Funding, Deals & Partnerships: BIOLOGICS & MEDICAL DEVICES; BioMed e-Series; Medicine and Life Sciences Scientific Journal – http://PharmaceuticalIntelligence.com
37th Annual J.P. Morgan HEALTHCARE CONFERENCE: #JPM2019 for Jan. 8, 2019; Opening Videos, Novartis expands Cell Therapies, January 7 – 10, 2019, Westin St. Francis Hotel | San Francisco, California, Volume 2 (Volume Two: Latest in Genomics Methodologies for Therapeutics: Gene Editing, NGS and BioInformatics, Simulations and the Genome Ontology), Part 1: Next Generation Sequencing (NGS)
37th Annual J.P. Morgan HEALTHCARE CONFERENCE: #JPM2019 for Jan. 8, 2019; Opening Videos, Novartis expands Cell Therapies, January 7 – 10, 2019, Westin St. Francis Hotel | San Francisco, California
Reporter: Stephen J. Williams, PhD
The annual J.P. Morgan Healthcare Conference is the largest and most informative healthcare investment symposium in the industry, bringing together industry leaders, emerging fast-growth companies, innovative technology creators, and members of the investment community.
Joe Biden on the Fight Against Cancer
Former Vice President of the United States joined the J.P. Morgan Healthcare Conference to discuss cancer initiatives.
Novartis Talks Move to Cell and Gene Therapies at JPM
Published: Jan 08, 2019By Alex Keown from Biospace.com
Denis Linine / Shutterstock
Following a strong post-hoc analysis of mid-stage data in the fall of 2018, Novartis announced this morning the company’s experimental humanized anti-P-selectin monoclonal antibody was crizanlizumab granted Breakthrough Therapy Status by the U.S.Food and Drug Administration (FDA).
Crizanlizumab received the designation as a treatment for the prevention of vaso-occlusive crises (VOCs) in patients of all genotypes with sickle cell disease (SCD). VOCs, which can be extremely painful for patients, happen when multiple blood cells stick to each other and to blood vessels, causing blockages.
The designation was awarded following results from the Phase II SUSTAIN trial, which showed that crizanlizumab reduced the median annual rate of VOCs leading to health care visits by 45.3 percent compared to placebo. The SUSTAIN study also showed that crizanlizumab significantly increased the percentage of patients who did not experience any VOCs vs placebo, 35.8 percent vs. 16.9 percent.
The FDA designation came one day after the Swiss pharma giant laid out its map for a future of success, sustainability and, if things work out, respect from consumers. In an interview with CNBC Monday, Novartis Chief Executive Officer Vas Narasimhan noted that the company is looking to become an entity that doesn’t draw its profits from treating disease, but will make money by providing cures. He pointed to the moves Novartis has made toward gene and cellular therapies that have the potential to cure patients of various diseases in what many researchers hope could be a “one-and-done” treatment. Narasimhan told CNBC that cures are what society wants and that is something they will value. The challenge will be determining the payment system.
As an example, the company is eying potential approval of a gene therapy for spinal muscular atrophy (SMA), a fatal genetic disease marked by progressive, debilitating muscle weakness in infants and toddlers. Novartis’ gene therapy Zolgensma is expected to be approved by the FDA this year and could have a price tag of between $4 and $5 million. While significantly high, non-profit SMA groups have already suggested that the gene therapy treatment could be more cost-effective than Spinraza, the only approved SMA treatment on the market.
During its presentation at J.P. Morgan, Novartis pointed to the moves it has made as the company pivots to this future of gene and cell therapies. The presentation noted that over the course of 2018, the company made several deals to sell off non-essential businesses, such as the $13 billion sale of its share of a consumer health business to partner GlaxoSmithKline. Not only that, but Novartis also made significant acquisitions to reshape its portfolio, including the $8.7 billion acquisition of AveXis for the SMA gene therapy. The deal for AveXis wasn’t the only gene therapy deal the company struck. Novartis began 2018 with a deal for Spark Therapeutics’ gene therapy Luxturna, a one-time gene therapy to restore functional vision in children and adult patients with biallelic mutations of the RPE65 (retinal pigment epithelial 65 kDa protein) gene.
In his interview with CNBC, Narasimhan said the company is about “platforms,” which also includes radio-ligand therapy. The company forged ahead in that area with two acquisitions, Advanced Accelerator Applications and Endocyte. Radiopharmaceuticals like Endocyte’s Lu-PSMA-617 are innovative medicinal formulations containing radioisotopes used clinically for both diagnosis and therapy. When the Endocyte deal was announced, Novartis noted the field is expected to become an increasingly important treatment option for patients, as well as a key growth driver for the company’s oncology business.
Other posts on the JP Morgan 2019 Healthcare Conference on this Open Access Journal include:
NIH SBIR Funding Early Ventures: September 26, 2018 sponsored by Pennovation
Stephen J. Williams PhD, Reporter
Penn Center for Innovation (Pennovation) sponsored a “Meet with NCI SBIR” program directors at University of Pennsylvania Medicine Smilow Center for Translational Research with a presentation on advice on preparing a successful SBIR/STTR application to the NCI as well as discussion of NCI SBIR current funding opportunities. Time was allotted in the afternoon for one-on-one discussions with NCI SBIR program directors.
