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Friday, June 26, 2020

Ascendis Pharma files U.S. marketing application for expanded use of TransCon

Ascendis Pharma A/S (NASDAQ:ASND) submits a Biologics License Application to the FDA for TransCon hGH (lonapegsomatropin), a long-acting once-weekly prodrug of somatropin (human growth hormone or hGH) for the treatment for pediatric growth hormone deficiency (GHD).
Currently, there is no approved long-acting growth hormone treatment in the U.S. or Europe.
The company plans to submit a marketing authorisation application for TransCon hGH in Europe in Q3 and to initiate a Phase 3 trial in pediatric GHD in Japan in Q4.

Sarepta finalizes U.S. application for DMD med casimersen

Sarepta Therapeutics (NASDAQ:SRPT) completes its rolling New Drug Application (NDA) to the FDA seeking accelerated approval for casimersen (SRP-4045), for treatment of patients with Duchenne muscular dystrophy (DMD).
The NDA includes data from the casimersen arm of the ESSENCE study, evaluating efficacy and safety in patients amenable to skipping exons 45 and 53.
If accepted, the completed ESSENCE study will serve as a post-marketing confirmatory study.

Citius rallies on accelerated review prospects for stem cells for COVID-19

Nano cap Citius Pharmaceuticals (NASDAQ:CTXR) jumps 18% premarket on robust volume on the heels of FDA feedback that it could apply for Fast Track status for its induced mesenchymal stem cells to treat and reduce the severity of acute respiratory distress syndrome (ARDS) in COVID-19 patients.
The agency also provided requirements for chemistry, manufacturing and control (CMC) data for its proposed trials.
The company intends to follow-up on the regulator’s recommendations and with an IND application under the Coronavirus Treatment Acceleration Program.

Bristol Myers’ Reblozyl OK’d in Europe

June 26, 2020

As expected, the European Commission approves Bristol Myers Squibb’s (NYSE:BMY) Reblozyl (luspatercept) for the treatment of adults with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes with ring sideroblasts, who had an unsatisfactory response or are ineligible for erythropoietin-based therapy and adults with transfusion-dependent anemia associated with beta thalassemia.
About two months ago, the advisory group CHMP adopted a positive opinion backing approval.

FDA rejects Allergan application for wet AMD drug

June 26, 2020

AbbVie (NYSE:ABBV) unit Allergan has received a Complete Response Letter (CRL) from the FDA regarding its marketing application seeking approval of DARPin drug abicipar pegol for the treatment of neovascular age-related macular degeneration (wet AMD).
The agency cited an unfavorable benefit/risk ratio.
The company plans to meet with the regulator to discuss next steps.
Allergan in-licensed exclusive global rights to the candidate from Molecular Partners (OTCPK:MLLCF) in August 2021 in a deal valued as high as $420M.
Wet AMD-related tickers: REGN, NVS, RGNX, ADVM, OCUL, OTCPK:CKDXY, OTLK, ISEE, OTCPK:BAYRY, OTCQX:RHHBY

Vaxart +70% after Covid-19 ‘Warp Speed’ designation for its vaccine

Vaxart (NASDAQ:VXRT) is surging for the second day in a row, up 70% pre-market, after the U.S. Government selected their oral vaccine as eligible to participate in a non-human study organized and funded as part of Operation “Warp Speed.”
The stock surge is coming off a day in which the stock nearly doubled when signing a deal to enable large scale manufacturing of its COVID-19 vaccine. 
The CEO said, “We are very pleased to be one of the few companies selected by Operation Warp Speed, and that ours is the only oral vaccine being evaluated. ” He added, ” our vaccine is a room temperature-stable tablet, an enormous logistical advantage in large vaccination campaigns.”
Operation Warp Speed originally began when the White House selected 5 candidates in early June.  They included candidates from Moderna (NASDAQ:MRNA), AstraZeneca (NYSE:AZN), Johnson & Johnson (NYSE:JNJ), Merck (NYSE:MRK) and Pfizer (NYSE:PFE).
Vaxart’s market capitalization stood at $525M as of yesterday’s close. 

Thursday, June 25, 2020

Pandemic Exposes Multiple Follies of Medicaid Expansion

We’re getting mixed messages on health care these days.
At both the federal and state levels, the response to the COVID-19 pandemic required turning to the private sector. Governments suspended unnecessary regulations so patients could access the care they needed and providers could innovate to meet the crisis.
Paradoxically, some politicians continue to pitch the idea that the answer to our health care problems is more government, through expansion of Medicaid or even so-called Medicare for All (which, as health care policy experts have noted, would operate more like Medicaid for All).
Medicaid is an important part of the safety net, but it should be focused on those most in need, not expanded to include able-bodied adults with no children. That’s even more important now, with states confronting difficult spending choices as revenues decline in the wake of the coronavirus-induced lockdowns.
Both Democratic and Republican states are facing up to that reality and are reexamining their Medicaid expansion plans. This should not come as a surprise. Even before the pandemic, Medicaid was devouring state budgets, with several spending more than a quarter of state-generated revenue on the program. But now the tough choices have become unavoidable.
Efforts by North Carolina’s Democratic governor, Roy Cooper, to expand Medicaid have hit a wall as the pandemic turned a surplus into a multibillion-dollar revenue shortfall almost overnight.
In Kansas, a Republican state with a Democratic governor, a deal to use the Affordable Care Act to expand Medicaid coverage to those earning up to 138 percent of the federal poverty level was put on hold.
Next door in Oklahoma, Republican Governor Kevin Stitt vetoed what he considered an inadequate funding package for his own “SoonerCare” plan to expand Medicaid, though voters will decide the issue at the end of the month.
Similar cuts are being debated in New York, Ohio, and other states that expanded their Medicaid rolls after the last recession. Many of these states face deep shortfalls despite receiving billions in coronavirus and recession related emergency aid from Washington, including a $36 billion boost in no-strings federal Medicaid funding. But this unprecedented federal aid is temporary and states cannot rely on it. Instead, they must make responsible budget decisions independent of Washington and consistent with what their taxpayers can afford.
Even deep-blue California is now mulling cuts to its Medicaid program to help close a $54 billion budget deficit, its biggest ever.
In Colorado, where Democrats control the governorship and the legislature, a proposed public option designed to compete with existing private insurers is off the table, with lawmakers facing cuts in every other part of the state budget.
But cost is not the only negative effect of Medicaid expansion. Critically, the number of hospital beds per capita has fallen more than 6 percent since 2013 in states that expanded Medicaid, while non-expansion states increased bed capacity, according to the Foundation for Government Accountability.
As pandemic – and lockdown-induced economic woes – decimate state tax collections, these challenges will only increase.
So while states are now turning away from expanding government health care on the payment side of the ledger, what have they been doing on the delivery-of-care side? Going the opposite direction, loosening some of the misguided regulations that made it harder for health care professionals to treat patients.
More than 600 regulations at the federal and state levels have been waived in response to the pandemic. Governors across the political spectrum moved swiftly to empower health care providers and patients, from easing rules on telehealth to allowing doctors and nurses to work across state lines, to suspending certificate-of-need laws that blocked expansion of needed equipment and services.
It has been a real-time demonstration project in empowering doctors, nurses, is less bureaucracy means better, faster care. Seeing these positive outcomes, lawmakers in many states are looking to make the changes permanent. Governors, state legislators, and Congress should respond accordingly.
Far from being a greenlight for more government control of health care, the coronavirus pandemic has been a clear lesson in what can be accomplished when we get bureaucracy and red tape out of the way.