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Karl Storz Has New Non-Muscle Invasive Bladder Cancer Detection System

 KARL STORZ Endoscopy-America, Inc., announces the commercial launch of the PDD Blue Light Flexible Video Cystoscopy System for enhanced detection of non-muscle invasive bladder cancer (NMIBC). This announcement follows the approval of a supplemental new drug application and a Premarket Approval supplement from the U.S. Food & Drug Administration (FDA) extending the indication for Blue Light Cystoscopy with Cysview® (BLCC) to include use of the new KARL STORZ PDD Blue Light Flexible Video Cystoscope. Also included in the approval is an expanded indication for the repetitive use of Cysview within the same patient and for the identification of Carcinoma in Situ (CIS); one of the most challenging types of bladder cancers to detect. These new indications greatly increase the treatment possibilities of this innovative therapy, which has rapidly become the standard of care for bladder cancer treatment at major medical institutions across the United States.

KARL STORZ PDD Blue Light Flexible Video Cystoscopy completes the 2-part system, which also includes ... KARL STORZ PDD Blue Light Flexible Video Cystoscopy completes the 2-part system, which also includes Rigid Blue Light Cystoscopy. As shown above, Blue Light Cystoscopy with Cysview® enables cancerous tumors to fluoresce in a bright pink color, improving tumor visibility and enhancing florescence guided resection. (Graphic: Business Wire)

Bladder Cancer affects over 708,444 patients in the US. Treatment is difficult because NMIBC tumors can look similar to normal healthy tissue, and can be missed or incompletely removed. Unfortunately, patients have a high probability of cancer recurrence, and in 2018 it is estimated that there will be 17,240 patient deaths due to bladder cancer1KARL STORZ PDD Blue Light Flexible Video Cystoscopy completes the 2-part system, which also includes Rigid Blue Light Cystoscopy. The flexible and rigid PDD Blue Light Cystoscopy systems are designed to improve the visualization and resection of deadly NMIBC tumors. The new flexible system expands upon the rigid platform, enabling comfortable, anesthesia free examinations to confirm suspected lesions from a previous cystoscopy and for ongoing monitoring of NMIBC when used in conjunction with Cysview® an optical imaging agent manufactured and distributed in the U.S. by Photocure. Blue Light Cystoscopy with Cysview® enables cancerous tumors to fluoresce in a bright pink color improving tumor visibility and enhancing florescence guided resection with the KARL STORZ UH 400 electrosurgical system.

The FDA approval of KARL STORZ PDD Blue Light Flexible Cystoscopy with Cysview® (BLFCC) is based on results from a large Phase III study using the KARL STORZ blue-light-enabled rigid and flexible cystoscopes and blue light video system. During the Phase III clinical trial for Blue Light Flexible Cystoscopy, the following significant clinical findings were documented:2

  • BLFCC significantly improves the detection of patients with recurrence of bladder cancer (20.6%, p<0.0001).
  • BLCC significantly improves the detection of patients who recurred with CIS (34.6%, p<0.0001).
  • Repeat use of BLCC improves tumor detection in 46.2% of the patients and is safe.

“Use of the KARL STORZ PDD system for Blue Light Cystoscopy with Cysview® procedures continues to offer vital new capabilities to physicians,” says John Martineau Director Marketing Urology, KARL STORZ Endoscopy-America, Inc. “It is also the only FDA-approved technology shown to improve detection of NMIBC tumors, leading to improved and more comprehensive florescence guided tumor resection. In the 2016 Guidelines for the management of NMIBC, the American Urological Association and Society for Urologic Oncology included Blue Light Cystoscopy as recommended for increasing the detection and reducing recurrence of NMIBC. We are certain that use of Blue Light Cystoscopy with Cysview® will be positioned to provide enhanced diagnostic and treatment capabilities to physicians and support improved outcomes for patients.”

