Category Archives: Biotechnology

SUNNYVALE, Calif., April 3, 2017 /PRNewswire-iReach/ -- Three business and scientific leaders with early-stage investors today announced the formation of Apostle Inc, a biotechnology company developing a novel bioinformatics-enabled nanotechnology aimed for early cancer detection. This new approach will enable the early assessment of the cancerous signals in human peripheral blood plasma, which is believed to have a significant impact on the global healthcare landscape in both developed countries and emerging markets.

Dr. David Dongliang Ge, an experienced business and scientific leader who was President of BioSciKin Co. and Director of Bioinformatics at Gilead Sciences, will lead the new company. He is joined by two colleagues as co-founders of Apostle and his investment partners. "Biotechnologies, especially those focusing on novel diagnostic or therapeutic advancements aiming for cancer, are among the key focuses in the global economy for the next 5-20 years. By 2020, the market size ofcancerdiagnosis is estimated to reach $168.6 billion. Apostle represents one of these focuses." Dr. Ge said. "With a groundbreaking bioinformatics-enabled nanotechnology approachwe want to inform the general population that we are able to help them identify cancer signals, earlier and more accurate than conventional techniques, and potentially advise their doctors to take highly effective surgical actions. "

"It's been a great pleasure to have the opportunity to work with David and his team on this amazing venture. We're thrilled to work with this scientifically imaginative and visionary company." One of the investors said. Apostleis funded by Amino Capital, ShangBay Capital, Westlake Ventures in the Silicon Valley and a group of individual investors from both the Silicon Valley and China. Apostle is advised by Dr. Charles Cantor, an American molecular geneticist, former director of the Department of Energy Human Genome Project, a member of the National Academy of Sciences, as well as Dr. Hongyu Zhao, the Ira V. Hiscock Professor of Biostatistics and Professor of Statistics and Genetics, Chair of the Biostatistics Department and the Co-Director of Graduate Studies of the Inter-Departmental Program in Computational Biology and Bioinformatics at Yale University.

About Apostle Inc.

Apostle Inc is a biotechnology company in Sunnyvale, CA. It's in the business of the research, development, licensing, and sales of novel bioinformatics-enabled nanotechnologies and the related intellectual properties, products, and services for diagnosis and treatment of human diseases

About the founder team of Apostle Inc.

Dr. David Dongliang Geis CEO and President of Apostle. Previously, he was President of BioSciKin Co. and Simcere Diagnostics Co., two global biotechnology companies headquartered in Nanjing, China. Between 2011 and 2016, he was Director of Bioinformatics at Gilead Sciences, where he founded and provided leadership to the bioinformatics group. Dr. Ge and his group led the phylogenomic analytical support for the critical regulatory approval of Sovaldi, a world-leading anti-HCV drug. In 2014 and 2015, Dr. Ge was invited to be a member of the U.S. NHGRI Special Emphasis Panel. He was appointed as Assistant Professor of Biostatistics and Bioinformatics at Duke University School of Medicine. He received his Ph.D.ofBiostatistics and Genetic Epidemiology from Peking Union Medical College and Chinese Academy of Medical Sciences in 2004. Dr. Ge's work in discovering the IL28B genetic variants associated with the clinical treatment responses, published in Nature in 2009, has received over 3000 times of citations with the U.S. FDA's citation in its several guidance for industry. The invention was licensed to LabCorp and QuestDiagnostics,and has become clinical diagnostic services since then (LabCorp 480630 and Quest AccuType IL28).Dr. Ge has authored over 70 original articles, including 5 in Nature and 1 in Science, in total receiving over 15,000 citations. Dr. Ge was named by the U.S. Genome Technology magazine as one of the "Rising Stars" in 2009, and by the U.K. Phacilitate as one of the "Top 50 Most Influential People in Big Data" in 2015.

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Bo Zhang, Ph.D.is VP of Chemistry of Apostle. Dr. Zhang received his Ph.D.ofChemistry from Stanford University in 2015 and received his B.S.ofChemistry from Peking University in 2010. Dr. Zhang has won the Gold Medal of National Chemistry Olympiad of China in 2006. Dr. Zhang has 10 years of experience in nanotechnology research, with outstanding achievements in developing novel nanomaterials with unique fluorescence characteristics. Dr. Zhang published over 30 original paperson Nature Medicine, Nature Materials, Nature Photonics, Nature Communications, etc. Dr. Zhang's two articles in Nature Medicine about novel nano-platform for type 1 diabetes and Zika virus infection diagnosis have attracted worldwide attention. Dr. Zhang has been PI for research projects funded by the NIH. He holds many patents. Dr. Zhang was the recipient of Materials Research Society Awards, Mona M. Burgess Fellow, William S. Johnson Fellowship, etc.

Xin Guo, Ph.D.is VP of Bioinformatics of Apostle. Previously, Dr. Guo was group leader at Gilead Sciences, in charge of the clinical phylogenomic program for developing Sovaldi. Dr. Guo received his Ph.D. in Computer Sciences from Duke University and M.S. in Informatics from Max Planck Institute of Germany. He received his B.S. in Informatics from Chiba Institute of Technology of Japan. Dr. Guo has over 10 years of experience in the R&D ofhigh performancecomputing, machinelearningand artificial intelligence. Dr. Guo has extensive experience in product development of complex algorithms and databases, with applications in genomic big data.

Wenqi Zeng, MD,PhD, FACMGis Chief Medical Advisor of Apostle. He is Chief Medical Officer of Simcere Diagnostics Co. Previously, Dr. Zeng was Senior Director of Molecular Genetics at Quest Diagnostics and was Director of Clinical Genomics at Ambry Genetics. Dr. Zeng was fellow of Clinical Molecular Genetics and Medical Genetics at Harvard. He received his M.D. from Xiang-Ya Medical School in China and Ph.D. in MolecularPatholgy/Molecular Genetics fromUniversityof Otago. He holds Diploma of American Board of Medical Genetics and Genomics(ABMGG),and is a qualified CAP inspection team leader, and a qualified CAP CLIA lab director in CA,FLand MD. He also has NY state COQ in molecular genetics and molecular oncology.

