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Wednesday, July 31, 2019

PRA Health Sciences EPS beats by $0.05, misses on revenue

PRA Health Sciences (NASDAQ:PRAH): Q2 Non-GAAP EPS of $1.22 beats by $0.05; GAAP EPS of $0.62 misses by $0.22.
Revenue of $763.31M (+5.6% Y/Y) misses by $1.64M.
Shares +1.24%.

Cross Country Healthcare EPS beats by $0.01, beats on revenue

Cross Country Healthcare (NASDAQ:CCRN): Q2 Non-GAAP EPS of $0.01 beats by $0.01; GAAP EPS of -$1.44 misses by $1.43.
Revenue of $202.76M (-0.9% Y/Y) beats by $3.3M.

China drugmaker Chipscreen’s STAR Market IPO sees feverish demand

Shenzhen Chipscreen Biosciences, which plans to list on China’s red-hot new tech board, said its newly issued shares were almost 3,000-times oversubscribed among retail investors, despite an eye-popping offer pricing of 468-times earnings.

While the scramble reflects a strong appetite for listings on Shanghai’s Nasdaq-style STAR Market, which was launched in June, it also underlines the challenge for bankers to value IPOs for tech start-ups as regulators let market prices play their role in setting prices.
Chipscreen, which develops original drugs for tumours and diabetes, said in an exchange filing on Thursday that shares reserved for individual investors – accounting for about one fifth of the IPO – were 2,956.25 times oversubscribed.
The company has set the price for its initial public offering (IPO) at 20.43 yuan per share, or 467.51 times 2018 earnings, the highest so far for a STAR Market company.
In contrast, China-listed pharmaceutical firms, traded at an average earnings multiple of 30.79 over the past month.
The feverish demand values Chipscreen at 8.38 billion yuan ($1.21 billion), and would allow the company to raise 1 billion yuan – 27% more than originally planned.
In a roadshow on Tuesday, Chipscreen founder Lu Xianping said the company will use the IPO proceeds to “strengthen competitiveness, expand market share, and develop new products in a bid to make growth sustainable”.
Chipscreen’s share offering came a week after the frenzied debut of the first batch of 25 companies on the science and technology board on July 22. The companies surged roughly 140% on average on that day.
Chipscreen has not disclosed when it will debut on the STAR Market, which has attracted a slew of Chinese drugmakers to list. One drug developer, Suzhou Zelgen Biopharmaceuticals Co, plans to list before it sells any products to patients, potentially testing investors’ risk tolerance.
The new tech board potentially competes with Hong Kong, which last year revised listing rules to allow so-called pre-revenue, or pre-profit biotech firms to go public in the city.
Under the new listing regime, 10 biotech firms have floated in Hong Kong as of May, raising over HK$33 billion ($4 billion).
Shares of Ascletis Pharma, the first biotech firm to list in Hong Kong under the new rules, are trading about 66% below is IPO price, while performances of the rest are mixed.

Biotech Investors: Mark Your Calendar For These August FDA Dates

Though light in terms of PDUFA events, July yielded mostly positive results. Quiet a few approvals came through, including Eli Lilly And Co LLY 0.25%‘s nasally administered low blood sugar drugKaryopharm Therapeutics Inc KPTI 0.84%‘s Selinexor and a handful of generic drugs.
With three new molecular entity approvals in July, the total number of NME approvals this year rose to 15, which pales before the 25 approvals that came through the same time last year.
PDUFA dates are deadlines for the FDA to review new drugs. The FDA is normally given 10 months to review new drugs. If a drug is selected for priority review, the FDA is allotted six months to review the drug. These time frames begin on the date that an NDA is accepted by the FDA as complete.
Here are the key PDUFA catalysts for the upcoming month.

Kala Eyes Drug Approval

  • Company: Kala Pharmaceuticals Inc KALA 0.34%
  • Type of Application: NDA
  • Candidate: KPI-121, 0.25%
  • Indication: dry eye disease
  • Date: Aug. 15
KPI-121, 0.25%, is Kala’s candidate for the temporary relief of signs and symptoms of dry eye disease using a two-week course therapy. It will likely also work for dry eye flare.
The company said its product candidate, if approved, will be the first safe and effective short-term treatment option for dry eye disease.