To find similar presentations and one-on-one discussions with NCI/SBIR program directors in an area nearest to you please go to their page at:
For more complete information on the NCI SBIR and STTR programs please go to their web page at: https://sbir.cancer.gov/about
A few notes from the meeting are given below:
In 2016 the SBIR/STTR 2016 funded $2.5 billion (US) of early stage companies; this is compared to the $6.6 billion invested in early stage ventures by venture capital firms so the NCI program is very competitive with alternate sources of funding
It was stressed that the SBIR programs are flexible as far as ownership of a company; SBIR allows now that >50% of the sponsoring company can be owned by other ventures; In addition they are looking more favorably on using outside contractors and giving leeway on budgetary constraints so AS THEY SUGGEST ALWAYS talk to the program director about any questions you may have well before (at least 1 month) you submit. More on eligibility criteria is found at: https://sbir.cancer.gov/about/eligibilitycriteria
STTR should have strong preliminary data since more competitive; if don’t have enough go for an R21 emerging technologies grant which usually does not require preliminary data
For entities outside the US need a STRONG reason for needing to do work outside the US
Budget levels were discussed as well as the waiver program, which allows for additional funds to be requested based on criteria set by NCI (usually for work that is deemed high priority or of a specialized nature which could not be covered sufficiently under the standard funding limits) as below:
Phase I: 150K standard but you can get waivers for certain work up to 300K
Phase II: 1M with waiver up to 2M
Phase IIB waiver up to 4M
You don’t need to apply for the waiver but grant offices may suggest citing a statement requesting a waiver as review panels will ask for this information
Fast Track was not discussed in the presentation but for more information of the Fast Track program please visit the website
NCI is working hard to cut review times to 7 months between initial review to funding however at beginning of the year they set pay lines and hope to fund 50% of the well scored grants
NCI SBIR is a Centralized system with center director and then program director with specific areas of expertise: Reach out to them
IMAT Program and Low-Resource Setting new programs more suitable for initial studies and also can have non US entities
Phase IIB Bridge funding to cross “valley of death” providing up to 4M for 2-3 years: most were for drug/biological but good amount for device and diagnostics
Also they have announced administrative supplements for promoting diversity within a project: can add to the budget
FY18 Contracts Areas
3 on biotherapies
2 imaging related
2 on health IT
4 on radiation therapy related: NOTE They spent alot of time discussing the contracts centered on radiation therapy and seems to be an area of emphasis of the NCI SBIR program this year
4 other varied topics
Breakdown of funding
>70% of NCI SBIR budget went to grants (for instance Omnibus grants); about 20-30% for contracts; 16% for phase I and 34 % for phase II ;
ALSO the success rate considerably higher for companies that talk to the program director BEFORE applying than not talking to them; also contracts more successful than Omnibus applications
Take Advantage of these useful Assistance Programs through the NIH SBIR Program (Available to all SBIR grantees)
The Niche Assessment Program is designed to help small businesses “jump start” their commercialization efforts. All active HHS (NIH, CDC, FDA) SBIR/STTR Phase I awardees and Phase I Fast-Track awardees (by grant or contract) are eligible to apply. Registration is on a first-come, first-serve basis!
The Niche Assessment Program provides market insight and data that can be used to help small businesses strategically position their technology in the marketplace. The results of this program can help small businesses develop their commercialization plans for their Phase II application, and be exposed to potential partners. Services are provided by Foresight Science & Technology of Providence, RI.
Technology Niche Analyses® (TNA®) are provided by Foresight, for one hundred and seventy-five (175), HHS SBIR/STTR Phase I awardees. These analyses assess potential applications for a technology and then for one viable application, it provides an assessment of the:
Needs and concerns of end-users;
Competing technologies and competing products;
Competitive advantage of the SBIR/STTR-developed technology;
Market size and potential market share (may include national and/or global markets);
Barriers to market entry (may include but is not limited to pricing, competition, government regulations, manufacturing challenges, capital requirements, etc.);
Market drivers;
Status of market and industry trends;
Potential customers, licensees, investors, or other commercialization partners; and,
NIH CAP is a 9-month program that is well-regarded for its combination of deep domain expertise and access to industry connections, which have resulted in measurable gains and accomplishments by participating companies. Offered since 2004 to address the commercialization objectives of companies across the spectrum of experience and stage, 1000+ companies have participated in the CAP. It is open only to HHS/NIH SBIR/STTR Phase II awardees, and 80 slots are available each year. The program enables participants to establish market and customer relevance, build commercial relationships, and focus on revenue opportunities available to them.
The I-Corps program provides funding, mentoring, and networking opportunities to help commercialize your promising biomedical technology. During this 8-week, hands-on program, you’ll learn how to focus your business plan and get the tools to bring your treatment to the patients who need it most.
Program benefits include:
Funding up to $50,000 to cover direct program costs
Training from biotech sector experts
Expanding your professional network
Building the confidence and skills to create a comprehensive business model
Gaining years of entrepreneurial skills in only weeks.
ICORPS is an Entrepreneurial Program (8 week course) to go out talk to customers, get assistance with business models, useful resource which can guide the new company where they should focus on for the commercialization aspect
The SBIR/STTR Applicant Assistance Program (AAP) is aimed at helping eligible small R&D businesses and individuals successfully apply for Phase I SBIR/STTR funding from the National Cancer Institute (NCI), National Institute for Neurological Disorders and Stroke (NINDS), National Heart, Lung and Blood Institute (NHLBI). Participation in the AAP will be funded by the NCI, NINDS, and NHLBI with NO COST TO PARTICIPANTS. The program will include the following services:
Needs Assessment/Small Business Mentoring
Phase I Application Preparation Support
Application Review
Team/Facilities Development
Market Research
Intellectual Property Consultation
For more details about the program, please refer to NIH Notice NOT-CA-18-072.