“This expanded approval in both rigid and flexible blue light cystoscopy (BLC) means that Cysview® can now be used during transurethral resection of bladder cancer surgery for diagnosis and staging, as well as with follow-up surveillance of NMIBC. Patients with NMIBC, especially high grade, require careful and frequent follow-up due to the high rate of recurrence and progression. Blue light cystoscopy with Cysview®, will enable physicians to provide appropriate and more accurate treatment earlier, which in my experience results in improved outcomes for my patients. In my high-risk NMIBC clinical practice, I recognize the benefit of using BLC with Cysview® to more readily detect carcinoma in-situ, i.e. aggressive high-grade flat lesions. In this study, an additional 35% of CIS patients were found by using BLC with Cysview®alone and missed with white light,” says Gary Steinberg, M.D., The Bruce and Beth White Family Professor, Vice Chairman and Director of Urologic Oncology, University of Chicago Medicine.

https://bit.ly/2rZLKLJ

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Uniqure Notches Bullish Initiation On Gene Therapy Prospects

Uniqure NV QURE 1.56% has managed to win the seal of approval from Wells Fargo Securities due to its gene therapy pipeline candidates that target multiple diseases.

The Analyst

Analyst Jim Birchenough initiated coverage of Uniqure with an Outperform rating and $47 price target.

The Thesis

Uniqure’s dominant gene therapy platform with an AAVS vector; its potential “best-in-class” lead gene therapy candidate AMT-061 for treating hemophilia B; and pipeline optionality for treating Huntington’s and genetically driven heart disease create a positive outlook, Birchenough said in the initiation note.

AMT-061 is in a Phase 3 trial for hemophilia B. The analyst expects a positive update on the candidate by the end of 2018 following dose confirmation and a Phase 3 enrollment update.

AMT-130, the company’s second gene therapy candidate, is being tested for Huntington’s disease, with preclinical data supporting extensive Huntington gene silencing in all regions of the brain, Birchenough said. Huntington’s disease is a $20-billion-plus opportunity, and AMT-130 is complementary to other gene-silencing approaches, he said.

Ionis Pharmaceuticals Inc IONS 1.85% and Roche Holdings AG Basel ADR Common Stock RHHBY 0.07% combined investigational gene therapy candidate IONIS-HTTRx was recently found to be effective in an ongoing Phase 1/2 clinical trial.

Uniqure is collaborating with Bristol-Myers Squibb Co BMY 0.06% for cardiovascular disease indications through its AMT-126, which is in preclinical development. Wells Fargo expects data by the end of the year that could support subsequent clinical development.

The relatively low expectations for this indication provide a favorable risk-reward, Birchenoughsaid.

https://bit.ly/2s013Uy

Dynavax hit on melanoma combo data

Dynavax (DVAX -13.4%) is down on increased volume in early trade in apparent response to the release of updated results from its ongoing Phase 1/2 study of SD-101 + Merck’s Keytruda (pembrolizumab) in PD-1-naive advanced melanoma patients.

In 25 subjects, the overall response rate was 60% (n=15/25). Preliminary data presented last year at ASCO (seven patients) showed a 100% response rate.

Previously: Combination of Dynavax’s SD-101 and Keytruda show 100% response in melanoma study; shares ahead 4% after hours (June 2, 2017)

#ASCO18

https://bit.ly/2wZwqV1

Bayer: Disappointing Verdict on Seed Treatment Restrictions

Bayer, which makes and markets imidacloprid and clothianidin, said it is disappointed with the verdict of the General Court of the European Union’s on Case T-429/13 Bayer CropScience v European Commission.

The Court has ruled that the European Commission’s decision from 2013, which restricted the use of certain neonicotinoids, was lawful. “Bayer will review the verdict in detail and assess its consequences and potential legal options,” the company said in a statement.

It continued: “Bayer decided to pursue legal action to gain clarity on the legal basis of the Commission’s decision, which – in Bayer’s opinion – was uncertain. Bayer remains convinced of the safety of its products when applied in accordance with the label instructions.”

The company’s statement said that the loss of neonicotinoid seed treatments “will have a huge impact on European sugar beet growers.”

https://bit.ly/2rVfxWO

Best Ways to Store Your Pet’s Food

Quick: Where do you store your pet’s food? Is it sitting in an open bag on the floor, perhaps? We pet people spend a lot of time, energy, and money selecting and buying our furry friends’ food, but where and how to store it becomes an afterthought.

To find out the best possible way to keep food fresh and your pets safe I talked with Dr. Jerry Klein, an emergency and critical care veterinarian in Chicago and chief veterinary officer for the American Kennel Club. Here’s what he had to say.