Media Contact: Public Relations, Apostle, Inc, Apostle, Inc, 650-483-5437, pr@apostlebio.com

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Apostle Inc, a Biotechnology Company for Early Cancer Detection, is Founded in the Silicon Valley - Yahoo Finance

(Photo: http://mma.prnewswire.com/media/485639/VSY.jpg )

VSY Biotechnology considers it important to note that this is a ruling by the German Court of First Instance. VSY Biotechnology has the right to and will appeal this judgement with the Higher District Court and if necessary with the Federal Supreme Court in Germany. It is expected that a final ruling on the infringement may take several years and German Court's ruling is only effective in Germany but not in all over the world.

VSY Biotechnology B.V. reserves its right to seek material and moral indemnity for all direct and indirect damages of VSY Biotechnology on basis of unfair competition and defamation due to incomplete press release of Carl Zeiss dated March 27, 2017. Further VSY Biotechnology will ask for all damages they may suffer from the decision of the First German Court in case of District Court or Federal Court rule in favor of VSY Biotechnology.

Additionally, VSY Biotechnology applied for invalidation of Zeiss Patent EP 2 377 493 B1 before EPO with the claim of Zeiss Patent has lack of novelty, lack of inventive step, therefore unpatentable.

VSY filed opposition against Zeiss patent

VSY Biotechnology B. V. (VSY) has, amongst others, filed an opposition against Carl Zeiss Meditec A.G.'s (Zeiss) European patent EP 2 377 493 B1 "Method for manufacturing aphakic intraocular lens" (EP'493) with the European Patent Office (EPO) on May 6, 2016. Zeiss acquired EP'493 from the German IP advisers IP Strategists GmbH who acquired the patent from the Japanese company Menicon Co..VSY is of the opinion that EP'493 does not fulfil the requirements of patentability as it lacks novelty and inventive step, is insufficiently disclosed and includes extension of subject matter.

The patent regards Trifocal intraocular lenses. Trifocal lenses of Zeiss are AT LISA tri and the AT LISA tri toric.

The opposition is still pending.

All details of the opposition can be found on: https://register.epo.org/application?number=EP09837427&lng=de&tab=doclist

About VSY Biotechnology BV

VSY Biotechnology is one of the leading companies in Industry of ophthalmology focusing on cataract surgery, producing premium intra ocular lenses and viscoelastics. For more information, please visit company website. http://www.vsybiotechnology.com.

SOURCE VSY Biotechnology

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Patent Case of Carl Zeiss Meditec AG and VSY Biotechnology B.V. - PR Newswire (press release)

LOS ANGELES--(BUSINESS WIRE)--Puma Biotechnology, Inc. (Nasdaq: PBYI), a biopharmaceutical company, announced that interim results from a Phase II clinical trial of Pumas investigational drug PB272 (neratinib) were presented at the 2017 American Association for Cancer Research Annual Meeting (AACR) that is currently taking place in Washington, D.C. The presentation, entitled Effects of adding budesonide or colestipol to loperamide prophylaxis on neratinib-associated diarrhea in patients with HER2-positive early stage breast cancer: the CONTROL trial, was presented as a poster presentation.

The main adverse event that has been seen to date in clinical trials of neratinib is diarrhea and more specifically grade 3 diarrhea. In the Phase III ExteNET trial of neratinib as extended adjuvant treatment of HER2-positive early stage breast cancer that has previously been treated with adjuvant Herceptin, 95.4% of the patients experienced all grade diarrhea and 39.8% of the patients experienced grade 3 or higher diarrhea (there was one event of grade 4 diarrhea). The CONTROL trial is an international, open-label, Phase II study investigating the use of loperamide prophylaxis with or without other agents in the reduction of neratinib-associated diarrhea that has a primary endpoint of the incidence of grade 3 diarrhea.

In the CONTROL trial, patients with HER2-positive early stage breast cancer who had completed trastuzumab-based adjuvant therapy received neratinib daily for a period of one year. High dose loperamide prophylaxis was given for the first 2 cycles (56 days) of treatment. Initially, the loperamide dosing used was 16 mg on day 1, then 12 mg on days 2 and 3 and then 6-8 mg on days 4-56 (original dosing). The protocol was later amended to simplify the regimen such that patients took 12 mg on days 1-14 and 8 mg on days 15-56 (modified dosing). The CONTROL trial was also expanded to include two additional cohorts. One cohort received the combination of loperamide and budesonide and the other cohort received the combination of loperamide plus colestipol. Budesonide is a locally acting corticosteroid that the Company believes targets the inflammation identified in a preclinical model of neratinib-induced diarrhea and colestipol is a bile acid sequestrant that the Company believes targets the bile acid malabsorption also seen in preclinical models of neratinib-induced diarrhea.

The interim analysis of the trial presented in the poster included a total of 137 patients who received neratinib plus loperamide prophylaxis (28 patients taking the original dosing and 109 patients taking the modified dosing), 64 patients who received neratinib plus loperamide prophylaxis for 2 cycles and budesonide for 1 cycle, and 26 patients who received neratinib plus loperamide prophylaxis for 1 cycle and colestipol for 1 cycle.

The results of the trial showed that the incidence of grade 3 diarrhea for the 137 patients who received the loperamide prophylaxis was 30.7%. For the 137 patients who received the loperamide prophylaxis, the median number of grade 3 diarrhea episodes per patient was 1 and the median cumulative duration of grade 3 diarrhea was 3 days. For the 137 patients who received loperamide prophylaxis, 20.4% discontinued neratinib due to diarrhea.

For the 64 patients who received the combination of loperamide plus budesonide, the results of the trial showed that the incidence of grade 3 diarrhea was 23.4%. The median number of grade 3 diarrhea episodes per patient was 1 and the median cumulative duration of grade 3 diarrhea was 2 days. For the 64 patients who received loperamide plus budesonide prophylaxis, 9.4% discontinued neratinib due to diarrhea.

