Shares of Akari Therapeutics PLC AKTX, +9.83% more than doubled–soaring 145%–toward a 15-month high in very active morning trade Wednesday, enough to pace all the gainers on the Nasdaq exchange, after the company said it plans to commence trials in European and U.S. pediatric patients this year for its treatment of pediatric thrombotic microangiopathy. Trading volume rocketed to 22.3 million shares, compared with the full-day average of about 20,000 shares. The company said it had a “successful,” pre-investigational new drug (IND) meeting with the Food and Drug Administration regarding its pivotal clinical trial program for pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA), which is an orphan condition with an estimated fatality rate of 80%. Chief Executive Clive Richardson said trials in HSCT-TMA patients are planned to begin in the fourth quarter. “We see HSCT-TMA as a gateway indication into a range of other poorly treated orphan TMAs, and are enthusiastic about the potential of Coversin to offer an improved standard of care for patients with these rare and usually fatal conditions,” Richardson said. The stock has now run up 93% over the past 12 months, while the iShares Nasdaq Biotechnology ETF IBB, +1.31% has slipped 1.1% and the S&P 500 SPX, +0.69%gained 0.9%.
Wednesday, March 13, 2019
Guardant Health up 25% on Q4 beat
Guardant Health (GH +25.3%) is up on 4x normal volume following its Q4 report released after the close yesterday that beat expectations. Highlights:
Revenue was up 64% to $32.9M; oncology testing revenue was up 98% to $28.1M.
Net loss was ($25.1M).
Company working with AstraZeneca on developing Guardant360 and GuardantOMNI tests as companion diagnostics for Tagrisso and Imfinzi, respectively.
2019 guidance: Revenue: $130M – 135M; net loss: ($129M – 126M).
Kerrisdale targets iRhythm in short attack, claiming tenuous reimbursements
Short seller Kerrisdale Capital has launched a new attack on iRhythm Technologies, manufacturers of the Zio heart monitor patch, based on what it describes as a looming crisis over the reimbursement structure for its main product.
Describing iRhythm’s revenue base as “exceedingly generous, but increasingly fragile,” the firm said that the rapid growth of the patch’s use over the past few years has not only attracted competition from other device companies, but that it could also lead to cutbacks in Medicare and commercial reimbursements.
The Zio patch—a single-lead, water-resistant ECG worn over the heart, used to help diagnose occasional arrhythmias over periods as long as two weeks—is currently reimbursed via a temporary CPT code, with a permanent code expected to be established for 2021, Kerrisdale said in its report(PDF).
“In the process, we anticipate reimbursement levels for the Zio patch will fall by over a third, and potentially more than 50%,” the firm wrote.
In addition, most of the company’s revenue is based on billing for performing analyses of the scans and delivering ECG reports from iRhythm’s own diagnostic facilities, where the patch is mailed for study after use. The Zio is not able to upload data to another device on its own.
iRhythm, a 2014 Fierce 15 company, and its Zio patch have been referred to as “the new standard” for remote cardiac monitoring. Studies have shown that the device was able to identify 57% more arrhythmias and was much more likely to provide a definitive diagnosis than the traditional multilead Holter monitor, which is worn around the neck for 24 to 48 hours.
In addition, 94% of patients reported preferring to wear the Zio patch compared to Holter monitoring. iRhythm says their simpler product also encourages patient adherence.
However, the Zio patch was first cleared by the FDA in 2009, and Kerrisdale says that superior devices have hit the market since then, including ones with features that allow data to be uploaded to the cloud much faster.
Kerrisdale aims to drive down the value of iRhythm’s stock and profit from the bets and put options it has placed against the company. Shortly after the release of the report, iRhythm’s stock was trading down about 3%, at just over $84.