Storing Dry Pet Food

The most important rules of dry food is get it off the floor and seal it. Bags left on the floor and unsealed are not only tempting to vermin (eww) but also to your pet, particularly dogs. “As an emergency vet for more than 30 years we’ve seen many cases where dogs get into an open bag of food and get bloat,” Dr. Klein says. That’s scary stuff involving too much gas in the tummy, which enlarges the stomach and makes it hard for the dog to breathe. So seal the bag, pick it up off the floor, and, better still, place it inside another container.

Why not just pour it out into another container altogether? Well, you can, the vet says, but it turns out the lining of those bags may be designed to retain important fat content as well as flavor. Plastic containers may even change the flavor for Fido or Fluffy’s kibble, although, to be honest, he says, that’s how he stores his dogs’ food. A stainless steel container, on the other hand, would retain nutrients without altering flavor. (I use vintage glass apothecary jars to store the kibble that supplements my dogs’ homemade diet, and that storage method got the OK from the vet.)

Lastly, keep the food in a temperature-controlled environment, says Dr. Klein, and check the use-by date found on the bag, usually located near the SKU code. When in doubt? It’s best to toss it. Better safe than sorry.

Storing Canned Pet Food

Like dry food, canned pet food comes with a use-by date, so always check that, Dr. Klein says. Once you’ve opened it, store it in the fridge for no more than five to seven days, and be sure it’s sealed with a plastic lid – or the old plastic-wrap-plus-rubber-band trick.

 

Let’s say you’ve served it and your pup or kitty is being picky. Dispose of it after four hours at room temp and wash the bowl.

Storing Raw Pet Food

If you’ve joined the raw food movement, you’re going to treat your pet’s stuff just like human food, Dr. Klein says. Note: The practice of feeding your pet raw food is controversial with passionate advocates for it and others who hotly dispute it. Dr. Klein doesn’t say “yay or nay,” adding that we’re talking about a lot of pathogenic bacteria, like salmonella, listeria, and E. coli.

The concern for many vets, it seems, is more related to the health of the person handling the raw meats and their family, particularly if there are children or immuno-compromised members of the family.

So to be safe, store all raw meats in secure packaging in the fridge or freezer. If you refrigerate the food, it has to be consistently below 40°F, Dr. Klein says. Should there be a power outage or issue that disrupts refrigeration, discard the meat — even if it smells and looks OK, you can’t go by that, he says. If you’re buying the raw products sold in the freezer section of pet food stores, you still want to follow the same good practices for handling raw meat. Wash your hands and any tools or surfaces that come into contact with the meat.

Storing Homemade Pet Food

Homemade food also gets the same treatment as people food — after all, that’s what it is. Store it in an airtight container in the fridge for up to three to five days. Dr. Klein adds that if you’re going the “people food” route, be sure to discuss it with your own vet.

https://bit.ly/2EvQxgi

British PM May calls on health, tech sectors to work on cancer

Prime Minister Theresa May will call on Britain’s health service, charities and artificial intelligence sector to work together to better identify patients with the early stages of cancer and stop thousands dying each year.

May, who is struggling to unite her top ministers over plans to leave the European Union, wants to broaden her agenda to try to show she is more than a leader just overseeing Brexit talks, which have all but stalled over customs arrangements.

In a speech in northern England on Monday, May will unveil plans which she says should see at least 50,000 people each year diagnosed at an early stage of prostate, ovarian, lung or bowel cancer – people who would have otherwise been diagnosed at a later and more deadly stage.

“Late diagnosis of otherwise treatable illnesses is one of the biggest causes of avoidable deaths,” she will say, according to excerpts of her speech.

“And the development of smart technologies to analyze great quantities of data quickly and with a higher degree of accuracy than is possible by human beings opens up a whole new field of medical research and gives us a new weapon in our armory in the fight against disease.”

Using the data on people’s genetics, habits and medical records, doctors would then be able to make referrals to an oncologist earlier, she will say.

She will also say that the health innovation is part of her government’s industrial strategy, part of measures to make sure that Britain is at the forefront of the development of new technology as it leaves the EU.