For the 26 patients who received the combination of loperamide plus colestipol, the results of the trial showed that the incidence of grade 3 diarrhea was 11.5%. The median number of grade 3 diarrhea episodes per patient was 2 and the median cumulative duration of grade 3 diarrhea was 2 days. For the 26 patients who received loperamide plus colestipol prophylaxis, no patient (0%) discontinued neratinib due to diarrhea. Further information is provided in Table 1 below:

Table 1: Characteristics of Treatment-Emergent Diarrhea

Loperamide

(original +

modified)

loperamide

loperamide

Loperamide

prn

a

In the ExteNET trial, higher grade (grade 2 and grade 3) diarrhea occurred early and persisted throughout the duration of the 12-month treatment period. In the CONTROL trial, in the loperamide prophylaxis, loperamide plus budesonide prophylaxis and loperamide plus colestipol prophylaxis arms, the results showed that higher grade diarrhea (grades 2 and 3) occurred early but did not typically recur. This is shown in more detail in Figure 1: Treatment Emergent Diarrhea, which is attached to this news release. In addition, a full copy of the poster that was presented at AACR is available on the Puma Biotechnology website.

During the course of the CONTROL trial there has been an increase in the proportion of patients previously treated with pertuzumab (mainly in the neoadjuvant setting). For the 55 patients in the loperamide prophylaxis cohort who received prior pertuzumab, the grade 3 diarrhea rate was 38.2% (Table 2). For the 82 patients who did not receive prior pertuzumab, the grade 3 diarrhea rate was 25.6%. For the 39 patients in the budesonide cohort who received prior pertuzumab, the grade 3 diarrhea rate was 10.3%. For the 25 patients in the budesonide cohort who did not receive prior pertuzumab, the grade 3 diarrhea rate was 36.0%. This analysis suggests that prior pertuzumab exposure may have led to a higher rate of grade 3 diarrhea in the CONTROL trial that was not effectively managed by loperamide prophylaxis alone but was more effectively managed by loperamide plus budesonide.

Table 2: Incidence of Grade 3 Diarrhea in CONTROL by Prior Pertuzumab Treatment

Loperamide Cohort

Budesonide Cohort

(n = 55)

(n = 82)

(n = 39)

(n = 25)

Dr. Carlos H. Barcenas, Assistant Professor, Department of Breast Medical Oncology and Associate Medical Director, Breast Cancer Survivorship Clinic for the University of Texas MD Anderson Cancer Center, said, We are pleased to see the reduction in incidence, severity and duration of neratinib-associated diarrhea when using the three antidiarrheal prophylaxis regimens tested so far in this study. When using either the loperamide prophylaxis, the loperamide plus budesonide prophylaxis or the loperamide plus colestipol prophylaxis, there appears to be a reduction in the incidence and severity of grade 3 diarrhea with neratinib. Importantly, the severe grade 2 and grade 3 diarrhea, when using the prophylaxis, appears to be acute, self-limiting and manageable. Although the study is still ongoing, we are encouraged that the addition of budesonide or colestipol appears to greatly improve the tolerability of neratinib as well. We look forward to completing the loperamide plus colestipol cohort.

Alan H. Auerbach, Chief Executive Officer and President of Puma Biotechnology, said, We are pleased to see the reductions in the incidence of severe neratinib-related diarrhea in the CONTROL trial when using the loperamide, loperamide plus budesonide or loperamide plus colestipol regimens. The severe diarrhea appears to become more acute, whereby it does not typically recur after the first month. We are also very encouraged by the improvements in tolerability that have been seen to date in the budesonide and the colestipol cohorts. This is a marked improvement in tolerability over what was seen in the ExteNET trial and we look forward to continuing to monitor this in the loperamide plus colestipol cohort.

About Puma Biotechnology

Puma Biotechnology, Inc. is a biopharmaceutical company with a focus on the development and commercialization of innovative products to enhance cancer care. The Company in-licenses the global development and commercialization rights to three drug candidatesPB272 (neratinib (oral)), PB272 (neratinib (intravenous)) and PB357. Neratinib is a potent irreversible tyrosine kinase inhibitor that blocks signal transduction through the epidermal growth factor receptors, HER1, HER2 and HER4. Currently, the Company is primarily focused on the development of the oral version of neratinib, and its most advanced drug candidates are directed at the treatment of HER2-positive breast cancer. The Company believes that neratinib has clinical application in the treatment of several other cancers as well, including non-small cell lung cancer and other tumor types that over-express or have a mutation in HER2. Further information about Puma Biotechnology can be found at http://www.pumabiotechnology.com.

Forward-Looking Statements:

This press release contains forward-looking statements, including statements regarding the development of the Companys drug candidates. All forward-looking statements included in this press release involve risks and uncertainties that could cause the Company's actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on current expectations, forecasts and assumptions, and actual outcomes and results could differ materially from these statements due to a number of factors, which include, but are not limited to, the fact that the Company has no product revenue and no products approved for marketing; the Company's dependence on PB272, which is still under development and may never receive regulatory approval; the challenges associated with conducting and enrolling clinical trials; the risk that the results of clinical trials may not support the Company's drug candidate claims; even if approved, the risk that physicians and patients may not accept or use the Company's products; the Company's reliance on third parties to conduct its clinical trials and to formulate and manufacture its drug candidates; the Company's dependence on licensed intellectual property; and the other risk factors disclosed in the periodic and current reports filed by the Company with the Securities and Exchange Commission from time to time, including the Company's Annual Report on Form 10-K for the year ended December 31, 2016. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. The Company assumes no obligation to update these forward-looking statements, except as required by law.

