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Sunday, January 6, 2019

Biocartis Announces Achieving its 2018 Key Business Objectives


Biocartis Group NV (the ‘Company’ or ‘Biocartis’), an innovative molecular diagnostics company (Euronext Brussels: BCART), today announces to have achieved its 2018 key business objectives.
Biocartis’ key business objectives for 2018 were focused on three performance indicators: installed base expansion of its Idylla™ molecular diagnostics platform, cartridge volume growth and the Company’s year-end cash position.
Based on non-audited numbers, Biocartis now reports meeting or exceeding these business objectives:
  •  Installed base – Biocartis realized 326 new instrument placements in 2018, exceeding the latest guidance of 300 new instrument placements. Biocartis’ installed base as per 31 December 2018 consequently grew to around 970 Idylla™ instruments.
  •  Cartridge volume – In 2018, Biocartis realized a commercial volume of approx. 133k cartridges, in line with the latest guidance of 130k – 135k cartridges.Biocartis’ 2018 commercial cartridge volume represents a year-over-year increase of approx. 87%.
  •  Cash position – As per 31 December 2018, Biocartis’ cash position amounted to EUR 64m (non-audited number) versus the latest guidance of around EUR 55m. No drawdowns on the Company’s multiple purpose credit facility of EUR 27.5m were made as per year-end 2018.
Other key achievements in 2018 that are expected to support further business growth, include amongst others:
  •  US – Successful initial roll-out of the Idylla™ platform in the US during the first full year of commercialization in that market, attracting reference customers such as Memorial Sloan Kettering Cancer Center (New York) and Dartmouth -Hitchcock Medical Center (New Hampshire);
  •  China – Announcement of joint venture with Wondfo for commercialization of the Idylla™ platform in China;
  •  Japan – Selection of commercialization partner for Japan, resulting into signing of a distribution agreement with Nichirei Bioscience as announced on 7 January 2019;
  •  Menu of molecular diagnostic tests – Continued menu expansion with the launch of the Idylla™ MSI Assay (RUO1);
  •  Test content partnerships – Expansion of the collaboration with Genomic Health Inc. into the field of urology with the planned development of an Idylla™ Oncotype DX GPS test and potentially additional cancer tests. Progression of the Idylla™ Oncotype Breast Recurrence Score® test towards launch; and
  •  Partnerships with pharmaceutical partners – New agreement with AstraZeneca aimed at demonstrating that Idylla™ can overcome the long turnaround time forlung cancer biomarker results.
Herman Verrelst, Chief Executive Officer of Biocartis, comments: “I am pleased we can announce today that we have achieved our key 2018 objectives. The growth in instrument installed base and the year-over-year ramp-up of cartridge volume are clear indicators of the commercial growth curve that we are on. We have significantly strengthened our global commercial footprint in 2018. With the recent signing of the commercialization agreement for the Japanese market we now have a commercial network in all major markets worldwide. As we furthermore continue to grow the assay menu on the Idylla™ platform and expand our partner network, Biocartis is in a solid position to execute upon its mission to offer rapid and easy molecular diagnostic solutions aimed at enabling faster and more accurate treatment decisions for oncology patients across the globe.”
Biocartis will publish its 2018 full year results and 2019 guidance on 28 February 2019. Next to a live webcast that day, Biocartis will also host a Capital Markets Day tailored for institutional investors, research analysts and sector journalists to provide amongst others an update on the Company’s test menu strategy. On 10 January 2019,
Biocartis will present at the 37th Annual J.P. Morgan Healthcare conference in San Francisco, California (US) at 10:30 PST /19:30 CET.

Anthem (ANTM) Upgraded at Evercore


Evercore ISI upgraded shares of Anthem (NYSE:ANTM) from an in-line rating to an outperform rating in a report issued on Thursday morning, MarketBeat reports. The firm currently has $255.02 target price on the stock.