These programs are free for first time grant applicants and must not have been awarded previous SBIR
Peer Learning Webinar Series goal to improve peer learning .Also they are starting to provide Regulatory Assistance (see below)
NIH also provides Mentoring programs for CEOS and C level
Application tips
Start early: and obtain letters of collaboration
Build a great team: PI multi PI, consider other partners to fill gaps (academic, consultants, seasoned entrepreneurs (don’t need to be paid)
They will pre review 1 month before due date, use NIH Project Reporter to view previous funded grants
Specify study section in SF to specify areas of expertise for review
Specific aims are very important; some of the 20 reviewers focus on this page (describes goals and milestones as well; spend as much time on this page as the rest of the application
Letters of support from KOLs are important to have; necessary from consultants and collaborators; helpful from clinicians
Have a phase II commercialization plan
Note for non US clinical trials: They will not fund nonUS clinical trials; the company must have a FWA
SBIR budgets defined by direct costs; can request a 7% fee as an indirect cost; and they have a 5,000 $ technical assistance program like regulatory consultants but if requested can’t participate in NIH technical assistance programs so most people don’t apply for TAP
They are trying to change the definition of innovation as also using innovative methods (previously reviewers liked tried and true methodology)
Discussion with Monique Pond, Ph.D. on Establishment of a Regulatory Assistance Program for NCI SBIR
I was able to sit down with Dr. Monique Pond, AAAS Science & Technology Policy Fellow, Health Scientist within the NCI SBIR Development Center to discuss the new assistance program in regulatory affairs she is developing for the NCI SBIR program. Dr Pond had received her PhD in chemistry from the Pennsylvania State University, completed a postdoctoral fellow at NIST and then spent many years as a regulatory writer and consultant in the private sector. She applied through the AAAS for this fellowship and will bring her experience and expertise in regulatory affairs from the private sector to the SBIR program. Dr. Pond discussed the difficulties that new ventures have in formulating regulatory procedures for their companies, the difficulties in getting face time with FDA regulators and helping young companies start thinking about regulatory issues such as pharmacovigilence, oversight, compliance, and navigating the complex regulatory landscape.
In addition Dr. Pond discussed the AAAS fellowship program and alternative career paths for PhD scientists.
A formal interview will follow on this same post.
Other articles on this OPEN ACCESS JOURNAL on Funding for Startups and Early Ventures are given below:
Economic Potential of a Drug Invention (Prof. Zelig Eshhar, Weitzman Institute, registered the patent) versus a Cancer Drug in Clinical Trials: CAR-T as a Case in Point, developed by Kite Pharma, under Arie Belldegrun, CEO, acquired by Gilead for $11.9 billion, 8/2017.
Curator: Aviva Lev-Ari, PhD, RN
Article ID #245: Economic Potential of a Drug Invention (Prof. Zelig Eshhar, Weitzman Institute, registered the patent) versus a Cancer Drug in Clinical Trials: CAR-T as a Case in Point, developed by Kite Pharma, under Arie Belldegrun, CEO, acquired by Gilead for $11.9 billion, 8/2017. Published on 10/4/2017
WordCloud Image Produced by Adam Tubman
UPDATED on 2/21/2021
The Announcement of the 2021 Dan David Prize Laureates – YouTube – PRIZE $1MM per Winner for contributors to CAR-T MOA leading to development of immunotherapy anti cancer drugs
Prof. Zelig Eshhar – Weitzmann Institute his student Arie S. Belldegrun was CEO at Kate Pharmaceutics sold to Gilead for $12Bil
Prof. Carl June of UPenn
Steven Rosenberg, PhD, NIH
UPDATED on 10/15/2020
Hooked by the science, Arie Belldegrun joins a group of influentials who believe Dewpoint may have the key to the next big thing in biotech John Carroll Editor & Founder Amir Nashat knew he had years of preclinical work to do when he talked to me at the beginning of 2019 about Dewpoint Therapeutics and its rare focus on the role bimolecular condensates could play in crafting a wide-ranging pipeline of therapeutics.
The Cambridge, MA-based Dewpoint team, which will now double in size over the next year, doesn’t have a late-stage preclinical program it can shove into the clinic. The biotech is investing in neurodegeneration, cancer, cardiovascular and other areas for a platform that could, eventually, have extensive applications. But asked about a timeline to proof-of-concept data, Nashat frankly estimates that it will take 4-5 years to birth some hard human data. The money should get them through 3 years and a considerable de-risking approach to their preclinical efforts
The CSO is Mark Murcko, an experienced and well known startup player.
Mark Murcko “When I think about new companies a lot of it is about timing; is it too soon or too late?” Murcko notes enthusiastically in our interview. “Is there enough information available to make you think you can take that and use it toward new drugs? Five years ago it was too early, too nascent.”
Now, Murcko adds, seems like a great time to give this a go.
Gilead is writing off its anti-BCMA CAR-T for multiple myeloma, eliminating one of the many efforts focused on that target and driving a big part of the company’s $820 million impairment charge for R&D in the 4th quarter of last year. But this could just be a taste of what’s to come.
A $12 billion buyout of Kite Pharma in 2017 brought with it the CAR-T therapy Yescarta (axicabtagene ciloleucel) and a foothold in immuno-oncology. But last year’s results make clear that return on that investment will be slow to materialize.
Buried in among Gilead’s fourth-quarter results statement is a line revealing it has abandoned an anti-BCMA cell therapy for multiple myeloma, part of its $12 billion acquisition of Kite Pharma.
The failed KITE-585 program and other costs associated with the acquisition resulted in a whopping $820 million impairment charge in the quarter and add to analyst speculation that with sales of approved CAR-T Yescarta still disappointing, Gilead may have to write down the value of the Kite deal entirely, according to a Bloomberg report.
Gilead’s decision to drop the KITE-585 CAR-T program reflects the increasing competition in the anti-BCMA category and doesn’t come out of the blue. The company said at the J.P. Morgan conference (JPM) last month that it would only press ahead with development of KITE-585 if its profile was very compelling.
Robert W. Baird & Co. analyst Brian Skorney says Gilead may have to write down the deal, which the company values at $11.9 billion. That means lowering the projections on its balance sheet, if the multi billion-dollar sales Wall Street expects don’t materialize. The long-time bull cut his rating on the stock to neutral in July following the management exodus.
It’s also clear that public investors did quite well in these deals – unlike some outcomes, both private and public investors can only be happy with these deals. Kite’s IPO investors made over a whopping 10x, and Juno’s nearly a 3.6x (in 3 years, so still a very strong public market return). Even the follow-on financing participants made handsome returns: both Kite’s and Juno’s follow-on financings about 4-6 months prior to acquisition delivered a 2x return in a short period. What’s clear is that participating at any point only these price curves was a positive for investors. Obviously that doesn’t always happen, but great to see when it does.