Vanda Sleepless Over Jet Lag Disorder Approval

  • Company: Vanda Pharmaceuticals Inc. VNDA 1.58%
  • Type of Application: sNDA
  • Candidate: Hetlioz
  • Indication: jet lag disorder
  • Date: Aug, 16
Vanda’s Hetlioz is a melatonin receptor agonist, which was initially approved in 2014 to treat non-24-hour sleep-wake disorder in totally blind individuals.
Non-24 is a chronic body clock disorder in the blind that causes problems with the timing of sleep.
The company’s sNDA for the drug was accepted in December of 2018, with the PDUFA date fixed for Aug. 16. However, the company said July 22 it received a notification from the FDA that the sNDA has deficiencies that preclude discussion of labeling and post-marketing requirements/commitments, although the agency did not disclose what the deficiencies were.
The FDA, however, said the notification does not reflect a final decision on the information under review.

Roche Seeks Approval For Personalized Cancer Medicine

  • Company: Roche Holdings AG Basel ADR RHHBY 0.21%
  • Type of Application: NDA
  • Candidate: Entrectinib
  • Indication: solid tumors and non-small cell lung cancer, or NSCLC
  • Date: Aug. 18
Entrectinib is being evaluated for treating adult and pediatric patients with neurotrophic tropomyosin receptor kinase, or NTRK, fusion-positive locally advanced or metastatic solid tumors as well as for the treating people with metastatic, ROS1-positive NSCLC.
“By combining comprehensive genomic profiling with actionable targeted therapies, like entrectinib, we are advancing our personalised healthcare goal to find the right treatment for each patient,” the company said in its release announcing FDA acceptance of the NDA.

Will Nabriva’s Antibiotic Pass The FDA Muster?

  • Company: Nabriva Therapeutics PLC – ADR NBRV 0.63%
  • Type of Application: NDA
  • Candidate: intravenous and oral formulations of Lefamulin
  • Indication: community-acquired bacterial pneumonia, or CABP
  • Date: Aug. 19
Lefamulin is a semi-synthetic pleuromutilin antibiotic evaluated for CABP. The NDAs – for the IV and oral formulations – have a priority review status, Fast Track designations as well as Qualified Infectious Disease Product designations.
While accepting the application, the FDA said it hasn’t planned an advisory committee meeting to discuss the applications.

AbbVie’s Rheumatoid Arthiritis Drug

  • Company: AbbVie Inc ABBV 0.82%
  • Type of Application: NDA
  • Candidate: upadacitinib
  • Indication: rheumatoid arthritis
  • Date: Q3 (likely Aug. 19/20 assuming a six-month review period)
AbbVie’s upadacitinib is an investigational once-daily JAK1-selective inhibitor being studied for multiple immune-mediated diseases. The company is now seeking approval of the drug for treating adult patients with moderate to severe rheumatoid arthritis.

Sarepta Seeks Approval Of Drug For DMD Patients With Specific Mutations

  • Company: Sarepta Therapeutics Inc SRPT 0.58%
  • Type of Application: NDA
  • Candidate: Golodirsen
  • Indication: Duchenne muscular dystrophy, or DMD
  • Date: Aug. 19
Golodirsen is being studied for the treatment of exon 53 amenable patients, who account for about 8% of DMD patients. It’s a phosphodiamidate morpholino oligomer engineered to treat DMD patients who have generic mutations, subject to skipping exon 53 of the dystrophin gene.

Can Second Time Be Charm For IntelliPharmaCeutics?

  • Company: IntelliPharmaCeutics International Inc. IPCIF 3.01%
  • Type of Application: NDA
  • Candidate: Rexista
  • Indication: abuse-deterrent pain medication
  • Date: Aug. 28
Rexista is Oxycodone Hydrochloride extended-release tablets. The FDA issued a complete response letter to the original NDA in September of 2017, with the agency providing recommendations and requesting for information, including the relevant Category 2 and Category 3 studies to assess the abuse-deterrent properties of Oxycodone ER by the oral and nasal routes of administration.
The company resubmitted the application March 4, 2019, with the FDA accepting the resubmitted application on March 29, deeming it to be a complete response.

