Gene si­lenc­ing com­pa­ny NeuBase Ther­a­peu­tics to re­verse merge its way on­to Nas­daq via trou­bled mi­cro­cap Ohr

Di­et­rich Stephan

Di­et­rich Stephan, one of the pi­o­neers in the field of pre­ci­sion med­i­cine, was in process of rais­ing ven­ture funds for his gene-si­lenc­ing com­pa­ny NeuBase Ther­a­peu­tics when he was in­tro­duced to the team at the strug­gling eye drug de­vel­op­er Ohr Phar­ma­ceu­ti­cal $OHRP. Ini­tial con­ver­sa­tions com­menced less than a month ago, and on Thurs­day, the two sides came to an agree­ment — NeuBase would cat­a­pult on­to the Nas­daq via a re­verse merg­er with Ohr, and pro­pel the de­vel­op­ment of its rare ge­net­ic dis­ease drugs.

NeuBase’s mod­u­lar an­ti­sense pep­tide nu­cle­ic acid (PNA) plat­form — dubbed PA­TrOL — is de­signed to im­prove gene si­lenc­ing ther­a­pies by fus­ing the ad­van­tages of syn­thet­ic ap­proach­es with the pre­ci­sion of an­ti­sense tech­nolo­gies, which in­hib­it the pro­duc­tion of dis­ease-caus­ing pro­teins.

NeuBase’s tech­nol­o­gy has sev­er­al ad­van­tages over tra­di­tion­al an­ti­sense oligonu­cleotides, ac­cord­ing to Stephan, who cur­rent­ly serves as a pro­fes­sor of hu­man ge­net­ics at the Uni­ver­si­ty of Pitts­burgh and has found­ed and co-found­ed at least 13 com­pa­nies, in­clud­ing NeuBase.

Ja­son Slak­ter

NeuBase’s tech­nol­o­gy al­lows for rapid drug de­sign due to the mod­u­lar na­ture of the PA­TrOL plat­form, se­lec­tiv­i­ty for spe­cif­ic RNA/DNA se­quences, the abil­i­ty to pen­e­trate the blood brain bar­ri­er, broad sys­temic dis­tri­b­u­tion for mul­ti-tis­sue dis­ease and high cell per­me­abil­i­ty. Ear­ly in vi­vo da­ta have al­so in­di­cat­ed no im­mune re­sponse and a low cost of goods, which could be es­sen­tial for scal­a­bil­i­ty in po­ten­tial­ly ad­dress­ing a wide range of ge­net­ic dis­eases, in­clud­ing can­cer.

But at this point, NeuBase is still in the process of val­i­dat­ing its ap­proach and has much work ahead — it is still con­duct­ing pre­clin­i­cal stud­ies, and ex­pects to en­ter the clin­ic with­in the next few years.

“Io­n­is has gen­er­at­ed aware­ness around the po­ten­tial for an­ti­sense tech­nolo­gies, but we are con­fi­dent that our PA­TrOL plat­form has sig­nif­i­cant ad­van­tages that will al­low us to ex­pand be­yond the space that Io­n­is $IONS has cre­at­ed,” Stephan told End­points News.

The com­pa­ny plans to file an IND for a drug for Hunt­ing­ton’s dis­ease in 2020 and one for my­oton­ic dy­s­tro­phy in 2021.

If the deal with Ohr is con­sum­mat­ed, NeuBase has pro­posed trad­ing un­der the sym­bol $NB­SE and will re­tain the ma­jor­i­ty 80% stake of the com­bined com­pa­ny, while Ohr will get a 20% stake. Stephan will lead the com­bined com­pa­ny.

“Fol­low­ing a com­pre­hen­sive re­view of strate­gic al­ter­na­tives, Ohr’s Board of Di­rec­tors con­clud­ed that the pro­posed trans­ac­tion with NeuBase is in the best in­ter­est of our stock­hold­ers…we in­tend to hold a spe­cial meet­ing of Ohr share­hold­ers in the first half of 2019 to vote on this merg­er,” Ohr CEO Ja­son Slak­ter said in a state­ment on Thurs­day.

Ohr’s shares $OHRP sky­rock­et­ed near­ly 82% be­fore the bell on the news.

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

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Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.