FDA Approves Genentech Xolair Prefilled Syringe Formulation

– Xolair, the only biologic approved for both allergic asthma and chronic idiopathic urticaria indications, is now also approved in a prefilled syringe (PFS) formulation –

– More than 330,000 patients have been treated with Xolair in the last 15 years –

– The 75 mg/0.5 mL and 150 mg/1 mL single-dose prefilled syringes are expected to be available by the end of 2018 –

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved 75 mg/0.5 mL and 150 mg/1 mL single-dose prefilled syringes (PFS) for Xolair^® (omalizumab) as an additional formulation for both allergic asthma and chronic idiopathic urticaria (CIU) indications. The new Xolair PFS formulation is expected to be available by the end of this year for the first time in the U.S. Xolair is currently available in a 150 mg single-dose vial with lyophilized, sterile powder for reconstitution.

“Xolair has long been an important treatment option for people with allergic asthma and CIU,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “The prefilled syringe formulation reflects our continued commitment to provide healthcare professionals with choices to best support each patient’s unique needs.”

The Xolair PFS eliminates the need for healthcare providers to procure Sterile Water for Injection (SWFI) and reconstitute Xolair before administering the medicine.

Xolair is approved for the treatment of moderate to severe persistent allergic asthma in people six years of age or older whose asthma symptoms are not controlled by inhaled corticosteroids, and for CIU in people 12 years of age and older who continue to have hives that are not controlled by H1 antihistamines. Over 330,000 people in the U.S. have been treated with Xolair since its initial approval for people 12 years and older with allergic asthma in 2003.

https://www.businesswire.com/news/home/20180928005624/en/FDA-Approves-Genentechs-Xolair-Omalizumab-Prefilled-Syringe

Insmed shares halted pending news

Nasdaq has suspended trading in Insmed (NASDAQ:INSM) pending the release of news, a near certainty that it is related to the pending FDA approval for ALIS for nontuberculous mycobacteria (NTM) lung disease.

https://seekingalpha.com/news/3393867-insmed-shares-halted-pending-news

FDA OKs Regeneron and Sanofi’s Libtayo for type of skin cancer

The FDA approves Regeneron Pharmaceuticals (NASDAQ:REGN) and Sanofi’s (NYSE:SNY) PD-1 inhibitor Libtayo (cemiplimab-rwlc) for the treatment of patients with metastatic cutaneous squamous cell carcinoma, the second most common type of skin cancer.

The U.S. list price will be $9,100 per three-week cycle.

https://seekingalpha.com/news/3393864-fda-oks-regeneron-sanofis-libtayo-type-skin-cancer

American Shared Hospital Services Won’t Stay Cheap For Long?

American Shared Hospital Services leases radiosurgery and radiotherapy equipment to cancer centers.

It’s a very predictable, high margin business that will likely see significant growth in the coming years.

At under 10x EV/EBIT, the stock offers a very attractive risk/reward opportunity with 60+% upside potential over the next 12-24 months.

With the market hitting new highs almost weekly, finding cheap stocks is like looking for a shrinking needle in a large haystack. You have to sort through hundreds of stocks just to maybe find a couple worth investing in. One of these increasingly rare finds is a little company called American Shared Hospital Services (AMS).

Business

AMS provides state-of-the-art radiosurgery and radiotherapy equipment to medical centers. It does so by financing the equipment and then entering into 10-year contractual agreements with each medical center. The medical center operates the equipment and pays AMS a fee, either on a fixed per use or revenue sharing basis.

The Gamma Knife, a precise, noninvasive tool that applies radiation beams for tumor treatment, accounts for just over 70% of revenue. Most of the remaining revenue comes from Proton Beam Radiation Therapy, or PBRT for short, which is similar to the Gamma Knife except that it uses proton rather than photon radiation.

The Gamma Knife business is well-established with minimal growth potential. The PBRT business, on the other hand, has only been around since early 2016 and is growing rapidly. AMS currently has one PBRT system in operation. Management plans to launch two more systems within the next couple of years, which have the potential to be significant growth drivers.

Growth

AMS’ revenue has been hovering in the $15 million to $20 million range for over a decade. Despite being very profitable during these years, this lack of growth is likely a big reason why the company’s stock has stayed off of most investors’ radars. However, I expect this to change going forward, and the PBRT system will be the catalyst.

https://seekingalpha.com/article/4208326-american-shared-hospital-services-stay-cheap-long-shares-offer-60-plus-percent-upside

60% of pharma companies using or trying blockchain – survey

Six out of ten pharma companies are using or experimenting with blockchain, according to a new study.

According to the not-for-profit organisation The Pistoia Alliance, 60% of pharmaceutical and life science professionals are either using or experimenting with blockchain today, compared to 22% when asked in 2017.