Nektar Waits With Bated Breath For Opioid Pain Medication Approval

  • Company: Nektar Therapeutics NKTR 2.77%
  • Type of Application: NDA
  • Candidate: NKTR-181
  • Indication: chronic low back pain in adult patients new to opioid therapy
  • Date: Aug. 29
NKTR-181 is a novel mu-opioid analgesic drug candidate. On July 23, the company said it received a General Advice letter from the FDA regarding its NDA, communicating the postponement of an Adcom meeting scheduled for Aug. 21, as the regulatory agency continues to consider a number of scientific and policy issues relating to this class of drugs.
Although the postponement is not unique to NKTR-181, the FDA did say in the communication the PDUFA goal date may not be met.

HHS Outlines Goals for Possible Future Canadian Drug Imports

The Trump administration on Wednesday outlined ideas for potential future steps toward allowing importation of certain drugs from Canada into the United States.
The US Department of Health and Human Services (HHS) did not release a firm timeline for making formal proposals on drug importation. In fact, a future publication of draft regulatory proposals would mark only a first concrete step toward opening serious debate on ideas being considered by HHS.
It thus is unclear when — or if — consumers may get access to cheaper drugs through Canada due to any of the ideas HHS discussed Wednesday with reporters.
Still, HHS Secretary Alex Azar on Wednesday did emphasize the Trump administration’s support for the concept of allowing consumers to buy cheaper versions of pharmaceuticals in Canada.
Changes in the business landscape have prompted a new look at the question of importation, Azar said. With expanding international operations, drug distribution firms and pharmacy chains may be able to manage secure cross-border transactions, he said.
“We owe it to the American people to keep challenging our assumptions, challenging our orthodoxies, challenging our systems,” Azar told reporters during a telebriefing.
He said the announcement on drug importation represents an important “mindshift” in the Trump administration. HHS wants pharmacy chains, states, and wholesalers to build the case for importation, Azar said.
“We’re saying here’s the roadmap. We’re open,” he said. “Work with us and convince us that you have a game plan that can work.”

Two Pathways

The outline HHS released Wednesday, which the department calls its “action plan,” envisions two regulatory pathways for safe importation of drugs.
HHS and the US Food and Drug Administration (FDA) could use a notice of process called notice of proposed rulemaking (NPRM) to authorize demonstration projects. These could be developed by states, drug wholesalers, and pharmacists and submitted for HHS review.
The would-be test groups would make a case to HHS about how they could safely import certain drugs from Canada that are versions of FDA-approved drugs manufactured consistent with the FDA approval.
In a press release, HHS said its planned future NPRM for the demonstration projects would also seek “significant cost savings” from importation for consumers.
And the FDA could use what is called guidance to give recommendations to drug makers who might want to import into the United States versions of those drugs they sell in foreign countries. In theory, this second pathway would potentially broaden the chances for importation to include medications like insulin used to treat diabetes, as well as those used to treat rheumatoid arthritis, cardiovascular disorders, and cancer, HHS said.

“Too Dangerous”

Drug makers seem unlikely to embrace this option.
The pharmaceutical lobbying group issued an immediate criticism of HHS’ outline. In a statement, Stephen J. Ubl, chief executive officer of the Pharmaceutical Research and Manufacturers of America (PhRMA), called the aim of the HHS outline “far too dangerous for American patients.”
“Law enforcement has repeatedly warned that importation schemes could worsen the opioid crisis and jeopardize public safety,” Ubl said. “Moreover, Canadian officials have said that the policy is unworkable, and they will not risk shortages by diverting their medicine supply to the United States.”
John J. Leppard, an analyst with Washington Analysis, which advises investors about federal policy changes, wrote a Wednesday report on the HHS announcement. It was titled “Rx Pricing: The Red Herring of Reimportation & Supply Chain Implications.”
In the report, Leppard noted that the pharmaceutical industry is “unlikely to provide sufficient supply to foreign nations or pursue a demonstration project that would materially undercut its own domestic market and profitability.”
Leppard also observed that there is opposition in Canada to allowing more of the national drug supply to flow toward the United States. There have been reports of the nation’s medical system, Health Canada, preparing to take actions to ensure its citizens have uninterrupted access to the drugs they need, Leppard wrote.
“As reimportation has garnered greater attention in the U.S., Canadian officials have increasingly pushed back against the policy,” Leppard wrote.