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Puma Biotechnology Presents Interim Results of Phase II CONTROL Trial of PB272 in Extended Adjuvant Treatment of ... - Business Wire (press release)

Cellect Biotechnology Gets European Patent (APOP) Investopedia secured a European patent covering a method of treatment from the European Patent Office. The patent covers cell-based therapeutics of Cellect's technology and method that have the potential to treat diseases like diabetes, graft-versus-host disease, ... BRIEF-Cellect Biotechnology receives formal notice of intention to grant for a patent from European patent officeReuters Cellect Biotechnology (APOP) Granted European Patents Protecting ...StreetInsider.com Cellect Biotechnology Ltd. (NASDAQ:APOP) reported an European patent grantBenchmark Monitor GlobeNewswire (press release) all 9 news articles »

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Cellect Biotechnology Gets European Patent (APOP) - Investopedia

Breakthrough Therapy Designation is described on the FDA website as "a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s)." It was signed into law in 2012, with the first full approval of a breakthrough designation candidate being only one year later. As of March 30, 2017, FDA has granted breakthrough therapy designation to 170 of the 505 total requests, and eventually approved 55 breakthrough therapy designated products.

The concept makes sense. If a new drug that aims to treat serious and life-threatening conditions shows unprecedented effect in early clinical trials, patients should not have to wait to receive treatment. A group called Friends of Cancer Research worked with partners in advocacy, regulation, drug development, and bipartisan congressional champions to take breakthrough therapy designation from concept to an expedited FDA development program. This ensures patient access to revolutionary drugs is as quick and effective as possible. A drug that receives breakthrough therapy designation is eligible for all FDA fast track features, intensive FDA guidance on an efficient development program (as early as phase 1), and organizational commitment involving FDA senior project managers.

FDA bases its decision to grant breakthrough therapy designation on scientific support for clinical endpoint(s). Studies that demonstrate a drug's effect on a surrogate or intermediate clinical endpoint must be "adequate and well controlled" as required by the FD&C Act. Using surrogate or intermediate endpoints can save valuable time in the drug approval process. Consider an example of cancer patients. Rather than wait for a study to complete a potentially lengthy overall survival measurement, FDA may approve a drug based on evidence of tumor shrinkage, associated with a shorter measure such as progression-free survival, because it is considered reasonably likely to predict clinical benefit (and hence prolonged survival of patients).

This well written article about the drug development process addresses the process and is worth a quick read. A cancer drug development company will still need to conduct studies to confirm that tumor shrinkage actually predicts that patients will live longer, which is sometimes a phase 3 or even a phase 4 clinical trial. One common question often asked by StrongBio stock club members (send email to join, its free) is, "will a company have to wait to market a drug until trials assessing secondary endpoint data or confirmational long-term effects of drugs on a patient population are completed"? So the answer can be yes or it can be no. The industry currently anticipates that primary consideration for marketing approval by FDA is primary endpoint result(s). So it depends what the primary endpoint of the trial is predetermined to be with FDA, in an advanced declaration called a special protocol assessment. Though caution is warranted when predicting clinical trial result interpretation by FDA, it is clear that shorter endpoint measures such as progression free survival were specifically developed by scientists and clinicians (over many years with massive resource allocation) as reliable metrics for early approval.

As a biotechnology stock investor, one valuable tool for learning about what therapeutic candidates FDA might favor, is the Friends of Cancer Research list of FDA Breakthrough Therapy Designations. If you have not included this or Knect365 lists as part of your due diligence, please consider doing it now. These lists consist of many indications in medicine, not just cancer. Many, but not all, of the stocks on this list have performed well, and having FDA in a supportive role behind a drug candidate has proven to have boosted confidence in these stocks when reviewed in articles analyzing how they are priced by the Street. Since excitement surrounds the designation, an increased stock price can be used to generate funds for the company to begin early manufacturing initiatives. In this way investors help serve those in need of life-saving therapies by funding faster production in hopes for a future payoff. Everyone can win in this positive scenario.

Genentech [Roche Holdings, (OTCQX:RHHBY)], with 15 breakthrough therapy designations, is a leader in the breakthrough designation category, with several rare and specialty indications. But with its multiple players and large market cap, it's more difficult to measure an effect of a breakthrough therapy on stock price. Factors that an investor should consider when looking at the designation is the potential market compared to company market cap. So Immunomedic's (NASDAQ:IMMU) Sacituzumab Govitecan breakthrough therapy designation for triple negative breast cancer with a 6 billion dollar market and only 600 million dollar market cap might be seen as more attractive in contrast to RHHBY (market cap 216 billion) rituximab designation for pemphigus vulgaris, a rare autoimmune disease causing skin and mucosal membrane blisters, with estimated market in the low hundreds of millions. At the time (February 2016) IMMU reported its breakthrough therapy designation it was trading at about 2 dollars per share, and is now about 6.50 about a year later (after several futile attempts by short-sellers to discredit the company ultimately at breast cancer victim's expense), compared to RHHBY, which has moved from about 31 dollars per share since its Mar 21, 2017 announcement to 32 dollars this week. It is extremely unlikely rituximab will drive RHHBY to 105 dollars one year from now to match IMMU's market performance of Sacituzumab Govitecan. However, one should never underestimate the ability of big pharma to gain an expedited product approval and then have that product used for other indications "off label" when other treatments fail. For instance, rituximab has implications in treating Lupus, with an increasing 3.5 billion dollar industry by 2025. For the purpose of breakthrough therapy designation affecting stock price, StrongBio will focus on a few publicly traded (Novimmune, Rebiotix, and Rhythm are interesting but privately held companies) small market cap representatives in this article in order to make some conclusions, referring to larger market cap companies as a point of reference.

