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Saturday, January 5, 2019

Crowdfunding Homeopathy for Cancer: A Very American Thing


“Crowdfunding” campaigns for the homeopathic treatment of cancer are overwhelmingly an American phenomenon and can be highly successful in terms of dollars raised, despite no evidence of efficacy, indicates a new study published January 1 in the Lancet Oncology.
A June 2018 analysis of the crowdfunding platform GoFundMe identified 220 unique campaigns for cancer patients seeking financial support for homeopathy.
The vast majority (186; 85%) of the campaigners were in the United States, with some in Canada (23; 10%) and the United Kingdom (8; 4%), and just one campaign each in Germany, Ireland, and Spain.
The campaigns requested US $5.8 million and were pledged US $1.4 million (24% of the total requested amount) by 13,621 donors.
There are three outstanding reasons that this group of 200-plus cancer patients turned to homeopathy, report the study authors, led by Jeremy Snyder, PhD, of Simon Fraser University, Burnaby, British Columbia, Canada.
Cancer patients intended to use homeopathy as a complement to standard cancer treatment (83; 38%), rejected standard treatment (63; 29%), or could not afford/accommodate standard therapy (69; 31%).
There is “broad consensus” in the scientific community that homeopathy is “ineffective,” write the study authors.
“These treatments are the bunkiest of the bunk, just complete garbage,” Snyder commented to the science blog, Gizmodo.
Snyder told Medscape Medical News that crowdfunding “opens up a new world of possibilities by providing people with a source of funding to pursue these unproven treatments — it also serves to normalize it.”
He added that complementary and alternative medicine providers “may be pushing their clients to crowdfund; this is something I’ve documented in stem cell clinics.”
Skyler Johnson, MD, Yale Cancer Center in New Haven, Connecticut, who was not involved with the new study, occasionally searches GoFundMe as part of his research interest in alternative treatments for cancer.
“The patient stories are heartbreaking to say the least. Most are terminal cancer patients raising money for bogus treatments — with no possible chance of working — only to maintain hope and put money in the pockets of charlatans,” he told Medscape Medical News.
Indeed, the new study indicated that more than a quarter of the cancer patients (62; 28%) died following the start of their homeopathy-based campaigns. The number is very likely an underestimate because of underreporting, said Snyder.
Additionally, Yale’s Johnson pointed out that even better prognosis, early stage cancer patients who reject standard treatment and exclusively opt for alternative therapies dramatically increase their mortality risk, as demonstratedin his 2017 study.
In the current study, the investigators report that about a third of the campaigns (63; 29%) made unsubstantiated, positive claims about the activity of homeopathy for cancer.
The claims included personal anecdotes and “sweeping activity” statements. Only 1% cited uncertainty about homeopathy’s efficacy.
The study authors would like to see professional organizations partner with crowdfunding platforms “to combat the worst forms of harm and misinformation.”
Johnson agrees, but is sanguine about the possibilities: “The direct financial conflicts of interest of these platforms give me little hope much will be done.”
Synder and Johnson have disclosed no relevant financial relationships.
Lancet Oncol. 2018;20:28-29. Abstract

Sojournix Completes $44 Million Series C Financing


SJX-653, a Novel Selective NK3 Antagonist in Clinical Development as a Once-Daily Non-Hormonal Treatment for Menopausal Vasomotor Symptoms, or “Hot Flashes”
Premier Healthcare Investors RA Capital Management, Boxer Capital of the Tavistock Group, Frazier Healthcare Partners, Venrock Healthcare Capital Partners and Janus Henderson Investors Participate in Financing
Sojournix, a clinical stage pharmaceutical company focused on creating new medicines to transform the treatment of women’s health and neuroendocrine disorders, announced today the completion of a $44 million Series C financing.  Proceeds from the financing will be used to advance SJX-653, a novel, potent, and selective neurokinin-3 (NK3) receptor antagonist in clinical development as a once-daily non-hormonal treatment for menopausal vasomotor symptoms (commonly referred to as “hot flashes”).
Leading healthcare investors participated in the Series C financing, including RA Capital Management, Boxer Capital of the Tavistock Group, Frazier Healthcare Partners, Venrock Healthcare Capital Partners and Janus Henderson Investors.  To date, Sojournix has raised a total of $70 million in capital, including $7 million in a Series A financing, $19 million in a Series B financing and $44 million in the Series C financing announced today.
“With this financing we are well-positioned with an outstanding syndicate of investors to rapidly progress clinical development of SJX-653 for the treatment of moderate to severe vasomotor symptoms due to menopause and additional potential indications,” said Dan Grau, President, CEO, and co-founder of Sojournix.  “By advancing SJX-653, we hope to address the large and well-recognized unmet medical need among patients for a safe and effective non-hormonal treatment option.”
“Our philosophy is to invest in companies that are developing the best agents, from the most promising emerging drug classes, spearheaded by outstanding management teams – and all three of these elements are present with Sojournix,” said Jamie Topper, M.D., Ph.D., Managing General Partner of Frazier Healthcare Partners’ Life Sciences team.  “We look forward to working with Dan and the Sojournix team as they develop the full clinical and commercial potential of SJX-653.”