Democratic Disapproval

The ranking Democrat on the health subcommittee of the Senate Finance Committee is also pushing back against the policy.
“This ‘action plan’ isn’t a real plan and lacks any real action. It sets up undefined ‘demonstrations’ that are limited in scope and it could take years to finalize rules, all while U.S citizens continue to pay the highest prescription drug prices in the world,” said Sen. Debbie Stabenow (D-MI), in a statement mailed to Medscape Medical News.
“If this Administration was serious about lowering drug prices, it should support passage of our legislation allowing Medicare to negotiate drug prices right now. The Administration should also support legislation I cosponsored, the Affordable and Safe Prescription Drug Importation Act, which will allow Americans to import safe and affordable prescription drugs,” she added.

Appeals court revives lab challenge of multibillion-dollar Medicare cut

A federal appeals court on Tuesday revived a group of clinical laboratories’ challenge to billions of dollars in lost Medicare revenue.
In a unanimous decision, the three-judge panel of the U.S. Court of Appeals for the District of Columbia Circuit overturned a lower court ruling that the laboratories couldn’t dispute the lost reimbursement funding. The Medicare changes stemmed from the Protecting Access to Medicare Act of 2014, or PAMA, which required certain clinical laboratories to give the CMS private payer data that could be used to set new reimbursement rates.
Although portions of the law aren’t reviewable by the courts, the D.C. Circuit determined it could review the data collection provisions and resurrected the American Clinical Laboratory Association’s lawsuit.
“Several features of the statute suggest that Congress meant to bar challenges to the ‘establishment of payment amounts’ but not to prevent review of the rule delineating the data collection practices that precede and inform the setting of those amounts,” U.S. Circuit Judge Cornelia Pillard wrote for the panel.
A District Court judge in September 2018 dismissed the case citing the judicial review clause of the law. The case will be sent back to the district court level for further review.
The ACLA, a trade group for clinical laboratories, sued the CMS in December 2017 over the planned cuts, alleging the agency ignored congressional intent and instituted a flawed data-reporting process.
The group’s president, Julie Khani, said in a statement that she was encouraged by the D.C. Circuit’s decision.
“HHS’ continued flawed data collection process poses a direct challenge to the rule of law and PAMA’s intent to support a sustainable, market-based laboratory market for millions of seniors,” she said. “On behalf of those who have already seen the consequences of this painful overreach, we urge the district court to act quickly to rule on the merits of ACLA’s case.”
The Congressional Budget Office in 2017 estimated PAMA would cut Medicare spending by $100 million in the first year of the new rates and $2.5 billion over a decade. However, that assumed that more labs would be surveyed under the clinical laboratory definition. Most hospital laboratories are exempt from the law.
Medicare’s fee schedule for lab tests has been largely unchanged since it was established in 1984. Each lab determines its own rates based on market prices. Medicare has historically paid 18% to 30% more than other insurers for some tests, HHS’ Office of Inspector General found. The program shells out about $7 billion a year for clinical diagnostic laboratory tests.