Like IMMU, Adaptimmune (NASDAQ:ADAP) received breakthrough therapy status, but for affinity enhanced T-cell therapy targeting NY-ESO in synovial sarcoma in February 2016. With a market cap of under 400 million and a potential market in the low hundreds of millions per year in synovial (and other off label or future indication) sarcomas, this is certainly an attractive investment to StrongBio if weakness in market price should occur. Some investors regard ADAP as some of the best cancer work being done right now, and recommends it as a good investment in the 200 billion dollar immunotherapy cancer space. Having raised some funds recently and having strong partners, the stock has decreased in value from about 7 dollars per share to about 5.50, but has some recent positive momentum after raising funds in an offering at just over 4 per share. It was first covered by StrongBio at 4 dollars earlier this year. IMMU and ADAP have both shown to be somewhat volatile following breakthrough therapy designation, warranting scrutiny of timing before entering a long position based upon breakthrough designation.

Another stock, like ADAP, that has trended down since its breakthrough designation by FDA is Trevena (NASDAQ:TRVN). TRVN has dropped over 50% since designation, February 22, 2016, shooting from 7 dollars per share to about 9 dollars per share, but is now trading at 3.65 per share one year later. TRVN's oliceridine, an acute pain control drug for treating moderate to severe post-bunionectomy or abdominoplasty, even met its phase 3 trial endpoints for pain control. But as this article summarizes well, may be seen as too expensive compared to morphine and other pain killers. This article by the Street makes a good case for TRVN in how oliceridine met pain control endpoints and potentially offers an advantage in a profile of side-effects including nausea, vomiting, and depressed breathing. However some of the side effects were not (yet) reported as statistically significant. With analyst price targets of 12 dollars and slew of legal class action threats that mimic the ones that IMMU had before its rapid price ascension, it is probably likely that once short sellers have covered TRVN will reach its targets. Certainly one to watch given this share weakness if one likes to use a contrarian approach. We know from StrongBio research done on the plastic surgery industry in previous articles that the industry is rapidly growing, and people don't seem to mind paying expensive prices. A more expensive but better pain killer may not be seen as a negative after all.

Kite Pharma (NASDAQ:KITE), market cap 1.8 billion, received FDA breakthrough therapy designation in December, 2015 for its refractory aggressive non Hodgkin lymphoma candidate KTE-C19. The cancer market is in the 100 billion range, so this stock should be on just about every biotechnology enthusiast's radar. At that time KITE was trading at about 75 dollars per share after a ramp up from about 50 per share earlier that fall. The price of KITE settled back to about 40 to 50 per share from January 2016 to January 2017, only recently increasing to its current 78 dollar level. KTE-C19 is therapy in which a patient's T cells are genetically modified to express a chimeric antigen receptor targeting the CD19 maker, a protein expressed on the cell surface of B cell lymphomas and leukemia. Its breakthrough designation includes treatment of diffuse large B cell lymphoma, primary mediastinal B cell lymphoma, and transformed follicular lymphoma. Investors are optimistic due to early studies which have demonstrated 31% "curative" complete responses and remission in chemorefractory diffuse large B cell lymphoma patients after a single treatment. Analyst targets for KITE are at an average of 86 dollars per share. Timing is critical for a long position in this company, and as volatile as the share price has been over the last 2 years, anything is possible. Ironically, this stock has remained flat in spite of the volatility since breakthrough designation.

Juno pharmaceuticals (NASDAQ:JUNO) [breakthrough designation December 2016 for JCAR017 for treatment of relapsed/refractory aggressive large B-cell maladies], Novartis, (NYSE:NVS) [forPKC412 midostaurin for adults with FLT3+ AML and LEE011 combination with letrozole for treatment of HR+/HER2- advanced or metastatic breast cancer], and Bellicum pharmaceuticals (BLCM) [no breakthrough candidate] are also operating in this B cell cancer area, with emphasis being put on safety by limiting graft versus host risks of these robust anti-cancer therapies. Juno, with a 2.5 billion dollar market cap might be undervalued. Having recently discontinued development of its nicely active anti-cancer JCAR015 for ALL, due to safety issues, combined with its early FDA favor for JCAR017 plus immunosuppression regimen, and strong cash position of nearly 1 billion, one can make a good investment case on a further pullback. Immunosuppression drugs, cytokines, T-Cell, and stem cell therapies are all approaches designed to make these therapies less likely to attack healthy tissues. BLCM just raised money in an offering at 12 dollars per share, has a market cap of 330 million, and analysts average target at 27.50. StrongBio regards this option as potentially the best potential market to market cap ratio, should BLCM succeed in pulling down a share. Uniquely, BLCM technology involves modifying donor immune cells with a "kill switch" that is activated with administration of rimiducid. In an ongoing trial with 122 pediatric patients receiving partially matched donor stem cell transplants, five cases of uncontrolled graft versus host disease were resolved by the rimiducid-activated kill switch. Juno, Novartis, and Kite are good investment candidates with plenty of up-side, but the dominant market share will go to the company that best suits safety needs in graft versus host reaction. NVS is a giant with a monster market cap of 174 billion, making it more difficult to obtain a high return due to any sole candidate. So in this case perhaps riding the wing of an FDA breakthrough therapy status candidate could provide the best investment opportunity, via BLCM acquisition or stand-alone data. This creates a new category of investment candidates and fits the StrongBio contrarian outlook.

Exelixis Inc. (NASDAQ:EXEL), with a 6 billion market cap, was granted breakthrough therapy designation by FDA for renal cell carcinoma with one prior therapy. As a more mature representative of the speculative designation class, its chart reflects a little more of a steady classic upward stair than a volatile up and down nausea inducer. Since the launch of its kidney drug, Cabometyx, has grown to about 27% of the 1 billion dollar market in renal cancer. At the time of designation, it traded at about 9 dollars per share, and now trades at about 21 dollars per share 18 months later. Having made efforts to decrease its debt, and expand its pipeline, and make international partners including Takeda Pharmaceuticals in Japan, the company is stabilizing and poised for stock growth, even with its fairly large market cap. The value of stability and income can never be underappreciated, nor can a nice stepwise ascension in stock price over 100% per year.

uniQure N.V. (NASDAQ:QURE), a Dutch gene therapy company, received breakthrough therapy designation for AMT-060, its investigatory gene therapy in patients with severe hemophilia B. With a market cap of only 145 million dollars, its stock price is relatively flat since the announcement Jan 30, 2017. QURE has received regulatory approval in the European Union and carries a relatively low valuation compared to its peers, including Bluebird bio (NASDAQ:BLUE), Avalanche (AAVL), and most recently Spark Therapeutics (NASDAQ:ONCE). Briefly, gene therapy offers curative potential by inserting a gene encoding the expression of a therapeutic protein with a single administration. When a gene encoding a blood clotting protein is missing or mutated in hemophilia B patients, QURE technology introduces a copy of the proper gene to restore the presence of the clotting factor, preventing uncontrolled bleeding. Given its small market cap and high reward to risk ratio in this 10 billion full market for hemophilia, QURE could have some potential upward volatility in its future.