Aldeyra Promotes David McMullin to Position of Chief Commercial Officer


Aldeyra Therapeutics (Nasdaq: ALDX) (Aldeyra), a biotechnology company devoted to development of next-generation medicines to improve the lives of patients with immune-mediated diseases, today announced that David McMullin has been promoted to the position of Chief Commercial Officer, effective immediately. In his new role, Mr. McMullin will oversee Aldeyra’s strategic initiatives, commercial planning activities, marketing, and commercial infrastructure development.
“Based on his deep understanding of orphan and large pharmaceutical markets, Dave is well positioned to lead our commercial development efforts,” commented Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. “I look forward to working closely with Dave as he prepares for commercialization of our late-stage clinical pipeline.”
Mr. McMullin joined Aldeyra in May 2018 as Senior Vice President and Head of Corporate Development and Strategy, and has more than 20 years of experience leading commercialization, business development, and strategic planning operations in the biopharmaceutical industry.  Prior to joining Aldeyra, Mr. McMullin was Group Vice President and Head of U.S. Internal Medicine at Shire plc, a $1.4 billioncommercial business unit.  Mr. McMullin also ran Global Commercial Operations at Shire Plc and was an in-line Committee member, responsible for commercial performance and execution worldwide.  Prior to his work at Shire, Mr. McMullin held roles of increasing responsibility in corporate strategy, marketing, sales, and operations at Novartis International AG. Mr. McMullin received an M.B.A. from Harvard Business School, and a Bachelor of Science degree from Brigham Young University.
Aldeyra Therapeutics is developing next-generation medicines to improve the lives of patients with immune-mediated diseases. Aldeyra’s lead product candidate, reproxalap, is a first-in-class treatment in late-stage development for dry eye disease, allergic conjunctivitis, noninfectious anterior uveitis, and Sjögren-Larsson Syndrome. The company is also developing other product candidates for autoimmune disease, post-transplant lymphoproliferative disease, retinal inflammation, metabolic disease, and cancer.  None of Aldeyra’s product candidates have been approved for sale in the U.S. or elsewhere.

Lipocine Trial Results Selected for Presentation at NASH-TAG Conference


Lipocine Inc. (NASDAQ: LPCN), a specialty pharmaceutical company, announced today that it has been selected to present LPCN 1144 results as part of the 2019 NASH-TAG Conference. The presentation will highlight data from multiple clinical trials of LPCN 1144 in potential non-alcoholic fatty liver disease (“NAFLD”) / non-alcoholic steatohepatitis (“NASH”) patients. The NASH-TAG Conference 2019 is being held January 3rd – 5th in Park City, UT.
Presentation Details
Title:
LPCN 1144 Improves NAFLD/NASH Biomarkers in Subjects At-Risk of NAFLD/NASH
Date:
Saturday, January 5th
Location:
The Chateaux Deer Valley, Park City, UT
Time:
3:50 pm – 4:05 pm
“The observed reduction in serum liver enzymes and lipids highlight the promise of LPCN 1144 as a potential therapy for NASH,” stated Mahesh Patel, Ph.D., Lipocine’s Chairman, President and Chief Executive Officer. “We look forward to sharing the results from our clinical trials with LPCN 1144 as part of the 2019 NASH-TAG Conference,” Dr. Patel further stated.

Computers turn neural signals into speech


For many people who are paralyzed and unable to speak, signals of what they’d like to say hide in their brains. No one has been able to decipher those signals directly. But three research teams recently made progress in turning data from electrodes surgically placed on the brain into computer-generated speech. These teams haven’t yet managed to re-create speech that people merely imagine. But by monitoring parts of the brain as participants either read aloud, silently mouthed speech, or listened to recordings, the researchers were able to use computational models called neural networks to reconstruct words and sentences that were close enough, in some cases, to be intelligible to human listeners.
Science  04 Jan 2019:
Vol. 363, Issue 6422, pp. 14
DOI: 10.1126/science.363.6422.14