Congress panics on potential heparin shortage due to diseased China swine

A decade ago Congress was up in arms about Chinese crude heparin in the U.S. supply chain. Now member are highly concerned there may not be enough of it.
A group of six congressional leaders from both parties are asking FDA Acting Commissioner Norman Sharpless to give them some assurances the U.S. heparin supply is not in danger of drying up as a result of African swine fever which is wreaking havoc on pig herds in China, the primary source of crude heparin.
“U.S. dependence on Chinese heparin and on one animal source raises risks of shortages,” the representatives wrote in a letter to the Sharpless. “Pharmaceutical researchers are raising concerns that the African swine fever outbreak in China ‘has the potential to cause an unprecedented shortage of heparin’s raw material.'”
Crude heparin, which is used to manufacture the essential anticoagulant used in kidney dialysis and open heart surgeries, comes from pig intestines and China has reportedly already lost 150 million of its 440 million swine, the letter says.
FDA rules require heparin be manufactured only from pig intestines, because when “ruminant” animals like cattle are used, there is a chance the raw material could be contaminated with bovine spongiform encephalopathy or oversulfated chondroitin sulfate (OSCS), a cheap filler that saves money but that can be deadly to patients. In 2008, Chinese heparin contaminated with OSCS was tied to the deaths of 80 dialysis patients in the U.S. As recently as last November, European regulators banned a Chinese supplier after finding contamination risks
The committee members pointed out that, since the disease was discovered in Chinese herds last year, the country has lost a reported 150 million hogs to the outbreak. While there is no immediate indication that China’s hog herd problems are impacting the U.S. heparin levels, the letter points out that the current supply is already stressed. It has faced periodic shortages, like when a Baxter International plant was taken out of production by Hurricane Maria in 2017.
It was a decade ago that the FDA got whiplashed by Congressional leaders over tainted Chinese heparin in the U.S. supply. The investigation began after heparin batches were tied to hundreds of allergic reactions, some that proved fatal. The blood thinner had been tainted somewhere along the supply chain, which stretched into some mom-and-pop workshops in the Chinese countryside.

Gilead ups forecasts as new HIV drug boosts sales

Gilead has become the latest pharma company to raise its full year sales forecasts following stronger than expected Q2 results.
AstraZeneca and Sanofi have already upped their forecasts for this year following their Q2 results, and the US pharma is also on the front foot following higher sales of HIV treatments.
Gilead said it now expects 2019 sales of $21.6 billion to rise to $22.1 billion in 2019, up from its previous forecast of $21.3 billion to $21.8 billion.
Sales of HIV drugs accounted for 71% of total sales and rose to $4.04 billion from $3.67 billion last year.
This was driven by Biktarvy, a three-drug cocktail in a single pill that keeps HIV, designed to be more tolerable and convenient than its older combinations typically based around the “backbone” of Truvada plus another pill.
Biktarvy was only approved in the US in February last year, but is already a blockbuster with sales of almost $1.2 billion.
Hepatitis C drug sales continued their decline, caused by tough competition in a shrinking market where many patients have been cured over the last few years, thanks to drugs from Gilead and its competitors including AbbVie.
Sales fell to $842 million from $1 billion, but this was still better than the $714 million predicted by analysts.
Total revenue was up slightly to $5.69 billion, from $5.65 billion, ahead of analysts’ estimates of $5.53 billion. Net income was almost $1.9 billion, up 3.5% from last year’s Q2.
Gilead’s CAR-T cancer cell therapy Yescarta is also gathering momentum, with sales of $120 million in Q2, compared with $68 million in last year’s Q2.
The results come as new CEO Daniel O’Day is making his mark on the business by taking it in new directions.
O’Day has already made several changes to the company’s senior management team, last month poaching Christi Shaw from Eli Lilly to run its cancer immunotherapy unit, Kite.
Gilead has also invested $5.1 billion in the Belgian biotech Galapagos to develop inflammatory disease and rheumatoid arthritis drugs.

Alexandria Real Estate Equities Plans Another Boston Life Sciences Lab Facility

Alexandria Real Estate Equities is planning another life sciences development in Boston. The firm has 250 properties, about 25% of which are in the Boston area, with the rest in San Francisco, New York and San Diego.
Alexandria indicates that it could develop a site of up to 650,000 square feet at 5, 10 and 15 Necco Street in Boston’s Fort Point neighborhood. At the company’s recent second-quarter earnings call, Peter Moglia, co-chief executive officer and co-chief investment officer of Alexandria, discussed the plans for a laboratory facility to be built next to the future site of General Electric’s future world headquarters.
The site “represents a strategic opportunity to expand Alexandria’s unparalleled, world-class” life science development in the Boston area, Moglia stated.