FluMist Flopped for Kids During Recent Flu Seasons


Live attenuated influenza vaccine (LAIV4), or FluMist, was less effective at preventing flu, especially influenza A/H1N1, in recent flu seasons among kids than inactivated influenza vaccine (IIV) was, researchers found.
A pooled analysis found that vaccine effectiveness for quadrivalent live attenuated influenza vaccine among children was 20% (95% CI -6% to 39%) for children ages 2-17 against influenza A/H1N1 (pdm09) compared with 67% (95% CI 62%-72%) for the inactivated influenza vaccine, reported Jessie Chung, MPH, of the CDC, and colleagues.
Moreover, patients who received LAIV4 had significantly higher odds of contracting the H1N1 strain compared with those who received IIV (OR 2.66, 95% CI 2.06-3.44), the authors wrote in Pediatrics.
When combining data from the 2013-2014 to the 2015-2016 flu seasons, overall vaccine effectiveness against any flu was 26% (95% CI 15%-36%) for LAIV4 compared with 51% (95% CI 47%-54%) for IIV, and recipients of LAIV4 had higher odds of testing positive for any flu (OR 1.48, 95% CI 1.28-1.70), they noted.
FluMist has had a roller coaster of highs and lows in recent years — going from the CDC’s Advisory Committee on Immunization Practices (ACIP) giving a preferential recommendation to this type of vaccine during the 2014-2015 season, to kicking it off the roster entirely during the 2016-2017 flu season, to adding it back as an option during the 2018-2019 flu season.
Regardless, the American Academy of Pediatrics (AAP) recently encouraged clinicians to use inactivated influenza vaccine as the “primary choice” when possible, and AAP liaison members spoke out against reinstating FluMist at the February 2018 ACIP meeting.
In this study, the authors raised questions about the accuracy of prior LAIV4 vaccine efficacy estimates, citing “heterogeneous findings,” as well as potential differences between U.S. and European vaccine effectiveness, where prior vaccination in the U.S. may have had an impact.
Researchers examined pooled individual patient-level data from five studies ranging from 2013-2014 to 2015-2016 in the CDC’s U.S. Influenza Vaccine Effectiveness Network.
Overall, data from over 17,000 children ages 2-17 was included, with an average age of around 7. One-fourth of patients tested positive for influenza, and among them 37% were infected with influenza A/H3N2, with 25% infected with influenza A/H1N1 (pdm09), and 25% infected with influenza B.
One third of patients were vaccinated and 30% of those received quadrivalent live attenuated influenza vaccine, the authors said. The authors noted that LAIV4 and IIV had similar effectiveness against influenza A/H3N2 and B viruses.
An accompanying editorial by Pedro Piedra, MD, of Baylor College of Medicine in Houston, said that the results of these five combined observational studies “provide a compelling argument why ACIP made the interim recommendation against… use” of LAIV4 in two recent flu seasons.
Piedra also reviewed potential hypotheses from the World Health Organization, including “methodological study differences; inadequate vaccine handling at vaccine distribution centers; intrinsic virological characteristics of the novel A/H1N1pdm09… used in the initial formulations of LAIV4.”
Other considerations included improved coverage across age groups since the ACIP made a universal influenza vaccine recommendation in 2010 and potential “viral interference” from the second B strain when the vaccine changed from a trivalent to a quadrivalent during the 2013-2014 season, Piedra said.
He cited an interim analysis on vaccine effectiveness from England, which found an interim end-of-season adjusted vaccine effectiveness for LAIV4 in children ages 2-17 of 90.3% (95% CI 16.4%-98.9%) against influenza A/H1N1 (pdm09) for the 2017-2018 season. Piedra characterized this result as “encouraging.”
Limitations to the data cited by Chung and colleagues, they said, included that “descriptive patient information beyond age, sex, and geographic region of enrollment” was not available for all studies, which could result in unmeasured confounding. The authors also said that historical vaccination data was limited to the season prior to enrollment as opposed to an entire vaccination history for most patients.
The study was supported by the National Institutes of Health (NIH).
The U.S. Influenza Vaccine Effectiveness Network was supported by the Centers for Disease Control and Prevention, the University of Michigan, Kaiser Permanente Washington Health Research Institute, Marshfield Clinic Research Institute, the University of Pittsburgh, Baylor Scott & White Health (U01 IP000473), and the NIH.
The Influenza Clinical Investigation for Children was supported by MedImmune.
Chung disclosed no conflicts of interest; other co-authors disclosed being employees of AstraZeneca.
Piedra disclosed support from AstraZeneca, Sanofi Pasteur, GlaxoSmithKline, Merck Sharp and Dohme, and Seqirus.
LAST UPDATED 

