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.

Con­quer­ing a silent killer: HDV and Eiger Bio­Phar­ma­ceu­ti­cals

Hepatitis delta, also known as hepatitis D, is a liver infection caused by the hepatitis delta virus (HDV) that results in the most severe form of human viral hepatitis for which there is no approved therapy.

HDV is a single-stranded, circular RNA virus that requires the envelope protein (HBsAg) of the hepatitis B virus (HBV) for its own assembly. As a result, hepatitis delta virus (HDV) infection occurs only as a co-infection in individuals infected with HBV. However, HDV/HBV co-infections lead to more serious liver disease than HBV infection alone. HDV is associated with faster progression to liver fibrosis (progressing to cirrhosis in about 80% of individuals in 5-10 years), increased risk of liver cancer, and early decompensated cirrhosis and liver failure.
HDV is the most severe form of viral hepatitis with no approved treatment.
Approved nucleos(t)ide treatments for HBV only suppress HBV DNA, do not appreciably impact HBsAg and have no impact on HDV. Investigational agents in development for HBV target multiple new mechanisms. Aspirations are high, but a functional cure for HBV has not been achieved nor is one anticipated in the forseeable future. Without clearance of HBsAg, anti-HBV investigational treatments are not expected to impact the deadly course of HDV infection anytime soon.

Am­gen chops 172 more staffers in R&D, op­er­a­tions and sales amid neu­ro­science ex­it, rev­enue down­turn

Neuroscience wasn’t the only unit that’s being hit by a reorganization underway at Amgen. As well as axing 149 employees in its Cambridge office, the company has disclosed that 172 others nationwide, including some from its Thousand Oaks, CA headquarters, are being let go.

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Ahead of strate­gic up­date, new Sanofi CEO mulls op­tions for con­sumer health­care arm — re­ports

Big pharma has made moves to sharpen its focus on developing new medicines, while slow-growing consumer health divisions fall by the wayside. Looks like another large drugmaker is considering a similar move. On Thursday, reports citing sources indicated that Sanofi is reportedly mulling a joint venture, sale, or a public listing of its consumer health arm.

The French group is in discussions for options that could value the division at $30 billion, Bloomberg and Reuters reported, citing sources familiar with the matter.

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The triple crown in biotech: An all-or-noth­ing bet on an FDA ap­proval of 3 drugs over 16 months starts to­day

Bristol-Myers Squibb’s $74 billion Celgene deal closed as expected Wednesday evening. And now a new clock has begun to tick down for Celgene shareholders who came away from the deal with CVRs — contingent value rights — worth $9 or nothing. Those CVRs start trading today as $BMYRT.

The new deadline they have is the end of March 2021, a little more than 16 months from now, when Bristol-Myers will need to gain approvals on 3 late-stage drugs it’s picking up in the buyout: Ozanimod and liso-cel (JCAR017) are due up at the end of 2020, with bb2121 deadlined at the end of Q1 in 2021.

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Genap­sys fi­nal­ly un­veils vaunt­ed se­quencer, but can it dent Il­lu­mi­na?

Hesaam Esfandyarpour holds what looks like a mini-cooler up to the computer screen in his California office.

Esfandyarpour is in his late-30s, with crows feet creeping up against a youthful face. He wears a gray polo and the device in his hand — with its hard plastic-looking shell, blue-and-white pattern, and a white plastic paddle resembling a handle jutting out the front — might contain diced strawberries and peanut-butter sandwiches to meet mom and the kids at a SoCal park. Instead, Esfandyarpour tells me it’s going to change medicine and biopharma research.

UP­DAT­ED: Make that 2 ap­proved RNAi drugs at Al­ny­lam af­ter the FDA of­fers a speedy OK on ul­tra-rare dis­ease drug

Seventeen years into the game, Alnylam’s pivot into commercial operations is picking up speed.
The bellwether biotech $ALNY has nabbed their second FDA OK for an RNAi drug, this time for givosiran, the only therapy now approved for acute hepatic porphyria. This second approval came months ahead of the February deadline — even after winning priority review following their ‘breakthrough’ title earlier.
AHP is an extremely rare disease, with some 3,000 patients in Europe and the US, not all diagnosed, and analysts have projected peak revenue of $600 million to $700 million a year. The drug will be sold as Givlaari.

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David Ricks. Eli Lilly

Eli Lil­ly touts $400M man­u­fac­tur­ing ex­pan­sion, 100 new jobs to much fan­fare in In­di­anapo­lis — even though it's been chop­ping staff

Eli Lilly is pouring in $400 million to beef up manufacturing facilities at its home base of Indianapolis. The investment, which was lauded by the city’s mayor, is expected to create 100 new jobs.

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Brii Bio backs in­fec­tious dis­ease start­up while ink­ing deal for its lead TB drug, dou­bling down on an­tibi­otics

Almost two years after leaving GSK to launch Brii Bio with a whopping $260 million in funding, Zhi Hong is seeing the trans-Pacific infectious disease specialist he set out to build take shape.

“Our pipeline is coming together,” he told Endpoints News, with 12 partnered assets plus some internal programs.

As its latest partner, AN2 Therapeutics, comes into the limelight for the first time with a $12 million seed round, so is Brii’s plans in the antibiotics space. Brii has obtained China rights to AN2’s antibacterial targeting mycobacterium tuberculosis for multi-drug resistant TB, which it says is in the clinical stage.

No­var­tis, Bay­er, Long­wood back ge­nomics start­up to speed search for im­munother­a­py tar­gets

Nearly a century passed between the first proto-immunotherapy attempts in cancer — crude and obscure but nonetheless with some scientific basis — and Jim Allison’s first T cell paper. Thirty-plus years flipped between the discovery of CTLA-4 as an off-switch and the approval of Yervoy. Twenty-two rolled between PD-1’s isolation and Opdiva and Keytruda. 

Longwood co-founder Lea Hachigian is betting she can hasten that. It’s a bet on newly established single-cell genomic analysis tech and the ability to crunch endless troves of data at a rate few others can, and investors including Leaps by Bayer and Novartis Venture Fund just put $39 million behind it. They call it Immunitas.