In May, Alexandria and National Development, based in Newton, Massachusetts, bought a 2.7-acre plot of land fronting the Fort Point Channel. The price was $252 million. There is a renovated 95,000-square-foot warehouse on the property that GE has leased for 12 years. Zoning allows for a 12-story commercial building.
Aside from that investment, the two firms recently spent $81.1 million in March to buy a multi-level parking garage at 10 Necco Street. Alexandria also partnered with Anchor Line Partners to develop a nine-story laboratory facility in South Boston at 99 A Street.
According to Alexandria’s 10Q filing for the quarter, it invested $12.872 billion in real estate as of June 30, up from the same period the year before of $11.913 billion. Its total assets for the half-year period total $16.039 billion. Total liabilities, noncontrolling interests and equity hit $16.039 billion.
Revenues for the quarter totaled $373.9 million, and for the six-month period, $723.7 million. Total expenses for the quarter were $309.4 million and for the half-year mark, $616.2 million.
The firm also notes that in addition to its U.S. properties, it has three operating properties in Canada and one in China.

Of the Necco site, Moglia said Alexandria is planning an “iconic … world-class life-science building with a robust and vibrant set of ground-floor amenities. At this point, the company believes it will break ground in early 2020.
Other recent projects have included: in April, the launch of Phase 1 of the Alexandria Center for AgTech – Research Triangle, the first and only fully integrated, multi-tenant agtech R&D and greenhouse camps in Research Triangle; in June, a partnership with Columbia University to open its second Alexandria LaunchLabs in New York City, planned for the spring of 2020; and also in June, opened the first facilities with a tech-focused opioid rehabilitation campus in Dayton, Ohio in partnership with Verily Life Sciences.
Earlier this month Alexandria and TMG Partners won full project approval to develop the 88 Bluxome campus in the San Francisco area.
“88 Bluxome will bring an exceptional mix of much-needed community benefits and retail amenities to SoMa while it provides a dynamic campus environment to spur innovation,” stated Terezia Nemeth, senior vice president of real estate development and community relations at Alexandra. “We have worked closely with the City of San Francisco and our community partners to effect a new model for urban development, one that supports the world’s leading innovators, embraces excellence in sustainability and takes a holistic approach to engaging and activating the community. Alexandria is committed to making a meaningful and lasting impact in all of the communities where we develop and operate, and beyond.”

Fitbit cuts 2019 revenue view, Versa Lite sales lag; shares tank

Wearable device maker Fitbit Inc cut its 2019 revenue forecast on Wednesday, blaming disappointing sales of its newly launched cheapest smartwatch Versa Lite, sending its shares tumbling as much as 16% to a record low.

Fitbit in March launched Versa Lite, priced at $160, compared with the $200 the full version sells for. The watch, which can track workouts and heart rate, lack features such as the ability to store music directly.
“While Versa Lite received good present consumer reviews, we saw that consumers were willing to pay more for a smartwatch with additional features or look for discounting versus everyday value,” Chief Executive Officer James Park said during a post earnings call with analysts.
The company had moved into the smartwatch market to cushion the hit from slowing growth of its popular colorful fitness trackers, but has faced tough competition from deeper-pocket companies such as Apple Inc and Samsung Electronics.
Smartwatch revenue decreased 27% year-over-year in the second quarter.
On the call, Chief Financial Officer Ron Kisling also said the company anticipates burning cash in the first three quarters before generating cash in the fourth.
Fitbit lowered its 2019 revenue forecast to between $1.43 billion and $1.48 billion, compared with its prior expectations of $1.52 billion to $1.58 billion.
The disappointing performance of Versa Lite suggests the overall health of the business is not as strong as we had thought, DA Davidson analyst Thomas Forte said.
Versa Lite also weighed on average selling price, which fell 19% to $86 per device and missed Wall Street expectations of $91, according to research firm FactSet.
Some analysts also pointed to the weak performance of the company’s health business – widely seen as a growth driver – that connects users with doctors, hospitals and lifestyle coaches.
Fitbit has been partnering with health insurers and making tuck-in acquisitions in the healthcare market as part of efforts to diversify its revenue stream.
Wedbush Securities analyst Alicia Reese said the 16% growth in the business during the second quarter was underwhelming and investors would want to see more if the segment is the major growth driver for Fitbit.
The business reported a 70% growth in the preceding quarter to post revenue of $30.5 million.
Fitbit estimated revenue of between $335 million and $355 million for the third quarter, below analysts’ a
verage estimate of $399.4 million, according to IBES data from Refinitiv. (
The company also forecast an adjusted loss of between 9 cents and 11 cents per share. Analysts were expecting a profit of 2 cents per share.
Excluding items, the company lost 14 cents per share in the second quarter ended June 30, smaller than estimates of 18 cents loss.
Revenue rose about 5% to $313.6 million, beating analysts’ average estimates of $312 million.
The company’s shares have fallen 15.5% this year to Wednesday’s close.

