Search This Blog

Friday, June 21, 2019

Vertex: FDA Approves SYMDEKO treatment for peds cystic fibrosis

-SYMDEKO is now approved for patients 6 years of age and older with two copies of the F508del mutation or one copy of a responsive mutation-
-Vertex’s third medicine approved to treat the underlying cause of CF in eligible patients in this age range-
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) for use in children with cystic fibrosis ages 6 through 11 years who have two copies of the F508del-CFTR mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to SYMDEKO. It was previously approved by the FDA for use in patients with cystic fibrosis 12 years and older with two copies of the F508del mutation or one copy of a responsive mutation in the U.S. An additional dosage strength of SYMDEKO tablets is now available (tezacaftor 50 mg/ivacaftor 75 mg and ivacaftor 75 mg) in connection with this approval.
“Today’s expanded approval of SYMDEKO in children ages 6 through 11 is an important step in our efforts to continue to bring treatment options to the youngest patients possible and importantly brings us closer to our goal of developing medicines for all people living with CF,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex.

Analysts to Bayer CEO: Start with the ‘simple things,’ then split up

Bayer CEO Werner Baumann has already put the company on a major overhaul mission to focus on life sciences. But apparently, investors aren’t satisfied yet.
Shareholders are still complaining about how Bayer management is handling the Roundup weedkiller litigation and executives’ lack of communication with the market over business information and future stock-moving events, Bernstein analysts Wimal Kapadia and Gunther Zechmann wrote in a letter to Baumann.
Their suggestions? “Start with the simple things (communication) and keep going” by settling the Roundup suits, addressing the pharma patent cliff and, last but not least, splitting up.
The Bernstein analysts’ observation echoes a rare no-confidence vote in management that came out of the company’s annual general meeting in April. There, more than half of shareholders voted against ratifying the Baumann-led executive team’s actions in 2018.
One reason? Bayer has made little effort at offering information on the standalone performance progress of Monsanto and the pre-merger crop business, the analysts said. And that doesn’t give investors the peace of mind that everything is going according to plan.
“In general, information availability vs. peers and willingness to disclose non-material information, is an opportunity for improvement,” they argued. “This matters more than you think.”

On the pharma side, Kapadia has long warned of the patent cliff for blood clot-buster Xarelto and eye drug Eylea, which could make up about 70% of Bayer’s pharma earnings in 2023, right before their protection expires. Despite the importance of those drugs in Bayer’s portfolio, the vicinity of the cliff and the lack of strength in the company’s late-stage pipeline, “investors do not sense urgency” from management, he said.
As no internal in-development drug is significant enough to help soften the blow, the Bernstein analysts suggest Bayer go after the in-licensing route; just two to three assets would give investors comfort.
“Given the capital constraints due to potential litigation settlement and the Monsanto acquisition, Bayer should approach U.S. Biotech with limited infrastructure (out of U.S.) and offer to be a partner of choice (like Eylea and original Vitrakvi deal with Loxo) in exchange for a manageable upfront and royalty payment,” they said.
That way, investors will notice Bayer is working to mend things, and the company can avoid competing with cash-rich bigger pharma peers for more mature assets.
For now, that seems to be the direction Bayer is moving. In its restructuring announcement last December, the company said it was cutting 12,000 jobs, selling two consumer health care brands and hiving off its animal health business. The savings from the reduction would go toward “investment in collaborative research models and external innovations,” the company said at the time.

But again, in those Bernstein analysts’ opinion, licensing is just a near-term remedy. For the long term, a separation of pharma and crop would create value for shareholders, they said.
“We struggle to see major synergies between the two divisions,” they explained. “Yes, they are both ‘research’ focused, but we suspect the overlap and tech transfer are minimal and provides no competitive advantage to peers.”
This is not the first time Bernstein has rooted for a split at Bayer. Before the makeover, they noticed Bayer’s shares were hurt by its multiple divisions and said a two-way separation could be its best option to restore the pharma division to trade at fair multiples.
As for the mounting Roundup lawsuits that have taken a $33 billion toll on Bayer’s stock, the Bernstein analysts are asking management to “settle the litigation and move on.”
“Past practices of ‘campaigning’ to defend the chemical must no longer be pursued and a genuine dialogue with regulators sought,” the Bernstein team suggested.
Despite recent defeats in U.S. state and federal trials that come with damages in the millions of dollars, Bayer hasn’t budged, saying it “continues to believe that it has meritorious defenses and intends to defend itself vigorously in all of these lawsuits.” By Bayer’s last count in April, the number of suits that claim Roundup caused cancer has swelled to 13,400.

Tough road seen for Juul: Gottlieb

Former FDA Commissioner Scott Gottlieb thinks Juul (JUUL) is in a tough bind as high-level government scrutiny ramps up.
“They have so much historical youth use with their product. I don’t know how Juul gets through an application process,” stated Gottlieb.
Even if the long-term plan at Juul is to sell a kid-proof product, Gottlieb noted that the documents to be reviewed by the FDA will reflect the marketing practices and selling trends from the last couple of years.
Behind the scenes, Altria (MO -4.3%) holds a key 35% stake in Juul.

Marker Therapeutics to report updated results from MultiTAA therapy trial

Market Therapeutics (MRKR +5.7%) surges to seven-month highs on heavy volume following yesterday’s announcement that updated clinical results from a Phase 1/2 trial with MultiTAA therapy in patients with pancreatic adenocarcinoma were selected for oral presentation during a plenary session at an upcoming American Association for Cancer Research conference.
MRKR is seeking to develop next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications.