A final takeaway is that there is “no one size fits all” for how to build business models that can work in biotech these days, even to get to similar product and patient outcomes. While Kite and Juno have remarkably similar products, similar platforms, and similar overall acquisition valuations, the stories were built quite differently when it comes to financing their growth.
Kite Pharma, under Arie Belldegrun, CEO, acquired by Gilead for $11.9 billion, 8/2017.
Kite’s Yescarta™ (Axicabtagene Ciloleucel) Becomes First CAR T Therapy Approved by the FDA for the Treatment of Adult Patients With Relapsed or Refractory Large B-Cell Lymphoma After Two or More Lines of Systemic Therapy
— Manufacturing Success Rate of 99 Percent in ZUMA-1 Pivotal Trial with a Median 17 Day Turnaround Time —
CAR T therapy is a breakthrough in hematologic cancer treatment in which a patient’s own T cells are engineered to seek and destroy cancer cells. CAR T therapy is manufactured specifically for each individual patient.
“The FDA approval of Yescarta is a landmark for patients with relapsed or refractory large B-cell lymphoma. This approval would not have been possible without the courageous commitment of patients and clinicians, as well as the ongoing dedication of Kite’s employees,” said Arie Belldegrun, MD, FACS, Founder of Kite. “We must also recognize the FDA for their ability to embrace and support transformational new technologies that treat life-threatening illnesses. We believe this is only the beginning for CAR T therapies.”
“Today is an important day for patients with relapsed or refractory large B-cell lymphoma who have run out of options and have been waiting for new treatments that may help them in their fight against cancer,” said John Milligan, PhD, President and Chief Executive Officer of Gilead Sciences. “With the combined innovation, talent and drive of the Kite and Gilead teams, we will rapidly advance cell therapy research and aim to bring new options to patients with many other types of cancer.”
The list price of Yescarta in the United States is $373,000.
Yescarta has been granted Priority Medicines (PRIME) regulatory support for DLBCL in the European Union. A Marketing Authorization Application (MAA) for axicabtagene ciloleucel is currently under review with the European Medicines Agency (EMA) and potential approval is expected in the first half of 2018.
The approval of Yescarta is supported by data from the ZUMA-1 pivotal trial. In this study, 72 percent of patients treated with a single infusion of Yescarta (n=101) responded to therapy (overall response rate) including 51 percent of patients who had no detectable cancer remaining (complete remission; 95% CI: 41, 62). At a median follow-up of 7.9 months, patients who had achieved a complete remission had not reached the estimated median duration of response (95% CI: 8.1 months, not estimable [NE]).
In the study, 13 percent of patients experienced grade 3 or higher cytokine release syndrome (CRS) and 31 percent experienced neurologic toxicities. The most common (≥ 10%) Grade 3 or higher reactions include febrile neutropenia, fever, CRS, encephalopathy, infections-pathogen unspecified, hypotension, hypoxia and lung infections. Serious adverse reactions occurred in 52% of patients and included CRS, neurologic toxicity, prolonged cytopenias (including neutropenia, thrombocytopenia and anemia), and serious infections. Fatal cases of CRS and neurologic toxicity occurred. FDA approved Yescarta with a Risk Evaluation and Mitigation Strategy.
Yescarta Indication
Yescarta is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma.
Diffuse large B-cell lymphoma (DLBCL) is the most common aggressive non-Hodgkin lymphoma (NHL), accounting for three out of every five cases. In the United States each year, there are approximately 7,500 patients with refractory DLBCL who are eligible for CAR T therapy. Historically, when treated with the current standard of care, patients with refractory large B-cell lymphoma had a median overall survival of approximately six months, with only seven percent attaining a complete response. Currently, patients with large B-cell lymphoma in second or later lines of therapy have poor outcomes and greater unmet need, since nearly half of them either do not respond or relapse shortly after transplant.
“With CAR T therapy, we are reengineering a patient’s own immune system to detect and kill cancer cells, and the results have been impressive,” said Frederick L. Locke, MD, ZUMA-1 Co-Lead Investigator and Vice Chair of the Department of Blood and Marrow Transplant and Cellular Immunotherapy at Moffitt Cancer Center in Tampa, Florida. “Many of the patients that received CAR T therapy had already relapsed several times with traditional treatments such as chemotherapy or hematopoietic stem cell transplant. Now, thanks to this new therapy many patients are in remission for months.”
“This therapy is a new option for patients with relapsed or refractory large B-cell lymphoma who have run out of treatment options and face a dire prognosis,” said Louis J. DeGennaro, PhD, President and Chief Executive Officer of The Leukemia & Lymphoma Society (LLS). “Early on, LLS recognized the potential of CAR T therapy and we are proud to be part of making this historic approval possible.”
“Engineered cell therapies like Yescarta represent the potential for a changing treatment paradigm for cancer patients,” said David Chang, MD, PhD, Worldwide Head of Research and Development and Chief Medical Officer at Kite. “Together, Gilead and Kite will accelerate studies of CAR T therapy in multiple blood cancers and advance other cell therapy approaches for solid tumors, with the goal of helping patients with diverse cancers benefit from this new era of personalized cancer therapy.”
ABOUT Drug Invention (Prof. Zelig Eshhar, Weitzman Institute, registered the patent)
ABOUT Gilead’s $12 billion buy of Kite Pharma
ABOUT the Drug Development process and the COMMERCIALIZATION GENIUS of Arie Belldegrun – Interviewed by Globes
ABOUT the Perspective of Drug Invention (Prof. Zelig Eshhar, Weitzman Institute, registered the patent) following the Gilead’s $12 billion buy of Kite Pharma – Interviewed by Globes
ABOUT the Economic significance of Kite Pharma Acquisition for the Venture Capital Investment in Biotech in Israel
I agree with Prof. Zelig Eshhar that this Case in Point is “one more invention, or parts of an invention, came from an Israeli laboratory (at the Weizmann Institute in this case) and fell into foreign hands. It is another enormous missed opportunity in the field of biomedicine and ethical drugs.”