Harpel Kumar, chief executive of Cancer Research said Britain “must remain an attractive place for the life sciences industry to invest”, something that some fear is under threat because of Brexit.

“If this platform unites government, academia, the charity sector, and industry, we will be primed to accelerate innovation and lead the healthcare sector to new heights.”

https://reut.rs/2KHgVDg

Pfenex: A Buy For Therapeutic Proteins Platform

On May 14, Pfenex announced positive topline results from a phase 3 trial which showed their lead candidate, PF708-301 had comparable overall profiles with Eli Lilly’s Forteo in osteoporosis.

The company expects to file an NDA in Q3 2018, with a potential commercial launch in the US in Q3, 2019 at the earliest, if approved.

The stock closed up 31% at $8.00 on May 15.

I take a closer look at this under-covered, small cap biotech company that develops therapeutic proteins.

Pfenex is a BUY for PF708-301 in osteoporosis, and other therapeutic proteins in the pipeline.

Pfenex (PFNX), is a clinical stage development and licensing biotech company producing therapeutic proteins, using their proprietary Pfenex Expression Technology platform.

On Monday, May 14, the company announced positive topline results from a phase 3 trial which showed their lead candidate, PF708-301 had comparable overall profiles with Eli Lilly’s Forteo in osteoporosis.

In the same announcement, Eef Schimmelpennink, CEO of Pfenex commented:

“We expect that these results from the PF708-301 study, along with the previously announced bioequivalence findings from the PF708-101 study in healthy subjects, will support the PF708 NDA submission. We are on-track for submission to the FDA in the third quarter of 2018, with a potential commercial launch in the United States as early as the third quarter of 2019, subject, of course, to FDA approval of the application.”

The market response was a very favorable 31% increase in stock price (from $6.10 on May 14 to $8.00 on May 15).

(Source: Seeking Alpha: 5 day chart ending on May 16, 2018)

(Source: Google Finance: 1 year chart ending on May 16, 2018)

Pfenex’s Drug Pipeline & Partnership

Pfenex uses their Pfenex Expression Technology® platform to develope a pipeline of therapeutic equivalents, vaccines, biologics and biosimilars. Below is an overview of their drug pipeline

(Source: Corporate Presentation)

1. The wholly-owned therapeutic proteins.

Regulatory pathway Indication Reference Drug 2017 Global Sales & its makers
PF708 PF708 is a therapeutic equivalent candidate to Forteo®, and is being developed pursuant to the 505(b)(2) regulatory pathway in the US. Osteoporosis $1.7B by Eli Lilly
PF582 PF582 is a biosimilar candidate to Lucentis® (ranibizumab) and is being developed as a biosimilar pursuant to the 351(k) regulatory pathway in the US. Retinal Diseases $3.3B by La Roche and Novartis
PF529 PF529, is a biosimilar candidate to Neulasta® (pegfilgrastim) and is being developed as a biosimilar pursuant to the 351(k) regulatory pathway in the US. Chemotherapy induced neutropenia $4.5B by Amgen
PF690 PF690 is a biosimilar product candidate to Oncaspar®. Jazz Pharmaceuticals currently has an option to negotiate a license to PF690. Acute lymphoblastic leukemia $0.2B by Shire

2. Collaboration with Jazz Pharmaceuticals

There are two drug candidates in this group so far: PF743 and PF745.

According to the lastest SEC form, Pfnex granted Jazz Pharmaceutical (JAZZ) worldwide rights to develop and commercialize these hematology/oncology drug candidates.

The partnership highlights:

  • up to $224.5M in upfront & potential milestone payments.
  • Tiered royalties on net sales.
  • $13.5M development achievement & 5M upfront payment received in Q4, 2017.
  • On track to meet 2018 development milestones.

3. Novel Anthrax Vaccine Candidates

Px563L and RPA563 are novel anthrax vaccine candidates.

The development of Px563L and RPA563 is currently fully funded by the U.S. Department of Health and Human Services, through the Biomedical Advanced Research and Development Authority, or BARDA, via a $143.5 million advanced development contract.

4. CRM 197, a carrier protein in vaccines.

CRM 197 is being investigated as carrier protein in vaccines for diseases including pneumococcal and meningitis bacterial infection.