Glaukos Tech in Numerous Presentations at 2019 American Glaucoma Society
Glaukos Corporation (NYSE: GKOS), an ophthalmic medical technology and pharmaceutical company focused on the development and commercialization of novel surgical devices and sustained pharmaceutical therapies designed to transform the treatment of glaucoma, announced today that its products will be featured in various presentations at the American Glaucoma Society (AGS) Annual Meeting on March 14-17, 2019 at the Marriott Marquis Hotel in San Francisco.
Glaukos is sponsoring an educational symposium for surgeons during AGS entitled “The New Dawn of MIGS: Devices, Data and Techniques” on March 15, 2019 at 1:15 to 2:15 PDT in the Soma Room of the Marriott Marquis Hotel. The faculty includes Ike Ahmed, MD (Moderator); Mark Gallardo, MD; Davinder Grover, MD; and Nate Radcliffe, MD. Go here to register.
Glaukos will also be exhibiting on the showroom floor throughout AGS at booth #B6.
In addition, key surgeon poster presentations available for viewing on March 14, 2019 include:
- One-Year Outcomes of Combined Phaco and Collector Channel Targeting with Two Second-Generation Trabecular Bypass Stents (iStent inject®) (PO001)
Author: Paul Harasymowycz, MD - Trabecular Micro-Bypass Stent Implantation with Cataract Extraction in Pseudoexfoliative Glaucoma: Long-Term Results (PO004)
Authors: Tanner J. Ferguson, MD; Vance Thomson, MD; John Berdahl, MD - Five-Year Outcomes of a Study Evaluating One, Two or Three Trabecular Micro-Bypass Stents for Open-Angle Glaucoma (OAG) (PO017)
Author: Oluwatosin U. Smith, MD - Three-Year Outcomes of Phacoemulsification with Implantation of Two First-Generation Trabecular Micro-Bypass Stents in Patients with OAG (PO018)
Authors: Lucy Ma, Won I. Kim, MD - Investigating Pre-Operative Factors that Predict Trabecular Meshwork Bypass Procedure Outcomes (PO022)
Authors: Jason Flamendorf, MD; Jonathan S. Myers, MD; Daniel Lee, MD - Long-Term Performance of Second-Generation Trabecular Micro-Bypass Stents (iStent inject) Implanted in Patients with Glaucoma: Four-Year Outcomes (PO025)
Authors: Fritz H. Hengerer, MD; Jay L. Katz, MD - Intermediate-Term Outcomes of First-Generation Trabecular Micro-Bypass Stents and Second-Generation Stents in Combination with Phacoemulsification (PO026)
Authors: Taylor T. Lukasik, MD; Ike K. Ahmed, MD - Single-Surgeon Experience with iStent® Trabecular Micro-Bypass Implanted in Conjunction with Cataract Surgery: Long-Term Outcomes (PO035)
Authors: Richard A. Supnet, MS; Mark J. Gallardo, MD - Medium-Term Outcomes of Multiple Trabecular Micro-Bypass Stents in Combination with Phacoemulsification (PO038)
Authors: Nicholas H. Andrew, MBBS; Ike K. Ahmed, MD - A Prospective, Randomized Pivotal Study of Second-Generation Trabecular Micro-Bypass Stents (iStent inject) Implanted in Conjunction with Cataract Surgery (PO042)
Author: Inder P. Singh, MD - Evaluating Minimally Invasive Glaucoma Surgery with Trabecular Micro-Bypass Stents in African American Glaucoma Patients (PO044)
Authors: Albert Bargoud, BA; Selena J. An, MSPH; Leon W. Herndon, MD; Albert S. Khouri, MD
Glaukos pioneered MIGS, which involves insertion of a micro-scale device from within the eye’s anterior chamber through a small corneal incision. Glaukos MIGS devices are designed to reduce IOP by restoring the natural outflow pathways for aqueous humor. In 2012, Glaukos received U.S. Food and Drug Administration (FDA) approval and launched its first MIGS device, the iStent Trabecular Micro-Bypass Stent.