However, 40% are not currently looking at implementing, or have no plans to implement blockchain, according to the survey of 170 senior pharma and life science professionals this year.

The biggest barriers identified to adoption are access to skilled blockchain personnel (55%), and that blockchain is too difficult to understand (16%).

These factors underline why The Pistoia Alliance is calling for the life science and pharmaceutical industries to collaborate over the development and implementation of blockchain.

Blockchain is an open, distributed ledger of information that is saved across several different servers, and is constantly growing as computers cryptographically discover the next “block” of information in the chain.

Data is protected in any given block as they cannot be altered retroactively without alteration of blocks, requiring consensus of the majority of the network.

Famously employed to administer the bitcoin cryptocurrency, blockchain can be used to create a secure repository for sensitive healthcare information such as clinical trial data.

The survey showed life science and pharmaceutical professionals are becoming more aware of the capabilities of blockchain.

Respondents believed the greatest opportunities for using blockchain lie in the medical supply chain (30%), electronic medical records (25%), clinical trials management (20%), and scientific data sharing (15%).

Of the benefits of blockchain, life science and pharmaceutical professionals believe the most significant is the immutability of data (73%). Significantly, for an industry with tight regulations, 39% also believe the transparency of the blockchain system is its best feature.

However, almost a fifth (18%) of professionals believe using blockchain adds no value beyond a traditional database, showing there is some reluctance in the industry to use the technology.

The Pistoia Alliance said that some of the misconceptions about blockchain can be overcome with greater education of those in industry.

Richard Shute, consultant for The Pistoia Alliance, said “We are currently focusing on educating scientists and researchers about the potential uses of blockchain technologies outside of the supply chain, particularly in R&D. At The Pistoia Alliance, we want to support our members’ initiatives in blockchain, as well as provide a secure global forum for partnerships and collaboration.”

• The Pistoia Alliance is holding a Blockchain Bootcamp on 8^th – 9^th October in Boston as part of its drive to educate the life science industry about the technology. For more information on the event and to register, see here.

https://pharmaphorum.com/news/60-of-pharma-companies-using-or-trying-blockchain-survey/

Alnylam rare disease drug may be delayed

Alnylam may have to put hold off plans for an early FDA approval for its gene-silencing drug givosiran – because recruitment to a rare disease trial is going better than expected.

Rare disease trials are notoriously difficult to conduct because of a paucity of available patients, but Alnylam said that the trial of givosiran in acute hepatic porphyria has attracted more participants than expected.

The firm had hoped to seek an accelerated approval from the FDA on early data before the trial completes – but because of the fast pace of enrolment, Alnylam said the regulator may choose to wait for full results.

Citing a company conference call, Reuters reported that this would push a filing back by four to six months.

“It is possible the FDA could ask [the] company to wait another 6 months until the full data is released,” an unnamed executive said.

Another concern could be safety – there were no deaths on the trial, but 22% of those on the drug reported serious side effects, compared with 10% of patients given a placebo.

There are no approved treatments for acute hepatic porphyria and the Cambridge, Massachusetts-based biotech reckons about 1,000 patients have a severe form of the disease, without around 5,000 suffering less frequent attacks.

Interim data revealed this week showed patients with the condition that were given givosiran had a significant reduction in aminolevulinic acid (ALA) protein in their urine.

High levels of ALA are considered to trigger an increase in the “attacks” experienced by patients.

There were no other details about efficacy, or further data on the drug’s safety, according to Reuters.

In August, Alnylam’s Onpattro (patisiran) became the first ever RNA interference drug approved by the FDA to treat the nerve damage caused by the rare disease hereditary transthyretin-mediated amyloidosis (hATTR) in adults.

The drug’s $450,000 per year price tag is expected to be mitigated by discounts and “value-based” deals with insurers.

Alnylam beat competition from biotech Ionis to get the approval, which is developing a rival hATTR drug, Tegsedi (inotersen).

https://pharmaphorum.com/news/alnylam-rare-disease-drug-may-be-delayed/

SEC charges Stryker for second time for FCPA violations

The Securities and Exchange Commission charged Stryker Corp. with violating the books and records and internal accounting controls provisions of the Foreign Corrupt Practices Act, or FCPA, with the regulator stating that this is the second time the SEC has brought an FCPA action against the medical device company. Stryker agreed to settle the charges and pay a $7.8M penalty. The SEC’s order found that Stryker’s internal accounting controls were not sufficient to detect the risk of improper payments in sales of Stryker products in India, China, and Kuwait, and that Stryker’s India subsidiary failed to maintain complete and accurate books and records.

https://thefly.com/landingPageNews.php?id=2797201