Ultragenyx’s DTX401 shows positive action in glycogen storage disease


Ultragenyx Pharmaceutical (NASDAQ:RARE) is up 4% premarket, albeit on only 100 shares, following its announcement of preliminary data from a Phase 1/2 trial evaluating gene therapy DTX401 in patients with glycogen storage disease type Ia, an inherited disorder characterized by the build up of sugar (glycogen) in cells leading to impaired organ function.
Three patients in the lowest dose cohort all experienced improved glucose control and increased time to hypoglycemia (low blood sugar) during fasting while two experienced clinically meaningful improvements in time to hypoglycemia during a controlled fasting challenge.
No infusion-related adverse events and no serious treatment-related adverse events have been reported. All adverse events to date have been mild or moderate.
Enrollment is the second cohort will begin this month with interim data expected mid-year.

Top 20 Life Science Startups to Watch in 2019


BioSpace is proud to present its NextGen Bio “Class of 2019,” a list of 20 up-and-coming life science companies in North America that launched* no earlier than 2017.
The NextGen Bio Class of 2019 is a stellar group of companies that are already making an enormous impact on the industry now and will continue into the future. Congratulations to this group!
RANKCOMPANYLAUNCHEDLOCATIONPOINTS
1Allogene Therapeutics2018California59
2Gossamer Bio2018California41
3Viela Bio2018Maryland33
4SpringWorks Therapeutics2017New York28
5NanOlogy2017Texas19
6Cullinan Oncology2017Massachusetts18
7Ambys Medicines2018California18
8Skyhawk Therapeutics2018Massachusetts17
9The Bill & Melinda Gates Medical Research Institute (MRI)2018Massachusetts17
10Ansun Biopharma2018California16
11Magnolia Neurosciences218New York16
12Compass Therapeutics2018Massachusetts15
13Beam Therapeutics2018Massachusetts15
14Joyn Bio2018Massachusetts14
15Partner Therapeutics2018Massachusetts14
16DataVant2017California13
17Genevant Sciences2018Massachusetts13
18Akouos2018Massachusetts12
19Akero Therapeutics2018Massachusetts / California12
20Rheos Medicines, Inc.2018Massachusetts11

Allogene
Points: 59
Launched: 2018
Location: South San Francisco, CA
Notable:
• Allogene closed a $300 million Series A venture round in April 2018, which included investments from Pfizer and Gilead Sciences.
• The company is focused on developing off-the-shelf CAR-T products.
• Allogene has access to 16 preclinical CAR-T licenses that Pfizer licensed from Cellectis and Servier.
• One of the licensed assets, UCART19, is expected to begin Phase II clinical trials in 2019.
• The company completed a $120 million private financing of convertible notes.
• Allogene raised $324 million in IPO, second highest IPO behind Moderna.
• Pfizer has a 25 percent stake in Allogene.

Gossamer Bio
Points: 41
Launched: 2018
Location: San Diego, CA
Notable:
• Gossamer Bio launched in January 2018 with a $100 million Series A financing, which was followed in July by a $230 million Series B financing.
• Gossamer Bio focuses on immunology and fibrosis, inflammation and immuno-oncology.
• The company currently has two products in Phase I clinical trials, two in Phase II, and two in preclinical studies.

Viela Bio
Points: 33
Launched: 2018
Location: Gaithersburg, MD
Notable:
• Viela Bio spun out of MedImmune in 2018.
• In February 2018, the company closed a Series A financing worth $282.3 million.
• Viela Bio focuses on severe inflammation and autoimmune diseases by targeting shared critical pathways.
• The company launched with three clinical and three pre-clinical drugs from MedImmune. These drugs include inebilizumab which is currently in Phase II for neuromyelitis optica, a rare condition of the optic nerve and spinal cord.