I agree with Prof. Zelig Eshhar that this Case in Point should have been a TEVA commercialization effort. It is a regrettable reality that the development and the manufacturing will not benefit the State of Israel, home of the Weitzman Institute where the Patentable invention took place by Prof. Zelig Eshhar.
It is to be acknowledged that for CAR-T – the process of treatment using the drug – personalized genetic engineering of each patient’s cells – a grafting process with no precedent in the pharmaceutical industry (Juno has related process) – is bringing to the Oncology arena a NOVEL treatment for hematological malignancies cancer patients
I agree with Prof. Zelig Eshhar that the Barriers in the pharmaceutical industry are especially high. Developing ethical drugs is a process requiring huge amounts of time, patience, money, and failures. It is exactly, therefore, all need to acknowledge that the Drug Development process and the COMMERCIALIZATION GENIUS of Arie Belldegrun is inseparable from the breakthrough invention of Prof. Zelig Eshhar to develop the drug from the Lab bench to the FDA accelerated process of Drug approval.
The Biotech industry in Israel needs to develop more MDs, PhDs with the level of training of Arie Belldegrun and with his entrepreneur acumen, keenness and depth of perception, discernment, discrimination especially in practical aspects of Translation Medicine, Clinical Research, Clinical Trial Design and abilities to engage in innovating the FDA processes.
The Biotech industry in US needs to develop more MDs, PhDs with the level of training of Prof. Zelig Eshhar to carry the scientific gravitas and the creativity to become inventors of novel drugs.
ABOUT Drug Invention (Prof. Zelig Eshhar, Weitzman Institute, registered the patent)
Pioneers of Cancer Cell Therapy: Turbocharging the Immune System to Battle Cancer Cells — Success in Hematological Cancers vs. Solid Tumors
FDA has approved the world’s first CAR-T therapy, Novartis for Kymriah (tisagenlecleucel) and Gilead’s $12 billion buy of Kite Pharma, no approved drug and Canakinumab for Lung Cancer (may be?)
ABOUT the Perspective of Drug Invention (Prof. Zelig Eshhar, Weitzman Institute, registered the patent) following the Gilead’s $12 billion buy of Kite Pharma – Interviewed by Globes
Kite Pharma was a $12b missed opportunity for Israel – Interview with Professor Zelig Eshhar
Some Israeli media headlines depicted Kite as an Israeli exit. But it is a US company that does no business in Israel and has no employees here.
Professor Zelig Eshhar is the man who registered the patent on the cancer treatment drug developed by Kite Pharma, recently acquired by Gilead for $11.9 billion.
“Globes”: Do you believe that any party in Israel could have financed the product and brought it where it is today?
Eshhar: “On the one hand, yes. The level of investment in the product before it reached Nasdaq was something that an Israeli concern could certainly have financed. On the other hand, Kite Pharma founder Professor Arie Belldegrun, with his energy and connections, brought it to a completely different place (Eshhar previously tried to interest various concerns in Israel in financing the drug, but all of them told him that it was too early, or that the product was not effective enough, E.T.).
Was the development already in its final form in the 1980s?
“Almost. I went to the National Institutes of Health (NIH), where I met for the first time Professor Steven Rosenberg, who later became the first doctor to conduct clinical trials with the technology. Rosenberg heard about my technology, and offered me exceptional conditions. We set up a team there, and had the best of everything. I only wish I had it now.”
They say that Belldegrun didn’t want the product at first. Today, he’s devoting all his efforts to it.
“When Arie founded Cougar Biotechnology, which developed a drug for prostate cancer, and was eventually sold to Johnson & Johnson for $1 billion, I contacted him and offered him the technology, but he was busy with Cougar’s product, and maybe didn’t think that he had enough capital for such a production. Only after he sold Cougar did he get back to me with an offer to buy the rights to my patent. At that time (2009-2010), the technology was already arousing great interest, and there were negotiations with several large companies.” (from an April 2015 “Globes” interview with Eshhar, who was awarded the Israel Prize).
Israelis can be very provincial. In at least some of the media headlines, Kite Pharma was portrayed as a “huge Israeli exit,” and the impression was given that it was an Israeli company. The truth is very different. Kite Pharma is not an Israeli company; it is a 100% US company. It does no business in Israel; its nearly $12 billion exit has no significance whatsoever for the Israeli economy, and will contribute nothing to it: no jobs, and the tax contribution will be marginal, and certainly not on the scale of Mobileye, for example. Let me say it again: Kite Pharma does not have even one employee in Israel (and has no reason to employ anyone here), and certainly does not pay taxes in Israel. There are no Israelis on the company’s management team or board of directors. This is a US company for all intents and purposes. The word “Israel” appears exactly once in the company’s full documents – where registration of the company’s patents is concerned. The fact that every story about the company mentions the small holdings of several Israeli financial institutions in it is a bad joke. Everyone should remember that Israeli financial institutions are of course entitled to invest in any foreign share, such as Google, Amazon, Facebook, Apple Computers, and so forth. Kite Pharma is one of those foreign shares, and nothing more.
Of course, there is cause for pride in the fact that Eshhar, owner of the patent for Kite Pharma’s drug is “one of ours,” i.e. an Israeli researcher at the Weizmann Institute of Science. Another source of pride is Kite Pharma founder and CEO Arie Belldegrun, a graduate of the Hebrew University Medical School who did his post-doctorate at the Weizmann Institute, where he met Eshhar, and Kite Pharma later bought his patent for the cancer drug. Belldegrun was also a director at Teva Pharmaceutical Industries Ltd. (NYSE: TEVA; TASE: TEVA) until recently, resigning at the peak of that company’s crisis. Beyond this Israeli connection, however, the Kite Pharma exit has no great significance for Israel. All it means is that one more invention, or parts of an invention, came from an Israeli laboratory (at the Weizmann Institute in this case) and fell into foreign hands. It is another enormous missed opportunity in the field of biomedicine and ethical drugs.