Kayak soothes nervous Boeing flyers with airplane search filte
Travel website Kayak is making changes to let customers exclude specific aircraft types from searches, and booking sites are looking to reroute passengers, after an unexplained Boeing jet crash that killed 157 people in Ethiopia.
The first move by one of the big U.S. travel websites to adapt its service came as hundreds of jittery customers of Southwest, United and American Airlines took to social media to seek flights on planes other than the Boeing 737 MAX, which was involved in the fatal crash on Sunday.
Other travel agents and websites reported a raft of re-bookings as a result of cancellations caused by the grounding of two-thirds of 737 MAX jets by a long list of global authorities and airlines outside North America.
“We’ve recently received feedback to make Kayak’s filters more granular in order to exclude particular aircraft models from search queries,” a spokeswoman for the website told Reuters in an email responding to questions.
“We are releasing that enhancement this week and are committed to providing our customers with all the information they need to travel with confidence.”
Kayak is a sister site of Booking.com and Agoda.com, used by millions of travelers each year to book flights and hotels.
Carlson Wagonlit Travel, which manages travel for big global businesses, said some clients had asked to explore the possibility of temporarily restricting travel on Boeing 737 MAX 8 planes.
“This could potentially result in travelers being left with fewer flight options and susceptible to higher airfares, depending on the route,” CWT said. “We are working with clients to understand the practicality and the implications of putting such restrictions in place.”
The Twitter accounts of Southwest and American Airlines were swamped with customers asking for refunds or to be moved to other flights, and complaints about long hold times before being connected to a representative.
Twitter user @johnpauli6 wrote to Southwest that he does not want to be on a Boeing 737 MAX 8 when he travels with his wife on Saturday. “Get ready for a lot of lawsuits” if anything happens, the user said.
American Airlines Group Inc said late on Wednesday it was working to re-book customers due to fly on the 737 MAX as quickly as possible after the United States banned the aircraft.
Southwest, the world’s largest operator of the 737 MAX 8 with 34 jets, said it is waiving any fare-difference charges for customers who wish to switch to another aircraft following bans across much of the world.
Boeing has been mentioned more than 590,000 times on Twitter, a 2,300 percent increase from median daily mentions figures.
The crash in Ethiopia was the second in the last five months involving a Boeing 737 MAX 8. Last year, a Lion Air jet went down in Indonesia, killing 189 people.
Though there is no proof of links, multiple nations and the European Union have suspended the 737 MAX.
Boeing has said it has no reason to doubt the airplane, and Southwest and American have both said their fleet data showed the plane was safe.
“While the situation has not stopped people traveling on business, many want to know what plane they’ll be flying on,” said a spokesman for American Express Global Business Travel, another major corporate provider.
“When a traveler books a trip, we know at that moment what aircraft type is assigned to service the flight. However, that can change at an airline’s discretion up until the time of departure.”
‘PHONE CALLS KEEP COMING’
Temporary bouts of nerves among travelers are a familiar part of the reaction to major crashes, and agents booking travel for major executives have remained cautious about making changes to booking systems in response to the Boeing row.
Norwegian travel agent Berg-Hansen, dealing with cancellations of flights on Norwegian Air’s 737 MAX planes, said clients were mainly concerned with whether their flights were still scheduled to fly and the need to re-book if so.
“We have increased our staff from last night, through the night and now,” Berg-Hansen Chief Executive Officer Per-Arne Villadsen said.
“Remarkably we have less phone calls than we expected, although they are more than usual. We had around 100 phone calls from midnight to 7 a.m. this morning and they keep coming.”
He said the company was using alternative airlines including SAS, AirFrance and KLM to re-book and that he believed customers would come back to Norwegian Air if they reopened flights on the MAX in the future.
“We can confirm a rising interest on the aircraft types we are operating,” a spokesman for Swedish-based SAS said. “I can guarantee you that we have not even considered changing any pricing policy due to the tragic situation.”