Immunocore’s extended runway


The $100 million upfront payment from Immunocore Ltd.’s most recent deal with Roche’s Genentech Inc. unit gives it enough cash to read out pivotal data from lead program IMCgp100, a catalyst that could help justify its lofty valuation and open up additional financing opportunities.
However, the fact it needed the partnering cash to begin with highlights how high valuations and big raises aren’t guarantees of easy access to future financing.
Immunocore splashed onto the scene in July 2015 with a $320 million series A round backed by Woodford Investment Management, Malin Corp. plc, Baker Brothers, RTW Investments, Eli Lilly and Co. and other undisclosed investors. At the time, it was the largest-ever European venture financing.
What followed was a period of rapid expansion, both in terms of headcount, physical footprint and valuation, which peaked at $1.3 billion, not including potential exercise of share options.
But that expansion cost money. At the end of 2017, Immunocore reported a cash position of £117 million ($158.3 million) and a 12-month operating loss of £67.5 million ($91.3 million) — giving it less than two years’ runway.
The Genentech cash allows Immunocore to stretch its runway beyond 2020, when IMCgp100 (ImmTAC-gp100) could be ready for commercial launch in uveal melanoma.
Since the start of the year, Immunocore has also seen a C-suite exodus, with five departures, headlined by that of CEO Eliot Forster in February.

NantCell Announces New Celgene Investment


Immunotherapy Company Will Present Findings at the JP Morgan Healthcare Conference in San Francisco on January 7th
Second Round Crossover Funding Brings Celgene’s Investment to $105M in NantCell Valued at $4 Billion
Currently Enrolling Patients in Advanced Stage Trials in 15 Indications for Registration Intent
Deep Pipeline of 28 Unique Molecules with 14 First in Human Studies in Over 1,000 Patients to Date
NantCell and it’s founder Dr. Patrick Soon-Shiong announced today that Celgene has completed its crossover investment in NantCell. Dr. Soon-Shiong will be introducing the company at the 37th Annual JP Morgan Healthcare Conference at the Westin St. Francis Hotel, San Francisco on Monday, January 7th at 8:30am.
NantCell is a privately held immunotherapy company, whose goal is to employ a broad portfolio of biological molecules that will enable it to develop a cancer vaccine to combat multiple tumor types without the use of high-dose chemotherapy. NantCell has one of the most comprehensive late stage clinical pipelines of an integrated platform of immunotherapy technologies addressing both the innate (activated macrophage and natural killer cell) and the adaptive immune system (dendritic, CD4 and CD8 killer T cells). Currently the company is actively enrolling patients for registration trials in 15 indications.
On December 19, 2018, Celgene completed a crossover funding round of $30 million in NantCell at a $4 billion valuation, bringing its overall investment in the company to $105 million with a 2.8% ownership in the company. This follows the May 2015 Celgene initial investment of $75 million in NantCell.
“We have partnered with Dr. Soon-Shiong and his mission to change the course of cancer from the very beginning,” said Mark Alles, Chairman and CEO of Celgene. “From his invention of Abraxane, to acquiring his company in 2010, to launching this protein nanoparticle drug as the backbone of immunotherapy to its current blockbuster status, and now to supporting his vision at NantCell of developing a chemo free cancer vaccine utilizing the body’s own immune system. Celgene invested in NantCell since its inception in 2015 and we are excited to extend this partnership today with the significant clinical progress he has made in developing cytokines and bispecific proteins in the ongoing quest to conquer this disease,” said Alles. Celgene announced on January 3, 2018 that it would be acquired by Bristol-Myers Squibb for $74 billion.
“To our knowledge,” said Soon-Shiong, “there is no other biotech or large pharma company with NantCell’s broad pipeline of bispecific and trispecific fusion cytokine proteins, peptides, mRNA, monoclonal antibodies, neoepitope and tumor associated vaccine delivery and cell therapy products, all in clinical phase of development, across multiple indications, for the treatment of cancer and infectious disease. We are very pleased with Celgene’s continued investment in the company and our shared vision of developing a chemotherapy free cancer vaccine.”
“With the clinical advances of the technology platforms across multiple tumor types at NantCell, the company is now poised to integrate the technologies developed at the two early stage immunotherapy public companies, NantHealth and NantKwest,” said Dr. Soon-Shiong, founder of all three companies. “The adenovirus and yeast vector delivery systems in NantCell compliments the tumor associated antigen and neoepitope discovery engine (GPS CancerTM) developed by NantHealth, enabling the subcutaneous delivery of the neoepitopes to enable the recruitment of T cells that target only expressed cancer mutations. The bispecific fusion cytokine proteins of NantCell stimulates the patient’s autologous primary NK and T cells, thereby supplementing the off-the-shelf, cryopreserved haNK cells developed by NantKwest. Collectively the immunotherapy platforms in NantCell, NantHealth and NantKwest serve as a comprehensive path to the development of a cancer vaccine,” said Soon-Shiong.