It is necessary to realize that while Belldegrun is indeed a big biomedical brain with many achievements in the field, he is a brain that has left Israel, and we all have to ask ourselves why he left, why Kite Pharma is not an Israeli company, and why its (as yet non-existent) product was not developed in Israel and will not be manufactured there. The headline in Israel for the Kite Pharma exit should ask why Israel lost out on it, even though the patent came from Israeli laboratories, albeit with US cooperation.
Belldegrun is likely to keep his experiences on the Teva board of directors to himself. Of all the directors in the company, what he has to say is the most interesting, but he is unlikely to divulge what happened there with the inflated deal with Allergan, and exactly what he said at the board of directors meeting that approved the deal that led Teva into its current major crisis. The Kite Pharma exit and his other exits only highlight the lost opportunity. Kite Pharma, still without a product and without approval for a product, was sold for $11.9 billion in cash. Teva yesterday hit another low point, with a market cap of $16 billion. It is simply inconceivable: a company with an enormous potential, but no product, is worth three quarters of a huge veteran company with at least dozens of products, including products in the ethical drug sector. Kite Pharma is actually one of the indirect reasons for Teva’s decline – for the fact that Teva, which could have been a hothouse for developments like Copaxone, chose a huge inflated gamble on the generics market – a gamble that is now jeopardizing Teva’s future and very existence.
It is true that developing drugs is a very long process, requires huge amounts of capital, and involves many failures, but Teva decided to neglect it, and when a major company like Teva neglects Israeli developments, there are enough competitors in the pharma industry ready to turn Israeli research into gold. Kite Pharma is one example of this research.
The Weizmann Institute is a fruitful source of biomedical research. According to previous estimates published in “Globes,” the Weizmann Institute gets NIS 1 billion each year in royalties on medical and other developments, amounting to half of its budget. Directly and indirectly, the Weizmann Institute, together with other universities in Israel, is responsible for tens of billions of pharmaceutical sales. Only a few billions of this, however, results from drugs developed in Israel, like Copaxone, and far less than that is also made in Israel. The reports by Yeda R&D Company Ltd., the technology transfer arm of the Weizmann Institute of Science, are top secret, and there is a good reason for that. Exposing them will only highlight the scale of the missed opportunities. Instead of these inventions providing a base for a major pharmaceutical industry here, the commercialization companies are benefiting only the inventors and the Weizmann Institute itself (that is certainly natural and legitimate, and they are entitled to it), even though the research infrastructure from which they sprung is Israeli know-how, as in the case of Eshhar.
Barriers in the pharmaceutical industry are especially high. Developing ethical drugs is a process requiring huge amounts of time, patience, money, and failures. When it succeeds, however, the profit is enormous – for the industry, the employees, and the state (provided that some tax is paid). For example, Pfizer’s peak sales of Lipitor, a very popular drug for reducing cholesterol and fat in the bloodstream, reached $11 billion, and its profit on the drug was $9 billion, before competition from a generic version began. In addition to money, a great deal of experience and marketing power is required, and that is the reason why most developments wind up in the hands of major companies like Pfizer, Merck, and others at some stage. After all these qualifying statements, everyone who celebrated Kite Pharma’s exit should weep over it – it is another part of the sale of Israeli know-how overseas for a mess of pottage. Instead of consolidating a splendid pharma industry here, Israel is selling the brains with their know-how to foreigners. More than anything else, Teva’s decline and the Kite Pharma exit epitomize this sad and dangerous trend.
Published by Globes [online], Israel Business News – www.globes-online.com – on August 30, 2017
ABOUT the Economic significance of Kite Pharma Acquisition for the Venture Capital Investment in Biotech in Israel
Israeli investors profit from $11.9b Kite acquisition
Pontifax fund and Israeli institutional investors will profit from the US personalized cancer drug company’s huge sale. Part of the technology was developed at the Weizmann Institute
Pharmaceutical company Gilead Sciences Inc. has announced that it will acquire US company Kite Pharma Inc., developer of personalized cancer treatment drugs, at a company value of $11.9 billion. This is one of the biggest ever acquisitions of a company whose products have not yet been approved for marketing. The company value for the acquisition reflects a 29% premium on the market price.
Kite Pharma has developed a new method for genetically engineering immune system cells, so that they will make a focused attack on the malignant tumor. The company was founded in the US by Israeli-American Professor Arie Belldegrun, who already has two exits to his credit. He is also a former director at Teva Pharmaceutical Industries Ltd. (NYSE: TEVA; TASE: TEVA) (whose current value is not much more than the value at which Kite Pharma, a company with no products approved for marketing yet, is being acquired).
A significant part of the technology on which the product is based was developed by Professor Zelig Eshhar of the Weizmann Institute of Science.
Kite Pharma is waiting for marketing approval of its first product, following a successful trial on 100 patients on a very abbreviated track for innovative cancer products. The product was initially designed for treatment of blood cancer, but it is now hoped that its use can later be expanded to treatment of other types of cancer. Gilead is making a big gamble, first of all that the US Food and Drug Administration (FDA) will fulfill its commitment to approve the product, even though the development plan it devised, together with the company, was very short and limited. The second gamble involves the process of treatment using the drug – personalized genetic engineering of each patient’s cells – a grafting process with no precedent in the pharmaceutical industry.
Speaking about the talks to sell Kite, Prof. Arie Belldegrun told “Globes.” “We handled like in the IDF 669 unit. Nobody knew anything. Nobody heard anything. We held meetings in places where nobody would see us. And before we announced it only five employees knew about it.”
