Target $20
Wednesday, October 6, 2021
AIM ImmunoTech: Ampligen has significant potential as intranasal therapeutic for COVID-19.
AIM ImmunoTech Inc. (NYSE American: AIM) today released detailed safety data from a Phase 1 clinical study which supports the company’s belief that its drug Ampligen has significant potential as an intranasal therapeutic for COVID-19.
AIM previously announced that the study to assess the safety, tolerability and biological activity of Ampligen as a potential intranasal therapy was completed, and that a Safety Report found the drug to be generally well tolerated and reported no Severe Adverse Events. More details are now being published on both the AIM website and on the website for the Centre for Human Drug Research (CHDR). A total of 40 healthy subjects received either Ampligen or a placebo in the trial, with the Ampligen given at four escalating dosages across four cohorts, to a maximum level of 1,250 micrograms. The poster was recently presented at the FIGON Conference in the Netherlands. Dosing was administered every other day for 7 doses.
4D Molecular Therapeutics Gets FDA Clearance for Two IND Applications
4D Molecular Therapeutics Inc. said the U.S. Food and Drug Administration has cleared the investigational new-drug application for 4D-150 for wet age-related macular degeneration.
The company said the active IND enables the initiation of 4D-150 Phase 1/2 clinical trial sites, which is expected before year-end.
4D Molecular Therapeutics also said the FDA has cleared the IND application for 4D-710 for the treatment of patients with cystic fibrosis. This active IND enables the initiation of 4D-710 Phase 1/2 clinical study sites, which is also expected before the end of the year, the company said.
The Phase 1/2 clinical trial for 4D-150 is a dose-escalation and randomized, controlled, masked expansion trial, expected to enroll 60 adults with wet AMD. The primary endpoints of the study are safety and tolerability, the company said.
Secondary endpoints include the number of supplemental aflibercept injections received, and change from baseline in best corrected visual acuity over time, the company said.
The Phase 1/2 clinical trial for 4D-710 is dose-expansion trial of 4D-710 in patients with cystic fibrosis who are ineligible for cystic fibrosis transmembrane conductance regulator, or CFTR, modulator therapy or who have discontinued therapy due to adverse effects. The primary endpoint of the study is safety and tolerability, the company said.
As a result of the IND clearance for 4D-710, the Cystic Fibrosis Foundation will buy 126,000 shares of 4D common stock for proceeds of $4 million, in accordance with the terms of the company's collaboration with the foundation. The proceeds of the CF Foundation's equity investment will be used to further support development of 4D-710, the company said.
Bayer in first trial win over Roundup
Shares in Bayer AG rose 2.5% in pre-market trade on Wednesday after the German agricultural and pharmaceuticals firm won its first trial over claims its Roundup weedkiller causes cancer.
A California jury found that the herbicide was not a substantial cause of a child's rare form of non-Hodgkin's lymphoma, the company said on Tuesday. The verdict is the fourth involving Roundup and the first in the company's favor.
Roundup-related lawsuits have dogged Bayer since it acquired the brand as part of its $63 billion purchase of agricultural seeds and pesticides maker Monsanto in 2018.
Boston Scientific to Acquire Baylis Medical Co. for $1.75B
Boston Scientific Corporation (NYSE: BSX) today announced that it has entered into a definitive agreement to acquire Baylis Medical Company Inc. for an upfront payment of $1.75 billion, subject to closing adjustments. The acquisition will expand the Boston Scientific electrophysiology and structural heart product portfolios to include the radiofrequency (RF) NRG® and VersaCross® Transseptal Platforms as well as a family of guidewires, sheaths and dilators used to support left heart access. These platforms have advanced transseptal puncture and are clinically proven to enhance safety, efficacy and efficiency when crossing the atrial septum to deliver therapies in the left side of the heart, such as atrial fibrillation ablation, left atrial appendage closure (LAAC) and mitral valve interventions.i,ii,iii Baylis Medical Company is expected to generate net sales approaching $200 million in 2022, having achieved double-digit year-over-year sales growth during each of the past five years.
Merck & Co justifies paying up for Acceleron
Once Acceleron added clinical validation with sotatercept to a promising launch for its first drug, Reblozyl, it became an obvious takeover target. The big question today is why it should have fallen not to Bristol Myers Squibb but to Merck & Co, noted Jacob Plieth on Evaluate Vantage.
It was Bristol, after all, that inherited a key deal with Acceleron – one of the few Celgene transactions to have truly delivered – and this should have given it first-mover M&A advantage. Bristol did feature in early speculation over who would buy Acceleron, but appears to have been unwilling to part with the $11.5 billion that Merck paid up.
As investment cases go, Acceleron’s was pretty simple. The group was focused on TGF-β superfamily biology, with three assets that targeted it in slightly different ways: Reblozyl, approved for anaemia in beta-thalassaemia and myelodysplastic syndromes (MDS); sotatercept, in pivotal development for pulmonary arterial hypertension (PAH); and ACE-1334, an early project that did not feature in sell-side models. A legacy Celgene deal concerned Reblozyl and sotatercept, resulting in Bristol owing Acceleron a low to mid-20% royalty on the former, and Acceleron owing Bristol a low-20% interest on the latter.