BofAML names Sarepta as top pick

Bank of America Merrill Lynch names Sarepta (SRPT -0.2%) as the top small- to mid-cap pick in biotech for H2 and sees several near-term catalysts for the company.
The firm cites upside potential from Sarepta’s LGMD gene therapy pipeline, which isn’t priced into the current valuation.
Analyst Tazeen Ahmad says LGMD2E clinical data is encouraging and notes that the treatment doesn’t have competition.
Ahmad also calls Sarepta a front-runner in DMD with plans to initiate a pivotal commercial supply study by the end of the year.
Pfizer will present data for its dystrophin DMD gene therapy at the end of the month. Ahmad writes that in the worst-case scenario, Pfizer would look competitive with Sarepta, an outcome that wouldn’t change the analyst’s estimates.
SRPT has an Outperform average Sell Side rating.

Daiichi Sankyo gets CRL on leukemia med candidate

Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) today announced that the company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) of quizartinib for the treatment of adults with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML).
“Daiichi Sankyo is evaluating the Complete Response Letter and will determine next steps in the U.S.,” said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo.
About Quizartinib
Quizartinib, an oral selective type II FLT3 inhibitor, is the lead product in the AML Franchise of Daiichi Sankyo. Quizartinib was approved by the Ministry of Health, Labor and Welfare (MHLW) of Japan under the brand name of VANFLYTA® for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, as detected by an MHLW-approved test, on June 18, 2019.
A broad and comprehensive development program is underway with quizartinib including phase 3 development in combination with standard chemotherapy in newly diagnosed FLT3-ITD AML (QuANTUM-First) in the U.S., EU and Japan; phase 1/2 development for pediatric and young adult relapsed/refractory FLT3-ITD AML in North America and the EU; and phase 1 development in combination with an investigational MDM2 inhibitor, milademetan, for relapsed/refractory FLT3-ITD AML and newly-diagnosed FLT3-ITD AML unfit for intensive chemotherapy in the U.S.
Milademetan is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established. Quizartinib is only approved for use in Japan.

European Medicines Agency Accepted First ‘China-Developed’ Biosimilar

Henlius trastuzumab biosimilar HLX02 has been accepted by the European Medicines Agency (EMA) for reviewing its Marketing Authorization Application (MAA). HLX02 has established three “Firsts” both in China and globally.
  • First China biosimilar conducted a multi-center, international phase 3 clinical trial
  • First trastuzumab developed in China following the National Medical Products Administration (NMPA) technical guideline for biosimilar with the New Drug Application (NDA) received the acceptance for review from the NMPA
  • First China-developed trastuzumab biosimilar accepted for MAA review by the EMA
Henlius, a leading biotech company in China developing both biosimilar and innovative biologics, announced today that the European Medicines Agency (EMA) has officially accepted to review the Marketing Authorization Application (MAA) of its trastuzumab biosimilar HLX02. The European rights of this product have been out-licensed to its business partner Accord Healthcare. HLX02 is indicated for human epidermal growth factor receptor 2-positive (HER2+) early-stagebreast cancer, HER2+ metastatic breast cancer, and untreated HER2+ metastatic gastric and gastroesophageal junction (GEJ) cancer.
The New Drug Application (NDA) of HLX02, a biosimilar of a classical targeted therapy for cancers, has been accepted for review by the National Medical Products Administration (NMPA) in China. The acceptance for review by the EMA demonstrates that the “China-developed” biosimilar has entered the global stage with the hope to benefit patients globally.
“We are very pleased to receive the EMA acceptance to review of our trastuzumab biosimilar HLX02 MAA submission,” said Dr. Scott Liu, co-Founder, President and CEO at Henlius. “It shows that our clinical, medical, regulatory affairs and quality system capabilities have been internationally recognized. We plan to work closely with EMA on this HLX02 MAA and hope to benefit HER2+ cancer patients globally with its high-quality and affordability.”
The Phase 3 study aimed for head-to-head similarity evaluation in efficacy, safety and immunogenicity profiles between HLX02 and reference trastuzumab sourced from the European Union (EU) in 649 previously untreated patients with HER2+ metastatic breast cancer in mainland ChinaUkrainePoland and the Philippines. The Phase 1 study has successfully demonstrated the equivalence in pharmacokinetics and safety profiles between HLX02 and reference trastuzumab sourced from both EU and China.
Apart from the R&D and innovation efforts, Henlius is also actively creating new business models and expanding its global presence. The acceptance of the regulatory submission of trastuzumab biosimilar HLX02 MAA in Europe is another example of strong progress Henlius continues to make across the robust biosimilars portfolio. The acceptance of HLX02 MAA review marks a key milestone for the collaboration between Henlius and its partner Accord. In June 2018, Henlius signed the licensing and supply agreements with Accord. Under this partnership, Accord is exclusively authorized to commercialize HLX02 in 53 countries including UK, FranceGermany, and Italy in Europe; 17 countries in Middle EastNorth Africa (“MENA”) and certain countries in Commonwealth of Independent States (“CIS”).
Henlius has strong capabilities in integrated innovation. Its first rituximab injection (HLX01) has been successfully launched in China. Currently, both HLX02 (trastuzumab biosimilar) and HLX03 (adalimumab biosimilar) NDAs are under NMPA review. Henlius has a robust R&D pipeline with IND/CTA filings for 13 products and two combination therapies in 23 indications.