Viking 2018 financials and corporate update
Viking Therapeutics, Inc. (Viking) (NASDAQ: VKTX), a clinical-stage biopharmaceutical company focused on the development of novel therapies for metabolic and endocrine disorders, today announced its financial results for the fourth quarter and year ended December 31, 2018, and provided an update on its clinical pipeline and other corporate developments.
Highlights from the Quarter, and Subsequent to December 31, 2018:
“The past year has been a transformative period for Viking, and we are excited to continue this momentum in 2019,” stated Brian Lian, Ph.D., chief executive officer of Viking Therapeutics. “We were particularly pleased in 2018 to have presented positive Phase 2 data from our novel thyroid receptor beta agonist VK2809 in non-alcoholic fatty liver disease at the AASLD conference, and honored that these results were featured in the Best of AASLD conference highlights. We plan to present additional, new results from this study at the upcoming EASL conference in April, and remain on-track to file an IND to initiate a study in biopsy-confirmed NASH later this year. In addition, pre-IND work for our second thyroid receptor agonist VK0214 continues to progress, and we plan to file an IND to initiate clinical development by the end of the year. Thanks to successful fundraising efforts in 2018, we ended the year with more than $300 million on our balance sheet, and are now capitalized to reach major inflection points for multiple programs. Entering 2019, we are focused on executing our operating plans and enthusiastic about the future development of our pipeline.”
Pipeline and Corporate Highlights
- Phase 2 study of VK2809 in patients with NAFLD and elevated LDL-C highlighted at 2018 AASLD. VK2809 is a novel, orally available small molecule thyroid receptor antagonist that possesses selectivity for liver tissue, as well as the beta receptor subtype, suggesting promise in certain metabolic and liver diseases, including non-alcoholic steatohepatitis (NASH). In November 2018, the company’s positive top-line results from a 12-week Phase 2 study of VK2809 were presented at the annual meeting of the American Association for the Study of Liver Diseases (AASLD) as part of the Late-Breaking Abstract Oral Session. The trial findings were also named to the Best of AASLD, which highlighted the contributions of particular importance at the annual meeting. The company is currently preparing to initiate a Phase 2b study of VK2809 in biopsy-confirmed NASH, which is anticipated to begin in the second half of 2019.
- Additional VK2809 Phase 2 data to be presented at the Late-Breaker poster session of the upcoming 2019 EASL conference. The company recently received additional data from the low-dose 5 mg cohort in the Phase 2 trial of VK2809 trial in NAFLD and hypercholesterolemia. The results demonstrated that patients receiving VK2809 experienced statistically significant reductions in liver fat content relative to placebo, as well as statistically significant improvements in response rates, defined by the proportion of patients experiencing at least a 30% or 50% relative reduction in liver fat, compared with placebo. Consistent with prior data from the 10 mg cohorts, VK2809 was well tolerated when dosed at 5 mg daily, and no serious adverse events were reported among patients receiving either VK2809 or placebo. These results will be presented at the Late-Breaker poster session at the upcoming annual meeting of the European Association for the Study of Liver (EASL), April 11-14, 2019 in Vienna, Austria.
- IND-enabling work for VK0214 in X-linked adrenoleukodystrophy (X-ALD) progressing, IND filing planned in 2019. VK0214 is a novel, orally available small molecule thyroid receptor agonist that possesses selectivity for the beta receptor subtype. The company is continuing to progress VK0214 through IND-enabling work, with a current goal of filing an IND in 2019 to allow initiation of a proof-of-concept study in X-ALD.