Published by Globes [online], Israel Business News – www.globes-online.com – on August 28, 2017
Cancer Therapies: Metabolic, Genomics, Interventional, Immunotherapy and Nanotechnology in Therapy Delivery (Series C Book 2) – on Amazon since 5/18/2017
The top five in the business saw their collective spending jump by more than $5 billion, from 2015 to 2016, based on the annual numbers filed largely — though not entirely — with the SEC and gathered by Endpoints News. Two of those companies,
Roche and the new number 2, a hard charging
Merck, accounted for the lion’s share of the increase. (To be sure, some onetime non-R&D spending, such as Merck’s patent settlement with Bristol-Myers on Keytruda, figured in. But so did bread and butter spending.)
Gilead also saw a significant increase in research costs, with
Eli Lilly — now off course following two bad setbacks for solanezumab and baricitinib — and the ever aggressive
Celgene joining the action as they pressed the accelerator on new drug programs.
The Data Reflects Several Key Trends Key Takeaways
Raising a Series A led by an institutional VC remains difficult, but female founders in NYC continued to be the most successful (compared to those in other cities) in 2016.
At 17%, the percentage of total A rounds led by female CEOs in 2016 represents the highest total percentage since Female Founders Fund started tracking the data in 2013.
In addition, New York saw a record number of Series B and C rounds led by female founders in 2016.
Female Founders Fund remains the most active institutional VC firm investing in early-stage female-led companies.
Funds that have traditionally been uninterested in e-commerce have renewed interest in the e-commerce sector following the Dollar Shave Club and Jet.com acquisitions by large strategic investors.
2016 Series A Rounds — NYC — Female CEO Rockets of Awesome — $12.5 million — December — Rachel Blumenthal
Ellevest — $9.0 million — September — Sallie Krawcheck
CoheroHealth — $9.0 million — November — Melissa Manice
Away — $8.5 million — September — Steph Korey
Primary– $8.0 million — June — Galyn Bernard
goTenna — $7.5 million — March — Daniela Perdomo
LOLA — $7.0 million — December — Alex Friedman and Jordana Kier
Uncharted Play — $7.0 million — September — Jessica Matthews
Thrive Global — $7.0 million — August — Arianna Huffington
Everplans — $6.4 million — June — Abby Schneiderman
pymetrics — $6.1 million — February — Frida Polli
Sakara Life — $4.8 million — January — Whitney Tingle, Danielle DuBoise
Shoppable — $3.5 million — August — Heather Marie
MMLaFleur — Sarah LaFleur
2016 Series A Rounds — Bay Area — Female CEO
Cortexyme — $15.0 million — January — Casey Lynch
FOVE — $11.0 million — March — Yuka Kojima
Front — $10.0 million — May — Mathilde Collin
REBBL — $10.0 million — December — Sheryl O’Loughlin
Nima — $9.2 million — May — Shireen Yates
Rocksbox — $8.7 million — March — Meaghan Rose
LaunchDarkly — $8.7 million — December — Edith Harbaugh
Modsy — $8.0 million — February — Shanna Tellerman
ThirdLove — $8.0 million — February — Heidi Zak
Node — $7.5 million — June — Falon Fatemi
Shippo — $7.0 million — September — Laura Behrens Wu
Mobilize — $6.5 million — September — Sharon Savariego
Neurotrack — $6.5 million — January — Elli Kaplan
Sourcery — $5.0 million — September — Na’ama Moran
Luka — $4.4 million — April — Eugenia Kuyda
SupportPay — $4.1 million — December — Sheri Atwood
Zybooks — $4.0 million — February — Smita Bakshi
Schoola — $3.6 million — May — Stacey Boyd
The total number of Series A rounds in the U.S. decreased by 10% in 2016. Of the seven regions that we track in the U.S., Seattle is the only region that experienced an increase in the number of Series A raises in 2016, at 4%.
While overall Series A activity declined slightly in Los Angeles, there were two large Series A raises for female-led businesses — (i) HopSkipDrive, led by CEO Joanna McFarland, which raised $10.2 million in January 2016 from Upfront Ventures and FirstMark Capital; and (ii) Hutch, led by CEO Beatrice Fischel-Bock, which raised $5 million in July 2016 from Founders Fund. Los Angeles remains among the most female entrepreneur-friendly cities in the U.S.
On Investment Platforms for Private Funds and Investment Platforms for Private Placements – SEC Update
Reporter: Aviva Lev-Ari, PhD, RN
SEC Update
December 2015
Finders and Unregistered Broker-Dealers
In the last few years, we have seen a number of important developments in the securities laws related to finders and broker-dealer registration requirements. Below we provide an overview of the broker-dealer registration requirement as it relates to finders who assist in matching issuers with investors or buyers and the latest developments in this area.
Overview
The distinction between being classified as a finder and a broker-dealer can have significant consequences. An unregistered broker-dealer may face sanctions from the Securities and Exchange Commission (SEC), and it may be unable to enforce payment for its services. In addition, transactions involving an unregistered broker-dealer may create a right of rescission in favor of the investors, allowing the investors the right to require the issuer to return the money invested. One example of the consequences of an unregistered broker-dealer occurred in the Ranieri Partners SEC enforcement action. In that action the SEC brought charges against a private-equity firm, its managing director, and a consultant because of the consultant’s failure to register as a broker-dealer. The SEC’s order found that the private equity firm paid transaction-based fees to a consultant, who was not registered as a broker-dealer, for soliciting investors for private fund investments.1
The federal securities laws do not specifically define the term “finder” or outline what finders can do. Instead, finders must avoid being deemed a broker or dealer under the federal securities laws unless they register as such with the SEC and the Financial Industry Regulatory Authority (FINRA). A broker is defined as “any person engaged in the business of effecting transactions in securities for the accounts of others.”2 A dealer is defined as a person that is “engaged in the business of buying and selling securities … for such person’s own account,” but excludes a person that buys and sells securities for its own account, but not as part of a regular business.3Because the broker definition is the one that finders have the most trouble with, this discussion is focused on what activities may cause a finder to fall within the definition of a broker required to register with the SEC and FINRA.