Points: 28
Launched: 2017
Location: New York, NY
Notable:
• SpringWorks Therapeutics launched with a $103 million Series A round, with Bain Capital, OrbiMed, and Pfizer as investors.
• SpringWorks holds the rights to four clinical-stage therapies from Pfizer.
• SpringWorks has partnerships in place with Desmoid Tumor Research Foundation, Children’s Tumor Foundation, Cohen Veterans Bioscience, University of California David, Boston Children’s Hospital, the Alzheimer’s Drug Discovery Foundation and BeiGene.
• The company’s most advanced drug candidate, nirogacestat, received orphan drug designation from the U.S. Food and Drug Administration (FDA) for desmoid tumors and PD-0325901 received orphan drug designation from the FDA for neurofibromatosis type 1.

NanOlogy
Points: 19
Launched: 2017
Location: Fort Worth, TX
Notable:
• NanOlogy was formed by DFB Pharmaceuticals, CritiTech, and US Biotest with seed funding of $2 million.
• NanOlogy is focused on developing naked nanoparticle forms of paclitaxel and docetaxel for local delivery of the therapeutics to treat cancer and other diseases.
• The company is currently running Phase I trials for prostate cancer, pancreatic cancer, pancreatic mucinous cysts and others.

Cullinan Oncology
Points: 18
Launched: 2017
Location: Cambridge, MA
Notable:
• Cullinan Oncology launched in October 2017 with a Series A financing worth $150 million.
• Cullinan is developing a venture fund/biotech business model where it plans to have 8 to 12 drugs in development with each drug having a dedicated chief executive. But instead of having a research team for each drug, Cullinan scientists will share company resources.
• Cullinan has three assets in its pipeline with one licensed from an unidentified university.
• The company signed a collaboration deal with MAB Discovery GmbH in February 2018.

Ambys Medicines
Points: 18
Launched: 2018
Location: Redwood City, CA
Notable:
• Ambys launched in August 2018 in a Series A financing round worth $60 million, funded by Third Rock Ventures and Takeda.
• As part of the launch, Ambys signed a strategic partnership with Takeda with an additional $80 million commitment to fund research.
• Ambys will focus on chronic liver disease, including cell therapy for hepatocyte transplantation, gene therapy for liver regeneration, and drug therapy to replace lost protein function.

Skyhawk Therapeutics
Points: 17
Launched: 2018
Location: Waltham, MA
Notable:
• In June 2018, Skyhawk Therapeutics closed a $40 million equity investment round and then, in an unusual deal, signed a five-year collaborative agreement with Celgene that included an upfront $60 million.
• Skyhawk focuses on developing small molecule therapeutics that correct RNA mis-splicing, what is usually called exon skipping.
• The company expects to move its first oncology treatment into the clinic in 2019.
• Skyhawk currently has four targets in oncology and three in neuro, which are in lead selection but past preclinical IND filing.

The Bill & Melinda Gates Medical Research Institute (MRI)

Points: 17
Launched: 2018
Location: Cambridge, MA
Notable:
• In 2018, The Bill & Melinda Gates Foundation decided to launch a biotech company named The Bill & Melinda Gates Medical Research Institute (MRI).
• MRI will focus on vaccines, especially malaria, tuberculosis and enteric diseases.
• The company expects to work with academic laboratories and biotech firms to shepherd programs through the early-to-middle stage of development then hunt for commercial partners.
• Via the Foundation, MRI has an annual operating budget of $100 million.
• MRI’s lead project is determining if the well-documented BCG vaccine given to adolescents can increase their resistance to tuberculosis.

Ansun Biopharma

Points: 16
Launched: 2018
Location: San Diego, CA
Notable:
• Ansun Biopharma launched on May 14, 2018, with $85 million in Series A financing from Sinopharm Healthcare Fund and Lilly Asia Ventures. Additional new investors include Lyfe Capital, Yuanming Capital, Matrix Partners China, 3e Bioventures Capital, Oceanpine Capital, VI Ventures and Joincap Investment.
• The company has two products in Phase II clinical trials: 1) Fludase is a first-in-class therapeutic to treat all forms of influenza; 2) Paradase is a nebulized drug for parainfluenza viruses.

