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Monday, December 2, 2019

Go or no go? Vascepa’s finale and Allergan’s pain relief

Amarin’s Vascepa and Allergan’s migraine project ubrogepant are the high-profile pending FDA approval decisions as the sector heads into the last month of the year.
No one could have predicted that November would see such a wave of early approvals by the FDA. Two sickle cell drugs – Adakveo and Oxbryta – the BTK inhibitor Brukinsa and RNAi therapy Givlaari all won through months ahead of schedule.
Whether the streak continues is questionable and December is often a quiet month. Some of the more interesting upcoming decisions are supplemental approvals, the most highly anticipated of which is Amarin’s Vascepa. The purified fish-oil product is looking for a broader label, mentioning cardiovascular risk reduction, after gaining a unanimous vote by an FDA advisory panel in November.
Just how broad a label it might get is a big question. Restriction to a secondary prevention setting, namely only those patients with established cardiovascular disease, is possible. A benefit was seen in a primary prevention setting – those considered at risk of future cardiac problems – but this was much less pronounced.
For now, consensus from EvaluatePharma shows 2024 sales at $2.2bn, giving an NPV of $5.9bn; if Vascepa obtains a cardiovascular risk reduction label analysts believe that its sales could peak at double that figure.
Another notable supplemental approval expected this month is that of Lynparza in a maintenance setting in pancreatic cancer. Results from the Polo trial strongly favoured the Astrazeneca/Merck & Co Parp on progression-free survival, but this was overshadowed by the absence of an overall survival benefit. Few successes have been seen in pancreatic cancer, and as current therapies involve toxic platinum chemotherapies the regulator might look favourably on Lynparza’s offering.
Meanwhile Pfizer will hope to gain ground on its prostate cancer rival J&J: it is due an approval decision on Xtandi in metastatic hormone-sensitive disease, for which J&J’s Erleada was approved in September.
SUPPLEMENTARY AND OTHER NOTABLE APPROVAL DECISIONS DUE IN DECEMBER
ProductCompanyEvent typeDate
TecentriqRochesBLA + Abraxane for first-line non-squamous NSCLC (Impower-130)Dec 2
ABP 710AmgenBiosimilar RemicadeDec 14
VascepaAmarinsNDA for Cardiovascular outcomes (Reduce-It)Dec 28
XtandiPfizer/Astellas PharmasNDA for metastatic hormone sensitive prostate cancer (Arches)Q4
LynparzaAstrazenecasNDA for BRCAm pancreatic cancer (Polo)Q4
Source: EvaluatePharma.
First-timers
Allergans’s oral CGRP antagonist ubrogepant will likely gain approval in an acute migraine setting by the end of the year. The product is backed by positive late-stage trials and has a clean safety profile. Ubrogepant is Allergan’s most valuable pipeline project with 2024 sales forecast to reach $302m, according to EvaluatePharma consensus.
Hot on ubrogepant’s heels is Biohaven’s orally dissolving tablet formulation of rimegepant, also known as Zydis ODT, with a PDUFA date in the first quarter of next year.
A decision is due for Shionogi/GSK’s HIV doublet cabotegravir and rilpivirine, a long-acting injectable treatment dosed monthly. Concerns about drug resistance mean that many remain to be convinced by the doublet strategy; three-drug regimens represent the current standard of care and Gilead’s oral once-daily triplet Biktarvy dominates the market with forecasts of $9.1bn by 2024, according to EvaluatePharma consensus.
Enzyvant’s RVT-802 is a one-off treatment for the severe immune deficiency that results from rare childhood diseases, such as complete DiGeorge anomaly. The regenerative therapy could be the first approval for Vivek Ramaswamy’s Roivant stable of companies. A strategic alliance with Sumitomo Dainippon that includes Enzyvant was announced in September.
Less likely to receive the FDA’s backing will be Intra-Cellular’s lumateperone in schizophrenia. A decision is now due after a three-month delay to a previous PDUFA date after the FDA cancelled an advisory meeting and requested further preclinical data to address toxicity findings in previous animal studies; phase III results have also been mixed.
NOTABLE FIRST-TIME US APPROVAL DECISIONS DUE IN DECEMBER
ProjectCompanyPDUFA dateProduct NPV ($m)
UbrogepantAllerganDecember701
RVT-802EnzyvantDecember–
AV001AvadelDec 156
IDP-123/tazaroteneBausch Health CompaniesDec 223
BrinavessCorrevio PharmaDec 24 (advisory committee Dec 10)504
LumateperoneIntra-Cellular TherapeuticsDec 272,046
LemborexantEisaiDec 27617
Cabotegravir & rilpivirineShionogi/GSKDec 291,792
Source: EvaluatePharma.
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Keeping corporations healthy is good business for Livongo