Merck and Serum Institute India are among companies with which Pfenex has royalty bearing partnership in place for CRM197’s development and commercialization.

Pfenex’s Near-Term Milestones

According to the company, there are 3 most significant near-term milestones.

(Source: Corporate Presentation)

As stated in the beginning of this article, Pfenex reported positive topline results from their phase 3 study which shows that PF708 is comparable in safety and efficacy with its reference drug, Eli Lilly’s Forteo.

As PF708 is the most advanced, wholly owned drug candidate, it represents the most significant value driver for this stock.

(Source: Corporate Presentation)

According to the company, there are several patents of Forteo that had expired in Dec. 2018 and in August 2019. If PF708’s NDA is successful and it’s approved, the company plans to launch in the US in Q3, 2019 at the earliest.

The market for biosimilar (or in this case therapeutic equivalent) drugs changes rapidly in the recent years, ranging from 1% achieved sale ($2B) of the global biologic market ($200B) in 2016, to an estimated 10% in the US or 25% to 62% of market share in the EU in 2015 (source, page 10).

The table below is an conservative estimation of the global peak sale of PF708 as a percentage of its reference drug’s 2017 achieved sale of $1.7B.

1% 10% 25%
PF708 global peak sale $17M $170M $425M

1% is probably too low an estimation, given the unfavorable market condition (i.e. loss of significant patents) for Forteo after Aug. 2019.

In their presentation, Pfenex indicates that the company values commercial partnership and are actively exploring ways for US and ex-US market of PF708 to be commercially partnered.

For example, the company has recently established a development and license agreement with NT pharma for PF708 in China and selective Asian countries with up to $25M in milestones and double digit royalties on net sale.

A Best scenario estimation for a potential revenue

If PF708 is successfully approved, launched, and reaches a 25% of annual sale of its mostly-off-patent reference drug, Forteo, and other partnered drug candidates continue to meet their developmental milestones, this table below estimates what the potential revenue will be like for Pfenex in a near to medium term.

PF708 reaches 25% of Forteo 2017 global sale $425M
Remaining milestone payments from JAZZ partnership $189.3M*

(=224.5-35.2)

BARDA development contract for Anthrax vaccine $143M
Milestone payments from NT pharma for PF708 $25M
Total $782.3M

*To date, Pfenes has already received $35.2M from JAZZ, thus it is subtracted from the total payment.

This above estimation only covers the disclosed development milestone amounts from the partners and does not include the undisclosed licensing/loyalty for CRM197. It does not include the tiered royalties on net sales of the partnered drugs, if/when they are successfully developed and go to market. It also does not include a possible contract of Anthrax vaccine by the BARDA, if/when the Anthrax vaccine candidates are successfully developed and approved.

The current market cap is ~$175M (May 18, 2018), which represents ~22.4% of the potential revenue of $782.3M. In other words, there is considerable room for the stock to appreciate, if the company successfully execute its business plan and increase its revenues.

Investment Thesis

Pfenex is a speculative buy for investors who have done their own DD on Pfenex and find the risk/reward scenario favorable to their investment goal and risk tolerance.

Pfenex is a solid company that has formed very significant partnerships for their extended, advanced pipeline. Looking at their history of timely execution of their clinical trials, it is highly likely that the company will continue to reach the developmental and commercial milestones in various programs and thus increase the revenues steadily and significantly.

According to Yahoo Finance, the institutional ownership is over 80%.

Cash Position

At the end of March 31, 2018, the company has $47M in cash or cash equivalent. The burn rate is $11M per Q. The company projects the available cash to fund its operation for the next 12 months, including the filing of a NDA for PF708 anticipated to take place in Q3 2018.

Risks

The most significant risks include but not limited to the regulatory failure of PF708 in its indication as a therapeutic equivalent drug in osteoporosis; any significant delay or failure in PF708’s commercial launch, if approved to go to market; lower than expected revenue generated by PF708 in the US and Ex-US; failures in the clinical developments of one or more drug candidates in partnership with JAZZ, BARDA, or other companies.

The financial risk of a dilution at the near term cannot be ruled out, if the company does not establish further commercial partnership for PF708 or no further non-dilutive fundings secured in a near term.

https://bit.ly/2GB6qPh