Magnolia Neurosciences

Points: 16
Launched: 2018
Location: New York, NY
Notable:
• Magnolia Neurosciences Corporation was co-founded by Accelerator Life Science Partners and The University of Texas MD Anderson Cancer Center.
• The company launched in August 2018 with a $31 million Series A financing, which included AbbVie Ventures, Alexandria Venture Investments, ARCH Venture Partners, Eli Lilly and Co., Innovative NY Fund, Johnson & Johnson Innovation – JJDC, Inc., the Partnership Fund for New York City, Pfizer Ventures, Watson Fund, and WuXi, AppTec’s Corporate Venture Fund and 180 Degree Capital Corp.
• The company’s subsidiary, Korysso Therapeutics, received a $19.95 million grant from the Cancer Prevention and Research Institute of Texas in August 2018.

Compass Therapeutics

Points: 15
Launched: 2018
Location: Cambridge, MA
Notable:
• Launched with the final $49 million of a $132 million Series A financing in July 2018, led by OrbiMed Advisors and included F-Prime Capital, Cowen Healthcare Investment, Thiel Capital, Biomatics Capital, Ulysses Holdings, Borealis Ventures, Alexandria Ventures Investments and Biomed Realty Ventures.
• Currently has 15 therapeutic candidates advancing through preclinical programs for cancer, inflammation and autoimmune diseases.


Beam Therapeutics
Points: 15
Launched: 2018
Location: Cambridge, MA
Notable:
• Beam Therapeutics launched with up to $87 million in cumulative Series A funding led by F-Prime Capital Partners and ARCH Venture Partners in May 2018.
• Beam focuses on multiple DNA base editor platforms developed in the lab of David Liu and the RNA base editor platform developed by Feng Zhang at the Broad Institute of MIT and Harvard.
• The company has licensed tech from Harvard for two base editing platforms.
• Beam entered into a licensing and option agreement with Editas Medicine.

14. Joyn Bio
Joyn Bio [square]

Points: 14
Launched: 2018
Location: Boston, MA
Notable:
• Joyn Bio is a joint venture between Bayer AG and Ginkgo Bioworks.
• Joyn Bio was launched with a $100 million Series A financing led by Bayer, Ginkgo BIoworks and Viking Global Investors.
• In addition to the financing, Ginkgo is offering Joyn access to technology, laboratory and office space, and is building a new facility just for Joyn.
• Bayer is providing access to proprietary microbial strains.
• Joyn Bio is focused on synthetic biology for agricultural applications.

Partner Therapeutics
Points: 14
Launched: 2018
Location: Lexington, MA
Notable:
• Partner Therapeutics launched in February 2018 with a $60 million Series A financing led by Perceptive Advisors, Adams St. Partners and MidCap Financial.
• The company acquired rights to Leukine (sargramostim) from Sanofi in February 2018, which was already approved, and will develop the drug for new indications.
• Partner Therapeutics signed a distribution deal with Tanner Pharmaceuticals for Leukine in May 2018.
•  In June, the FDA approved Leukine for the treatment of acute radiation syndrome and in November Leukine was granted orphan drug designation for pulmonary alveolar proteinosis.

16. Datavant
Datavant

Points: 13
Launched: 2017
Location: San Francisco, CA
Notable:
• Datavant is a part of Roivant Sciences, one of Vivek Ramaswamy’s numerous companies.
• The company focuses on utilizing artificial intelligence to improve the clinical trial process and is led by Travis May, a Silicon Valley tech veteran who was co-founder and chief executive officer of LiveRamp.
• Datavant had a $40 million financing round in April 2018 led by Roivant Sciences and Travis May.
• In April 2018, Datavant acquired Universal Patient Key (UPK), a leading provider of HIPAA-compliant de-identification services for healthcare data.
• In May 2018, Datavant teamed with Worldwide Clinical Trials, a Contract Research Organization (CRO).