Tonix Pharmaceuticals (NASDAQ:TNXP) is a unique company because it received breakthrough therapy designation in December 2016 for its post-traumatic stress disorder drug candidate, TNX-02 SL. TNX-102 SL is a protective eutectic oral formulation of cyclobenzaprine that allows for a rapid systemic exposure and increased bioavailability through transmucosal delivery to modulate sleeping cycles. Scientific mechanism of action is to block serotonin receptor 2A, associated with an increase in restorative slow wave sleep and a decrease in waking after sleep onset. With a market cap around only 18 million, there would seem to be a lot of upside to this reward to risk analysis. Tonix has advanced its candidate TNX-02 SL to phase 3. But TNXP is also plagued by typical early stage biotechnology company challenges, including having recently reverse split its stock to stay listed on the Nasdaq exchange, and recently raised 8 million in capital in an offering, providing the company with approximately 30 million in operating capital. Recently TNXP enrolled its first military related post-traumatic stress disorder patient in its study. Because this stock has not made much of a move yet, some potential for upward volatility remains for TNXP stock. Because there is a likelihood of another raise of cash, StrongBio would advise caution in investing heavily in spite of the wonderful cause.

The full scope of breakthrough designation grants is beyond the scope of a Seeking Alpha article, but demonstrated is the main point that FDA breakthrough therapy designation lists yield fascinating investment candidates. Remember there are no sure things in the stock market, and that investing in biotechnology includes a lot of variability in results, risks, and volatility. Investment strategies previously published by StrongBio best include timing entries of positions, making good critical selections of key growth, and optimizing foreseeable market favor. One way to maximize the chances of an optimal result for market favor is to diversify the portfolio with companies that have obtained FDA breakthrough therapy designation, especially when perceived as undervalued. Though there are no guarantees that the market is going to reward investors short term, in the long run it appears that most breakthrough designation candidates meet with success eventually. The charts also caution that volatility may be associated with these picks. StrongBio recommends putting candidates with FDA breakthrough therapy designation on the watch list and hedge in on pullbacks.

Disclosure: I am/we are long IMMU.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: Secondary tickers include RHHBY, IMMU, ADAP, TRVN, KITE, BLCM, EXEL, JUNO, QURE, BLUE, AAVL, ONCE, TNXP but were not listed as options

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Impact Of FDA Breakthrough Therapy Designation Speculative Biotechnology Stock Performance - Seeking Alpha

The president of DSM Food Specialties, Ilona Haaijer, has told FoodBev that shes really excited to be able to offer laboratory and scale-up facilities as part of its new biotechnology centre in the Netherlands.

The company unveiled the Rosalind Franklin Biotechnology Center on its Delft campus earlier, where more than 400 scientists will advance the companys research into food enzymes, cultures, bio-preservatives and taste ingredients for the global food industry.

Alongside significant investments in robotics and automation, the centre includes space for start-ups to explore and scale up their concepts in partnership with DSM Food Specialties.

At the opening: Alex Clere

There was a general buzz, a sense of excitement at the unveiling of DSMs imposing glass biotechnology centre this morning. DSMs scientists had already moved in, keen to give visitors an impression of the new equipment some the result of millions of euros worth of investment and collaborative spaces in action.

Ahead of the unveiling ceremony, DSM Food Specialties president Ilona Haaijer told me which of the companys products she thought were particularly pertinent, given the state of the food industry at the moment.

Its MaxiLact product, which removes lactose from a product while delivering additional sugar reductions, was flying, she said. And the Preventase proprietary enzyme allows brands to limit the amount of acrylamide in bakery items amid widespread concern across Europe.

This new centre, a major achievement for DSM Food Specialties, will help the business continue to meet the industrys biggest trends and satisfy changes to consumer preference.

The Rosalind Franklin Biotechnology Center is part of DSM Food Specialties network of more than 30 laboratories in ten countries. According to Gerhard Wagner, the director of DSMs Biotechnology Center, the company has sought to align itself with local technology hubs like Boston, Massachusetts and put down roots close to its markets.

Haaijer also noted that the world was becoming an increasingly volatile, uncertain, complex and ambiguous (VUCA) place, which made the prediction of future trends difficult.

The process was no longer as linear as it had once been, becoming an art, not a science.

But in that challenge was the opportunity for DSM to increase its participation with external stakeholders, like Delft University of Technology and Corbion.

The biotechnology centre in Delft has also been designed with the help of DSMs scientists, and includes features and open spaces deliberately intended to encourage cooperation and collaboration between employees.

Haaijer claimed that it was more and more important to open up with chief operating officer Cindy Gerhardt adding that this was the only way companies can be early to market with a product.

The Rosalind Franklin Biotechnology Center will play a crucial role in that strategy.

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DSM 'excited' by biotechnology opportunites, president says - FoodBev.com

ALACHUA, Fla., April 3, 2017 /PRNewswire/ --Sid Martin Biotechnology Institute (SMBI), the leading biotechnology incubator at the University of Florida, has been awarded the Randall M. Whaley Incubator of the Year award for 2017, the highest award given by the International Business Innovation Association (InBIA). InBIA is the world's leading organization for advancing business incubation, acceleration and entrepreneurship. SMBI was named Incubator of the Year among more than 7,500 incubators worldwide. The annual award, sponsored by the Friends of the University Science Center in Philadelphia, recognizes the top global business incubation program and includes a cash prize.