However, sell-side forecasts never caught up with buyside expectations. Evaluate Pharma consensus suggests NPVs of $7.4 billion for Reblozyl – only a fraction of which would accrue to Acceleron – and $1.7 billion for sotatercept. Against this Acceleron reached a market cap of $8 billion before rumors of a takeover surfaced this month, and the $180 a share Merck agreed today amounts to a valuation of $11.5 billion.
Overall, Acceleron is the latest example of Merck putting to use the cash its Keytruda blockbuster is throwing off, following the acquisitions of Pandion, Peloton and Velosbio. The group told analysts that Acceleron would provide growth when Keytruda loses patent exclusivity later this decade, though low-cost competition could come even earlier.
Kyle Blenkenship, managing editor of EndPoints News noted that the acquisition doesn’t come at much of a premium: Shares of Acceleron closed Wednesday at around $175, suspiciously plummeting from a high of around $186 earlier in the day. The company’s stock had ticked up for days after reports from Bloomberg and the Wall Street Journal quoting insider sources mostly outlined the terms of the deal with Merck.
For Merck, the Acceleron buyout confirms the drugmaker’s intent to make a splash in rare disease as a new team of executives lead the way following the departures of CEO Ken Frazier and R&D czar Roger Perlmutter, the brain trust behind Merck’s massively successful Keytruda I/O franchise. Now, under CEO Rob Davis and R&D head Dean Li, formerly the head of early discovery R&D, Merck is looking outside of oncology for value as part of what could be a string of deals for the nascent leadership team.
Geoffrey Porges, analyst at SVB Leerink Research, said the price seemed fair given that a buyer would get 25% to 30% of the value of Reblozyl, which is likely to become a $2 billion-plus brand by the late 2020s. Merck would also get access to other drugs in Acceleron’s pipeline, including sotatercept, he said. “Acceleron will offer a buyer a relatively secure $1 billion to $1.5 billion in estimated revenue by 2025, increasing to an estimated $2.7 billion by 2030,” he said.
https://www.thepharmaletter.com/article/look-back-at-pharma-news-in-the-week-to-october-1-2021
Voyager looks to shake off tough year with Pfizer gene therapy pact worth $630M
Hoping to make gene therapies safer and looking to move on from a troubled 18 months, Pfizer is putting down as much as $630 million in an upfront/milestone-based payment for Voyager’s next-gen gene therapies.
Pfizer, which recently had to change the protocol for its ongoing Duchenne muscular dystrophy gene therapy trial after a series of safety concerns emerged, will pay $30 million upfront and $20 million in exercise fees for two options, with $580 million backloaded to use Voyager’s so-called TRACER AAV capsids specifically for neurologic and cardiovascular gene therapy programs.
These capsids have been sought out to target desired cells and tissues with greater specificity, at lower doses and with fewer off-target risks than conventional AAV, with enhanced blood-brain barrier penetration.
The hope is that these could prove safer in the long run and follows on from a similar $1.1 billion deal Takeda made this week with Selecta Biosciences, a pact that focuses on the biotech’s tolerogenic therapies that selectively lessen unwanted immune responses and can therefore also potentially make these therapies safer.
This also comes after Astellas has seen four deaths in young children from its now FDA halted gene therapy trial for AT132 in a rare neuromuscular disease, with three of the deaths appearing to be linked to the therapy and its affect on the liver. Gene therapies hold much promise but also come with risk; the future of their use will surely be focused heavily on making them safe for patients, so don’t be surprised to see more deals like this in the future.
“This transaction highlights the potential of our TRACER platform to identify novel AAV capsids that target desired cells and tissues with greater specificity at lower doses and with fewer off-target risks than conventional AAV serotypes,” said Michael Higgins, interim CEO of Voyager.
“We believe that our TRACER platform has the ability to produce not only enhanced blood-brain-barrier penetrant capsids, but also novel capsids with enhanced tropisms across a diversity of tissues and cell types, offering promise to unlock the fullest potential of gene therapies for a wide array of diseases with unmet medical need.”
This comes as Voyager has been dealing with its own issues: Last August, AbbVie axed its Alzheimer's and Parkinson’s disease pacts with Voyager after the Big Pharma paid $134 million to enter into the two partnerships but then dropped the projects after Voyager completed research activities.
That same year the biotech was slapped with a clinical hold on an IND for its gene therapy program, VY-HTT01, because of issues with chemistry, manufacturing and controls. This marked a miserable time for the program, coming one year after Sanofi’s Genzyme unit pulled out of a collab for it.
But in April, the biotech got a reprieve after the FDA lifted the hold, allowing the trial to continue after the issues were cleared up.
Just one month later, the biotech’s CEO Andre Turenne (replaced by Higgins for the time being) and its R&D chief Omar Khwaja, M.D., Ph.D., both left unexpectedly. This deal with Pfizer is some much needed good news.
“Our collaboration with Voyager will provide Pfizer with access to additional AAV capsids that may help further advance our industry-leading gene therapy portfolio,” added Seng Cheng, Ph.D., senior vice president and chief scientific officer of Pfizer's rare disease research unit.
“We are impressed with Voyager’s results to date and are enthusiastic about the potential to utilize these novel capsids to help accelerate the development of new therapeutic options for patients living with certain neurologic and cardiovascular diseases.”
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