Theravance Biopharma EPS beats by $0.50, beats on revenue

Theravance Biopharma (NASDAQ:TBPH): Q2 GAAP EPS of -$0.72 beats by $0.50.
Revenue of $26.15M (+11.4% Y/Y) beats by $14.22M.

Translate Bio’s MRT5005 encouraging in early-stage CF study

Preliminary results from a Phase 1/2 clinical trial, RESTORE-CF, evaluating single and ascending doses of Translate Bio’s (NASDAQ:TBIO) inhaled mRNA therapeutic MRT5005 in cystic fibrosis (CF) patients showed a positive effect at certain doses.
Data from the single ascending dose (SAD) portion in 12 CF patients (participants received either 8 mg, 16 mg, 24 mg or placebo) showed that the 16 mg dose was the most effective in terms of improved lung function (ppFEV1) with a mean maximum increase from baseline of 15.7%. All three subjects in this arm experienced maximum increases of 11.1 – 22.2%. The treatment effect in the 8 mg arm was not much better than placebo while the 24 mg arm was better than placebo but not as significant as the 16 mg arm.
On the safety front, the number of treatment-related adverse events in the three arms were 28, 25 and 33, respectively, compared to 11 in the control arm. All were mild or moderate. The most common were cough and headache.
The company plans to add a 20 mg dose cohort to the SAD portion. Preliminary data from this arm and the multiple ascending dose portion should be available in 2020.
Additional results from this initial dataset will be presented at the North American Cystic Fibrosis Conference in Nashville, TN, October 31 – November 2.
Orphan Drug-tagged MRT5005 is designed to address the underlying cause of CF by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung epithelial cells through nebulization.
Management will host a conference call today at 8:00 am ET to discuss the results.

Translate Bio misses on revenue

Translate Bio (NASDAQ:TBIO): Q2 GAAP EPS of -$0.57.
Revenue of $1.17M misses by $0.13M.

Regenxbio out-licenses gene therapy vector to Pfizer

REGENXBIO (NASDAQ:RGNX) inks an agreement with Pfizer (NYSE:PFE) granting it a non-exclusive global license, with sublicense rights, to its NAV AAV9 vector for the development and commercialization of gene therapies for the treatment of a rare inherited neurodegenerative movement disorder called Friedreich’s ataxia.
Under the terms of the deal, RGNX will receive an upfront payment, milestones and royalties on net sales. Specific financial terms are not disclosed.