- Phase 2 study results of VK5211 in patients recovering from hip fracture highlighted at ASBMR 2018 Annual Meeting. VK5211 is an orally available, non-steroidal selective androgen receptor modulator (SARM) designed to selectively stimulate muscle and bone formation with reduced activity in peripheral tissues such as skin and prostate. In the fourth quarter, Viking announced positive new findings from the company’s Phase 2 trial of VK5211 in patients recovering from hip fracture during the plenary session of the Annual Meeting of the American Society for Bone and Mineral Research (ASBMR). In addition, the abstract received the 2018 Most Outstanding Clinical Abstract Award from the conference organizers. Among the notable results from this study was the observation that patients receiving VK5211 gained weight, added muscle, and lost fat content compared with patients receiving placebo. The company is continuing to explore partnering and licensing opportunities that will allow us to optimize the value of VK5211.
- Balance sheet sufficient to support operations through at least 2021. In 2018, Viking raised approximately $315 million in gross proceeds through the issuance of common stock, and completed 2018 with approximately $302 million in cash, cash equivalents, and short-term investments. The company currently expects these resources to be sufficient to support operations through at least the 2021 timeframe.
- Upcoming investor events. Viking management will participate in the following March investor events:
31st Annual ROTH Conference
Updated Time/Date: 12:00 p.m. PT on Monday, March 18, 2019 (webcast available)
Location: Ritz-Carlton Laguna Niguel, Dana Point, CA
Room: Pink – Salon 5
Updated Time/Date: 12:00 p.m. PT on Monday, March 18, 2019 (webcast available)
Location: Ritz-Carlton Laguna Niguel, Dana Point, CA
Room: Pink – Salon 5
Oppenheimer’s 29th Annual Healthcare Conference
Time/Date: 11:30 a.m. ET on Wednesday, March 20, 2019
Location: Westin New York Grand Central Hotel, New York
Room: Consulate Room
Time/Date: 11:30 a.m. ET on Wednesday, March 20, 2019
Location: Westin New York Grand Central Hotel, New York
Room: Consulate Room
Q4 and Full-Year 2018 Financial Highlights
Fourth Quarter Ended December 31, 2018 and 2017
Research and development expenses for the three months ended December 31, 2018were $5.1 million compared to $3.0 million for the same period in 2017. The increase was primarily due to increased pre-clinical study efforts, manufacturing expenses related to our drug candidates, use of third party consultants and stock-based compensation, partially offset by a decrease in clinical study expenses.
General and administrative expenses for the three months ended December 31, 2018were $1.9 million compared to $1.4 million for the same period in 2017. The increase was primarily due to increased expenses related to stock-based compensation, salaries and benefits, and legal and patent expenses.
For the three months ended December 31, 2018, Viking reported a net loss of $5.2 million, or $0.07 per share, compared to a net loss of $4.1 million, or $0.14 per share, in the corresponding period in 2017. The increase in net loss for the three months ended December 31, 2018 was primarily due to the increase in research and development expenses noted previously, partially offset by an increase in other income related to the increase in interest income. The decrease in net loss per share for the three months ended December 31, 2018 is primarily driven by the additional shares outstanding at December 31, 2018 versus those outstanding at December 31, 2017, given the additional shares issued by the Company during 2018, primarily through public equity offerings.
Twelve Months Ended December 31, 2018 and 2017
Research and development expenses for the twelve months ended December 31, 2018 were $19.0 million compared to $13.7 million for the same period in 2017. The increase was primarily due to increased expenses related to pre-clinical study efforts, use of third party consultants, stock-based compensation, and manufacturing related to our drug candidates, partially offset by a decrease in clinical study expenses.
General and administrative expenses for the twelve months ended December 31, 2018 were $7.1 million compared to $5.3 million for the same period in 2017. The increase was primarily due to increased expenses related to stock-based compensation, salaries and benefits, professional services, use of third party consultants, insurance, legal and patent expenses, and franchise taxes.