The award was presented on March 28th at the InBIA's 31st Annual International Conference on Business Incubation. Accepting the award for SMBI were Mark S. Long, Director, and Merrie Shaw, Assistant Director. SMBI also received another award, the 2017 Technology/Science Entrepreneurship Center Program.

David L. Day, Assistant Vice President for Technology Transfer at the University of Florida, said, "We are honored for the Institute to be recognized as the best in the world incubator. It is a tribute to our staff and their outstanding efforts helping startups grow great innovations and new solutions into successful businesses that will make the world a better place."

SMBI has a biotechnology focus, and over the past 21 years has served more than 100 startup companies in biotechnology, biomedicine and bioagriculture. The Institute has created more than 2,200 high-tech jobs since its inception, and SMBI resident companies have accumulated over $1.62B in capital and M&A activity. There is a 93% survival rate for companies that entered the SMBI program since March of 2003, and an overall 78% survival rate for all companies served over the past 21 years.

Since becoming Director of SMBI in January 2016, Long has overseen the admission of 13 new companies, and the graduation of three companies. "We continue to see the growth of North Central Florida as a biotech hub," said Long. "As part of the University of Florida's Research Foundation, we are able to offer new biotechnology startups a tremendous wealth of resources, advisement and equipment. We are proud to be recognized by our peers as the top incubation program in the world."

About Sid Martin Biotechnology Institute at the University of Florida

The Sid Martin Biotechnology Institute (SMBI) is the leading biotechnology incubator headquartered at the University of Florida in Alachua, Florida at Progress Park. SMBI has been honored with national and international awards for incubator excellence and achievements in technology commercialization, funding access, job creation and technology-based economic development. It is dedicated to mentoring and accelerating the growth of innovative early-stage bioscience and biotechnology companies, and supporting the economic growth of the North Central Florida region. For more information, visit sidmartinbio.org.

Contact:Merrie Shaw, Assistant Director, Sid Martin Biotechnology Institute, 386-462-0880, mashaw@ufl.edu, sidmartinbio.org

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University of Florida/Sid Martin Biotechnology Institute Receives ... - PR Newswire (press release)

SDSU College of Agriculture and Biological Sciences

BROOKINGS The South Dakota State University Swine Club, along with various campus organizations and industry partners, will host a presentation by Julie Borlaug on April 12 to address the controversy surrounding the use of biotechnology in food production.

Her presentation takes place at 7:00 p.m. at the Performing Arts Center on the SDSU campus.

There is no charge to attend the event, but a free-will donation of canned goods for the Brookings Food Pantry is appreciated.

As society moves farther and farther away from modern production agriculture, it creates a potential disconnect between the people who raise the food and the ones that consume it, explains Madelyn Regier, SDSU Swine Club president and an agricultural education and animal science major. The SDSU Swine Club believes that it is essential for consumers and the general public to better understand modern agriculture so we can all work together in feeding the worlds growing population in a safe and sustainable manner.

Julie Borlaug is the granddaughter of Norman E. Borlaug, known as the father of the Green Revolution. She serves as the assistant director of partnerships at the Borlaug Institute for International Agriculture at Texas A&M University.

Since the passing of her grandfather, Julie has worked to continue his legacy through developing agricultural partnerships between public, private and philanthropic groups to further the Borlaug legacy and expand upon his mission to feed the worlds hungry. She has spent her career in the nonprofit sector and has worked for organizations such as the Salvation Army and the American Cancer Society as director of development. She recently transitioned into her new role as assistant director of partnerships in order to champion her grandfathers legacy and lend a voice to his desire to create more successful collaborative partnerships between the public and private sectors in order to ensure the continuation of breakthroughs in international agriculture.

Co-sponsors of this event include the South Dakota Pork Producers Council, the SDSU College of Agriculture and Biological Sciences, the South Dakota Farm Bureau, the South Dakota Soybean Research & Promotion Council, the South Dakota Wheat Commission, and SDSU Collegiate FFA.

The SDSU Swine Club is a student-led organization dedicated to generating interest, building understanding and providing opportunities for growth in the swine industry. For more information contact Madelyn Regier, SDSU Swine Club President, at [emailprotected], or (507) 822-5944, or Swine Club Advisor Robert Thaler, professor and SDSU Extension Swine Specialist at [emailprotected], (605) 688-5435.

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SDSU Speaker to address topic Biotechnology: Friend or Foe in Fighting World Hunger - Farm Forum

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Cellect Biotechnology Ltd. (NASDAQ: APOP) announced today that it has received a formal notice of Intention to Grant for a patent (Application No. 11751949.6-1466) covering a key method of treatment from the European Patent Office. The allowed claims relate to the engineering of regulatory immune cells with enhanced apoptotic activity to be used for immunomodulation in treating or preventing immune-related disorders.

Earlier this year, Cellect received a Notice of Allowance from the U.S. Patent & Trademark Office for the same patent.

The patent is expected to protect Cellects technology and method when used to treat multiple medical conditions with significant unmet needs, such as diabetes, inflammatory bowel disease, graft-versus-host disease and transplant rejection. The patent covers a segment of cell-based therapeutics that is separate from the one Cellect is currently working in, is relevant to the fast-growing class of immune therapies and Cellect believes may create an opportunity to enhance Cellects pipeline.

Shai Yarkoni, Cellects CEO, commented, Now covering both the U.S. and the EU, this patent is a base for the future commercialization of our global business. Cellect has seven families of patents and patent applications to protect its core assets for enabling stem cell regenerative medicine. With this patent, Cellect has the opportunity to diversify its pipeline and open up new commercialization routes for additional market segments.