Genevant

Points: 13
Launched: 2018
Location: Cambridge, MA
Notable:
• Genevant is one of Vivek Ramaswamy’s companies under the umbrella of Roivant Sciences.
• Genevant was formed by Arbutus Biopharma Corporation and Roivant Sciences as a jointly-operated company focused on discovery, development, and commercialization of RNA-based therapeutics enabled by Arbutus’ proprietary nanoparticle (LNP) and ligand conjugate delivery technologies.
• Roivant had a $116 million stake in Arbutus’ hepatitis B program.
• Genevant was launched with $37.5 million in seed capital from Roivant.
• Genevant signed a research deal with BioNTech to develop five products for rare diseases.

18. Akouos
Akouos

Points: 12
Launched: 2018
Location: Boston, MA
Notable:
• Launched in 2018 with a $50 million Series A co-led by 5AM Ventures and New Enterprise Associates, who were joined by existing seed investor Partners Innovation Fund and new investors Sofinnova Ventures, RA Capital Management and Novartis Venture Fund.
• The company will focus on developing AAV-based gene therapies for sensorineural hearing loss.
• The company has licensed technology from Massachusetts Eye and Ear, and Lonza.

Akero Therapeutics
Points: 12
Launched: 2018
Location: Cambridge, MA / San Francisco, CA
Notable:
• Akero Therapeutics launched in June 2018 with a $65 million Series A co-led by Apple Tree Partners, Atlas Venture, venBio Partners and Versant Ventures and most recently, closed $70 million Series B in December.
• The company’s focus is on non-alcoholic steatohepatitis (NASH) and other serious metabolic diseases.
• Akero’s lead clinical program is AKR-001, a long-acting fibroblast growth factor 21 (FGF21) analogue, which it licensed from Amgen and plans to initiate a Phase II clinical trial in NASH.

Rheos Medicines, Inc.

Points: 11
Launched: 2018
Location: Cambridge, MA
Notable:
• Rheos Medicines launched in March 2018 with a $60 million Series A financing backed by Third Rock Ventures.
• The company focuses on developing therapies for immune-mediated diseases by tuning metabolic pathways in immune cells.
• Rheos Medicines’ product engine is a proprietary Immune Cell Encyclopedia (ICE), which maps the metabolic pathways by using different types of immune cells to regulate their fate and function.

To come up with this Top 20, BioSpace sorted companies into that age grouping, and they were then weighted by a number of different categories and finally ranked in a cumulative fashion, based on the points awarded for each category. These categories were: Finance, Collaborations, Pipeline and Innovation.

Curaleaf Opens New Medical Cannabis Dispensary In New York


U.S. cannabis retailer Curaleaf Holdings (CSE: CURA) (OTCBB: CURLF) has opened its fourth dispensary in New York State, bringing the total number of the company’s dispensaries to 36.
Located at 255 Glen Cove Road in Carle Place, Long Island, the new location offers medical cannabis patients in Nassau and Suffolk counties easier and more convenient access to product, the company said in a press statement.
New Curaleaf Cannabis Location
According to Curaleaf, the new Carle Place location will offer a range of free wellness and medical marijuana educational programs such as health seminars led by medical professionals and yoga classes. The company plans to support local community events and initiatives such as the Susan G. Komen annual “Race For The Cure,” EPIC Long Island, and the Multiple Sclerosis Society of NY and Long Island.
“This new location will serve a community of patients in Long Island and provide them with high-quality medical cannabis used to help patients suffering from chronic pain, cancer, opioid addiction, PTSD and other ailments,” Curaleaf New York President Michelle Bodner said in the statement.
The dispensary’s operating hours are Tuesday through Thursday and Saturday, 10:30am – 6pm; Friday, 11:30am -7pm; closed on Sundays and Mondays. Throughout the year, discounts are offered to military veterans and recipients of Medicaid.
Curaleaf recently expanded its product line by adding new products and nine new SKU’s – Curaleaf Slim Vape Pen, Peppermint Flavored Tincture, and Lemon Flavored Tinctures. The company says that it made these products from cannabis grown in the state and processed at the company’s cultivation facility in Ravena, New York.
Curaleaf also operates medical cannabis dispensaries in Newburgh, Plattsburgh and Forest Hills, Queens.
The State of New York does not allow cannabis for recreational use. However, it is legal to use cannabis for medical purposes, while possession of small amounts is decriminalized in the state.