For the twelve months ended December 31, 2018, Viking reported a net loss of $22.1 million, or $0.38 per share, compared to a net loss of $20.6 million, or $0.79 per share, in the corresponding period in 2017. The increase in net loss for the twelve months ended December 31, 2018 was primarily due to the increase in research and development expenses and general and administrative expenses noted previously offset by an increase in other income related to the decrease in the fair value of the debt conversion feature liability, as well as an increase in interest income. The decrease in net loss per share for the twelve months ended December 31, 2018 is primarily driven by the additional shares outstanding at December 31, 2018 versus those outstanding at December 31, 2017, given the additional shares issued by the Company during 2018, primarily through public equity offerings.
FDA Broadens Cancer Trial Eligibility With 5 New Guidance Documents
Scott Gottlieb, the commissioner of the U.S. Food and Drug Administration (FDA) may be on his way out, but the agency continues its activities. The FDA, as part of efforts to update clinical trial eligibility criteria, published four draft guidance documents on cancer clinical trial criteria and one final draft on adolescents in adult oncology trials.
The four draft guidances were developed by the FDA with feedback from the American Society of Clinical Oncology and Friends of Cancer Research. The focus is largely on what the minimum age should be for pediatric patients, guidelines related to patients with HIV, hepatitis B or C, with organ dysfunction or previous cancers, and patients with cancer that has spread to the brain.
“Overly restrictive eligibility criteria may slow patient accrual, limit patients’ access to clinical trials and lead to trial results that don’t fully represent treatment effects in the patient population that will ultimately receive the drug,” stated Gottlieb.
The guidance on minimum age for pediatric patients describes how sponsors of clinical trials that want to include pediatric patients in adult cancer trials should evaluate pediatric formulations, accounting for age, size, physiologic condition and the treatment needs of the patients. This focuses on patients aged 2 years to 12, noting—and this seems obvious—that children under the age of two “may be particularly vulnerable to expected and unanticipated toxicity due to developmental concerns and age-dependent maturation of metabolic enzyme systems and organ functions,” and should not be included in adult cancer trials, except for in rare cases.
For patients with HIV, and Hepatitis B and C infections, the guidance tries to be more inclusive for this patient population in cancer trials, noting that it “is justified in many cases, and may accelerate the development of effective therapies in cancer patients with these chronic infections.”
In terms of the guidelines for organ dysfunction or previous or ongoing cancers, the agency’s eligibility criteria recommendations focus on the patients’ renal function, cardiac function and hepatic function. “By excluding individuals from cancer clinical trials who have major organ dysfunction or previous or concurrent cancers, trial recruitment favors younger patients, which may not be fully representative of the population for whom the drug will be indicated,” the FDA stated.
The guidelines for brain metastases noted that about 70,000 U.S. cancer patients are diagnosed with brain cancers. They discuss how these patients can be included in clinical trials, stating, “The incidence of brain metastases is increasing in patients with certain malignancies such as melanoma, lung cancer, and breast cancer. However, the patients with brain metastases have historically been excluded from clinical trials due to concerns of poor functional status, shortened life expectancy, or increased risk of toxicity.”
And finally, the final guidance on including adolescent patients in adult cancer trials gave recommendations for appropriate inclusion criteria, dosing and pharmacokinetic (PK) evaluations, safety monitoring, and ethical requirements. In particular, the agency says that this cohort should only be enrolled in first-in-human or dose-escalation trials after the first adult PK and toxicity data are acquired, and only in early phase trials if they have relapsed or refractory cancer, or a cancer with no standard curative treatments available.
On the one hand, you can argue, as the agency seems to be doing, that the earlier guidelines weren’t completely based on clear scientific or clinical rationales, so the new guidelines were long overdue. On the other hand, biopharma companies have an incentive to select patient populations that not only provide the best chances of a drug’s success, but also provide the cleanest, most easily analyzed data for a specific disease without possible complications from trial participants with other conditions and treatment histories. However, the new guidelines will likely make it easier for patients to gain entry to clinical trials and provide much-needed clarity for pediatric and adolescent patients’ participation in adult clinical cancer trials.
The guidelines for adolescents are final. The other four are draft guidance and as yet are not binding.
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