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Cellect Biotechnology (APOP) Granted European Patents Protecting Technology in Multiple Indications - StreetInsider.com

LOS ANGELES--(BUSINESS WIRE)--Puma Biotechnology, Inc. (Nasdaq: PBYI), a biopharmaceutical company, announced that results from an ongoing Phase II clinical trial of Puma's investigational drug PB272 (neratinib) were presented at the 2017 American Association for Cancer Research Annual Meeting (AACR) that is currently taking place in Washington, D.C. The presentation entitled, Neratinib in HER2 or HER3 mutant solid tumors: SUMMIT, a global, multi-histology, open-label, phase 2 basket study, was presented as a plenary session by David Hyman, M.D., Director of Developmental Therapeutics at Memorial Sloan Kettering Cancer Center (MSK), and principal investigator of the trial.

The Phase II SUMMIT basket trial is an open-label, multicenter, multinational study to evaluate the safety and efficacy of PB272 administered daily to patients who have solid tumors with activating HER2 or HER3 mutations. All patients received loperamide (16 mg per day initially) prophylactically for the first cycle of treatment in order to reduce the neratinib-related diarrhea.

Included in the presentation were data on 141 patients enrolled in the neratinib monotherapy arm of the trial, including 124 patients with HER2 mutations and 17 patients with HER3 mutations. This included patients with 21 unique tumor types, with the most common being breast, lung, bladder and colorectal cancer. There were also 30 distinct HER2 and 12 distinct HER3 mutations observed among these patients, with the most frequent HER2 variants involving S310, L755, A755_G776insYVMA and V777.

In the HER2-mutant cohort, clinical responses were observed in tumors with S310, L755, V777, P780_Y781insGSP and A775_G776insYVMA mutations. When stratified by tumor type, responses were observed in patients with breast, cervical, biliary, salivary and non-small-cell lung cancers, which led to cohort expansions in these tumor types. No activity was observed in the HER3-mutant cohort. A more detailed presentation of the data is presented in Table 1 below and a copy of the full presentation is available on the Puma Biotechnology website.

HER2mut

HER2mut

HER2mut

HER2mut

HER2mut

HER2mut

HER3mut

Lung

(95% CI)

rate, n (%)

(95% CI)

20.1

(95% CI)

(0.5--NA)

The neratinib safety profile observed in the SUMMIT study is consistent with that observed previously in metastatic patients with HER2 amplified tumors. With anti-diarrheal prophylaxis and management, diarrhea was not a treatment-limiting side effect in SUMMIT. The interim safety results of the study showed that the most frequently observed adverse event was diarrhea. For the 141 patients enrolled in the neratinib monotherapy arm with safety data available, 31 patients (22%) reported grade 3 diarrhea. The median duration of grade 3 diarrhea for those patients was 2 days. 4 patients (2.8%) permanently discontinued neratinib due to diarrhea and 21 patients (14.9%) temporarily discontinued neratinib due to diarrhea and then restarted after the diarrhea subsided.

Dr. David Hyman stated, "Neratinib showed signs of clinical activity in several of the cohorts in the SUMMIT trial. The safety profile of the drug was manageable and the diarrhea was not treatment-limiting with appropriate prophylaxis and management. We look forward to completing enrollment in the ongoing cohorts in the study and continuing to utilize the basket trial design to explore possible treatment options for these select patient populations.

"The basket-trial design we are utilizing for SUMMIT is enabling us to evaluate the clinical potential of neratinib in patients with specific mutation-types rather than limiting exploration to one tumor type at a time. It is an efficient approach that is generating clinically meaningful information to guide targeted therapy across a broad spectrum of tumor types with HER mutations, including in patients with rare tumors who may not otherwise have access to investigational therapies, said Alan H. Auerbach, Chief Executive Officer and President of Puma Biotechnology. We are very pleased with the preliminary activity seen with neratinib in the SUMMIT trial. We look forward to advancing this targeted compound into further clinical development in multiple HER2 mutant tumor types, both as monotherapy and in novel combinations."

About Puma Biotechnology

Puma Biotechnology, Inc. is a biopharmaceutical company with a focus on the development and commercialization of innovative products to enhance cancer care. The Company in-licenses the global development and commercialization rights to three drug candidatesPB272 (neratinib (oral)), PB272 (neratinib (intravenous)) and PB357. Neratinib is a potent irreversible tyrosine kinase inhibitor that blocks signal transduction through the epidermal growth factor receptors, HER1, HER2 and HER4. Currently, the Company is primarily focused on the development of the oral version of neratinib, and its most advanced drug candidates are directed at the treatment of HER2-positive breast cancer. The Company believes that neratinib has clinical application in the treatment of several other cancers as well, including non-small cell lung cancer and other tumor types that over-express or have a mutation in HER2. Further information about Puma Biotechnology can be found at http://www.pumabiotechnology.com.

Forward-Looking Statements:

This press release contains forward-looking statements, including statements regarding the development of the Companys drug candidates. All forward-looking statements included in this press release involve risks and uncertainties that could cause the Company's actual results to differ materially from the anticipated results and expectations expressed in these forward-looking statements. These statements are based on current expectations, forecasts and assumptions, and actual outcomes and results could differ materially from these statements due to a number of factors, which include, but are not limited to, the fact that the Company has no product revenue and no products approved for marketing; the Company's dependence on PB272, which is still under development and may never receive regulatory approval; the challenges associated with conducting and enrolling clinical trials; the risk that the results of clinical trials may not support the Company's drug candidate claims; even if approved, the risk that physicians and patients may not accept or use the Company's products; the Company's reliance on third parties to conduct its clinical trials and to formulate and manufacture its drug candidates; the Company's dependence on licensed intellectual property; and the other risk factors disclosed in the periodic and current reports filed by the Company with the Securities and Exchange Commission from time to time, including the Company's Annual Report on Form 10-K for the year ended December 31, 2016. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. The Company assumes no obligation to update these forward-looking statements, except as required by law.

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Puma Biotechnology Presents Results from the Phase II SUMMIT Trial of PB272 for ERBB2 (HER2) Mutant, HER2 Non ... - Business Wire (press release)