Livongo Health, which launched a highly successful IPO in July, claims to be one of the fastest-growing companies in Silicon Valley. Can it keep investors happy as growth rates slow?
Digital health group Livongo has pioneered a new business model, convincing multinationals, government bodies and pharmacy benefit managers to pay for its technology, which enables users to manage chronic conditions. This approach seems to have worked, at least so far, with its third quarter revenues topping expectations.
Its investors are as keen as its customers; this summer Livongo conducted one of the largest IPOs in medtech history, going out at a premium to its preannounced range. But it has yet to turn a profit. Perhaps as importantly, it could struggle to maintain its current growth trajectory.
Applying themselves
Several companies offer apps intended to help patients manage various disorders – Glooko’s diabetes-focused software, for example, or Pear Therapeutics’ addiction management apps. But Livongo’s technology, which covers conditions including diabetes and hypertension, is paid for by employers – and even then, only if the employees actually use it.
“In the US, the person with the chronic condition doesn’t always pay for it, so they don’t have a huge incentive to reduce that cost,” Glen Tullman, Livongo’s executive chairman, tells Vantage. “The person who does pay for it in the US is either large self-insured employers – big businesses – or the government.”
Livongo’s pitch is that its technology can keep a company’s workforce healthier, reducing the insurance costs incurred by sickness and boosting productivity since staff will require less time off. Mr Tullman says it has signed up more than 770 organisations, including “almost 25% of the Fortune 500”.
The technology itself is a combination of hardware – continuous glucose monitors for diabetes patients, sphygmomanometers for the blood pressure programme – and smartphone and tablet apps. Users are monitored and receive “nudges” to take their medication, for example, and they can also talk to or message coaches, who advise on nutrition and lifestyle as well as disease-specific matters.
“We’re taking information from people’s bodies, we’re aggregating all that information,” says Mr Tullman. “It might be from our meters [or] from a watch, from their pharmacy, from their electronic health record. And then we’re putting it back to them in the form of digital insights and nudges.”
The tech will also monitor patients’ use of consumables such as glucose test strips, and re-order them automatically when needed. And there is one further step: it tracks whether the nudging and advising actually improves users’ health. If it doesn’t, a new approach will be tried.
Livongo’s customers, the corporations and government groups, are only charged for active users.
“It is a subscription model, customers pay a monthly fee, but they only pay when people keep using it. We call it per participant per month, not per member per month,” Mr Tullman says.
The Amazon of healthcare?
The popularity of Livongo as a technology provider seems to be looking good, at least for now. Its popularity as an investment proposition has been more easy to track.
The group’s public debut was little short of spectacular; it started out by boosting the size of its IPO from an initial figure of $246m to $278m – and then beat even that, realising proceeds of $355m (Livongo enters the medtech float hall of fame, July 25, 2019). It closed its first day of trading up 36%.
Since then the stock’s value has slid, though a rally over the last month or so has left the shares slightly up from the IPO price. Mr Tullman says that one thing that drove investors’ demand was the prevalence of chronic conditions: in the US they for 90% of US healthcare expenditure – more than $1tn per year.
Livongo has certainly grown strongly: its revenues expanded 122% from 2017 to 2018, and 134% from 2018 to 2019.
LIVONGO AT A GLANCE
 WW annual sales ($m) 
Segment20182020e2022e2024eCAGR
Diabetes65226469759+51%
Hypertension12087156+158%
Prediabetes and weight management3142642+53%
Behavioural health–122746N/A
Total Company Revenues682736101,003+56%
Source: EvaluateMedTech.
The pace is forecast to slow, however, according to data compiled by EvaluateMedTech. Then there is the small matter of translating revenue into profit. The group is loss-making, and will remain so for some time, Mr Tullman says.
“If today we stopped investing … we would be highly profitable, because it’s a highly profitable business model. But because we’re growing so fast, that requires us to deliver great service, and that requires us to invest.”
He says the company expects to become profitable on the Ebitda line in 2021. Its investors, keen as they are, might not want to choose between growth and profit.
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PDS Bio up 16% ahead of Versamune presentation

Thinly traded nano cap PDS Biotechnology (PDSB +15.5%) is up on modestly higher volume, 100K shares, on no particular news.
On Wednesday, December 4, CMO Lauren Wood, M.D. will deliver a presentation on cancer immunotherapy platform Versamune at the World Vaccine & Immunotherapy Congress West Coast in San Francisco at 3 pm PT.
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Epizyme down ahead of tazemetostat FDA panel review

Epizyme (EPZM -5.9%) slips on average volume as broad market selling weighs on shares. The stock had rallied over 70% since early October before today’s action.
The FDA’s Oncologic Drugs Advisory Committee will meet on Wednesday, December 18, to review and discuss the company’s marketing application seeking accelerated approval of tazemetostat for metastatic or locally advanced epithelioid sarcoma patients not eligible for curative surgery.
The agency’s action date is January 23, 2020.
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Blueprint Medicines down 7% on bearish call at Deutsche Bank

Blueprint Medicines (BPMC -6.8%) was down as over 9% intraday in apparent response to a new “Sell Catalyst Call” at Deutsche Bank. Analyst Konstantinos Aprilakis lacks confidence that initial data from a Phase 2 study, PIONEER, evaluating avapritinib in a rare disorder called indolent systemic mastocytosis will be positive.
In late October, shares briefly sold off on the news that the FDA’s review of its application for avapritinib in third- and fourth line gastrointestinal stromal tumor (GIST) will likely be delayed past the February 14 2020 PDUFA date since the agency wants to include topline results from the Phase 3 VOYAGER trial, expected to be available in Q2 2020.
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CryoLife’s E-nya stent CE Mark’d

CryoLife (CRY -1.3%) announces CE Mark certification for its E-nya thoracic stent graft system for the minimally invasive repair of lesions of the descending thoracic aorta.
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Concert Pharma launches mid-stage study of CTP-692 in schizophrenia

Concert Pharmaceuticals (CNCE -3.4%) initiates a Phase 2 clinical trial evaluating CTP-692 for the adjunctive treatment of schizophrenia.
The primary endpoint is the change from baseline in a schizophrenia symptom scale called PANSS at week 12. Topline results should be available in about a year.
CTP-692 is a deuterium-modified form of an amino acid called D-serine. The company says it has the potential to restore NMDA receptor activity in key areas of the brain in schizophrenia sufferers, adding that it will be initially developed as an adjunctive therapy with standard antipsychotic medications.
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