Humana up 7% premarket on Q2 beat; raised 2019 guidance

Humana (HUMQ2 results: Revenues: $16,245M (+13.9%); Premiums: $15,776M (+15.0%); Services: $355M (-7.1%); Investment income: $114M (-30.5%).
Net Income: $940M; EPS: $6.94; Non-GAAP EPS: $6.05 (+52.8%).
Q2 Benefit ratio was 84.4%; Retail benefit ratio of 85.2%; Group and Specialty benefit ratio of 86.3%.
Individual Medicare Advantage membership was 3,484,500 (+15% Y/Y).
Group Medicare Advantage membership was 519,100 (+5% Y/Y).
2019 Guidance: Total Revenues: $64.2B – 64.8B from $63.3B – 63.9; GAAP EPS: ~$17.97 from ~$16.63 – 16.88; Non-GAAP EPS: ~$17.60 from ~$17.25 – 17.50; Individual Medicare Advantage: up 480K – 500K from 415K – 440K;  Group Medicare Advantage: up ~30,000 (unch); Retail Benefit Ratio: 86.4% – 86.8% from 86.4% – 87.4%; Group and Specialty segment Benefit Ratio: 82.4% – 82.8% from 81.3% – 81.8%.
Shares are up 7% premarket.

Late-stage study underway for Biohaven’s verdiperstat

The first subject has been enrolled in a Phase 3 clinical trial, M-STAR, evaluating Biohaven Pharmaceutical Holding Company’s (NYSE:BHVN) verdiperstat (BHV-3241) in patients with a rare degenerative neurological disorder called multiple system atrophy (MSA) characterized by Parkinson’s disease-like symptoms.
The primary endpoint is the change from baseline in an MSA scale called UMSARS at week 48 compared to placebo. The estimated completion date is October 2021.
Verdiperstat is an orally available, blood-brain barrier penetrant, irreversible inhibitor of an enzyme called myeloperoxidase that plays a key role in oxidative stress and inflammation in the brain.

Omeros secures long-term production deal for OMS721

Omeros (NASDAQ:OMER) inks an agreement with Basel, Switzerland-based Lonza Group (OTCPK:LZAGF) for the commercial production of narsoplimab (OMS721). Financial terms remain confidential.
The company is preparing marketing applications in the U.S. and Europe for hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA).

FDA calls key Impossible Foods ingredient safe

The FDA says heme looks safe and suitable for use in Impossible Foods (IMPSBL) products.
The soy leghemoglobin additive gives Impossible Burgers their meat-like flavor and red coloring.
Beyond Meat (BYND +3.5%) uses pea protein and beet extract instead of soy protein and soy leghemoglobin.

LivaNova Q2 top-line down 4%; reiterates guidance

LivaNova (LIVN +1.6%Q2 results: Revenues: $277.2M (-3.6%); Cardiovascular: $172.2M (-2.4%); Neuromodulation: $104.3M (-5.8%); Other: $0.7M (+75.0%).
Net Loss: ($29.4M); Loss Per Share: ($0.61); Non-GAAP Net Income: $34.3M (-27.8%); Non-GAAP EPS: $0.70 (-27.1%); CF Ops: ($17.4M) (-135.9%).
2019 guidance: Revenue growth: 1% – 3% (unch); non-GAAP EPS: $3.00 – 3.10 (unch).

TCR2 teams up with NCI in cancer study of proprietary T cells; shares up 3%

TCR2 Therapeutics (TCRR +2.7%) will collaborate with the National Cancer Institute (NCI) on a Phase 1/2 clinical trial evaluating its proprietary T cell receptor (TCR) Fusion Construct T cells (TRuC-T cells) (named TC-210 at present) as a cancer therapeutic agent against a protein called mesothelin that is overexpressed in certain cancers (e.g., mesothelioma, ovarian cancer, pancreatic adenocarcinoma).
NCI’s activities will be aimed at better understanding TC-210’s pharmacodynamics via translational studies, measuring potential biomarkers in treated patients.

DelMar Pharma up 43% on advancement of VAL-083

Nano cap DelMar Pharmaceuticals (DMPI +42.9%) is up more than a 5x surge in volume in reaction to its announcement that enrollment in its ongoing Phase 2 clinical trial evaluating VAL-083, combined with radiation therapy, in newly diagnosed patients with MGMT-unmethylated glioblastoma multiforme (brain cancer) is 2/3 (~20 subjects) of the way there.
Target enrollment in the China-based trial is 30. The estimated primary completion date is December.
Orphan Drug-tagged VAL-083 (dianhydrogalactitol) is a small molecule chemotherapeutic, specifically, a bifunctional alkylating agent that kills cancer cells by breaking up DNA.