M&A Brokers
FINRA Guidance
Investment Platforms for Private Placements
Investment Platforms for Private Funds
Crowdfunding
Potential Regulatory Action
Conclusion
A determination of whether an intermediary is acting as a finder or an unregistered broker-dealer is a very fact-specific analysis and can often be very complex. Unfortunately for unwary entrepreneurs, company executives, and equity fund sponsors, frequently a third party assisting with capital-raising will be acting as a broker-dealer, not a finder, and therefore should not be engaged unless properly registered. It is likely that we will see further clarification or new rules from regulators in the future; regardless, it is important to always carefully consider the involvement of finders or broker-dealers in any capital-raising endeavor.
If you have any questions regarding the use of finders, or capital raising in general, please contact the Venable lawyer with whom you work, one of the authors of this article, or a member of our Corporate Finance and Securities Group.
UPMC Enterprises, the commercialization arm of UPMC, is shaping the future of health care through innovation. Focused on generating impactful technology solutions, they invest in key focus areas: clinical tools, population health, consumer-centric health care, and business services and infrastructure. As the most active corporate venture investor of the year in digital health, UPMC contributed to several funding rounds including investments in Health Catalyst and Lantern.
Most Prolific VC
Khosla Ventures
Khosla Ventures helps entrepreneurs with large problems that are amenable to technology solutions. A longtime tech and healthcare investor, Khosla Ventures participated in five digital health deals this year, including Neurotrack and Color Genomics.
Best Performing IPO
Evolent Health
Evolent Health partners with health systems to accelerate their transformation to value-based care. By integrating the people, processes, and technology needed to drive clinical and financial growth, Evolent has found the secret to success—it is one of the digital health stocks with the biggest returns YTD, up 114% since the start of the year.
Crowdfunding Hero
BSX Technologies: LVL
LVL is the first wearable hydration monitor that gives users the complete picture of their health by also tracking activity, sleep, mood, and heart rate. Their wildly successful Kickstarter campaignresulted in over $1.1 million pledged of their $50,000 goal, and backers will soon be able to measure their hydration in real time. Fewer than 200 crowd funding campaigns have ever raised over $1 million.
Accel is an early & growth-stage venture capital firm and is known for its investments in Facebook, Slack, and Dropbox. This is the firm’s annual presentation on what the tech environment is like for founders today and what will happen in 2017, republished with permission.
It’s an incredible time to be a technology entrepreneur.
A rising “new guard” are officially the most valuable companies in the world: Apple, Alphabet/Google, Microsoft, Amazon, Facebook.
But of course, it’s important to stay disciplined.
We’re seeing an acceleration of M&A activity and a growing IPO pipeline through the end of 2016, but the bar remains high.
Business Forward Roundtable with John Podesta: Economic Growth and Opportunity
Reporter: Stephen J. Williams, Ph.D.
July 26, 2016 (Philadelphia, PA)
A Round Table and Q&A with the Entrepreneur Group Business Forward and John Podesta,Chairman and Founder of Center for American Progress on Policy, Economic Growth and Opportunity
With the help of more than 50 of the world’s most respected companies, Business Forward is making it easier for tens of thousands of business leaders from across America to advise Washington on how to create jobs and accelerate our economy. Business Forward is active in over 100 cities and works with more than 450 senior Administration officials, Members of Congress, mayors, and governors.
Business leaders who have participated in our briefings have seen their suggestions implemented in the Affordable Care Act, the Jobs Act, three trade agreements, and every one of the President’s budgets. Many have also shared their recommendations with their representatives in Congress and through op-eds and interviews with local media. Ninety-eight out of 100 business leaders who have participated in a Business Forward briefing would be interested in participating in another one.
Member Companies
Many of America’s largest and most respected firms – from America’s software, telecommunications, media, hospitality, financial services, manufacturing, apparel, defense and pharmaceutical industries – have already joined Business Forward.
Members include Aetna, American Airlines, AT&T, Comcast, Cheniere Energy, Deloitte, Dow, eBay Inc., Fidelity Investments, Facebook, Ford, Google, Intuit, Lockheed Martin, Microsoft, the National Restaurant Association, Pacific Gas & Electric, POET, Pricewaterhouse Cooper, Qualcomm, SAS, T-Mobile, Time Warner, Time Warner Cable, Verizon, Viacom, Visa, and Walmart.
These corporations work with Business Forward to identify, recruit and brief small business owners, venture capitalists and entrepreneurs of all kinds who are looking for a meaningful way to participate in policy debates.
John Podesta on Economic Policy, Equality and Growth
— Stephen J Williams (@StephenJWillia2) July 26, 2016
Business Forward Round Table on Economic Strategy and Opportunity Agenda with John Podesta: Policy
John Padesta (JP): We have had an economic bounce back from the recession however it is agreed that wages need to go up in US. The goal of policy is to return to a more equitable time such as during the 90’s. The Hillary Clinton campaign is actively reaching out to find out what is happening on all levels of the economy: from small startups to international trade and workers views.
JP: There are five main areas the Clinton campaign is focusing on with regard to economic growth policy
jobs, investment, create context to spur private-public partnership investments
skills training – human capital
invest in places left behind: promise zones
sustainable growth: allowing workers to share in productivity gains by tax reform, profit sharing
family policy – says they will define this policy later in the week
JP: want to get entrepreneurs more involved with policy decision. Clear directive from Hillary is that policy requires input from ALL stakeholders in economy in all sectors
There may be a focus on paid leave
Question from audience: What about the crisis in rural health.
Definitely a problem Ann O’Leary will be heading up the health policy for Clinton campaign
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