachusetts biotechs raised $1.2 billion, and the year’s only half over. And there are three biotech companies in Massachusetts that have filed to go public, but hadn’t done so yet. One of those includes Rubius Therapeutics.

The list of Massachusetts biotech companies with IPOs so far this year is listed below.

#1. Cue BioPharma. Headquartered in Cambridge, Mass., Cue focuses on injectable biologics engineered to modulate the immune system to treat cancer, chronic infectious diseases and autoimmune disorders. Its IPO on January 2 raised $66 million.

#2. Unum Therapeutics. Also based in Cambridge, Unum is also working in the immuno-oncology space, developing therapies based on an antibody-coupled T cell receptor. It is differentiated from CAR-T or TCR by not being restricted to a particular target and may have applications in a broad range of cancer types. The company’s IPO raised $69 million on March 29.

#3. Scholar Rock. Of course, based in Cambridge, Scholar Rock is focused on the biology of growth factor activation, targeting growth factors in the disease microenvironment. This has applications in neuromuscular disorders, cancer, fibrosis, and anemia. It raised $75 million on May 24.

#4. RestorBio. Headquartered in Boston, RestorBio is focusing on aging-related diseases. Its lead program, RTB101, focuses on the mechanistic target of rapamycin (mTOR) pathway, an evolutionarily conserved pathway that regulates aging. On January 26, the company raised $85 million.

#5. Evelo Biosciences. Located in Cambridge, Evelo Biosciences focuses on monoclonal microbials for oncology and inflammatory diseases. Its lead product is EDP1066, currently in a clinical trial in inflammatory diseases, such as psoriasis, atopic dermatitis, and ulcerative colitis/Crohn’s colitis. On May 9, the company raised $85 million.

#6. AvroBio. Headquartered in Cambridge, AvroBio is focused on lentiviral-based gene therapies for rare diseases. Its lead gene therapy is AVR-RD-01 for Fabry disease. It also has three more gene therapies for other lysosomal storage disorders. On June 21 it raised $99 million.

#7. Neon Therapeutics. Located in—you guessed it—Cambridge, Neon is working on directing the immune system using neoantigens to treat cancer. Neoantigens are specific genetic mutations that are common in cancer. The company’s June 27 IPO raised $100 million.

#8. Magenta Therapeutics. Magenta is based in Cambridge and is developing therapeutics for bone marrow transplant for patients with autoimmune diseases, blood cancers and genetic diseases. The company’s most advanced clinical product is MGTA-456, a cell therapy in a Phase II clinical trial in patients with inherited metabolic diseases. Magenta raised $100 million on June 21.

#9. Surface Oncology. Based in Cambridge, Surface Oncology, not surprisingly given its name, focuses on cancer. The differentiating factor is its focus on the tumor microenvironment. On June 12, it initiated a Phase I clinical trial of SRF373, a fully human antibody targeting CD73, the second of its immunotherapies to advance into this clinic this year. On April 19, its IPO raised $108 million.

#10. Translate Bio. Headquartered in Lexington, Mass., Translate focuses on messenger RNA (mRNA) to treat diseases caused by protein or gene dysfunction. On June 11, it signed a dealwith Sanofi Pasteur to develop up to five undisclosed infectious disease pathogens. The company raised $122 million on June 28.

#11. Solid Biosciences. Based in Cambridge, Solid is focused on a gene therapy for Duchenne muscular dystrophy (DMD). On June 18, the U.S. Food and Drug Administration (FDA) lifted a clinical hold on the company’s IGNITE DMD Phase I/II clinical trial of SGT-001 for DMD, acknowledging the company had addressed all the questions the agency had in its clinical hold. The company raised $125 million on January 26.

#12. Homology Medicines. Headquartered in Bedford, Mass., Homology is a genetic medicines company focused on rare genetic diseases. Its proprietary platform utilizes its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines either through a gene therapy or nuclease-free gene editing modality for a variety of genetic disorders. Its IPO on March 28 raised $144 million.

#13. Kiniksa Pharmaceuticals. Kiniksa has headquarters in Hamilton, Bermuda and Lexington, Mass. The company’s pipeline includes rilonacept for recurrent pericarditis, mavrilimumab for giant cell arteritis, KPL-716 for prurigo nodularis/atopic dermatitis, KPL-045 for T-cell dependent and B-cell-mediated indications, and KPL-404 for the same as KPL-045. On May 24, its IPO raised $153 million.

https://bit.ly/2IM2egH